February 28, 2011 is Rare Disease Day.
An estimated 30 million people are affected by one of about 7,000 rare diseases. Take a minute to pray for someone you might know and their daily fight to survive!!
Your fight is my fight! ♥
Showing posts with label Aplastic Anemia. Show all posts
Showing posts with label Aplastic Anemia. Show all posts
Friday, February 25, 2011
Tuesday, February 22, 2011
Leading To BMT
Aplastic Anemia is a bone marrow failure disease in which the blood stem cells in the bone marrow fail to produce sufficient life-sustaining blood cells. Failure to treat Aplastic Anemia is life-threatening. Aplastic Anemia is however a curable disease if the patient can receive a bone marrow transplant from a matched donor. (See Aplastic Anemia - Be The Match - Donate Marrow - Marrow.org)
During the initial stages of Aplastic Anemia, a common symptom is tiredness, but a person may not recognize any noticeable signs. As the disease progresses, the tiredness does, too. If labs including a complete blood count aren't run, the disease will continue without diagnosis.
As the disease progresses, blood stem cells in the bone marrow die, which drastically reduces the numbers of cells produced. The body recognizes the lack of cells, and reacts by producing blasts of immature cells that fail to meet the demands upon them. Tiredness turns to extreme exhaustion. As Neutrophils decline, so do the numbers of white cells, which results in vunerability to infection. The patient becomes neutropenic - unable to fight off infection.
If diagnosis hasn't been attempted up and until this point, an infection or extreme exhaustion often cause a patient to seek medical attention. A CBC will show the details of the progressing Aplastic Anemia. A regime of antibiotics, anti-viral, anti-fungal, and anti-bacterial drugs are prescribed quickly.
Very Severe Aplastic Anemia will reveal quick declining blood counts, which require transfusions to maintain life. If ATG or other immunosuppressant drugs haven't been attempted, they may be recommended. Alternatively, the recommendation may be for a bone marow transplant.
A bone marrow transplant is a transplant of blood stem cells taken from a closely-matched donor and infused into the patient. The blood stem cells make their way to the bone marrow, seat themselves in the soft boney tissue, grow and begin to produce blood cells (engraftment). Those cells are life.
A bone marrow transplant is a means by which patients with Aplastic Anemia are cured of the disease.
Do something really big: BE THE MATCH! Marrow.org.
During the initial stages of Aplastic Anemia, a common symptom is tiredness, but a person may not recognize any noticeable signs. As the disease progresses, the tiredness does, too. If labs including a complete blood count aren't run, the disease will continue without diagnosis.
As the disease progresses, blood stem cells in the bone marrow die, which drastically reduces the numbers of cells produced. The body recognizes the lack of cells, and reacts by producing blasts of immature cells that fail to meet the demands upon them. Tiredness turns to extreme exhaustion. As Neutrophils decline, so do the numbers of white cells, which results in vunerability to infection. The patient becomes neutropenic - unable to fight off infection.
If diagnosis hasn't been attempted up and until this point, an infection or extreme exhaustion often cause a patient to seek medical attention. A CBC will show the details of the progressing Aplastic Anemia. A regime of antibiotics, anti-viral, anti-fungal, and anti-bacterial drugs are prescribed quickly.
Very Severe Aplastic Anemia will reveal quick declining blood counts, which require transfusions to maintain life. If ATG or other immunosuppressant drugs haven't been attempted, they may be recommended. Alternatively, the recommendation may be for a bone marow transplant.
A bone marrow transplant is a transplant of blood stem cells taken from a closely-matched donor and infused into the patient. The blood stem cells make their way to the bone marrow, seat themselves in the soft boney tissue, grow and begin to produce blood cells (engraftment). Those cells are life.
A bone marrow transplant is a means by which patients with Aplastic Anemia are cured of the disease.
Do something really big: BE THE MATCH! Marrow.org.
Saturday, October 17, 2009
Misdiagnosed Aplastic Anemia
Hairy cell leukemia (HCL) is occasionally misdiagnosed as aplastic anemia when only a few leukemic cells are present in the circulation. Here, we describe a patient with HCL who initially presented with pancytopenia and received a diagnosis of aplastic anemia. The patient was treated with immunosuppressive therapy including cyclosporine A and anti-thymocyte globulin (ATG). No blood cell transfusion was required for approximately 3 years after ATG therapy. She was referred to our hospital because of an abdominal mass and requiring periodic blood transfusions. A bone marrow biopsy at this time revealed proliferation of lymphocytes with a fried egg appearance and an increase in reticulin fibers that are typical findings of HCL. It is notable that our patient with a presumably long history of HCL and an increase in marrow reticulin fibers showed good recovery of hematopoiesis after cladribine therapy. Some HCL patients may receive an initial diagnosis of aplastic anemia and may show a good response to ATG masking the underlying HCL.
http://www.ncbi.nlm.nih.gov/pubmed/19826898?ordinalpos=3&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_DefaultReportPanel.Pubmed_RVDocSum
http://www.ncbi.nlm.nih.gov/pubmed/19826898?ordinalpos=3&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_DefaultReportPanel.Pubmed_RVDocSum
Cyclosporine Warning
[Posted 07/14/2009]
The FDA is requiring the makers of certain immunosuppressant drugs [Sirolimus (Rapamune), Cyclosporine (Sandimmune and generics), Cyclosporine modified (Neoral and generics), Mycophenolate mofetil (Cellcept and generics), Mycophenolic acid (Myfortic)] to update their labeling to reflect that immunosuppressed patients are at increased risk for opportunistic infections, such as activation of latent viral infections, including BK virus-associated nephropathy. These immunosuppressant drugs are used to protect against the rejection of certain organ transplants. The association of BK virus-associated nephropathy has previously been reported for another immunosuppressant drug, tacrolimus (Prograf). Monitoring for this serious risk and early intervention by the health care provider is critical. Adjustments in immunosuppression therapy should be considered for patients who develop BK virus-associated nephropathy.
FDA is continuing to review the safety of immunosuppressant drug products used in renal transplantation. The FDA urges both healthcare professionals and patients to report side effects from the use of immunosuppressant drug products to the FDA's MedWatch Adverse Event Reporting program. For more information visit the FDA website at: http://www.fda.gov/Safety/MedWatch/SafetyInformation and http://www.fda.gov/Drugs/DrugSafety.
Important Warning:
Cyclosporine is available in its original form and as another product that has been modified (changed) so that the medication can be better absorbed in the body. Original cyclosporine and cyclosporine (modified) are absorbed by the body in different amounts, so they cannot be substituted for one another. Take only the type of cyclosporine that was prescribed by your doctor. When your doctor gives you a written prescription, check to be sure that he or she has specified the type of cyclosporine you should receive. Each time you have your prescription filled, look at the brand name printed on your prescription label to be sure that you have received the same type of cyclosporine. Talk to your pharmacist if the brand name is unfamiliar or you are not sure you have received the right type of cyclosporine.
Taking cyclosporine or cyclosporine (modified) may increase the risk that you will develop an infection or cancer, especially lymphoma (cancer of a part of the immune system) or skin cancer. This risk may be higher if you take cyclosporine or cyclosporine (modified) with other medications that decrease the functioning of the immune system such as azathioprine (Imuran), cancer chemotherapy, methotrexate (Rheumatrex), sirolimus (Rapamune), and tacrolimus (Prograf). Tell your doctor if you are taking any of these medications, and if you have or have ever had any type of cancer. To reduce your risk of skin cancer, plan to avoid unnecessary or prolonged exposure to sunlight and to wear protective clothing, sunglasses, and sunscreen during your treatment. If you experience any of the following symptoms, call your doctor immediately: sore throat, fever, chills, and other signs of infection; flu-like symptoms; coughing; difficulty urinating; pain when urinating; a red, raised, or swollen area on the skin; new sores or discoloration on the skin; lumps or masses anywhere in your body; night sweats; swollen glands in the neck, armpits, or groin; trouble breathing; chest pain; weakness or tiredness that does not go away; or pain, swelling, or fullness in the stomach.
Cyclosporine and cyclosporine (modified) may cause high blood pressure and kidney damage. Tell your doctor if you have or have ever had high blood pressure or kidney disease. Also tell your doctor if you are taking any of the following medications: amphotericin B (Amphotec, Fungizone); cimetidine (Tagamet); ciprofloxacin (Cipro); colchicine; fenofibrate (Antara, Lipophen, Tricor); gemfibrozil (Lopid); gentamicin; ketoconazole (Nizoral); melphalan (Alkeran); nonsteroidal anti-inflammatory drugs such as diclofenac (Cataflam, Voltaren), naproxen (Aleve, Naprosyn), and sulindac (Clinoril); ranitidine (Zantac); tobramycin (Tobi); trimethoprim with sulfamethoxazole (Bactrim, Septra); and vancomycin (Vancocin). If you experience any of the following symptoms, call your doctor immediately: dizziness; swelling of the arms, hands, feet, ankles, or lower legs; fast, shallow breathing; nausea; or irregular heartbeat.
If you have psoriasis, tell your doctor about all the psoriasis treatments and medications you are using or have used in the past. The risk that you will develop skin cancer is greater if you have ever been treated with PUVA (psoralen and UVA; treatment for psoriasis that combines an oral or topical medication with exposure to ultraviolet A light); methotrexate (Rheumatrex) or other medications that suppress the immune system; UVB (exposure to ultraviolet B light to treat psoriasis); coal tar; or radiation therapy. You should not be treated with PUVA, UVB, or medications that suppress the immune system while you are taking cyclosporine (modified) to treat psoriasis.
Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests to check your body's response to cyclosporine or cyclosporine (modified).
The FDA is requiring the makers of certain immunosuppressant drugs [Sirolimus (Rapamune), Cyclosporine (Sandimmune and generics), Cyclosporine modified (Neoral and generics), Mycophenolate mofetil (Cellcept and generics), Mycophenolic acid (Myfortic)] to update their labeling to reflect that immunosuppressed patients are at increased risk for opportunistic infections, such as activation of latent viral infections, including BK virus-associated nephropathy. These immunosuppressant drugs are used to protect against the rejection of certain organ transplants. The association of BK virus-associated nephropathy has previously been reported for another immunosuppressant drug, tacrolimus (Prograf). Monitoring for this serious risk and early intervention by the health care provider is critical. Adjustments in immunosuppression therapy should be considered for patients who develop BK virus-associated nephropathy.
