Aplastic Anemia Association of India
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FAQ

TREATMENT RELATED FAQ

  1. What are treatments available for Aplastic Anemia patients?
  2. What is Immunosuppressive therapy?
  3. What is difference between ATG and ALG?
  4. How does ATG/ALG work?
  5. How can I know whether Immunosuppressive Drugs is successful or not?
  6. Should a patient be treated with ATG/ALG second time?
  7. What are the side effects of ATG/ ALG?
  8. What is Cyclosporine (Neoral)?
  9. What are the side effects of Cyclosporine (Neoral)?
  10. What is Supportive Treatment?
  11. What is Blood Transfusion?
  12. What things to keep in mind for Blood Transfusion?
  13. Why it is required to use blood filters (Leukocyte filters)?
  14. Where can I buy the blood/platelets filters?
  15. What is the life of Red blood cells (RBC)?
  16. What is life of White Blood Cells (WBC)?
  17. What are side effects of blood transfusions?
  18. What is Iron Overloading?
  19. How is Iron removed?
  20. What is Complete Blood Count (CBC)?
  21. What is platelets transfusion?
  22. What are Growth Factors?
  23. Are Growth Factors successful in Aplastic Anemia Patients?
  24. What is BMT (Bone Marrow Transplant)?
  25. How to reduce the pain during the puncture for blood and platelets transfusion?
  26. What is EMLA gel?
  27. Where do you get EMLA gel in India?
  28. What are Clinical Trails?
  29. What are the website to learn more about clinical trials?

Q1: What are treatments available for Aplastic Anemia patients?

A:

  • Immunosuppressive therapy (ATG or ALG, Cyclosporine)
  • Supportive Treatment (platelets and blood transfusion)
  • Growth Factors
  • Bone marrow Transplant

Q2: What is Immunosuppressive therapy?

A: Immunosuppressive therapy means AntiThymocyte globulin (ATG) or AntiLymphocyte globulin (ALG) and/or cyclosporine.

Q3: What is difference between ATG and ALG?

A: ATG is horse serum while ALG is rabbit serum.

Q4: How does ATG/ALG work?

A: ATG/ALG targets T-lymphocytes—the cells that are responsible for destroying or suppressing the stem cells of patients with Aplastic anemia. Horse ATG is typically given intravenously over four days for approximately four hours a day and ALG for five days. This schedule may vary depending on your needs or the particular methods of your doctor or hospital. ATG/ALG therapy used alone is helpful about half the time. When it is used in conjunction with Cyclosporine, the chances of the treatment working increase to about 70 percent and higher.

Q5: How can I know whether Immunosuppressive Drugs is successful or not?

A: If it is successful, ATG/ALG will usually eliminate the need for transfusions within two to three months, and patients will feel well. Blood counts rise within several months, some rising dramatically, some more slowly. Response times can vary greatly, however, and some patients experienced delayed response times of up to nine months or even longer. Blood counts, however, may still be below normal.

Q6: Should a patient be treated with ATG/ALG second time?

A: Some patients who respond to ATG/ALG may need to have treatment again because of falling blood counts. Some patients who do not respond to ATG/ALG the first time will respond if the treatment is repeated. (Ask the doctor). Mostly, Doctors have opinion that since it didn’t work for first time, there are hardly chances for its success for the second time.

Q7: What are the side effects of ATG/ ALG?

A: ATG/ALG has a number of side effects. You should discuss these with your doctor. Less serious side effects include fever, chills, and hives. A rare but severe side effect is anaphylaxis, a life-threatening allergic reaction. Patients may be skin tested to determine if they are likely to develop allergies. If you are, you can still receive ATG/ALG therapy, but you will need to undergo a procedure called desensitization, in which small, gradually increasing doses of ATG/ALG are given, reducing the body’s allergic reaction to the drug. Serum sickness is another type of reaction against a foreign protein. It causes rash and joint and muscle aches. Corticosteroids are usually given to patients to reduce the chances of their getting serum sickness or to reduce its severity.

Q8: What is Cyclosporine (Neoral)?

A: Cyclosporine is given with ATG/ALG and is another immunosuppressive drug that targets Tlymphocyctes. Cyclosporine used alone is less successful than ATG /ALG alone, and less successful than ATG / ALG used in combination with cyclosporine. Cyclosporine comes in liquid and pill forms. The first doses will be based on your body weight. Subsequent doses will be adjusted based on the amount of the medicine in your blood. Too much of the drug will cause side effects and too little won’t be effective.

Q9: What are the side effects of Cyclosporine (Neoral)?

