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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.
CLINICAL TRIALS AND OBSERVATIONS Single-agent lenalidomide induces complete remission of acute myeloid leukemia in patients with isolated trisomy 131 Department of Internal Medicine, Division of Oncology, Siteman Cancer Center, Washington University, St Louis, MO; 2 Department of Medicine, Division of Hematology and Oncology, Comprehensive Cancer Center, 3 Division of Medicinal Chemistry, College of Pharmacy, and 4 Division of Pharmaceutics, College of Pharmacy, The Ohio State University, Columbus; and 5 Department of Pathology and Immunology, Siteman Cancer Center, Washington University, St Louis, MO 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 2 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 identifiers NCT00466895 [ClinicalTrials.gov] and NCT00546897 [ClinicalTrials.gov] .
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