Jeffrey J. Molldrem, MD; Eric Leifer, PhD; Erkut Bahceci, MD; Yogen Saunthararajah, MD; Mary Rivera, RN; Cynthia Dunbar, MD; Johnson Liu, MD; Riotoro Nakamura, MD; Neal S. Young, MD; A. John Barrett, MD
Molldrem JJ, Leifer E, Bahceci E, Saunthararajah Y, Rivera M, Dunbar C, et al. Antithymocyte Globulin for Treatment of the Bone Marrow Failure Associated with Myelodysplastic Syndromes. Ann Intern Med. 2002;137:156-163. doi: 10.7326/0003-4819-137-3-200208060-00007
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Published: Ann Intern Med. 2002;137(3):156-163.
Myelodysplastic syndromes are bone marrow disorders that are characterized by ineffective hematopoiesis and leukemic transformation.
Almost half of the deaths caused by these syndromes are from cytopenia.
Standard supportive care (red blood cell and platelet transfusions and hematopoietic growth factors) often fails.
This prospective case series found that one third of 61 patients with red blood cell transfusion-dependent myelodysplastic syndrome became transfusion independent within 8 months of a 4-day course of intravenous antithymocyte globulin.
Before instituting treatment changes on the basis of these exciting preliminary findings, physicians should watch for controlled studies that compare transfusion and survival outcomes in patients treated with antithymocyte globulin and patients given usual care (or other therapies).
Five-year probability of survival was 76% (95% CI, 64% to 88%). Dotted lines represent the upper and lower boundaries of confidence intervals. Covariate-adjusted survival for responders and nonresponders. Response status was ascertained at 8 months after treatment with antithymocyte globulin.
Five-year probability of remaining progression free was 90% (95% CI, 80% to 100%). Dotted lines represent the upper and lower boundaries of confidence intervals. Covariate-adjusted time to progression for responders and nonresponders. Response status was ascertained at 8 months after treatment with antithymocyte globulin.
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