A: Two of the most serious side effects are kidney damage and high blood pressure. Both of these problems, however, are usually easy to manage and are reversible. You may need to take a blood pressure lowering medication while on cyclosporine as well as a supplement of magnesium. You will generally need to continue taking cyclosporine for months or even years. If you have a stable, positive response to the drug, your doctor will gradually decrease the dosage.

Q10: What is Supportive Treatment?

A: Supportive treatment includes blood and platelets transfusion.

Q11: What is Blood Transfusion?

A: If red blood cell is low, patients may need to receive transfusions. Important advice about transfusions: do not ask close family members to donate red blood cells until after a bone marrow transplant has been done or ruled out. This is because if a family member turns out to be the best bone marrow donor, a prior donation from a family member could reduce the chances for a successful transplant.

Q12: What things to keep in mind for blood transfusion?

A: Blood transfusions may contain viruses or other infections that may be passed to the recipient. Blood products used for transfusion are usually treated in some way (such as being irradiated or filtered) to remove leukocytes. These measures reduce the risk of certain complications from transfusion— irradiation minimizes the risk of graft-versus-host disease.

Q13: Why it is required to use blood filters (Leukocyte filters)?

A: Leukocyte filters decrease the risk of sensitization to proteins present in the transfused blood product, and also reduce the risk of transmitting cytomegalovirus. Irradiation and filtering are important in cases where the patients are severely immunosuppressed.

Q14: Where can I buy the blood/platelets filters?

A: Blood/platelets filters are available from medical stores at Marine Lines, Bombay (as far as I know). Blood Filters are around Rs.1000 and Platelets filters around Rs.1700. It is suggested to buy these from wholesale medical dealers as you can get a good deal. Please contact me for more information

Q15: What is the life of Red blood cells (RBC)?

A: RBC has a life span of 120 days.

Q16: What is life of White Blood Cells (WBC)?

A: White blood cells have a very short life span of only a few hours.

Q17: What are side effects of blood transfusions?

A: One side effect of blood transfusion is Iron Overloading.

Q18: What is Iron Overloading?

A: If you receive regular red cell transfusions damaging amounts of iron will begin to collect in key organs such as the heart and liver. This is called iron overload (Hemochromatosis). Untreated, this can lead to severe organ damage that can be fatal.

Q19: How is Iron removed?

A: Iron can be removed by treatment with metal chelators like Desferal.

Q20: What is Complete Blood Count (CBC)?

A: Refer to this link to understand Complete Blood Count. http://www.cc.nih.gov/ccc/patient_education/pepubs/cbc97.pdf

Q21: What is platelets transfusion?

A: Refer the platelets FAQ.

Q22: What are Growth Factors?

A: Growth factors are normal body products that encourage production of blood cells. Granulocyte colony-stimulating factor (G-CSF) and granulocyte macrophage colony-stimulating factor (GM-CSF) have been the most effective growth factors for increasing white cell production.

Q23: Are Growth Factors successful in Aplastic Anemia Patients?

A: To date, use of growth factors like erythropoietin to increase red blood cells and platelets has not been particularly successful in patients with Aplastic anemia. It is important to remember that growth factors do not cure Aplastic anemia. Rather, they may help damaged bone marrow to work better until other treatments have a chance to reverse the processes causing the marrow damage.

Q24: What is BMT (Bone Marrow Transplant)?

A: Please refer Bone Marrow FAQ.

Q25: How to reduce the pain during the puncture for blood and platelets transfusion?

A: Use EMLA gel

Q26: What is EMLA gel?

A: EMLA (idocaine & prilocaine) is topical anesthetic used for those people who are very sensitive to pain It is a prescription gel that comes with its own bandaids. (In India, we get small EMLA tube). It is applied to the area of puncture at least one hour before needle insertion takes place. The result is decreased discomfort with the needle insertion.

Q27: Where do you get EMLA gel in India?

A: You get EMLA gel from medical stores at Marine Lines, Bombay (opposite to Bombay Hospital). Each tube cost nearly Rs.200. It is very expensive

Q28: What are Clinical Trails?

A: For patients who have not responded to standard treatments, new treatments may be tested without any comparison group. You should consider participating in a clinical trial only after your doctor has explained the trial’s specific purposes, risks, and benefits. Clinical trials offer some of the best medical care available. Trials not only provide the latest and best medical expertise, but patients receive close attention and monitoring. Most insurance companies cover treatment done within a clinical trial - if not, first treatment is free of charge at the National Institutes of Health.

Q29: What are the website to learn more about clinical trials?

A: www.aamds.org
    www.clinicaltrials.gov
    www.centerwatch.gov
    www.nih.gov

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