FDA is continuing to review the safety of immunosuppressant drug products used in renal transplantation. The FDA urges both healthcare professionals and patients to report side effects from the use of immunosuppressant drug products to the FDA's MedWatch Adverse Event Reporting program. For more information visit the FDA website at: http://www.fda.gov/Safety/MedWatch/SafetyInformation and http://www.fda.gov/Drugs/DrugSafety.
Important Warning:
Cyclosporine is available in its original form and as another product that has been modified (changed) so that the medication can be better absorbed in the body. Original cyclosporine and cyclosporine (modified) are absorbed by the body in different amounts, so they cannot be substituted for one another. Take only the type of cyclosporine that was prescribed by your doctor. When your doctor gives you a written prescription, check to be sure that he or she has specified the type of cyclosporine you should receive. Each time you have your prescription filled, look at the brand name printed on your prescription label to be sure that you have received the same type of cyclosporine. Talk to your pharmacist if the brand name is unfamiliar or you are not sure you have received the right type of cyclosporine.
Taking cyclosporine or cyclosporine (modified) may increase the risk that you will develop an infection or cancer, especially lymphoma (cancer of a part of the immune system) or skin cancer. This risk may be higher if you take cyclosporine or cyclosporine (modified) with other medications that decrease the functioning of the immune system such as azathioprine (Imuran), cancer chemotherapy, methotrexate (Rheumatrex), sirolimus (Rapamune), and tacrolimus (Prograf). Tell your doctor if you are taking any of these medications, and if you have or have ever had any type of cancer. To reduce your risk of skin cancer, plan to avoid unnecessary or prolonged exposure to sunlight and to wear protective clothing, sunglasses, and sunscreen during your treatment. If you experience any of the following symptoms, call your doctor immediately: sore throat, fever, chills, and other signs of infection; flu-like symptoms; coughing; difficulty urinating; pain when urinating; a red, raised, or swollen area on the skin; new sores or discoloration on the skin; lumps or masses anywhere in your body; night sweats; swollen glands in the neck, armpits, or groin; trouble breathing; chest pain; weakness or tiredness that does not go away; or pain, swelling, or fullness in the stomach.
Cyclosporine and cyclosporine (modified) may cause high blood pressure and kidney damage. Tell your doctor if you have or have ever had high blood pressure or kidney disease. Also tell your doctor if you are taking any of the following medications: amphotericin B (Amphotec, Fungizone); cimetidine (Tagamet); ciprofloxacin (Cipro); colchicine; fenofibrate (Antara, Lipophen, Tricor); gemfibrozil (Lopid); gentamicin; ketoconazole (Nizoral); melphalan (Alkeran); nonsteroidal anti-inflammatory drugs such as diclofenac (Cataflam, Voltaren), naproxen (Aleve, Naprosyn), and sulindac (Clinoril); ranitidine (Zantac); tobramycin (Tobi); trimethoprim with sulfamethoxazole (Bactrim, Septra); and vancomycin (Vancocin). If you experience any of the following symptoms, call your doctor immediately: dizziness; swelling of the arms, hands, feet, ankles, or lower legs; fast, shallow breathing; nausea; or irregular heartbeat.
If you have psoriasis, tell your doctor about all the psoriasis treatments and medications you are using or have used in the past. The risk that you will develop skin cancer is greater if you have ever been treated with PUVA (psoralen and UVA; treatment for psoriasis that combines an oral or topical medication with exposure to ultraviolet A light); methotrexate (Rheumatrex) or other medications that suppress the immune system; UVB (exposure to ultraviolet B light to treat psoriasis); coal tar; or radiation therapy. You should not be treated with PUVA, UVB, or medications that suppress the immune system while you are taking cyclosporine (modified) to treat psoriasis.
Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests to check your body's response to cyclosporine or cyclosporine (modified).
Friday, October 16, 2009
TOP CANCER HOSPITALS WITH APLASTIC ANEMIA FOCUS
U. S. News publishes the top cancer hospitals list:
http://health.usnews.com/health/best-hospitals/cancer-hospital-rankings/
Mayo Clinic, Rochester Minnesota
http://health.usnews.com/health/best-hospitals/mayo-clinic-5000000/cancer
Reputation Score: 27.8%
Ranking: #4
Score: 59.6
University of Washington Medical Center, Seattle, Washington
http://health.usnews.com/health/best-hospitals/univ-of-washington-medical-ctr-6910750/cancer
Reputation Score: 18.7%
Ranking: #6
Score: 47.7
University of Massachusetts General Hospital, Boston, Massachusetts
http://health.usnews.com/health/best-hospitals/massachusetts-general-hospital-6140430/cancer
Reputation Score: 14.2%
Ranking: #7
Score: 43.8
How top hospitals selected
http://health.usnews.com/health/best-hospitals/cancer-hospital-rankings/
Mayo Clinic, Rochester Minnesota
http://health.usnews.com/health/best-hospitals/mayo-clinic-5000000/cancer
Reputation Score: 27.8%
Ranking: #4
Score: 59.6
University of Washington Medical Center, Seattle, Washington
http://health.usnews.com/health/best-hospitals/univ-of-washington-medical-ctr-6910750/cancer
Reputation Score: 18.7%
Ranking: #6
Score: 47.7
University of Massachusetts General Hospital, Boston, Massachusetts
http://health.usnews.com/health/best-hospitals/massachusetts-general-hospital-6140430/cancer
Reputation Score: 14.2%
Ranking: #7
Score: 43.8
How top hospitals selected
The Aplastic Anaemia–Paroxysmal Nocturnal Haemoglobinuria Syndrome
http://bloodjournal.hematologylibrary.org/cgi/reprint/48/1/63 (2007 article – the key to successful treatment according to the study is early transplant (within 17-100 days or original diagnosis)). Further reading evidences that siblings were matched for all participants in the study. Patients were given prednisone (10 mg sq m/day) to detect underlying leukemia. Cyclophosphamide was used alone for conditioning for engraftment. ATG or ATS and procarbazine (PCB) were given. After transplant, methotrexate was given to ameliorate potential graft-versus-host disease.
Summary
Study included 17 females transplanted who had idiopathic etiology (severe aplastic anemia from an unknown cause). Diagnosis was made within 17 weeks. Initial, PMN range was 0-1500 (avg 200), 1-23 platelets (avg. 6k), .1-1.4 retriculocytes (avg. .1%), and 40-99 nonmyeloid marrow cells (avg. 85%).
26 patients received CY alone, 10 CY plus ATG and PCB in preparation for engraftment.
Immunosuppression began within 12-97 days after diagnosis (avg. 33 days). Delays in treatments were due to INCOMPATIBLE DONORS, WAITING FOR AN AVAILABLE BED, DIAGNOSIS, INFORMED CONSENT (OF PATIENT AND FAMILY).
36 patients were to be transplanted; 2 died of sepsis during pretransplant immunosuppression; 34 patients were transplanted; 67% (24) survived with complete marrow restoration after 4-20 months (median 9 months). Of the 24, 2 are severely limited by GVHD; all others returned to regular activities within 3-12 months after transplantation.
No correlation was made to age, sex, etiology of aplasia, transplant cnter or transplant regimen.
Of the 34 engrafted, 10 died: 4 from infection, 2 from sepsis GVHD, 1 pneumonia and sepsis, 1 tuberculosis; 9 rejected graft (1 died before second transplant, 7 were retransplanted and of those 5 died for transplant failure and 2 were successfully engrafted).
Of the 24 that were successfully transplanted, 12 died. Of those, 8 rejected their grants within 4-5 weeks of transplant, 1 lost marrow activity during adenine arabinoside therapy begun 23 days post-transplant for disseminated herpes zoster, and 1 died before a second transplant could be attempted. 7 patients who rejected their initial grafts were re-transplanted; 2 were success.
31 patients were not transplanted. 7 improved (1 completely, 5 partially, 1 partially temporarily). Responses were seen within 1 to 3 months post treatment. Responses were positive in all three cell lines, but compared to transplant patients the responses were lesser. 19 patients died within 1-11 months due to 10 hemorrahaging, 7 from sepsis, and 2 from a combination of hemorrhage and sepsis.
Within 4 months, 67% of transplanted patients survived while only 36% of non-transplanted patients did so. Within 12 months, 75% of transplanted patients survived while only 22% of non-transplanted patients did so. At month 20, 75% of transplanted patients were still surviving while 1 non-transplanted patient survived, but has relapsed after 4 months and remains aplastic.
Notably, they say that the quick transplant choice of the patient played a key role in survival, and patients who were immediately transplanted without a prior non-transplant treatment failure clearly had much higher survival rates.
Study did not evaluate the role of intensive support in the management of severe aplastic anemia that is required during pretransplant treatment and during the 2-3 week period of the transplanted marrow beginning to function after transplant.
Some transplanted patients remained in protective environments and received oral non-absorbable antibiotics.
In the Seattle study of 23 aplastic anemia patients who survived more than 1 year after allogeneic mrrow transplantation, only one patient died, one has chronic GVHD, and one has almost completely recovered from chronic GVHD. The OTHER 20 PATIENTS ARE IN GOOD HEALTH WITH NORMAL MARROW FUNCTION AFTER 2 TO 4.5 YEARS POST TRANSPLANT.
STUDY SHOWS THAT PROMPT BONE MARROW TRANSPLANTATION SIGNIFICANTLY DECREASES EARLY MORTALITY OF SEVERE APLASTIC ANEMIA.
