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Hemato-Oncology-25-Myelodysplastic Syndrome-Treatment (1)



Welcome to the twenty fifth part of a series on Hemato-oncology.

Question: Dr. Chiragbhai, thank you for explaining the details of making MDS diagnosis. What are the treatment options available for this disease?

Answer: MDS is a difficult disorder to treat. Only curative option is Stem Cell Transplant/Bone Marrow Transplant. Other options provide temporary relief in a number of cases. Three new options have been recently approved – all non curative however, and some of them very expensive.

Goal of treatment is to provide hematological improvement in LOW risk cases and try to alter natural history of disease in HIGH risk cases. Many prognostic scoring systems are available to help in making these decisions. Most commonly used now is IPSS – based on clinical criteria i.e. blood counts, cytogenetics, and blast count. Low risk cases (low and INT-1 risk categories) are offered LOW intensity therapies, and High risk (INT-2, and high risk categories) are offered HIGH intensity therapies.

LOW intensity therapies include erythropoietin, G-CSF, cyclosporine, anabolic steroids, blood transfusions, azacytidine, decitabine, lenalidomide, ATG.

HIGH intensity therapies include Stem Cell Transplant if feasible (preferred option), chemotherapy in doses used for leukemia.

Most patients are old, with frequent comorbidities. Hence it is important to evaluate these and treat them along side. Certain scoring systems help in evaluating the likely risk from these comorbidities, such as diabetes, heart disease, hypertension…This is especially important for high intensity therapies.

Que: Why do you say that MDS is difficult to treat?

Ans: Yes, even though there are many options to choose from now, only curative option remains Transplant. It is not feasible for many old patients, especially with comorbidities. Recently a number of centers in USA are treating such old patients with RIC transplant i.e. transplant using milder and less chemotherapy. Still cure rate from transplant is not more than 40%. Other options are all non curative and response rates are in range of only about 20-50%.


For some patients, e.g. those with del 5q cytogenetic abnormality, thalidomide or lenalidomide is very effective in controlling disease for a long time with just one tablet per day. Also, milder forms of MDS e.g. RA, RARS also live long with minimal treatment. MDS with hypoplastic marrow responds better to treatments used for aplastic anemia, such as ATG and cyclosporine.


New medicine Azacytidine has shown significant increase in survival in high risk patients, from 15 months to 25 months. Decitabine, other new medicine has good response rate but no increase in survival. Lenalidomide is the 3rd new medicine.



Dr. Chirag A. Shah; M.D. Oncology/Hematology (USA), 98243 12144, 98988 31496

Diplomate American Board of Oncology and Hematology. Ahmedabad. Shyam Hem-Onc Clinic. 402 Galaxy, Near Shivranjani, Opp Jhansi ki Rani BRTS, Ahmedabad.