Tuesday, March 15, 2011

Send A Letter & Possibly Save A Life

On February 10, 2011, Representative Doris O. Matsui (D-CA) formally introduced the “Bone Marrow Failure Disease Research and Treatment Act” (H.R. 640).

H. R. 640 is invaluable legislation. It brings together resources from several Federal agencies to advance understanding of and treatment of bone marrow failure diseases such as Aplastic Anemia, Myelodysplastic Syndromes (MDS) and ParoPNH (PNH). As such, it and the bone marrow registry legislative bills are the single most important and valuable bills yet to be introduced to save lives of people with bone marrow failure diseases.

Senator John McCain, however, is proposing a bill to eliminate the funding to Bone Marrow Failure Research & Treatment that was allocated in February 2011. His purpose in doing this is obvious. That funding is under the Department of Defense & Research; he intends to redirect funding intended to save lives towards his war agenda. He wants those funds to take the lives of the thousands upon thousands of people in the USA who die from bone marrow failure diseases and the lives of our boys and girls whom are sent to fight for oil tycoons. That is just wrong.

YOU CAN HELP! Please take a few moments to visit the Grassroots page at AAMDS.org and send a letter to Senator McCain opposing his bill, and ask for your Senators' support. It's real easy. There are prewritten letters for you to use, just fill in your name and address, or you can write your own. Please send a letter and possibly save a life.

Aplastic Anemia affects approximately 10,000 to 12,000 people in the world each year. Leukemia affects approximately 245,000 people in the United States. Although Aplastic Anemia is non-cancerous, both it and Leukemia can be treated with a bone marrow transplant. The research and treatment funding to save those lives is in jeopardy with Senator John McCain's intended bill. Write to McCain and tell him that you oppose his bill. Write to your Senators and ask them to help save this funding in D.C. It only takes a minute to send these letters online.

Click here to go to the AAMDS.org Take Action page.

Or click here to go the AAMDS.org website.

YOU CAN ALSO WRITE TO:

Senator John McCain•Senator John McCain's Phoenix Office:
5353 North 16th Street
Suite 105
Phoenix, AZ 85016
Main: (602) 952-2410
Fax: (602) 952-8702

•Senator John McCain's Prescott Office:
122 North Cortez Street
Suite 108
Prescott, AZ 86301
Main: (928) 445-0833
Fax: (928) 445-8594

•Senator John McCain's Tempe Office:
4703 South Lakeshore Drive
Suite 1
Tempe, AZ 85282
Main: (480) 897-6289
Fax: (480) 897-8389

•Senator John McCain's Tucson Office:
407 West Congress Street
Suite 103
Tucson, AZ 85701
Main: (520) 670-6334
Fax: (520) 670-6637

•Senator John McCain's Washington Office:
241 Russell Senate Office Building
Washington, DC 20510
Main: (202) 224-2235
Fax: (202) 228-2862


Senator Doris MatsuiWashington Office
222 Cannon Building
Washington, DC 20515
-or-
Senator Doris Matsui
Sacramento Office
501 I Street, Suite 12-600
Sacramento, CA 95814




Send a letter to your Senator: AAMDS.org; Donate to AAMDS.

Saturday, March 12, 2011

Summation - Death of People! & Civil Rights?

Send a letter to your Senator: AAMDS.org; Donate to AAMDS.

In addition to revocation of collective bargaining rights, Governor Walker also encapsulated another hidden agenda in his bill, and has plans for a final death hand. The hidden agenda is to reform Medical Assistance; the death hand is two-fold; first, he intends to spin off UW-Madison hospital and clinics who are a national leader in life-saving research and treatment, and second, he intends to reform medical services at the state level.

The bill directs the department of public health services to 'assess' the medical assistance program and propose means by which it can be made more effective. As the spokesperson from the National Bone Marrow Donation Association put it, "That's not a bad thing. Despite how much you may like or dislike Governor Walker, to assess programs for effeciency is a good thing." Yes, it is; however, it goes on to say that the PHS can make law by means of medical emergency legislative paths. As well, what is written there in invisible ink is the call for reform and exclusions. One exclusion that other states have made during their assessments is of waiver programs; these are special programs to provide life-saving services such as bone marrow transplants. Without bone marrow transplants, people with Aplastic Anemia, as well as many other diseases (Leukemia, PNH, MDS, EB, and the list goes on and on and on) will die. That, Governor Walker, is a sentence to death by legislative pen.

Add to that legislative pen the stroke of deaths of millions with the spinning off of UW-Madison to make it act as a business on its own two feet. UW-Madison is a national leader in stem cell research. Those stem cells aren't fetuses, but rather blood stem cells donated from the marrow of people just like you and me, from the cords of babies donated by their parents to save a life, and form donors who are dying. They are on the brink of finding a solution for Alzheimer's. Do these Senators not realize how many millions of dollars could be saved by the Medicare and Medicaid programs by simply not confining an Alzheimer's patient to a nursing home? Multiples of millions in Wisconsin, to the billions of dollars nationwide, would be saved.