Cardeza Foundation, Philadelphia
Children’s Hospital, Boston
Children’s Hospital, Los Angeles
Children’s Hospital, Milwaukee
Children’s Hospital, Philadelphia
Children’s Hospital, St. Louis
Emory University, Atlanta
Hammersmith Hospital, London
Hematology-Oncology Associates PA.,
Plainfield
Hematology-Oncology Associates,
Providence
James Whitcomb Riley Hospital,
Indianapolis
J. HilIis Miller Health Center,
Gainesville
Johns Hopkins Oncology Center,
Baltimore
Milwaukee County General Hospital
Mount Sinai Hospital, New York City
Naval Medical Center, Bethesda
New Jersey Medical School, Newark
Peter Bent Brigham Hospital, Boston
Rhode Island Hospital, Providence
San Francisco Medical Center
St. Jude Children’s Research Hospital,
Memphis
UCLA
University of Capetown
University of Minnesota, Minneapolis
University of Kentucky, Lexington
University of Washington, Seattle
Yale University, New Haven
West Side V.A. Hospital, Chicago
A. Erslev
B. Camitta, D. Nathan,
R. Parkman
S. Siegel
J. Casper
S. Friedman, E. Schwartz
R. Ducos, A. Ragab,
H. Zarkowsky
T. Heffner, J. Keller
E.C. Gordon-Smith
D. Fnimmer
R. Damico
R. Baehner, A. Provisor
T. Dellinger
G. Santos
A. Pisciotta
R. Taub, R. Zalusky
R. Cahill, R. Hartzman,
K. Sell
T. Walters
J. Rappeport
E. Forman
J. Klock, S. Shohet
R. Strauss
S. Feig, R. Gale
D. Dubovsky, P. Jacobs
J. Kersey, W. Knivit
M. Greenwood, P. Holland
ED. Thomas, R. Storb
S. McIntosh, R. O’Brien
R. Epstein, W. Fried
USPHS FR-00l28
USPHS AM-05581
Tommy Fund
USPHS 5-M0l-RR-00888
USPHS CA-l7995
ALSAC
USPHS CA-12800-
RR00865
Minnesota Medical
Foundation
SMF-155-75
USPHS CA-10895
Summary
Study included 17 females transplanted who had idiopathic etiology (severe aplastic anemia from an unknown cause). Diagnosis was made within 17 weeks. Initial, PMN range was 0-1500 (avg 200), 1-23 platelets (avg. 6k), .1-1.4 retriculocytes (avg. .1%), and 40-99 nonmyeloid marrow cells (avg. 85%).
26 patients received CY alone, 10 CY plus ATG and PCB in preparation for engraftment.
Immunosuppression began within 12-97 days after diagnosis (avg. 33 days). Delays in treatments were due to INCOMPATIBLE DONORS, WAITING FOR AN AVAILABLE BED, DIAGNOSIS, INFORMED CONSENT (OF PATIENT AND FAMILY).
36 patients were to be transplanted; 2 died of sepsis during pretransplant immunosuppression; 34 patients were transplanted; 67% (24) survived with complete marrow restoration after 4-20 months (median 9 months). Of the 24, 2 are severely limited by GVHD; all others returned to regular activities within 3-12 months after transplantation.
No correlation was made to age, sex, etiology of aplasia, transplant cnter or transplant regimen.
Of the 34 engrafted, 10 died: 4 from infection, 2 from sepsis GVHD, 1 pneumonia and sepsis, 1 tuberculosis; 9 rejected graft (1 died before second transplant, 7 were retransplanted and of those 5 died for transplant failure and 2 were successfully engrafted).
Of the 24 that were successfully transplanted, 12 died. Of those, 8 rejected their grants within 4-5 weeks of transplant, 1 lost marrow activity during adenine arabinoside therapy begun 23 days post-transplant for disseminated herpes zoster, and 1 died before a second transplant could be attempted. 7 patients who rejected their initial grafts were re-transplanted; 2 were success.
31 patients were not transplanted. 7 improved (1 completely, 5 partially, 1 partially temporarily). Responses were seen within 1 to 3 months post treatment. Responses were positive in all three cell lines, but compared to transplant patients the responses were lesser. 19 patients died within 1-11 months due to 10 hemorrahaging, 7 from sepsis, and 2 from a combination of hemorrhage and sepsis.
Within 4 months, 67% of transplanted patients survived while only 36% of non-transplanted patients did so. Within 12 months, 75% of transplanted patients survived while only 22% of non-transplanted patients did so. At month 20, 75% of transplanted patients were still surviving while 1 non-transplanted patient survived, but has relapsed after 4 months and remains aplastic.
Notably, they say that the quick transplant choice of the patient played a key role in survival, and patients who were immediately transplanted without a prior non-transplant treatment failure clearly had much higher survival rates.
Study did not evaluate the role of intensive support in the management of severe aplastic anemia that is required during pretransplant treatment and during the 2-3 week period of the transplanted marrow beginning to function after transplant.
Some transplanted patients remained in protective environments and received oral non-absorbable antibiotics.
In the Seattle study of 23 aplastic anemia patients who survived more than 1 year after allogeneic mrrow transplantation, only one patient died, one has chronic GVHD, and one has almost completely recovered from chronic GVHD. The OTHER 20 PATIENTS ARE IN GOOD HEALTH WITH NORMAL MARROW FUNCTION AFTER 2 TO 4.5 YEARS POST TRANSPLANT.
STUDY SHOWS THAT PROMPT BONE MARROW TRANSPLANTATION SIGNIFICANTLY DECREASES EARLY MORTALITY OF SEVERE APLASTIC ANEMIA.
Cardeza Foundation, Philadelphia
Children’s Hospital, Boston
Children’s Hospital, Los Angeles
Children’s Hospital, Milwaukee
Children’s Hospital, Philadelphia
Children’s Hospital, St. Louis
Emory University, Atlanta
Hammersmith Hospital, London
Hematology-Oncology Associates PA.,
Plainfield
Hematology-Oncology Associates,
Providence
James Whitcomb Riley Hospital,
Indianapolis
J. HilIis Miller Health Center,
Gainesville
Johns Hopkins Oncology Center,
Baltimore
Milwaukee County General Hospital
Mount Sinai Hospital, New York City
Naval Medical Center, Bethesda
New Jersey Medical School, Newark
Peter Bent Brigham Hospital, Boston
Rhode Island Hospital, Providence
San Francisco Medical Center
St. Jude Children’s Research Hospital,
Memphis
UCLA
University of Capetown
University of Minnesota, Minneapolis
University of Kentucky, Lexington
University of Washington, Seattle
Yale University, New Haven
West Side V.A. Hospital, Chicago
A. Erslev
B. Camitta, D. Nathan,
R. Parkman
S. Siegel
J. Casper
S. Friedman, E. Schwartz
R. Ducos, A. Ragab,
H. Zarkowsky
T. Heffner, J. Keller
E.C. Gordon-Smith
D. Fnimmer
R. Damico
R. Baehner, A. Provisor
T. Dellinger
G. Santos
A. Pisciotta
R. Taub, R. Zalusky
R. Cahill, R. Hartzman,
K. Sell
T. Walters
J. Rappeport
E. Forman
J. Klock, S. Shohet
R. Strauss
S. Feig, R. Gale
D. Dubovsky, P. Jacobs
J. Kersey, W. Knivit
M. Greenwood, P. Holland
ED. Thomas, R. Storb
S. McIntosh, R. O’Brien
R. Epstein, W. Fried
USPHS FR-00l28
USPHS AM-05581
Tommy Fund
USPHS 5-M0l-RR-00888
USPHS CA-l7995
ALSAC
USPHS CA-12800-
RR00865
Minnesota Medical
Foundation
SMF-155-75
USPHS CA-10895
Thursday, October 15, 2009
Clinical Definition of Severe Aplastic Anemia
Severe Aplastic Anemia is defined as follows:
Bone marrow cellularity < 25%, or marrow cellularity < 50% but with < 30% residual hematopoietic cells.
Two out of three of the following (in peripheral blood): neutrophils < 0.5 x 10^9/L; platelets < 20 x 10^9/L; reticulocytes < 20 x 10^9/L. SAA diagnostic criteria may be applied to assessment at initial diagnosis or to the follow-up assessments.
Bone marrow cellularity < 25%, or marrow cellularity < 50% but with < 30% residual hematopoietic cells.
Two out of three of the following (in peripheral blood): neutrophils < 0.5 x 10^9/L; platelets < 20 x 10^9/L; reticulocytes < 20 x 10^9/L. SAA diagnostic criteria may be applied to assessment at initial diagnosis or to the follow-up assessments.
Aplastic Anemia References
Ades L, Mary JY, Robin M, et al. Long-term outcome after bone marrow transplantation for severe aplastic anemia. Blood. 2004;103:2490-2497.
Bagby GC, Liptin JM, Sloand EM, Schiffer CA. Marrow failure. In Broudy VC, Berliner N, Larson RA, Leung LL, eds. Hematology 2004. American Society of Hematology Education Program Book. 318-336. Accessed at www.asheducationbook.org on June 20, 2005.
Broksky RA, Jones RJ. Aplastic anemia. Lancet 2005;365:1647-1656.
Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003; 289:1130-1135.
Young NS. Acquired aplastic anemia. Ann Intern Med 2002;136:534-546.
Young NS, Maciejewski J. The pathophysiology of acquired aplastic anemia. N Engl J Med. 1997;336:1365-1372.
Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006 Oct 15;108:2509-2519.
Young NS, Maciejewski JP. Aplastic anemia. In: Hematology: Basic Principles and Practice. 5th edition. Hoffman R, Benz E Jr, Shattil S, Furie B, Silberstein L, McClave P, and Heslop H, editors. Philadelphia, PA. Elsevier, 2009: 359-383.
Brodsky RA. New insights into paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program. 2006;24-8, 516.
Teramura M, Kimura A, Iwase S, Yonemura Y, Nakao S, Urabe A, Omine M, Mizoguchi H. Treatment of severe aplastic anemia with antithymocyte globulin and cyclosporin A with or without G-CSF in adults: a multicenter randomized study in Japan. Blood. 2007;110:1756-1761.
Castro-Malaspina, Hugo, and Richard J. O'Reilly. "Aplastic Anemia and Myelodysplastic Syndromes." In Harrison's Principles of Internal Medicine, ed. Anthony S. Fauci, et al. New York: McGraw-Hill, 1997.
Doney, Kristine, et al. "Primary Treatment of Acquired Aplastic Anemia: Outcomes with Bone Marrow Transplantation and Immunosuppressive Therapy." Annals of Internal Medicine 126 (15 Jan. 1997): 107+.
Young, Neal. "Aplastic Anaemia." The Lancet 346 (22 July 1995): 228+.
Young, Neal, and Jaroslaw Maciejewski. "The Pathophysiology of Acquired Aplastic Anemia." The New England Journal of Medicine 336 (8 May 1997): 1365+.
