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.
Showing posts with label ATG. Show all posts
Showing posts with label ATG. Show all posts
Tuesday, February 22, 2011
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
Thursday, October 15, 2009
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.
Treatment
In secondary aplastic anemia, removing the cause is critical. In some cases it can lead to recovery.
Mild Aplastic Anemia
Mild cases of aplastic anemia may be treated with supportive care, or may not need treatment. Blood and platelet transfusions will help correct the abnormal blood counts and relieve some symptoms in moderate cases.
Mild Aplastic Anemia
Severe aplastic anemia, which causes a very low blood-cell count, is a life-threatening condition. Younger patients with a severe case of the disease will need a bone marrow transplant. Older patients, or those who do not have a matched bone marrow donor, can be treated with medications that suppress the immune system, such as antithymocyte globulin (ATG), tacrolimus, or cyclosporine.
ATG - Antithymocyte Globulin
ATG consists of antibodies made in horses or rabbits against a type of white blood cell in humans called T cells. It is used to suppress the body's immune system, allowing the bone marrow to start generating blood cells again.
Cyclosporine, Cytoxan
Other medications to suppress the immune system, such as cyclosporine, tacrolimus, and cyclophosphamide (Cytoxan) also may be used. Corticosteroids and androgens have been used as well.
http://healthtools.aarp.org/adamcontent/secondary-aplastic-anemia
Mild Aplastic Anemia
Mild cases of aplastic anemia may be treated with supportive care, or may not need treatment. Blood and platelet transfusions will help correct the abnormal blood counts and relieve some symptoms in moderate cases.
Mild Aplastic Anemia
Severe aplastic anemia, which causes a very low blood-cell count, is a life-threatening condition. Younger patients with a severe case of the disease will need a bone marrow transplant. Older patients, or those who do not have a matched bone marrow donor, can be treated with medications that suppress the immune system, such as antithymocyte globulin (ATG), tacrolimus, or cyclosporine.
ATG - Antithymocyte Globulin
ATG consists of antibodies made in horses or rabbits against a type of white blood cell in humans called T cells. It is used to suppress the body's immune system, allowing the bone marrow to start generating blood cells again.
Cyclosporine, Cytoxan
Other medications to suppress the immune system, such as cyclosporine, tacrolimus, and cyclophosphamide (Cytoxan) also may be used. Corticosteroids and androgens have been used as well.
http://healthtools.aarp.org/adamcontent/secondary-aplastic-anemia
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