It's not only a Wisconsin problem. Senator John McCain is sponsoring a bill that will terminate funding to bone marrow research. That research is housed under the same arena as is the defense funding. Obviously, pro-war Senator McCain is seeking to stop dollars from going to save lives to put dollars towards the effort to take lives. We expect this given his history. McCain: there is no honor in killing Americans through a silent holocost on the medical assistance and research funding to fight for their freedom. We strongly disagree, and encourage everyone to write to Senator McCain to tell him not to kill the funding to bone marrow research.

PLEAS:

Whoever you are, you don't know that you won't one day need a bone marrow transplant. If the research is ended, where will you go? The US is the leader in medical research. Write to your Senator and to Senator John McCain to "Oppose any Amendment to Eliminate CDMRP Funding". You might also want to write to Doris O. Matsui (D-CA) to tell her that you support her and the “Bone Marrow Failure Disease Research and Treatment Act” (H.R. 640). This legislation would bring together the resources of several federal agencies to advance our understanding of, and treatments for aplastic anemia, myelodysplastic syndromes (MDS), and ParoPNH. We need your support now! It is simple to send that letter, just go to AA & MDS and fill in your name and submit the letter.

TO THOSE PROTESTING IN WISCONSIN: Please keep the reform appeals in Walker's bill in your minds and hearts as you fight for civil rights; this fight is for life.

Lent: One of my lenten sacrafices is to give two hours of my off time every day to helping further the fight of those who are fighting for their lives recovering from a bone marrow transplant.

Dear Senator McCain:

Senator McCain:

Last year I almost died from a bone marrow failure disease. Because of research they found out that I had Aplastic Anemia. Because of research they were able to give me a bone marrow transplant. Because of research, the research you intend to take away, I am 1 year post transplant and am able to be with my children. Because of research my 2 and 3 year old children did not have to put their mother in the ground.

To all readers:

I am asking for your valuable and immediate support. Last year, Bone Marrow Failure Research & Treatment received funding; today, Sen John McCain supports a bill to eliminate that life-saving funding. Please go to http://AAMDS.org/ & support saving lives. It'll take 1 minute.

Send a letter to your Senator: AAMDS.org; Donate to AAMDS.

U.S. Held In Highest Regard For Cure

If there will be a cure found for Aplastic Anemia, and we believe that there will one day be a cure, that cure will come from the United States. Senator McCain, you have the power to kill thousands.

Dear Senator McCain I am not a U.S resident but have the utmost respect for you country as you have always been great allies to England I am asking you to please not take away the funding into bone marrow disease research I dont have to rem...ind you of these terribble diseases as I know you are aware and I know resources are difficult but you are a leading country and others look upto you so your research in the U.S is paramount to continue if a cure is to be found I beg you not to take this funding away forget about statisitics its real people many many children are dying I have this disease myself and to be honest it grieves me more for the children it affects they are the ones with the disease and the ones which we as adults leave behind when we die from this disease as we will without the funding. I thankyou for taking the time to read my request respectfully yours Julie Anne Theobald.
Read more about an issue by clicking on a link below. For Advocacy issues (where we suggest you take action with your elected officials) you can follow the instructions on the screen to easily communicate with your elected officials.

Oppose McCain Amendment to Eliminate Funding for Research
The Aplastic Anemia & MDS International Foundation has received word that Senator John McCain (R-AZ) may be offering an amendment, as early as next week, to eliminate all fiscal year 2011 funding for (more)


Help Get Cosponsors for Bone Marrow Failure Legislation
On February 10, 2011, Representative Doris O. Matsui (D-CA) formally introduced the “Bone Marrow Failure Disease Research and Treatment Act” (H.R. 640)... (more)

Wisconsin:
Johnson, Ron - (R - WI) Class III
2 RUSSELL COURTYARD WASHINGTON DC 20510
(202) 224-5323
Web Form: ronjohnson.senate.gov/public/index.cfm/contact

Kohl, Herb - (D - WI) Class I
330 HART SENATE OFFICE BUILDING WASHINGTON DC 20510
(202) 224-5653
Web Form: kohl.senate.gov/contact.cfm

On February 10, 2011, Representative Doris O. Matsui (D-CA) formally introduced the “Bone Marrow Failure Disease Research and Treatment Act” (H.R. 640). This legislation would bring together the resources of several federal agencies to advance our understanding of, and treatments for aplastic anemia, myelodysplastic syndromes (MDS), and ParoPNH. We need your support now!

Suggested letter:
I am writing to ask you to cosponsor the "Bone Marrow Failure Disease Research and Treatment Act" (H.R. 640), introduced by Representative Doris Matsui (D-CA) on February 10, 2011. We need your support. As a constituent coping with bone marrow failure disease, I ask that you support this new comprehensive approach to combating these devastating diseases.

Every year approximately 20,000 to 30,000 Americans are diagnosed with aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH), and other acquired bone marrow failure diseases.

The Bone Marrow Failure Disease Research and Treatment Act would leverage the expertise and resources of several agencies within the Department of Health and Human Services (HHS), as well as patient advocacy organizations, in a new comprehensive approach to combat bone marrow failure diseases. H.R. 640 increases the Federal Government's commitment to researching and treating aplastic anemia, MDS, PNH, and other acquired bone marrow failure diseases. The legislation directs the Department of Health and Human Services (HHS) to combat these diseases through a comprehensive strategy that includes creation of a national bone marrow failure disease registry so that researchers can combine their data in one place, yielding more effective research designs and better results and pilot studies through the Agency for Toxic Substances and Disease Registry to determine which environmental factors cause people to acquire bone marrow failure diseases.

