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Safety and efficacy of azacitidine in the treatment of elderly patients with myelodysplastic syndrome

Authors Ritchie E

Received 29 February 2012

Accepted for publication 16 April 2012

Published 19 June 2012 Volume 2012:7 Pages 165—173

DOI https://doi.org/10.2147/CIA.S24659

Review by Single-blind

Peer reviewer comments 4


Ellen K Ritchie

Leukemia Program, Weill Medical College of Cornell University and The New York Presbyterian Hospital, New York, NY, USA

Abstract: The goals of treating older patients with myelodysplastic syndrome (MDS) are different than for younger patients. Few elderly patients are able to pursue an allogeneic stem cell transplant for potential cure of the disease. The focus for the treatment of older patients with MDS is therefore not curative, but rather alleviation of symptoms, improvement in quality of life, maintenance or improvement of functional status, and continued independent living. Prolongation of survival is only important if functional status and quality of life can be maintained, and there is greater risk of losing these outcomes in elderly patients. Azacitidine is an important drug for the treatment of older patients with MDS. Data from the AZA-001 trial has shown a survival benefit for patients with high-risk disease treated with azacitidine. Importantly, treatment has also been shown to improve quality of life for MDS patients. Subset analysis of the data has shown that the drug can be used safely in even the oldest patients with MDS and is superior to treatment with other established regimens, such as low-dose cytarabine. Given the delay between the initiation of treatment and the clinical response, patients may need aggressive supportive care with antiemetics, prophylactic antibiotics, and transfusions to maintain them through therapy. Azacitidine provides a better quality of response when it is used beyond the first response, so ongoing treatment is generally recommended in responding patients. A new oral preparation of the drug is in development that will make the treatment more feasible and comfortable for elderly patients.

Keywords: geriatrics, myelodysplastic syndrome, acute myeloid leukemia, azacitidine, supportive care

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