Aplastic Anemia Foundation of America. P.O. Box 613, Annapolis, MD 21404. (800) 747-2820. http://www.aplastic.org/.
http://bloodjournal.hematologylibrary.org/
Bagby GC, Liptin JM, Sloand EM, Schiffer CA. Marrow failure. In Broudy VC, Berliner N, Larson RA, Leung LL, eds. Hematology 2004. American Society of Hematology Education Program Book. 318-336. Accessed at www.asheducationbook.org on June 20, 2005.
Broksky RA, Jones RJ. Aplastic anemia. Lancet 2005;365:1647-1656.
Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003; 289:1130-1135.
Young NS. Acquired aplastic anemia. Ann Intern Med 2002;136:534-546.
Young NS, Maciejewski J. The pathophysiology of acquired aplastic anemia. N Engl J Med. 1997;336:1365-1372.
Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006 Oct 15;108:2509-2519.
Young NS, Maciejewski JP. Aplastic anemia. In: Hematology: Basic Principles and Practice. 5th edition. Hoffman R, Benz E Jr, Shattil S, Furie B, Silberstein L, McClave P, and Heslop H, editors. Philadelphia, PA. Elsevier, 2009: 359-383.
Brodsky RA. New insights into paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program. 2006;24-8, 516.
Teramura M, Kimura A, Iwase S, Yonemura Y, Nakao S, Urabe A, Omine M, Mizoguchi H. Treatment of severe aplastic anemia with antithymocyte globulin and cyclosporin A with or without G-CSF in adults: a multicenter randomized study in Japan. Blood. 2007;110:1756-1761.
Castro-Malaspina, Hugo, and Richard J. O'Reilly. "Aplastic Anemia and Myelodysplastic Syndromes." In Harrison's Principles of Internal Medicine, ed. Anthony S. Fauci, et al. New York: McGraw-Hill, 1997.
Doney, Kristine, et al. "Primary Treatment of Acquired Aplastic Anemia: Outcomes with Bone Marrow Transplantation and Immunosuppressive Therapy." Annals of Internal Medicine 126 (15 Jan. 1997): 107+.
Young, Neal. "Aplastic Anaemia." The Lancet 346 (22 July 1995): 228+.
Young, Neal, and Jaroslaw Maciejewski. "The Pathophysiology of Acquired Aplastic Anemia." The New England Journal of Medicine 336 (8 May 1997): 1365+.
Aplastic Anemia Foundation of America. P.O. Box 613, Annapolis, MD 21404. (800) 747-2820. http://www.aplastic.org/.
http://bloodjournal.hematologylibrary.org/
Research In Treatments of Aplastic Anemia
In addition to bone marrow transplants (the preferred method of treatment), supportive treatments and immune system suppression, advancements are being made in studies of the immune system to treat aplastic anemia.
Researchers are working to improve the drugs used for immunosuppression and stem cell transplantation. The goal is to find treatments that have fewer side effects than the present treatments with better results.
Studies Into Acquired (or Secondary) Aplastic Anemia
One drug under study is called daclizumab (Zenapax®). This is a monoclonal antibody (a laboratory-made antibody that attaches to specific substances) that blocks the action of interleukin-2 (IL-2). IL-2 is a normal part of the immune system that stimulates the immune response. Blocking IL-2 suppresses the immune system in a way different from ATG. Allergic reactions to this drug are rare. It is currently being used as a part of stem cell transplants, and is under study as a treatment for aplastic anemia.
Stem Cell Research To Treat Aplastic Anemia
Doctors are also trying to make stem cell transplants safer and more available. One approach is to use stem cells that come from the umbilical cord blood of newborns. This is a very rich source of stem cells. Efforts are being made nationwide to develop storage facilities for cord blood. The advantage of cord blood stem cells is that they may lead to less graft-versus-host disease. The disadvantage is that there may be too few cells to successfully "take." In one case, doctors have overcome this by transplanting cord blood stem cells from 2 separate donors. This has proven successful and may have an even lower risk of graft versus host disease. This method is currently under study.
Research for Inherited Aplastic Anemia
Researchers are also studying the possibility of using gene therapy to treat some inherited forms of aplastic anemia. Another approach is to give a drug called amifostine to help protect the bone marrow in children with Fanconi anemia.
Researchers are working to improve the drugs used for immunosuppression and stem cell transplantation. The goal is to find treatments that have fewer side effects than the present treatments with better results.
Studies Into Acquired (or Secondary) Aplastic Anemia
One drug under study is called daclizumab (Zenapax®). This is a monoclonal antibody (a laboratory-made antibody that attaches to specific substances) that blocks the action of interleukin-2 (IL-2). IL-2 is a normal part of the immune system that stimulates the immune response. Blocking IL-2 suppresses the immune system in a way different from ATG. Allergic reactions to this drug are rare. It is currently being used as a part of stem cell transplants, and is under study as a treatment for aplastic anemia.
Stem Cell Research To Treat Aplastic Anemia
Doctors are also trying to make stem cell transplants safer and more available. One approach is to use stem cells that come from the umbilical cord blood of newborns. This is a very rich source of stem cells. Efforts are being made nationwide to develop storage facilities for cord blood. The advantage of cord blood stem cells is that they may lead to less graft-versus-host disease. The disadvantage is that there may be too few cells to successfully "take." In one case, doctors have overcome this by transplanting cord blood stem cells from 2 separate donors. This has proven successful and may have an even lower risk of graft versus host disease. This method is currently under study.
Research for Inherited Aplastic Anemia
Researchers are also studying the possibility of using gene therapy to treat some inherited forms of aplastic anemia. Another approach is to give a drug called amifostine to help protect the bone marrow in children with Fanconi anemia.
When Transplant Treatments Fail
Sometimes the first treatment isn't successful and will need to be repeated. Often the second treatment will be effective, even if the first wasn't. Also, the treatment may be only partially effective, and the blood counts may not return to normal. In this situation, many people can still lead a normal life and not need treatment. Their blood counts, although not normal, will be high enough for a normal life.
Aplastic Anemia Treatment
This information represents the views of the doctors and nurses serving on the American Cancer Society's Cancer Information Database Editorial Board. These views are based on their interpretation of studies published in medical journals, as well as their own professional experience.
The treatment information in this document is not official policy of the Society and is not intended as medical advice to replace the expertise and judgment of your cancer care team. It is intended to help you and your family make informed decisions, together with your doctor.
Your doctor may have reasons for suggesting a treatment plan different from these general treatment options. Don't hesitate to ask him or her questions about your treatment options.
Treatment for aplastic anemia is either primary or supportive. Primary treatment is aimed at curing the disease. Common primary treatments are stem cell transplant and immune suppression. Supportive treatment is aimed at helping the symptoms of aplastic anemia without actually trying to cure it. Treating anemia with transfusions is a type of supportive treatment.
Primary treatment
Allogeneic stem cell transplant
Allogeneic stem cell transplant is considered the best treatment for younger people with aplastic anemia. Transplantation works best in children and young adults. As people get older, it's harder for them to tolerate this procedure. For people older than 30 or 40, many doctors prefer using immune therapy as the first treatment. In order for transplant to be an option, you need to have a donor who is matched to you. Someone who is closely related to you, like a brother or sister, is generally the best choice. In aplastic anemia, this stem cell transplant is successful up to 80% to 90% of the time if cells from a matched related donor are used.
Matching is determined by a type of test called HLA typing, which is done in the laboratory. If there is no match from a brother or sister, sometimes an unrelated donor will match. A transplant from a matched-unrelated donor is riskier than using a sibling match. Another option is to use stem cells from the umbilical cord of a newborn baby. A nationwide registry of potential stem cell donors and stored umbilical cord blood has been developed (www.marrow.org). Many doctors recommend using a non-related donor only if immune treatments described below don't work.
For a stem cell transplant, you will first receive chemotherapy. The chemotherapy drugs used most often include cyclophosphamide (Cytoxan®) and fludarabine (Fludara®). A drug to suppress your immune system, like anti-thymocyte globulin (ATG) or alemtuzumab (Campath®), is also given. A newer drug, daclizumab (Zenapax®), may also be used. This immune treatment is important to keep your immune system from killing the new bone marrow (like it killed your original bone marrow). Immune treatment is also important to prevent the new bone marrow from attacking your body (this is called graft-versus-host disease and is discussed later in this section). You may also get a low dose of radiation therapy to your whole body.
Soon after the radiation and/or chemotherapy, you will receive a transfusion with blood-forming stem cells from a donor. The stem cells can be obtained from the donor by removing bone marrow in the operating room while he or she is under general anesthesia. Sometimes a procedure called apheresis is used to remove stem cells from the bloodstream. Apheresis uses a machine that is hooked up to a large vein (through a catheter) and removes stem cells from the blood (returning the other cells). No matter which way they are collected, the stem cells are infused through your vein into the blood and then travel to the bone marrow, where they will grow
Stem cell transplantation is a major procedure with many risks and side effects. Some people may die during this procedure. The most serious side effects often occur during the first few weeks after the transplant. But with advances in this treatment, death from these early side effects is less common. The chance of severe side effects increases with the age of the patient.
In the first few weeks after the transplant there are usually side effects from the chemotherapy or radiation therapy. Very low blood counts requiring red blood cell and platelet transfusions are common. You may develop nausea, vomiting, diarrhea, and mouth sores from the treatment. Serious infections can also occur and are treated with large doses of antibiotics. These all generally go away in 3 to 4 weeks when the transplanted blood-forming stem cells start to produce normal blood cells. If ATG is used, there is also a risk of serious allergic reactions during the infusion. These reactions can range from skin rashes to low blood pressure and problems breathing. Generally, these side effects can be controlled with medicines. This is discussed in more detail in the next section.
A very serious side effect of a stem cell transplant is graft-versus-host disease (GVHD). This occurs when the transplanted donor cells attack your own cells through an immune reaction. GVHD is more common with unrelated or mismatched donors. The risk of GVHD is also higher in older patients. GVHD can occur very early after the transplant. This is called acute GVHD. When GVHD develops later on or lasts a long time it is called chronic GVHD. Early signs of GVHD include skin rashes with severe itching and bowel disturbances such as diarrhea. There may be abnormal lab tests showing liver damage. GVHD is treated with medications (like prednisone, methotrexate, or tacrolimus) to try to suppress the immune system. In some cases, GVHD goes away and the medications can be stopped. In other cases, GVHD is only partly controlled, and medications are needed for a long time. GVHD can sometimes be very disabling and occasionally even cause death.