Other highlights include minority-focused programs to make information on treatment options and clinical trials available to minority communities, particularly Hispanic and Asian American communities and Agency for Healthcare Research and Quality grants to help improve diagnostic practices and quality of care for patients with bone marrow failure diseases.

Last year, the U.S. House of Representatives approved a similar but modified version of the bill.

Congresswoman Matsui will be sending you a Dear Colleague letter in the coming weeks. Please consider becoming a cosponsor of this important legislation and contact Joel Bailey, Senior Legislative Assistant, in the office of Representative Matsui at joel.bailey@mail.house.gov or at (202) 225-7163.

Thank you for your attention to this important matter.

Who Is My Senator?

Who is your Senator?

Look it up at Senate.

Strike Planned

As the old phrase states: Extraordinary times require extraordinary measures. These are indeed extraordinary times in Wisconsin. The Budget Repair Bill that was passed by Governor Scott Walker and State Republicans will strip public employees of the right to collectively bargain and threaten the very existence of unions in the state.

Read Strike Plans

URGENT - WRITE TO SEN JOHN MCCAIN BEFORE HE KILLS SOMEONE YOU LOVE

Here, we are reposting with permission a letter from a woman inflicted with Aplastic Anemia, PNH and Leukemia cells. If you haven't written to Senator John McCain, please take a moment to do so; the life that your letter may save may be yours or that of someone you love. If you cannot write a letter due to your medical or economic situation, please send us a message through this BLOG so that we can help you to do so.

Senator McCain,

My name is Exie Williams and I am a survivor of 2 forms of bone marrow failure. I am in remission, but I am not cured. The treatment options for people with bone marrow failure is minimal. By eliminating funding for the Congressionally-Directed Medical Research Programs at the Department of Defense for Bone Marrow Failure research, is without a doubt devestating to myself and many others. This is the world’s largest research program dedicated to finding a cure for bone marrow failure diseases like aplastic anemia, myelodysplastic syndromes, and PNH.

I ask that you take a moment and view a support group page and see the faces of the men, women, and children that this admendment will effect.

http://www.facebook.com/AplasticAnemia

I was diagnosed with Aplastic Anemia, PNH, and leukemia cells when I was 31 years old. I fought the fight of my life. There was a moment during my fight that it looked as if I was not responding to treatment and my father had to ask me what my wishes were for burial and for my young children. My 70 year old father had to face the fact that he may have to bury his daughter because we were running out of options. Through much prayer and faith, I pulled through and I won my fight and claimed remission. Everyday through the support group I created I see families who are losing their faith and their hope. Young children are dying or suffering, mothers and fathers are dying or suffering because there are no options left for them.

When I received this news today, I was heart broken for those who are not responding to treatment and who can not find suitable bone marrow donors.

I ask and pray that you will reconsider eliminating this last bit of hope that so many are holding on to. We need this research program to continue so that we can one day possibly have a cure.

I thank you for your time and consideration in this matter. This is so very important to so many who are suffering from bone marrow failure diseases.

Best Regards,

Exie Williams

----------------------------------------------------------------
Another letter to Senator John McCain:

Mr. McCain, I am the father of a 5 year old girl who has been diagnosed with MDS (myelodysplastic syndrome) This is a rare condition in which the body stops making blood or starts making malformed cells. This, as well as Aplastic Anemia, wh...ich she was originally diagnosed with, is a disease about which very little is known. 4 years ago, nothing was known, and they just supported the kids as best they could until they died. Leaps and bounds have been made though, and treatment options have become available, due in part to the DoD Bone Marrow Failure Disease Research program, which will be defunded as part of of an amendment you are adding to the Defense Appropriations Act for 2011. I understand that our deficit is out of control and spending must be cut, but when you cut medical research funding, you dont just cut the future of medical advancement, which is already under attack from the socialized medicine crowd. You also cut off the lives of people like my little girl, whose lives may depend on advances that have been and could be made on the strength of this research. There is so much waste in government as you pointed out in your campaign that could be cut or at least pursued before taking the knife to life saving research. I beg you for the sake of my daughter, Maelee Grace Sheehan, and on the behalf of so many others we have come to know as we fight this disease, please reconsider this portion of your cuts.

Friday, March 11, 2011

Wisconsin Bill Passes

Wisconsin passes the bill to remove collective bargaining rights AND DIRECTS THE DEPARTMENT OF PUBLIC HEALTH SERVICES TO REVIEW AND REFORM MEDICAL ASSISTANCE IN WISCONSIN.

UW-Madison, a leader in stem cell research, is being spun off from the UW System.

What does it mean to people with bone marrow failure diseases?

Death.

My dad used to say, "You can put lipstick on a pig, but it's still a pig." Befitting, I think, for Governor Scott Walker.

Pass the recall sheet, I'll sign it, because when we said we wanted you to be conservative, Governor Walker, we did not mean by killing people. UW is one of the leaders in stem cell research. Walker, do you have any idea of how many billions of dollars would be saved by not putting people in nursing homes who have Alzheimer's? Mind you, billions of dollars are paid by medicare and medicaid to Canadian owned nursing homes.

