Aplastic Anemia & MDS International Foundation, Inc.
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Prognosis & Disease Evolution


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  What To Expect When Illness Progresses
  Remission and Stem Cell Harvest
  Risks of Aplastic Anemia Developing Into MDS
  Reversing Chromosome Damage
  MDS Progressing to Leukemia
  Liver Problems Associated with Diseases
  Iron Supplements
  Spleen Removal in MDS

What To Expect When Illness Progresses

What can patients and family members expect when the illness progresses to the end-stages of a patient's life?

Patients and family members should express their concerns and direct their questions to their doctor because each case is unique. Also, contact our Patient Information Department at the Aplastic Anemia & MDS International Foundation who will discuss these issues and can put you in touch with other family members who will share their experiences.  Call our toll-free help line at 800.747.2820 or help@aamds.org.


Remission and Stem Cell Harvest

How do you define "remission" for a patient with aplastic anemia in terms of being able to harvest stem cells?

Remission means normal blood counts and normal numbers and functions of

Cells that are produced in the bone marrow and differentiate into red cells, white cells, and platelets.
stem cells. Many people believe that (in most persons) stem cells never return to normal in both numbers and functions. Therefore, harvesting one's own stem cells as "insurance" would be considered investigational.


Risks of Aplastic Anemia Developing Into MDS

What percentage of aplastic anemia develops into MDS?

It varies from among studies with the original treatment and follow-up. In some studies, 35-50% of patients who are followed for 10-20 years have developed MDS or AML. In other studies, the frequency of these complications is less than half of these numbers.


Reversing Chromosome Damage

Do damaged chromosomes reverse themselves after successfully being treated with medical therapy for MDS?

The chromosome changes may "disappear" in a patient who enters remission with medical therapy. This is probably not a cure but just a decrease in the level of abnormal cells to levels that are not detectable by standard methods. With transplantation, there may be true eradication of cells with abnormal chromosomes.


MDS Progressing to Leukemia

Does ATG treatment for MDS have anything to do with development of leukemia or does MDS progress to leukemia in  some patients?

The published studies of

ATG (Atgam, Thymoglobulin) is an immunosuppressant, a drug therapy that reduces the body's natural immunity. It is approved by U.S. Food and Drug Administration (FDA) for treating moderate to severe aplastic anemia patients. ATG is most commonly used with cyclosporine, an oral medicine. ATG/cyclosporine combination is sometimes used for treating myelodysplastic syndromes (MDS), but is not nearly as effective against MDS as it is aplastic anemia.
anti-thymocyte globulin(ATG) administration in myelodysplastic syndromes (MDS) patients have not reported an increased risk of disease progression.
The majority of case of MDS will not turn into acute leukemia. The risk factors present at the time of MDS diagnosis can predict the disease course in many patients. Those MDS patients with a high risk of developing AML (based on the percentage of bone marrow
Immature cells that mature into the three types of blood cells.
blasts
and the types of chromosome abnormalities present) probably will not benefit from ATG administration.


Liver Problems Associated with Diseases

Are problems with the liver or kidney function frequently associated with aplastic anemia and myelodysplastic syndromes?

Though liver or kidney problems are not typically caused by aplastic anemia or MDS, they can occur in patients for many reasons. Disease-related reasons could include iron overload from multiple blood transufions (liver) or infection or medication (e.g. ara-C can cause hepatoxicity). Liver or kidney injury can result from

Cyclosporine (Neoral, Sandimmune) is an immunosuppressant, a drug that reduces the body's natural immunity. Cyclosporine is used along with anti-thymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia.
cyclosporine, but these effects are at least partially reversible by dose adjustment. Most often, liver or kidney problems are un-related to the diseases and have to do with underlying disease conditions.


Iron Supplements

If a person was receiving oral iron supplements for anemia for several years before diagnosis of pure red cell aplasia, could supplemental iron contribute to bone marrow damage or other organ damage?

Doses of iron that are used in typical "tonics" do not contain enough iron to cause problems unless the patient has an inherited or acquired problem with iron absorption (h

An excess of iron deposits in the body, also known as iron overload.
emochromatosis) or
The oxygen carrying cells in our body. These bring oxygen to our tissues, and are the most numerous of the blood cells.
red blood cells
production (aplastic anemia, pure red cell aplasia). Once one of these problems is diagnosed then supplemental iron would be contraindicated.


Spleen Removal in MDS

When and why would spleen removal be recommended in MDS? What are the general risks and benefits of splenectomy in MDS?

In some subtypes of myelodysplastic syndromes (MDS), such as chronic myelomonocytic leukemia (CMML), the spleen can become very large. When it reaches 20 to 25 cm in length, it can cause reductions in peripheral blood counts. At that point, low dose radiation and/or removal of the spleen may be indicated, especially if it has become large enough to cause vascular problems.

Splenectomy is generally well tolerated in experienced hands, but requires preparation (respiratory exercise) and approximately a month of recovery time.


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