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HEMATOLOGY-ONCOLOGY -58-Hemato-Oncology Main Messages – Summary 9 Aplastic Anemia


(All the articles published in past are available at
Question: Dr. Chiragbhai, thank you very much for helping us summarize this complex topic. In last part we covered myeloproliferative neoplasms, including some interesting new medicines like Ruxolitinib. BEFORE WE PROCEED, LET ME CONGRATULATE YOU FOR YOUR BOOK ON CANCER AWARENESS in GUJARATI language, covering Blood, Breast, Gastrointestinal cancers. For our readers, it is available through publisher at 7926620472/09879500179.
Ans: Let us continue our discussion to cover Aplastic Anemia. This is a non cancer disease but frequently more deadly than many cancers. Let us review major points in diagnosis and treatment of this common disease, which can be treated very well in most cases, if timely diagnosed and if decisions are made in time regarding which treatment to choose.
It is important to remember that search for secondary causes should be done but briefly. Most cases are idiopathic and even with most drug induced aplastic anemias or post infectious aplastic anemias, standard aplastic anemia treatment should be started quickly, otherwise there is a serious risk of death from sepsis or hemorrhage. Trephine biopsy from bone marrow is mandatory for diagnosis.
STEROIDS should NOT be used for aplastic anemia treatment. This is to be strongly discouraged. Also, prophylactic platelet transfusions are widely over utilized in these patients. Platelet transfusions should be given only if there are significant bleeding manifestations (not just petechiae or bruises), or for platelet counts below 5000. SDP is preferred especially for patients awaiting transplant, to reduce exposure to number of donors. For red cell transfusions also, goal should be symptom relief, not some prespecified number. Thus, if patient is asymptomatic at Hb of 6, he should not be transfused. All blood products should preferably be leukoreduced, however cost is an important consideration in India. Treatment options are:
1. Transplant: for severe or very severe grade, and if patient is less than 50 (and fit) years age. Transplant long term success is 80-90% if done early. It also provides protection against development of long term secondary disorders like MDS, PNH etc which are seen after non transplant treatment options. Should be done early rather than late. Less the number of transfusions better the result. Minimize number of transfusions and use leukoreduction pre transplant to improve results. Timely referral is crucial in saving life of patients with severe aplastic anemia i.e. those with absolute neutrophil count <500, or platelet <20,000. These patients frequently die in first 2 months since diagnosis, and ATG response is often too slow to save their life. For patients who do not have a matched sibling, search should be done to find a voluntary stem cell donor through registries. Results of these MUD transplants are also almost equivalent to matched sibling transplants in experienced centers.
2. ATG and Cyclosporine: for patients who do not fit above, or where transplant is not feasible due to personal financial or other reasons, ATG followed by cyclosporine is the treatment of choice with long term success in about 60% patients. Main drawback is slow response, which may take up to 3 months. HORSE ATG is better than rabbit ATG based on a recent trial. Some patients need very long term cyclosporine continuation, and many patients have partial remissions with transfusion independence but not a complete normalization of their CBC.
3. Other options: for those who cannot afford ATG or very elderly (over 80 or over 60 but with serious comorbidities making them unfit for ATG), only cyclosporine is an option. It rarely provides complete durable remission however. Recently Eltrombopag, an oral TPO agonist drug, has been approved for aplastic anemia. It provides remission in some patients after a prolonged use of many months. Cost however is an issue.
4. Relapsed/Refractory aplastic anemia: for these patients, options include Transplant if not done earlier (or even a second transplant), second course of ATG (either rabbit or horse), alemtuzumab, eltrombopag, and various other immune suppressants or immune modulators.
December 14th, 2014.

Dr. Chirag A. Shah;M.D. Oncology/Hematology (USA), 079 26754001. Diplomate American Board of Oncology and Hematology. Ahmedabad.

Shyam Hem-Onc Clinic. 402 Galaxy, Near Shivranjani, Opp Jhansi ki Rani BRTS, Ahmedabad.

Comments (2)

  1. dear sir
    It is a relief to hear of an issue that has been bother some in my life my mother is 69 years diagnosed of anemia but can not find the cause and it’s been two years she has been loosing blood, the only reason she is still alive is because of transfusion, I am African from Zimbabwe and needs to find a cure for her, we have been in and out of doctors, please help!

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