Friday, March 4, 2011

"Recall Walker" Effort Begun

Under Wisconsin laws, a Governor can be recalled if 25% of the voting population that voted him into office signs a petition. That effort has begun in Wisconsin via a pre-recall effort. The Recall Walker website provides an information signature form that the group is asking people to complete.

Friday, February 25, 2011

28 February 2011 is Disease Day

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! ♥

$597 Per Voter Balances The Budget

Someone suggested that we just split the cost of balancing the budget, and move on with life.

Wisconsin reported a population of 5.6 million in 2009. Approximately 82% of Wisconsin are over the age of 18 years.

With a $2.7 billion deficit, a contribution of $588 would balance the budget.

Reduce the deficit by the $165 million that the reduction in benefits would amount to according to Walker's statements regarding the funding behind those benefits, and that makes the contribution $552 per voter to balance the budget.


Ah, but not everybody is working. While the reports show one thing, an estimated 270,000 people are out of work.

That puts it back to $586 PER VOTER, or $1173 per household, given that most households have two voters (and hopefully two workers).

A State Doesn't Live on Fed Aid Alone

Wisconsin had long been a thriving industrial state and the home to some of the top manufacturing companies in the nation.

Wisconsin's flip from a state that had excess funds from taxation to one which is in deep debt could be seen back in 2002 when manufacturers closed their doors to go out of business or move to another state where labor and taxes were cheaper.

I recall visiting West Bend, Wisconsin in 2000 to find three thriving manufacturers supplying the majority of employment for the local and closely surrounding area. In 2001, one of those, a major manufacturer of pots and pans, had closed its doors. The next year, another closed. And the next year, there was only one farming equipment manufacturer left, and he was running part time.

When the gross domestic product loses ground, people move from middle income earners to low income earners, and while taxes decrease, the burden on the government increases by way of aid to people. Wisconsin eliminated welfare some years ago, and all that is left to help people are a few payments of electricity, food pantries, food stamps and health care. Under special programs, local businesses in Wisconsin have created low-income services, such as dental care, albiet with the assistance of funding. But other funding, such as housing assistance is gone.

Peaceful, But Tense

At 10 East Doty Street, an office building just 'off of the square' and a stone throw from the capitol building, protestors marched on Koch Industries, Inc., who has an office on the 7th floor.

At 3:45 PM, the protestors marched from the capitol square down Martin Luther King Jr Blvd. towards the Doty entrance to 10 East Doty Street with drums beating loudly and chats heard over them, "Ho, ho, the bill must go." The core of the protest only lasted an hour, but protestors remained long after the close of business when I was escorted to my vehicle. Well into the evening hours, they could be seen standing in front of the Doty Street entrance shouting to cars as people were leaving the capital and local offices. Doty Street is one of the streets in the second square around the capitol, and a main drag for people on their ways home.

From early morning hours until at least 6 PM, building security personnel stood in groups at each ingress point and along hallways throughout the building. All of the elevators were locked down requiring people to have a security pass card to access any floor, but even those with a pass couldn't access the upper floors. Guards stood at the stairway doors. When I left at 6 PM, there were people standing near the doors trying to gain access to the building.

According to reports from several tenants of the building, there had been some incidences on the 7th floor where the Koch Industries office is located. Police were called to remove protestors.

Regardless of a person's stand on the issues, the current experience of Madison is one to behold. The protests are 'peaceful' as far as protests go for those we've witnessed on TV in other countries, but that doesn't eliminate the sense of fear, perhaps due to the mere gathering together of hundreds of people chanting, beating drugs and showing emotions in their faces if not with their fists and emphasis added to their messages by throwing their arm in the air. But there also is a sense of excitement. Several people commented on the protests remarking that they were pleased to see people standing up for their rights.

Thursday, February 24, 2011

One of the side effects of prolonged steroid usage such as a treatment for acute or chronic graph-versus-host disease is the possible infliction of Avasculor Necrosis (AVN), which is the death of bone (usually joints or long bones) due to the lack of blood supply.

Another side effect can be scar tissue on joints, particularly wrists, hands, fingers, hips, knees. Inflamation around these joins is similar to arthritis.

Treatments vary. One treatment path might include drugs, such as Sirolinus (a Rapamune drug).

Send a letter to your Senator: AAMDS.org; Donate to AAMDS.

Wednesday, February 23, 2011

Walker's Bill Proposes Permanent Changes

The items in Walker's bill in assembly at this moment are for permanent changes to Wisconsin laws.

The proposed changes will not end with his term.

Announcement of Recall Effort Ineffective, or Not?

Americans Against Immigrant Amnesty has filed electronic registration under the name American Recall Coalition in an effort to recall the eight Democractic Senators.

State law requires recalls to be filed by qualified electors from the legislator's district. A Utah based organization, person or company CANNOT legally file a petition for recall of a Senator in Wisconsin.

"The Government Accountability Board says that the group will need to find a 'sponsor' in each district, but that the 60-day clock started ticking with this group's filing of the paperwork." according to Fox 11.

The Democract Senators are:

Senators Fred Risser (D-Madison)
Mark Miller (D-Monona)
Jim Holperin (D-Conover)
Lena Taylor (D-Milwaukee)
Spencer Coggs (D-Milwaukee)
Robert Wirch (D-Pleasant Prairie)
Julie Lassa (D-Stevens Point) and
Dave Hansen (D-Green Bay).

