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Blood, 29 January 2009, Vol. 113, No. 5, pp. 1002-1005.
Prepublished online as a Blood First Edition Paper on September 29, 2008; DOI 10.1182/blood-2008-04-152678.
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Submitted April 24, 2008
Accepted September 5, 2008
Single agent lenalidomide induces complete remission of acute myeloid leukemia in patients with isolated trisomy 13
Todd A. Fehniger, John C. Byrd, Guido Marcucci, Camille N. Abboud, Cheryl Kefauver, Jacqueline E. Payton, Ravi Vij, and William Blum*
Department of Internal Medicine, Division of Oncology, Sitemen Cancer Center, Washington University, St. Louis, MO, United States
Division of Medicinal Chemistry, College of Pharmacy, The Ohio State University, Columbus, OH, United States
Division of Pharmaceutics, College of Pharmacy, The Ohio State University, Columbus, OH, United States
Department of Medicine, Division of Hematology/Oncology, Comprehensive Cancer Center, The Ohio State University, Columbus, OH, United States
Department of Pathology and Immunology, Sitemen Cancer Center, Washington University, St. Louis, MO, United States
* Corresponding author; email: william.blum{at}osumc.edu.
Patients with acute myeloid leukemia (AML) frequently fail chemotherapy due to refractory disease, relapse, or toxicity. Among older AML patients (age >60 years), there are few long-term survivors. Lenalidomide is a candidate for study in AML based on its clinical activity in a related disorder, myelodysplastic syndrome (MDS) with the 5q- chromosomal abnormality. We report induction of sustained morphologic and cytogenetic complete remission in two older AML patients treated with high-dose, single agent lenalidomide; each patient had trisomy 13 as the sole cytogenetic abnormality. We show for the first time that lenalidomide has clinical activity in this poor-risk cytogenetic subset of AML. The clinical trials described in this paper have been registered with www.clinicaltrials.gov under identifier NCT00466895 and NCT00546897.

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