For more information, please see our document, Bone Marrow and Peripheral Blood Stem Cell Transplants.
Immunosuppressive therapy
If you are not able to have a transplant (because of age or because you do not have a matched donor), doctors will recommend immunosuppressive treatment. Most cases of acquired aplastic anemia are caused by the immune system attacking the bone marrow. This treatment helps stop the immune system from killing the bone marrow cells. This type of treatment is not usually helpful for cases of inherited aplastic anemia because they are not caused by the immune system.
The major drugs used are antithymocyte globulin (ATG) and cyclosporine. ATG contains antibodies against human T-lymphocytes. This medicine is given in the hospital through an intravenous (IV) line. ATG decreases (suppresses) your immune system function by lowering the number of T-cells in the body. The antibodies in ATG come from an animal (like a horse or a rabbit), so there is a risk of a serious allergic reaction when the ATG is given. Sometimes patients getting ATG also receive a corticosteroid medicine (like prednisone) to reduce the chance that a serious reaction will occur. A newer drug, alemtuzumab (Campath®), is sometimes used instead of ATG. It has a lower risk of serious allergic reactions, but doctors aren't sure that it works as well as ATG. Often the drug called cyclosporine is given as well. This drug suppresses the immune system. Combining ATG and cyclosporine improves the blood counts in about 70% of patients with the most severe disease. The aplastic anemia may not actually be cured in most of these patients. Still, even when the blood counts do not become completely normal, they often improve enough for the patient to feel well and live a normal life. Often, after a period of remission, the aplastic anemia will come back. Usually, it will respond again to immune treatment, which can be safely repeated.
Some doctors now also give a drug called G-CSF along with ATG and cyclosporine. This drug is a growth factor for white blood cells -- it tells the bone marrow to make more of these cells. It is also known as filgrastim or Neupogen. Studies looking to see if G-CSF is helpful when given with immune therapy are ongoing.
Using high doses of cyclophosphamide (Cytoxan®) for immunosuppression is controversial. Cyclophosphamide is a chemotherapy drug that can suppress the immune system and damage T-lymphocytes. Although this treatment can be effective, many experts believe that it is more dangerous than ATG. Most doctors in the United States prefer to delay using cyclophosphamide until ATG and cyclosporine are no longer working.
Immunosuppressive therapy can have serious side effects. Holding back the immune system impairs the body's ability to fight infection. People on immunosuppression can get life-threatening infections with bacteria, viruses, and fungi. The drugs used in this therapy also have serious side effects. For example, ATG can cause serious allergic reactions with symptoms including skin rashes, low blood pressure, and problems breathing. Generally, these side effects can be controlled with medicines. Also, about 15% of patients develop leukemia or myelodysplasia several years after getting ATG. Myelodysplasia is a disorder of the bone marrow that is similar to leukemia (see the American Cancer Society document, Myelodysplastic Syndromes). Side effects of cyclosporine include high blood pressure as well as kidney and liver damage. To help prevent these problems, the doctor will check the level of cyclosporine in the blood regularly. Blood tests will also be done to check kidney and liver function. Corticosteroids (like prednisone) can cause increases in blood sugar (like diabetes), high blood pressure, weight gain, changes in mood, and weak bones.
Supportive treatment
Transfusion
While your blood counts are low, you may need transfusions of red blood cells or platelets. Although blood transfusions are generally safe, their long-term use creates problems. The main problem is that the red cells contain iron. The transfused cells only live a few weeks and iron from these cells builds up in the body. Eventually this will result in a high level of body iron, which is toxic. Although this can be treated with drugs, these aren't always successful and some organs, mainly the liver and heart, can become damaged. Also, getting tranfusions before a stem cell transplant increases the chance that the transplant won't work. This is why doctors avoid giving transfusions when a transplant is planned.
Antibiotics
Because of your low white blood cell count, you may develop an infection and need antibiotics. Infection is the major cause of death from aplastic anemia. Treatment with antibiotics should be started as soon as infection is suspected. Transfusing white blood cells is not generally practical. They survive only a very short time, and it is not possible to get enough white blood cells from normal donors to raise a person's white blood cell count.
Growth factors
Drugs can be given to increase the white blood cell count. These are called filgrastim (also called G-CSF or Neupogen®), pegfilgrastim (Neulasta®), and sargramostim (also called GM-CSF or Leukine®). These work only slightly for most patients with aplastic anemia.
Androgens
Some patients with early or mild aplastic anemia can be treated with androgens (instead of stem cell transplantation or ATG). Androgens are often the first treatment given to patients with inherited forms of aplastic anemia (like Fanconi anemia and dyskeratosis congenita). Androgens are male sex hormones that also stimulate blood production. They are the reason that men have higher red blood cell counts than do women. Although they can be effective in improving blood counts, they do not cure aplastic anemia. Also, using androgens for a long time has been linked to liver tumors and liver cancer. Androgens are male hormones, so women taking this medicine can develop masculine characteristics such as facial hair, balding, deepening voice, etc.
General considerations in treating aplastic anemia
Many years ago, aplastic anemia was considered a fatal disease unless the patient recovered without treatment (spontaneously). Now that we understand this is often an immune disease, treatments have been devised that have a high success rate.
Children with inherited causes of aplastic anemia are treated with supportive therapy (such as androgens) and stem cell transplant. Immunosuppressants don't help these patients.
Those with acquired aplastic anemia are treated with supportive care, followed by immunesuppressant therapy or stem cell transplant. Stem cell transplant is preferred when the patient has a matched, related donor, and is young and otherwise healthy. Immune treatments may be the first option for older patients and those without a matched donor.
A small number of people with aplastic anemia will develop leukemia or other cancers after several years. But it isn't clear if this is caused by the treatment, or just part of the disease itself. For example, many people with Fanconi anemia who don't get aplastic anemia, will still go on to develop leukemia.
Aplastic anemia is rare, and treating it is very specialized, so it may best be done at a major medical center where they have experience with this disease. This is particularly true for stem cell transplants.
Sometimes the first treatment isn't successful and will need to be repeated. Often the second treatment will be effective, even if the first wasn't. Also, the treatment may be only partially effective, and the blood counts may not return to normal. In this situation, many people can still lead a normal life and not need treatment. Their blood counts, although not normal, will be high enough for a normal life.
Sometimes, none of the treatments is successful. In this case a person with aplastic anemia might want to consider taking part in a clinical trial, that is, a study of a new therapy that hasn't yet been proven successful.
Adapted in part or whole from the American Cancer Society website.
The treatment information in this document is not official policy of the Society and is not intended as medical advice to replace the expertise and judgment of your cancer care team. It is intended to help you and your family make informed decisions, together with your doctor.
Your doctor may have reasons for suggesting a treatment plan different from these general treatment options. Don't hesitate to ask him or her questions about your treatment options.
Treatment for aplastic anemia is either primary or supportive. Primary treatment is aimed at curing the disease. Common primary treatments are stem cell transplant and immune suppression. Supportive treatment is aimed at helping the symptoms of aplastic anemia without actually trying to cure it. Treating anemia with transfusions is a type of supportive treatment.
Primary treatment
Allogeneic stem cell transplant
Allogeneic stem cell transplant is considered the best treatment for younger people with aplastic anemia. Transplantation works best in children and young adults. As people get older, it's harder for them to tolerate this procedure. For people older than 30 or 40, many doctors prefer using immune therapy as the first treatment. In order for transplant to be an option, you need to have a donor who is matched to you. Someone who is closely related to you, like a brother or sister, is generally the best choice. In aplastic anemia, this stem cell transplant is successful up to 80% to 90% of the time if cells from a matched related donor are used.
Matching is determined by a type of test called HLA typing, which is done in the laboratory. If there is no match from a brother or sister, sometimes an unrelated donor will match. A transplant from a matched-unrelated donor is riskier than using a sibling match. Another option is to use stem cells from the umbilical cord of a newborn baby. A nationwide registry of potential stem cell donors and stored umbilical cord blood has been developed (www.marrow.org). Many doctors recommend using a non-related donor only if immune treatments described below don't work.
For a stem cell transplant, you will first receive chemotherapy. The chemotherapy drugs used most often include cyclophosphamide (Cytoxan®) and fludarabine (Fludara®). A drug to suppress your immune system, like anti-thymocyte globulin (ATG) or alemtuzumab (Campath®), is also given. A newer drug, daclizumab (Zenapax®), may also be used. This immune treatment is important to keep your immune system from killing the new bone marrow (like it killed your original bone marrow). Immune treatment is also important to prevent the new bone marrow from attacking your body (this is called graft-versus-host disease and is discussed later in this section). You may also get a low dose of radiation therapy to your whole body.
Soon after the radiation and/or chemotherapy, you will receive a transfusion with blood-forming stem cells from a donor. The stem cells can be obtained from the donor by removing bone marrow in the operating room while he or she is under general anesthesia. Sometimes a procedure called apheresis is used to remove stem cells from the bloodstream. Apheresis uses a machine that is hooked up to a large vein (through a catheter) and removes stem cells from the blood (returning the other cells). No matter which way they are collected, the stem cells are infused through your vein into the blood and then travel to the bone marrow, where they will grow
Stem cell transplantation is a major procedure with many risks and side effects. Some people may die during this procedure. The most serious side effects often occur during the first few weeks after the transplant. But with advances in this treatment, death from these early side effects is less common. The chance of severe side effects increases with the age of the patient.
In the first few weeks after the transplant there are usually side effects from the chemotherapy or radiation therapy. Very low blood counts requiring red blood cell and platelet transfusions are common. You may develop nausea, vomiting, diarrhea, and mouth sores from the treatment. Serious infections can also occur and are treated with large doses of antibiotics. These all generally go away in 3 to 4 weeks when the transplanted blood-forming stem cells start to produce normal blood cells. If ATG is used, there is also a risk of serious allergic reactions during the infusion. These reactions can range from skin rashes to low blood pressure and problems breathing. Generally, these side effects can be controlled with medicines. This is discussed in more detail in the next section.
