Lenalidomide and prednisone for myelofibrosis: Eastern Cooperative Oncology Group (ECOG) phase 2 trial E4903

• Published in: Blood Vol. 116; no. 22; pp. 4436 - 4438
• Main Authors: Mesa, Ruben A., Yao, Xiaopan, Cripe, Larry D., Li, Chin Yang, Litzow, Mark, Paietta, Elisabeth, Rowe, Jacob M., Tefferi, Ayalew, Tallman, Martin S.
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 25.11.2010; Americain Society of Hematology; American Society of Hematology
• Series: Clinical Trials and Observations
• Subjects: Antineoplastic Agents - adverse effects; Antineoplastic Agents - therapeutic use; Antineoplastic Combined Chemotherapy Protocols; Biological and medical sciences; Clinical Trials and Observations; Follow-Up Studies; Hematologic and hematopoietic diseases; Humans; Lenalidomide; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Medical sciences; Prednisone - adverse effects; Prednisone - therapeutic use; Primary Myelofibrosis - drug therapy; Thalidomide - adverse effects; Thalidomide - analogs & derivatives; Thalidomide - therapeutic use; Antineoplastic agent; Corticosteroid; Myelofibrosis; Hematology; Lenalidomide; Hemopathy; Prednisone; Malignant tumor; Myeloproliferative syndrome; Cancerology; Adrenal hormone; Phase II trial; Cancer
• ISSN: 0006-4971; 1528-0020; 1528-0020
• DOI: 10.1182/blood-2010-05-287417

A multicenter Eastern Cooperative Group (ECOG) phase 2 trial assessed whether adding prednisone to lenalidomide would improve previously reported responses in persons with myelofibrosis (MF). Forty-eight subjects with anemia (42 evaluable) received lenalidomide, 10 mg/d, with a 3-month low-dose prednisone taper. Ten subjects received 3 months, and 25 received 6 months of therapy. Myelosuppression was the main toxicity with 88% with ≥ grade 3 hematologic toxicity and 45% ≥ grade 3 nonhematologic toxicity. There were responses in 10 subjects (23%) using the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT)–defined clinical improvement of anemia in 8 (19%) and/or decreased spleen size in 4 (10%). Serial bone marrow analysis showed no resolution of disease-related fibrosis or angiogenesis. With a median follow-up of 2.3 years, 23 subjects are alive. Lenali-domide and prednisone for myelofibro-sis evaluated through a multicentered-cooperative group mechanism is only modestly active and myelosuppre-sive. This study was registered at http://clinicaltrials.gov as NCT00227591.