Buffalo Beast

Walker talks offline to assumed supporter, BuffaloBeast reports it. Unfortunately, pinging that site shows that it was taken offline.

The conversation however is available at the daily mail: http://www.dailymail.co.uk/news/article-1359918/Wisconsin-Governor-Scott-Walker-duped-newspaper-editor-posing-billionaire-backer.html.

Contact Walker - Serve Wisconsin

Walker's contact information is:

Office of Governor Scott Walker
115 East Capitol
Madison WI 53702
Email: govgeneral@wisconsin.gov
Phone: (608) 266-1212

The Boards and Commissions for which the state of Wisconsin is seeking applicants is located at: Wisconsin Boards and Commissions. One of those commissions is the Emergency Medical Services Board, which is key to future changes to the Medical Assistance and Badger Care programs. Under Walker's bill, the Department of Health Services will have full control over the Medical Assistance program, and will be able to freely and carelessly create and pass laws under the Emergency Medical Services Board that effect benefits, including those needed for bone marrow transplants. If you can serve on that board, please apply right away.

Doctors, PA's, Nurses: your help is needed on that board; please consider serving it.

At minimum, everyone, please write Walker and voice your concerns over his Medical Assistance reform; tell him that we don't want Wisconsin playing God; we don't want any person in our state sentenced to death for lack of budgetary funds.

Medical Assistance Reform = Death Sentences By Legislative Pens?

An Arizona man was denied a liver that had been type-match perfectly to him after it was delivered to the hospital where he lay waiting for it to save his life because budget cuts to the medical assistance program couldn't afford to pay for it, nor could he as he was indigent.

See: Arizona man sentenced to death by legislative budget cuts

If you were thinking that wouldn't happen in Wisconsin, think again.

Governor Walker's proposed budget reform bill includes a medical assistance reform act that may limit or even eliminate Badger Care, Wisconsin's medical assistance program that pays for transplants.

Governor Walker's medical assistance reform act seeks to push through a change that doesn't appear all that harmful on its face, but when you read between the lines, it is a formula for deletion of the medical assistance benefits that save the lives of many, many people in Wisconsin. People who have lost their jobs, and thus lost their insurance benefits, often find themselves meeting the federal povery guidelines and qualifying for Badger Care. The bill cleverly excludes those who are disabled. But the reality of the situation of a person who needs a transplant is that they are not considered to be 'disabled' by the administrative definition of that word until the moment at which they receive the transplant, per the department of Social Security. His bill has the potential to eliminate transplants for Wisconsin people in need of them who could not afford to pay the price.

That person could be you.

Cut budgets, cut pay raises, increase the costs of pensions and insurance to state employees to equalize what the rest of the private sector is paying, but don't play God and take life from people who need transplants.

Obviously, people in need of joint or bone replacements would also fall under the 'excluded' list because neither would be considered to be 'disabled' until such time that they were receiving the replacement, hence no benefits would be available until after the transplant were done unless they already qualified due to being impoverished provided that those benefits were still available after the study and changes.

Solidarity

Firefighters joined teachers at the capital to protest Governor Walker's bill that removes some of the Teacher's Unions collective bargaining rights.

Meanwhile, Wisconsin National Guard members are visiting prisons to prepare to take over if prison guards walk off the job to join the protests.

Governor Walker's response in a public address this evening was to warn of layoff's if the Democrat legislators fail to return to work. Ultimately, Walker is placing the blame for some 5-6,000 layoffs anticipated immediately if the bill is not passed on the shoulders of the Democrats who ran away to Illinois.

Included in that bill is a Medical Assistance reform bill, noted below, which may affect those with diseases such as Aplastic Anemia and Leukemia whose lives are dependant upon costly medical procedures, namely bone marrow transplants.

2/3 Vote Signed Into Law For Tax Rate Increases

Current Wisconsin law requires a simple majority to pass tax rate increases.

Governor Walker signed a bill on Tuesday, February 22, 2011, requiring a two-thirds vote to pass tax rate increases.

Tuesday, February 22, 2011

Walker Addresses Wisconsin

Walker's address to Wisconsin.

One Life Matters - Aplastic Anemia Research: National Guard Preparedness

One Life Matters - Aplastic Anemia Research: National Guard Preparedness

Doctor's Note Might Get Jail Time

While teachers and the Teacher's Union are considering their options, the Governor has some answers.

One of the protestors claimed to be a doctor and offered 'sick notes' for teachers to get an excuse for being absent from work last Friday. While many of us dropped our jaws, there may be a judge dropping his gavel for criminal charges against that doctor and possibly against those receiving and using their 'sick notes'.

Dr. Pam Galloway, Wisconsin Senator, is a breast cancer surgeon. She has begun an investigation to determine if the doctors were acting alone, and found that they were in deed soliciting people for their sick notes.

The doctor claims that her investigation will cause the doctors to be investigated for criminal activity.

Walker Blocks Solidarity Website

Walker is accused of blocking a website that was being used to organize protestors.

Walker says, "I didn't block the website."

Let's keep the facts straight, folks!