A very serious side effect of a stem cell transplant is graft-versus-host disease (GVHD). This occurs when the transplanted donor cells attack your own cells through an immune reaction. GVHD is more common with unrelated or mismatched donors. The risk of GVHD is also higher in older patients. GVHD can occur very early after the transplant. This is called acute GVHD. When GVHD develops later on or lasts a long time it is called chronic GVHD. Early signs of GVHD include skin rashes with severe itching and bowel disturbances such as diarrhea. There may be abnormal lab tests showing liver damage. GVHD is treated with medications (like prednisone, methotrexate, or tacrolimus) to try to suppress the immune system. In some cases, GVHD goes away and the medications can be stopped. In other cases, GVHD is only partly controlled, and medications are needed for a long time. GVHD can sometimes be very disabling and occasionally even cause death.
For more information, please see our document, Bone Marrow and Peripheral Blood Stem Cell Transplants.
Immunosuppressive therapy
If you are not able to have a transplant (because of age or because you do not have a matched donor), doctors will recommend immunosuppressive treatment. Most cases of acquired aplastic anemia are caused by the immune system attacking the bone marrow. This treatment helps stop the immune system from killing the bone marrow cells. This type of treatment is not usually helpful for cases of inherited aplastic anemia because they are not caused by the immune system.
The major drugs used are antithymocyte globulin (ATG) and cyclosporine. ATG contains antibodies against human T-lymphocytes. This medicine is given in the hospital through an intravenous (IV) line. ATG decreases (suppresses) your immune system function by lowering the number of T-cells in the body. The antibodies in ATG come from an animal (like a horse or a rabbit), so there is a risk of a serious allergic reaction when the ATG is given. Sometimes patients getting ATG also receive a corticosteroid medicine (like prednisone) to reduce the chance that a serious reaction will occur. A newer drug, alemtuzumab (Campath®), is sometimes used instead of ATG. It has a lower risk of serious allergic reactions, but doctors aren't sure that it works as well as ATG. Often the drug called cyclosporine is given as well. This drug suppresses the immune system. Combining ATG and cyclosporine improves the blood counts in about 70% of patients with the most severe disease. The aplastic anemia may not actually be cured in most of these patients. Still, even when the blood counts do not become completely normal, they often improve enough for the patient to feel well and live a normal life. Often, after a period of remission, the aplastic anemia will come back. Usually, it will respond again to immune treatment, which can be safely repeated.
Some doctors now also give a drug called G-CSF along with ATG and cyclosporine. This drug is a growth factor for white blood cells -- it tells the bone marrow to make more of these cells. It is also known as filgrastim or Neupogen. Studies looking to see if G-CSF is helpful when given with immune therapy are ongoing.
Using high doses of cyclophosphamide (Cytoxan®) for immunosuppression is controversial. Cyclophosphamide is a chemotherapy drug that can suppress the immune system and damage T-lymphocytes. Although this treatment can be effective, many experts believe that it is more dangerous than ATG. Most doctors in the United States prefer to delay using cyclophosphamide until ATG and cyclosporine are no longer working.
Immunosuppressive therapy can have serious side effects. Holding back the immune system impairs the body's ability to fight infection. People on immunosuppression can get life-threatening infections with bacteria, viruses, and fungi. The drugs used in this therapy also have serious side effects. For example, ATG can cause serious allergic reactions with symptoms including skin rashes, low blood pressure, and problems breathing. Generally, these side effects can be controlled with medicines. Also, about 15% of patients develop leukemia or myelodysplasia several years after getting ATG. Myelodysplasia is a disorder of the bone marrow that is similar to leukemia (see the American Cancer Society document, Myelodysplastic Syndromes). Side effects of cyclosporine include high blood pressure as well as kidney and liver damage. To help prevent these problems, the doctor will check the level of cyclosporine in the blood regularly. Blood tests will also be done to check kidney and liver function. Corticosteroids (like prednisone) can cause increases in blood sugar (like diabetes), high blood pressure, weight gain, changes in mood, and weak bones.
Supportive treatment
Transfusion
While your blood counts are low, you may need transfusions of red blood cells or platelets. Although blood transfusions are generally safe, their long-term use creates problems. The main problem is that the red cells contain iron. The transfused cells only live a few weeks and iron from these cells builds up in the body. Eventually this will result in a high level of body iron, which is toxic. Although this can be treated with drugs, these aren't always successful and some organs, mainly the liver and heart, can become damaged. Also, getting tranfusions before a stem cell transplant increases the chance that the transplant won't work. This is why doctors avoid giving transfusions when a transplant is planned.
Antibiotics
Because of your low white blood cell count, you may develop an infection and need antibiotics. Infection is the major cause of death from aplastic anemia. Treatment with antibiotics should be started as soon as infection is suspected. Transfusing white blood cells is not generally practical. They survive only a very short time, and it is not possible to get enough white blood cells from normal donors to raise a person's white blood cell count.
Growth factors
Drugs can be given to increase the white blood cell count. These are called filgrastim (also called G-CSF or Neupogen®), pegfilgrastim (Neulasta®), and sargramostim (also called GM-CSF or Leukine®). These work only slightly for most patients with aplastic anemia.
Androgens
Some patients with early or mild aplastic anemia can be treated with androgens (instead of stem cell transplantation or ATG). Androgens are often the first treatment given to patients with inherited forms of aplastic anemia (like Fanconi anemia and dyskeratosis congenita). Androgens are male sex hormones that also stimulate blood production. They are the reason that men have higher red blood cell counts than do women. Although they can be effective in improving blood counts, they do not cure aplastic anemia. Also, using androgens for a long time has been linked to liver tumors and liver cancer. Androgens are male hormones, so women taking this medicine can develop masculine characteristics such as facial hair, balding, deepening voice, etc.
General considerations in treating aplastic anemia
Many years ago, aplastic anemia was considered a fatal disease unless the patient recovered without treatment (spontaneously). Now that we understand this is often an immune disease, treatments have been devised that have a high success rate.
Children with inherited causes of aplastic anemia are treated with supportive therapy (such as androgens) and stem cell transplant. Immunosuppressants don't help these patients.
Those with acquired aplastic anemia are treated with supportive care, followed by immunesuppressant therapy or stem cell transplant. Stem cell transplant is preferred when the patient has a matched, related donor, and is young and otherwise healthy. Immune treatments may be the first option for older patients and those without a matched donor.
A small number of people with aplastic anemia will develop leukemia or other cancers after several years. But it isn't clear if this is caused by the treatment, or just part of the disease itself. For example, many people with Fanconi anemia who don't get aplastic anemia, will still go on to develop leukemia.
Aplastic anemia is rare, and treating it is very specialized, so it may best be done at a major medical center where they have experience with this disease. This is particularly true for stem cell transplants.
Sometimes the first treatment isn't successful and will need to be repeated. Often the second treatment will be effective, even if the first wasn't. Also, the treatment may be only partially effective, and the blood counts may not return to normal. In this situation, many people can still lead a normal life and not need treatment. Their blood counts, although not normal, will be high enough for a normal life.
Sometimes, none of the treatments is successful. In this case a person with aplastic anemia might want to consider taking part in a clinical trial, that is, a study of a new therapy that hasn't yet been proven successful.
Adapted in part or whole from the American Cancer Society website.
Bone Marrow Transplants
The bone marrow—the sponge-like tissue found in the center of certain bones—contains stem cells that are the precursors of white blood cells, red blood cells, and platelets. These blood cells are vital for normal body functions, such as oxygen transport, defense against infection and disease, and clotting. Blood cells have a limited lifespan and are constantly being replaced; therefore, healthy stem cells are vital.
In association with certain diseases, stem cells may produce too many, too few, or otherwise abnormal blood cells. Also, medical treatments may destroy stem cells or alter blood cell production. The resultant blood cell abnormalities can be life threatening.
There are three types of bone marrow transplants:
Bone marrow transplantation involves extracting bone marrow containing normal stem cells from a healthy donor, and transferring it to a recipient whose body cannot manufacture proper quantities of normal blood cells. The goal of the transplant is to rebuild the recipient's blood cells and immune system and hopefully cure the underlying ailment.
In association with certain diseases, stem cells may produce too many, too few, or otherwise abnormal blood cells. Also, medical treatments may destroy stem cells or alter blood cell production. The resultant blood cell abnormalities can be life threatening.
There are three types of bone marrow transplants:
Bone marrow transplantation involves extracting bone marrow containing normal stem cells from a healthy donor, and transferring it to a recipient whose body cannot manufacture proper quantities of normal blood cells. The goal of the transplant is to rebuild the recipient's blood cells and immune system and hopefully cure the underlying ailment.
Aplastic Anemia
Aplastic anemia is a disorder in which the bone marrow greatly decreases or stops production of blood cells.
Description
The bone marrow (soft tissue which is located within the hard outer shell of the bones) is responsible for the production of all the types of blood cells. The mature forms of these cells include red blood cells, which carry oxygen throughout the body; white blood cells, which fight infection; and platelets, which are involved in clotting.
In aplastic anemia, the basic structure of the marrow becomes abnormal, and those cells responsible for generating blood cells (hematopoietic cells) are greatly decreased in number or absent. These hematopoietic cells are replaced by large quantities of fat.
Yearly, aplastic anemia strikes about 20,000-30,000 people, or 5-10 people in every one million. Although aplastic anemia strikes both males and females of all ages, there are two age groups that have an increased risk. Both young adults (between 15-30 years of age) and the elderly (over the age of 60) have higher rates of aplastic anemia than the general population. While the disorder occurs worldwide, young adults in Asia have a higher disease rate than do populations in North America and Europe.
Causes and symptoms
Aplastic anemia falls into three basic categories, based on the origin of its cause: idiopathic, acquired, and hereditary. In about (50% to) 60% of cases, aplastic anemia is considered to be idiopathic, meaning that the cause of the disorder is unknown.
Acquired Aplastic Anemia
Acquired aplastic anemia refers to those cases where certain environmental factors and physical conditions seem to be associated with development of the disease. Acquired aplastic anemia can be associated with:
Hereditary aplastic anemia is relatively rare, but does occur in Fanconi's anemia, Shwachman-Diamond syndrome, and dyskeratosis congenita.
