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Rebecca Olin, MD, MS


Is Lenalidomide Effective in Pediatric Patients With Resistant AML?

By: Vanessa A. Carter, BS
Posted: Tuesday, May 25, 2021

Anders Kolb, MD, of Nemours Center for Cancer and Blood Disorders, Alfred I. duPont Hospital for Children, Wilmington, Delaware, and colleagues conducted a study to whether lenalidomide would be a safe and effective treatment for pediatric patients with relapsed or refractory acute myeloid leukemia (AML). Although the safety data were reported to be similar to the safety profile of lenalidomide, only 1 of 17 patients responded to the therapy. Their findings were published in Pediatric Blood & Cancer.

The investigators focused on 17 patients with relapsed or refractory AML treated with two or more prior therapies and had at least 5% leukemic blasts in their bone marrow. The median patient age was 12 years (range, 5–18 years). Participants were administered lenalidomide for a maximum of 12 cycles, starting with 2 mg/kg/day for 21 days of a 28-day cycle. Treatment was continued until toxicity, adverse events, pregnancy, compliance withdrawal, or death occurred.

The median white blood cell count was 3.7 x 109/L, and the median peripheral blood blast count was 1.0 x 109/L. A majority of patients (76.5%) had at least one cytogenetic abnormality, and five patients had a complex karyotype with three or more abnormalities. Only one patient, a 13-year-old boy, achieved morphologic complete response after two treatment cycles, hematopoietic stem cell transplantation, and salvage chemotherapy; he has not displayed evidence of disease for 3 years. All other patients experienced treatment failure, mostly due to resistant disease (n = 12).

Grade 3 to 4 treatment-related adverse events affected every participant, with the most common being thrombocytopenia (58.8%), febrile neutropenia (47.1%), anemia (41.2%), and hypokalemia (41.2%). Serious treatment-related adverse events were observed in 13 patients, 3 of whom required dose reduction. One single fatal grade 5 event was reported.

Disclosure: For full disclosures of the study authors, visit

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