Amid reports that the site -- defendwisconsin.org - was inaccessible on Monday and Tuesday morning, Wisconsin Democratic Party press secretary Graeme Zielinski pointed the finger at Walker and other Republicans, accusing them of a "direct assault on the First Amendment." A liberal blogger at the website ThinkProgress accused Walker of "taking a page from former Egyptian Dictator Hosni Mubarak and cutting off internet access to key protest organizers within the state Capitol building."

If a website is new, there is a chance that the Wisconsin administrative office responsible for Internet access may block it. If a site has viruses, or publishes threats, if it uses such a large amount of band width, it may cause its own issues with people accessing it.

National Guard Preparedness

Although Fox 11 reports that a spokesperson for the Wisconsin National Guard admitted that the guard was visiting prisons, it was merely in preparedness but not at the request of Governor Walker.

Let's keep the facts straight!!

Last week, Governor Walker requested that the National Guard be prepared to handle any and all situations should the needs arise given the announcement of his bill.

Governor Walker stated in an interview on video at Fox 11 that he didn't expect all of the publicity that came about because of his bill, particularly the national vision.

We find that calling on the National Guard infers that one expects shit to hit the fan, regardless of whether its thrown from Waupaun, Neenah, Green Bay, Richmond or Chicago!

Walker's Medical Assistance Bill May Affect Helpless

Neatly tucked away in the confines of obscure language is the Medical Assistance reform portion of Walker's bill that if read closedly reveals the means by which he provides the path to elimination of - or at the very least extreme cut-back's to - the Medical Assistance program in Wisconsin, known as Badger Care.

While an estimated 70,000 people voice their opinions about being forced into a rule-aligned bargaining agreement with the state of Wisconsin, who is speaking for the victims of diseases, foreign wars, mental disabilities, and poverty due to loss of jobs or injury?

Nobody.

That is sad.

While the Associated Press continuously erroneously reports the situation in Wisconsin (NOTE: all of our schools are in session today, with the exception of those which were snowed-under and delayed 2 hours), and there weren't 200,000 in Madison's capital but rather about 70,000 in and outside of the grounds, protesting their rights to collective bargaining, nobody is speaking out about the helpless people of our society. What is wrong with this picture?

Arizona sentenced a man to death with a denial of a liver transplant after a matched donor had been found because the state couldn't afford it.

What is wrong with this picture?

Put yourself in his shoes. No, put yourself IN his shoes. Do you envision it yet? Do you see that your life will end because you lost your job, nearly lost your home but for hard work and good financial planning you did before you got sick, and now without insurance must tell your little girl that you will leave her before the year ends. Do you see it? Do you feel it?

What are you doing about it?

Read Walker's Bill.

Public Assistance Reform under Walker

Walker's bill provides appropriation of TANF program funds from Federal Government:

PUBLIC ASSISTANCE
Reflecting the receipt of emergency contingency funds under the Temporary Assistance for Needy Families (TANF) block grant program, this bill increases by $37,000,000 the amount of TANF moneys allocated for the earned income tax credit.

Walker's bill also spins off extensive medical research and medical provisions, including blood stem cell research, leukemia research, bone marrow transplant facilities, research and funding, and the list goes on as to those areas in which UW-Madison has contributed through UW Hospitals and Clinics:

OTHER HEALTH AND HUMAN SERVICES
This bill eliminates the UW Hospitals and Clinics Board, a state agency assigned the single duty to enter into a contractual services agreement with the UW Hospitals and Clinics Authority to provide the services of state employees who are in clerical, blue collar and nonbuilding trades, building trades crafts, security and public safety, and technical collective bargaining units. The bill also transfers all employees of the UW Hospitals and Clinics Board to the UW Hospitals and Clinics Authority.

Walker's Medical Assistance Reform Act

Walker's bill on Medical Assistance
Although the language is illusive, the outcome is this: current law requires changes to Badger Care to be manifested from the Federal Government and administered locally. The new bill removes Federal law governance and places the administration and law making into one body under the state Governor. This change has the distinct possibility of extensively reducing benefits to Badger Care insured persons or revoking them. The bill's MA provisions are quoted verbatim below.

It is one thing to make state employees pay for benefits that the private sector can only dream about (pensions are all but history in the private sector) and the insurance benefits long enjoyed by state employees would make you drule, but it is quite another thing to play God.

In Arizona, a family man in need of a liver transplant to survive was denied the operation after a matched donor had been found. He will die before the end of this years. At some point while the legislature that sentenced him to death is driving around in his mercedes benz or playing with his children on his back lawn, the family man that was denied a transplant will explain to his elementary-aged daughter that he will die this year. Congratulations, Arizona, we didn't know God had entered your legislature. Congratulations, Wisconsin, you are not far behind.


HEALTH AND HUMAN SERVICES
MEDICAL ASSISTANCE

Under current law, DHS administers the Medical Assistance (MA) program, which is a joint federal and state program that provides health services to individuals who have limited resources. Some services are provided through programs that operate under a waiver of federal laws related to medical assistance (MA waiver programs).