Symptoms
Symptoms of aplastic anemia tend to be those of other anemias, including fatigue, weakness, tiny reddish-purple marks (petechiae) on the skin (evidence of pinpoint hemorrhages into the skin), evidence of abnormal bruising, and bleeding from the gums, nose, intestine, or vagina. The patient is likely to appear pale. If the anemia progresses, decreased oxygen circulating in the blood may lead to an increase in heart rate and the sudden appearance of a new heart murmur.
Prognosis
Aplastic anemia is a life-threatening illness. Without treatment, it will almost surely progress to death. Survival depends on how severe the disease is at diagnosis, which type of treatment a patient is eligible for, and what kind of response their body has to that treatment. The worst-prognosis type of aplastic anemia is one associated with very low numbers of a particular type of white blood cell. These patients have a high chance of dying from overwhelming bacterial infections. In fact, 80% of all patients treated with blood transfusions alone die within 18 months to two years. Patients who undergo bone marrow transplantation have a 60-90% chance of being cured of the disease.
BOOKS:
Castro-Malaspina, Hugo, and Richard J. O'Reilly. "Aplastic Anemia and Myelodysplastic Syndromes." In Harrison's Principles of Internal Medicine, ed. Anthony S. Fauci, et al. New York: McGraw-Hill, 1997.
PERIODICALS:
Doney, Kristine, et al. "Primary Treatment of Acquired Aplastic Anemia: Outcomes with Bone Marrow Transplantation and Immunosuppressive Therapy." Annals of Internal Medicine 126 (15 Jan. 1997): 107+.
Young, Neal. "Aplastic Anaemia." The Lancet 346 (22 July 1995): 228+.
Young, Neal, and Jaroslaw Maciejewski. "The Pathophysiology of Acquired Aplastic Anemia." The New England Journal of Medicine 336 (8 May 1997): 1365+.
ORGANIZATIONS:
Aplastic Anemia Foundation of America. P.O. Box 613, Annapolis, MD 21404. (800) 747-2820. http://www.aplastic.org/.
GLOSSARY:
Bone marrow—A substance found in the cavities of bones, especially the long bones and the sternum (breast bone). The bone marrow contains those cells which are responsible for the production of the blood cells (red blood cells, white blood cells, and platelets).
Bone marrow transplant—A procedure in which a quantity of bone marrow is extracted through a needle from a donor, and then passed into a patient to replace the patient's diseased or absent bone marrow.
Hematopoietic cells—Those cells which are lodged within the bone marrow, and which are responsible for producing the cells which circulate in the blood (red blood cells, white blood cells, and platelets).
Description
The bone marrow (soft tissue which is located within the hard outer shell of the bones) is responsible for the production of all the types of blood cells. The mature forms of these cells include red blood cells, which carry oxygen throughout the body; white blood cells, which fight infection; and platelets, which are involved in clotting.
In aplastic anemia, the basic structure of the marrow becomes abnormal, and those cells responsible for generating blood cells (hematopoietic cells) are greatly decreased in number or absent. These hematopoietic cells are replaced by large quantities of fat.
Yearly, aplastic anemia strikes about 20,000-30,000 people, or 5-10 people in every one million. Although aplastic anemia strikes both males and females of all ages, there are two age groups that have an increased risk. Both young adults (between 15-30 years of age) and the elderly (over the age of 60) have higher rates of aplastic anemia than the general population. While the disorder occurs worldwide, young adults in Asia have a higher disease rate than do populations in North America and Europe.
Causes and symptoms
Aplastic anemia falls into three basic categories, based on the origin of its cause: idiopathic, acquired, and hereditary. In about (50% to) 60% of cases, aplastic anemia is considered to be idiopathic, meaning that the cause of the disorder is unknown.
Acquired Aplastic Anemia
Acquired aplastic anemia refers to those cases where certain environmental factors and physical conditions seem to be associated with development of the disease. Acquired aplastic anemia can be associated with:
- exposure to drugs, especially anti-cancer agents, antibiotics, anti-inflammatory agents, seizure medications, and antithyroid drugs (drugs given to stop the functioning of an overactive thyroid)
- exposure to radiation
- chemical exposure (especially to the organic solvent benzene and certain insecticides)
- infection with certain viruses (especially those causing viral hepatitis, as well as Epstein-Barr virus, parvovirus, and HIV, the virus which can cause AIDS)
- pregnancy
- certain other disorders, including a disease called paroxysmal nocturnal hemoglobinuria, an autoimmune reaction called graft-vs-host disease (which occurs when the body's immune system attacks and destroys the body's own cells), and certain connective tissue diseases
Hereditary aplastic anemia is relatively rare, but does occur in Fanconi's anemia, Shwachman-Diamond syndrome, and dyskeratosis congenita.
Symptoms
Symptoms of aplastic anemia tend to be those of other anemias, including fatigue, weakness, tiny reddish-purple marks (petechiae) on the skin (evidence of pinpoint hemorrhages into the skin), evidence of abnormal bruising, and bleeding from the gums, nose, intestine, or vagina. The patient is likely to appear pale. If the anemia progresses, decreased oxygen circulating in the blood may lead to an increase in heart rate and the sudden appearance of a new heart murmur.
Prognosis
Aplastic anemia is a life-threatening illness. Without treatment, it will almost surely progress to death. Survival depends on how severe the disease is at diagnosis, which type of treatment a patient is eligible for, and what kind of response their body has to that treatment. The worst-prognosis type of aplastic anemia is one associated with very low numbers of a particular type of white blood cell. These patients have a high chance of dying from overwhelming bacterial infections. In fact, 80% of all patients treated with blood transfusions alone die within 18 months to two years. Patients who undergo bone marrow transplantation have a 60-90% chance of being cured of the disease.
BOOKS:
Castro-Malaspina, Hugo, and Richard J. O'Reilly. "Aplastic Anemia and Myelodysplastic Syndromes." In Harrison's Principles of Internal Medicine, ed. Anthony S. Fauci, et al. New York: McGraw-Hill, 1997.
PERIODICALS:
Doney, Kristine, et al. "Primary Treatment of Acquired Aplastic Anemia: Outcomes with Bone Marrow Transplantation and Immunosuppressive Therapy." Annals of Internal Medicine 126 (15 Jan. 1997): 107+.
Young, Neal. "Aplastic Anaemia." The Lancet 346 (22 July 1995): 228+.
Young, Neal, and Jaroslaw Maciejewski. "The Pathophysiology of Acquired Aplastic Anemia." The New England Journal of Medicine 336 (8 May 1997): 1365+.
ORGANIZATIONS:
Aplastic Anemia Foundation of America. P.O. Box 613, Annapolis, MD 21404. (800) 747-2820. http://www.aplastic.org/.
GLOSSARY:
Bone marrow—A substance found in the cavities of bones, especially the long bones and the sternum (breast bone). The bone marrow contains those cells which are responsible for the production of the blood cells (red blood cells, white blood cells, and platelets).
Bone marrow transplant—A procedure in which a quantity of bone marrow is extracted through a needle from a donor, and then passed into a patient to replace the patient's diseased or absent bone marrow.
Hematopoietic cells—Those cells which are lodged within the bone marrow, and which are responsible for producing the cells which circulate in the blood (red blood cells, white blood cells, and platelets).
Causes Of Aplastic Anemia
50% of the people diagnosed with aplastic anemia do not know the cause, nor is the cause ever determined. When the cause is unknown, it is referred to as idiopathic aplastic anemia. The other half can trace the disease back to one of the following causes:
- Exposure to insecticides
- Exposure to pesticides
- Exposure to chemical toxins
- Ingestation of arsenic
- Certain drugs
- Chemotherapy
- Disorders present at birth (congenital disorders)
- Drug therapy to suppress the immune system
- Pregnancy
- Radiation therapy
- Toxins such as benzene or arsenic
Bone Marrow Transplants See Positive Improvements
http://health.usnews.com/usnews/health/healthday/080323/a-bone-marrow-disease-with-a-brighter-prognosis.htm
There's hope: Considered fatal as recently as two decades ago, aplastic anemia is becoming a far more manageable disease. Advances in drug therapies and improvements in the field of transplantation have slashed the death toll, allowing patients to live longer, fuller lives.
"We are getting better at treating aplastic anemia, either in getting rid of it or treating its symptoms," said Dr. Jaroslaw P. Maciejewski, with the Cleveland Clinic's Department of Hematologic Oncology and Blood Disorders.
There's hope: Considered fatal as recently as two decades ago, aplastic anemia is becoming a far more manageable disease. Advances in drug therapies and improvements in the field of transplantation have slashed the death toll, allowing patients to live longer, fuller lives.
"We are getting better at treating aplastic anemia, either in getting rid of it or treating its symptoms," said Dr. Jaroslaw P. Maciejewski, with the Cleveland Clinic's Department of Hematologic Oncology and Blood Disorders.
Wednesday, October 14, 2009
Potential Donors - Am I a donor? Can I be a donor?
When it is determined that a bone marrow transplant should or will take place, the next step is to find a suitable donor. The primary candidates for a suitable donor are the siblings of the intended recipient. There is a 25% chance of each sibling matching. If a sibling does not match, then the next step is to go to the National Bone Marrow Registry to seek a donor. Information about the process of donating, how bone marrow is matched and how to be a donor is provided below.
Listed Potential Donors
If you have been listed as a potential donor for a person who will need a bone marrow transplant, that person's cancer team will direct you to the appropriate facility for collection of your blood sample or buckle test (cheek swab), typing and communication of your typing. They will likely call you shortly after a determination has been made that a bone marrow transplant either is or may be done. They will give you the name and address of the location to which they want you to go to. When you arrive, they will process your paperwork, confirming your identity and proceed to take a sample of your blood or a swab sample from the incide of your cheek (buckle test). In almost all instances, it will be the original cancer team that will contact you to inform you of whether you are a bone marrow match.
Unlisted Potential Donors
If you are not listed as a potential donor on the recipient's donor list, it is likely because you are not the potential recipient's sibling; only siblings are listed and contacted in the first steps of seeking a matched donor. If a sibling does not match, then the next step is to seek a donor from the national registry, or if there are people who want to be considered as a donor then from their results.