This bill requires DHS to:

  1. study potential changes to the MA state plan and
  2. study potential changes to waivers of federal law relating to: medical assistance for certain purposes, including increasing the cost effectiveness and efficiency of care for the MA program and MA waiver programs and improving the health status of individuals who receive benefits under the MA program or an MA waiver program.
  3. If DHS determines, as a result of the study, that revision of existing statutes or rules would be necessary to advance any of the purposes for which the study was conducted, DHS may promulgate rules to implement certain changes, including making certain requirements, modifying benefits, revising provider reimbursement models, developing standards and methodologies for eligibility, and reducing income levels for purposes of determining eligibility.
  4. Before promulgating a rule, DHS must submit the proposed rule and any plan developed as a result of the study to JCF for review.
  5. DHS must submit an amendment to the state MA plan or request a waiver of federal laws related to medical assistance, if necessary, to the extent necessary to implement any proposal.
  6. If the federal Department of Health and Human Services does not allow the amendment or does not grant the waiver, DHS may not put the rule into effect or implement the proposal.
  7. To reduce the eligibility income levels to a certain amount, DHS must request a waiver from the secretary of the federal Department of Health and Human Services to permit DHS to have in effect eligibility standards, methodologies, and procedures that are more restrictive than those in place on March 23, 2010.
  8. If DHS does not receive approval for the waiver, DHS must reduce the eligibility income levels for MA programs and MA waiver programs to 133 percent of the federal poverty line for adults who are not pregnant and not disabled, as allowed under federal law.
  9. DHS may promulgate the rules as emergency rules.

PUBLIC ASSISTANCE
Reflecting the receipt of emergency contingency funds under the Temporary Assistance for Needy Families (TANF) block grant program, this bill increases by $37,000,000 the amount of TANF moneys allocated for the earned income tax credit.

Read the bill here (http://legis.wisconsin.gov/JR1SB-11.pdf)

Federal Poverty Levels (FPL)

The poverty income levels are used to determine enrollment for BadgerCare Plus. Those may be found at access.wi.gov or here.

One Life Matters - Aplastic Anemia Research: Walker Medical Assistance Hidden Agenda

One Life Matters - Aplastic Anemia Research: Walker Medical Assistance Hidden Agenda

Insurance Saves Lives

As Aplastic Anemia progresses to Severe Aplastic Anemia and then to Very Severe Aplastic Anemia, the patient will ultimately become 'disabled' although only for a period of time in the patient's lifetime.

As the patient's disease progresses, s/he will ultimately become unable to work due to the extreme exhaustion, very high susceptibility to infection, inability to concentrate and remember, and of course, anxiety. The typical patient will experience anxiousness, depression, and changes in blood pressure.

The average cost of a single bone marrow transplant is in excess of $150,000.00, and depending upon the treatments necessary before, during and after the transplant, often rise to the $200,000.00 to $300,000.00 ranges.

Medications necessary before, during and after the treatment are extensive. Typical costs range from $10,000.00 to $50,000.00.

A bone marrow transplant patient remains hospitalized in special facilities to protect their lives during the transplant when their immune systems are completely killed off and for approximately 20-30 days post transplant.

Patients attend clinic and receive blood draws regularly: usually two to three times per week immediately upon release from the hospital and for several months thereafter. Eventually, clinic appointments can be reduced to once to twice per week, and then eventually to once every other week, then monthly, then every three months, then every six months until they reach annual appointments at the four or five year mark.

From the time of release from the hospital until the patient is considered 'cured' of this terrible disease, the patient is on medication on a decreasing schedule. Some of those medications are as inexpensive as $1.00 each; most cost much more.

When a person receives a bone marrow transplant, they are considered temporarily disabled for Social Security purposes on the day of the transplant. They cannot return to work until released by their medical team. That release is subsequent to removal from immunosuppressant drugs.

Complications can increase costs, such as infections or development of Graph-versus-host disease.

Insurance, medical assistance programs, medical research, drug research, stem cell research and our wonderfully brilliant doctors save lives.

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.

Saturday, January 1, 2011

Federal Poverty Income Levels - Wisconsin 2011

Federal Poverty Levels (FPL)


The following income levels are
used to determine enrollment for BadgerCare Plus.  To see if you may be
able to enroll, go to access.wi.gov
and apply today.


Effective February 1, 2011












































































Family size 100% Monthly Income 150% Monthly income 200% Monthly income 300% Monthly income
1 $   907.50 $1,361.25 $1,815.00 $2,722.50
2 $1,225.83 $1,838.75 $2,451.67 $3,677.50
3 $1,544.17 $2,316.25 $3,088.33 $4,632.50
4 $1,862.50 $2,793.75 $3,725.00 $5,587.50
5 $2,180.83 $3,271.25 $4,361.67 $6,542.50
6 $2,499.17 $3,748.75 $4,998.33 $7,497.50
7 $2,817.50 $4,226.25 $5,635.00 $8,452.50
8 $3,135.83 $4,703.72 $6,271.67 $9,407.50

For each additional person, add:

$318.33 $477.50 $636.67 $955

Saturday, December 19, 2009

Hickmann Catheter Infection

Can an infection develop in the Hickmann catheter and not show up in a grown culture or on a slide?

Yes. Contrary to popular belief, infections can actively exist in the body (meaning that they are alive and growing) and still not be detectable in a set of cultures or on a slide.

There are several reasons why this can occur. One of those reasons is that the cultures and slides are not looking for the correct type of infection.

What is done?

If the infection is believed to be treatable, the catheter will likely be left in if at all possible so that medication can be infused into the body more easily.