Can I Be A Donor?
Perhaps. There are two types of donors - those who want to donate to a particular person, and those who want to donote to anyone with whom they match. The type of donor you wish to be determines the method for your testing.
Anonymous Donors Registry
If you wish to become a registered donor with the national registry, which would include being assessed for your desired recipient if time permits for your results to be entered into the national database in time for your intended recipient's search. The national registry process takes more time to process donor information, so that must be taken into consideration. But the national registry is free. The process is quite simple. You register, answer a few health questions for preliminary acceptance, and ask that a kit be sent to you; you swab the inside of your cheek, send the results back, and are listed in the registry. IF YOU WANT TO USE THE FREE SERVICES OF THE REGISTRY, BE SURE TO ASK THAT YOUR RESULTS BE MAILED TO YOU AS WELL AS ADDED TO THE REGISTRY. Remember, that listing with the national registry is a committment on your part stating that you will donate to anyone if asked. For private matching, please see private matching below. More information about the national registry is available at their website, http://www.marrow.org/.
In all instances, the testing process through the registry is free. If you are an intended recipient's sibling, it is very likely that the recipient's insurance company will cover the costs of the test.
Private Bone Marrow Donor Matching
If you wish to be tested for matching to a particular intended recipient, then the national registry is not the way to go. Rather, you should seek information on the website, http://www.bonemarrowtest.com/.
To be tested to match a particular intended recipient, you will need to find a collection center near you. Because the HL Testing process is unique, it is not done by most doctors or hospitals.
The second step is to ask your insurance company if they will cover the costs for you to be tested for an intended recipient (in most cases, they won't if you are not a sibling). The cost of the test is dependant upon the type of HLA test being conducted. In the preliminary testing process, it would likely be sufficient to test for Class 1 AB HLA Matching. If those two factors match, you can then be tested for the subsequent factors (Class 1 C, Class 2 DR, DQ and DP). If time is of the essence, you may want to be tested for Class 1 ABC, Class 2 DR; and if cost is not a factor for an additional one test, then also DQ; and lastly, if it is not cost prohibitive, then for DP.
Be sure to ask that your results be mailed to you. When you receive them, you can then fax them to the coordinator for the intended recipient's bone marrow matching.
See Bone Marrow Matching for more information.
Listed Potential Donors
If you have been listed as a potential donor for a person who will need a bone marrow transplant, that person's cancer team will direct you to the appropriate facility for collection of your blood sample or buckle test (cheek swab), typing and communication of your typing. They will likely call you shortly after a determination has been made that a bone marrow transplant either is or may be done. They will give you the name and address of the location to which they want you to go to. When you arrive, they will process your paperwork, confirming your identity and proceed to take a sample of your blood or a swab sample from the incide of your cheek (buckle test). In almost all instances, it will be the original cancer team that will contact you to inform you of whether you are a bone marrow match.
Unlisted Potential Donors
If you are not listed as a potential donor on the recipient's donor list, it is likely because you are not the potential recipient's sibling; only siblings are listed and contacted in the first steps of seeking a matched donor. If a sibling does not match, then the next step is to seek a donor from the national registry, or if there are people who want to be considered as a donor then from their results.
Can I Be A Donor?
Perhaps. There are two types of donors - those who want to donate to a particular person, and those who want to donote to anyone with whom they match. The type of donor you wish to be determines the method for your testing.
Anonymous Donors Registry
If you wish to become a registered donor with the national registry, which would include being assessed for your desired recipient if time permits for your results to be entered into the national database in time for your intended recipient's search. The national registry process takes more time to process donor information, so that must be taken into consideration. But the national registry is free. The process is quite simple. You register, answer a few health questions for preliminary acceptance, and ask that a kit be sent to you; you swab the inside of your cheek, send the results back, and are listed in the registry. IF YOU WANT TO USE THE FREE SERVICES OF THE REGISTRY, BE SURE TO ASK THAT YOUR RESULTS BE MAILED TO YOU AS WELL AS ADDED TO THE REGISTRY. Remember, that listing with the national registry is a committment on your part stating that you will donate to anyone if asked. For private matching, please see private matching below. More information about the national registry is available at their website, http://www.marrow.org/.
In all instances, the testing process through the registry is free. If you are an intended recipient's sibling, it is very likely that the recipient's insurance company will cover the costs of the test.
Private Bone Marrow Donor Matching
If you wish to be tested for matching to a particular intended recipient, then the national registry is not the way to go. Rather, you should seek information on the website, http://www.bonemarrowtest.com/.
To be tested to match a particular intended recipient, you will need to find a collection center near you. Because the HL Testing process is unique, it is not done by most doctors or hospitals.
The second step is to ask your insurance company if they will cover the costs for you to be tested for an intended recipient (in most cases, they won't if you are not a sibling). The cost of the test is dependant upon the type of HLA test being conducted. In the preliminary testing process, it would likely be sufficient to test for Class 1 AB HLA Matching. If those two factors match, you can then be tested for the subsequent factors (Class 1 C, Class 2 DR, DQ and DP). If time is of the essence, you may want to be tested for Class 1 ABC, Class 2 DR; and if cost is not a factor for an additional one test, then also DQ; and lastly, if it is not cost prohibitive, then for DP.
Be sure to ask that your results be mailed to you. When you receive them, you can then fax them to the coordinator for the intended recipient's bone marrow matching.
See Bone Marrow Matching for more information.
Blood Type & Bone Marrow Transplants
Blood types do not matter, and are not used in the bone marrow matching process; before marrow is transplanted, existing bone marrow and blood stem cells are destroyed. During marrow transplants, new blood stem cells are interveneously injected into the bloodstream of the recipient to make their way into the marrow. Remaining blood cells in the recipient die off within hours of the initiation of a transplant (as they were radiated prior to the transplant). The transplanted marrow then builds new blood stem cells, and in some situations the recipient's blood type will change.
Allogeneic Transplants
An allogeneic transplant is a transplant that comes from another person.
Bone Marrow or Stem Cell Transplant
Stem cells are cells in the bone marrow (the spongy, liquid center of certain bones) that constantly make blood cells for the body. Stem cell transplants are used to restock the bone marrow when it has been destroyed by chemotherapy, radiation, or disease. Stem cells can be taken from bone marrow or blood. Stem cells may be the patient's own (autologous), or they may come from someone else (allogeneic).
Bone marrow transplants (BMTs) were the first method for replacing stem cells, but are used less often today. Now, peripheral blood stem cell transplant (SCT) is the most common method. In peripheral blood stem cell donation, stem cells are taken from circulating blood. Before collection, the donor must take special medicines to cause stem cells to grow and enter the bloodstream.
Bone Marrow or Stem Cell Transplant
Stem cells are cells in the bone marrow (the spongy, liquid center of certain bones) that constantly make blood cells for the body. Stem cell transplants are used to restock the bone marrow when it has been destroyed by chemotherapy, radiation, or disease. Stem cells can be taken from bone marrow or blood. Stem cells may be the patient's own (autologous), or they may come from someone else (allogeneic).
Bone marrow transplants (BMTs) were the first method for replacing stem cells, but are used less often today. Now, peripheral blood stem cell transplant (SCT) is the most common method. In peripheral blood stem cell donation, stem cells are taken from circulating blood. Before collection, the donor must take special medicines to cause stem cells to grow and enter the bloodstream.
Aplastic Anemia Pre-transplant Radiation Treatment & Effects
Radiation is used for treatment of some types of cancers. It is also used to treat a patient prior to a bone marrow transplant. Prior to a bone marrow transplant (or stem cell transplant), total body radiation is used to kill the immune system and bone marrow. Typically, a level 12 radiation is used (comparatively, breast cancer radiation levels are usually at 40).
During Radiation
Extra precautions to take during and after radiation treatment include dietary changes, skin protection, clothing (not wearing tight clothes), and getting plenty of rest. For more information, see Radiation Treatment Care: http://www.cancer.org/docroot/MBC/content/MBC_2_3x_What_Can_I_Do_to_Take_Care_of_Myself_during_Therapy.asp?sitearea=MBC
Effects of Radiation
Radiation can cause fatigue, loss of hair, eating problems (particularly nauseau, sores in the mouth).
Brain Radiation
Stereotactic radiosurgery is often used when cancer has only spread to a few sites in the brain. Side effects vary depending on where the radiation is aimed, and usually become most serious 1 to 2 years after treatment. Talk with your radiation oncologist about what to watch for and when to call for help.
Sometimes the whole brain is treated with radiation when cancer has spread into many sites. This may also be done to prevent cancer from spreading to the brain. Whole brain radiation can cause some of the following side effects:
Stomach & Intestinal Radiation
http://www.cancer.org/docroot/MBC/content/MBC_2_3X_What_Side_Effects_Occur_With_Radiation_Therapy_to_the_Stomach_and_Abdomen_Area.asp?sitearea=MBC
During Radiation
Extra precautions to take during and after radiation treatment include dietary changes, skin protection, clothing (not wearing tight clothes), and getting plenty of rest. For more information, see Radiation Treatment Care: http://www.cancer.org/docroot/MBC/content/MBC_2_3x_What_Can_I_Do_to_Take_Care_of_Myself_during_Therapy.asp?sitearea=MBC
Effects of Radiation
Radiation can cause fatigue, loss of hair, eating problems (particularly nauseau, sores in the mouth).
Brain Radiation
Stereotactic radiosurgery is often used when cancer has only spread to a few sites in the brain. Side effects vary depending on where the radiation is aimed, and usually become most serious 1 to 2 years after treatment. Talk with your radiation oncologist about what to watch for and when to call for help.
Sometimes the whole brain is treated with radiation when cancer has spread into many sites. This may also be done to prevent cancer from spreading to the brain. Whole brain radiation can cause some of the following side effects:
- headaches
- swelling of the brain
- hair loss
- nausea
- vomiting
- fatigue
- hearing loss
- skin and scalp changes
- trouble with memory and speech
- seizures
Stomach & Intestinal Radiation
http://www.cancer.org/docroot/MBC/content/MBC_2_3X_What_Side_Effects_Occur_With_Radiation_Therapy_to_the_Stomach_and_Abdomen_Area.asp?sitearea=MBC
Subscribe to:
Posts (Atom)