If the infection either cannot be treated, or has reoccured, then the hickmann will likely be removed.

Options

One alternative to a hickmann is an IV. If the patient is being treated with chemotherapy or other drugs after chemo treatment, an IV may not be the ideal route because the veins may not be able to handle the intense infusion of drugs, as well as other reasons. Sometimes, an IV will be placed in each arm temporarily. Once the infection is cleared, a PIC line can be implemented, and the IV's removed.

Saturday, October 17, 2009

Lung Injury After Transplant

Transfusion related acute lung injury (TRALI) is an uncommon but potentially fatal adverse reaction to transfusion of plasma containing blood components. We describe a case of 10-year-old male child with aplastic anemia, platelet count of 7800/l, B positive blood group who developed fever (39.2C), difficulty in breathing and cyanosis within 2 hrs after transfusion of a random platelet concentrate. Despite the best resuscitative efforts, the child died within next 24 hrs. The present case highlights the fact that TRALI should be kept as a differential diagnosis in all patients developing acute respiratory discomfort within 6 hrs of transfusion. Without a 'gold standard' the diagnosis of TRALI relies on a high index of suspicion and on excluding other types of transfusion reactions. Notification to transfusion services is crucial to ensure that a proper investigation is carried out and at-risk donor and recipients can be identified, and risk reduction measures can be adopted.

http://www.ncbi.nlm.nih.gov/pubmed/19805974?ordinalpos=7&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_DefaultReportPanel.Pubmed_RVDocSum

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

Cyclophosphamide Results

OBJECTIVE:
To determine if high-dose cyclophosphamide is an effective therapy for children with refractory severe aplastic anemia (SAA).

BACKGROUND:
SAA is an illness characterized by the depletion of hematopoietic precursors associated with life-threatening complications. Hematopoietic stem cell transplant (HSCT) is the treatment of choice if a human leukocyte antigen (HLA)-related donor is available. Immunosuppression with anti-thymocyte globulin (ATG) and cyclosporine A (CSA) is an option for patients who are not HSCT candidates. Unrelated donor HSCT has been used with limited success. High-dose cyclophosphamide has been used successfully in the treatment of adults with SAA, but experience in children is limited.

PROCEDURE:
Five pediatric patients who had failed previous immunosuppressive therapy for SAA were treated with high-dose cyclophosphamide (45 mg/kg/day x 4 days).

RESULTS:
After 12 months of treatment, two of five patients experienced a complete response with high-dose cyclophosphamide therapy. The two complete responders achieved red cell recovery with a hematocrit of >36% at days 212 and 112 and platelet recovery with a platelet count of >100 x 10(9)/L at days 126 and 324. Of the remaining patients, one patient failed to respond, and two patients expired from infectious complications. CONCLUSIONS: High-dose cyclophosphamide can lead to complete responses in children with SAA who have failed to respond to traditional immunosuppressive therapy. Pediatr Blood Cancer (c) 2009 Wiley-Liss, Inc.

http://www.ncbi.nlm.nih.gov/pubmed/19827142?ordinalpos=2&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).

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

A Study - Transplant Treatment Severe Aplastic Anemia - Journal of American Society of Hemotology

Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality


BM Camitta, ED Thomas, DG Nathan, G Santos, EC Gordon-Smith, RP Gale, JM Rappeport and R Storb

A prospective randomized trial of therapy for severe aplastic anemia was designed to compare early bone marrow transplantation with conventional treatments. All patients with a sibling matched at the major histocompatibility region were transplanted. Transplantation was performed with 17-100 (median 33) days of original diagnosis. Conventional treatments included transfusion support with or without androgens. Twenty-four of 36 patients intered on the transplant arm are alive after 4-20 (median 9) mo with full marrow reconstitution. Only two are limited by chronic graft-versus-host disease. In contrast only 12 of 31 conventionally treated patients are alive. Six of these survivors have improved, five incompletely. The 19 nontransplant deaths have occurred within 1-11 (median 3) mo of diagnosis. Compared to nontransplant regimens, early transplantation more effectively restores normal marrow function and decreases the acute mortality of severe marrow aplasia (p = 0.006). Pending longer follow-up, early marrow transplantation appears to be the most effective available treatment for severe aplastic anemia.

SUMMARY
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, an excellent citation. 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.


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).

Initial complication was sepsis (https://www.google.com/health/ref/Sepsis).

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

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.

Allogeneic Stem Cell Transplant

What is a stem cell transplant?

Most stem cells are in your bone marrow. You also have some that circulate from your marrow into your blood. Bone marrow stem cells turn into red blood cells, white blood cells, or platelets to help your body stay healthy. If your bone marrow is damaged or destroyed, it can no longer make normal blood cells. In a stem cell transplant, healthy stem cells are placed in your body through an IV to help your bone marrow start to work right.

When the stem cells come from another person, it is called an allogeneic transplant. The donor may be a relative or a complete stranger. The important thing is that the donor's immune system markers are closely matched to yours. This is more likely when the donor is your brother or sister.

When the stem cells come from your own blood or bone marrow, it is called an autologous transplant.

Stem cells can also be found in your bloodstream and in the blood inside a newborn's umbilical cord.

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/

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.

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.

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.

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:
  • 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
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).