Modest activity of pomalidomide in patients with myelofibrosis and significant anemia.

Abstract

We evaluated single agent pomalidomide for myelofibrosis-associated anemia. First, 21 patients received pomalidomide 3.0mg/day on 21-day-on/7-day-off schedule. Due to poor tolerance the study was quickly suspended. Second, 29 patients received pomalidomide 0.5mg/day continuously. Three patients (10%) experienced clinical improvement in hemoglobin per International-Working-Group criteria (median time to response 1.6 months; median response duration 6.7 months). Ten patients were RBC-transfusion-dependent per Delphi criteria; 2 (20%) achieved RBC-transfusion-independence (time to response 0.9 months in both; response duration of 8.3 and 15 months). One grade 3/4 toxicity (neutropenia) occurred. Pomalidomide at low dose is well tolerated but has modest clinical activity in myelofibrosis.

• Authors: Naval Daver, Aditi Shastri, Tapan Kadia, Alfonso Quintas-Cardama, Elias Jabbour, Marina Konopleva, Susan O'Brien, Sherry Pierce, Lingsha Zhou, Jorge Cortes, Hagop Kantarjian, Srdan Verstovsek
• Journal: Leukemia research (Leuk Res) Vol. 37 Issue 11 Pg. 1440-4 (Nov 2013) ISSN: 1873-5835 [Electronic] England
• PMID: 23890523 (Publication Type: Clinical Trial, Phase II, Journal Article, Research Support, N.I.H., Extramural)
• Copyright: Copyright © 2013 Elsevier Ltd. All rights reserved.

Chemical References

• Angiogenesis Inhibitors
• Thalidomide
• pomalidomide

Topics

• Aged
• Anemia (chemically induced, drug therapy)
• Angiogenesis Inhibitors (therapeutic use)
• Antineoplastic Combined Chemotherapy Protocols (adverse effects)
• Female
• Follow-Up Studies
• Humans
• Male
• Maximum Tolerated Dose
• Middle Aged
• Primary Myelofibrosis (drug therapy)
• Prospective Studies
• Thalidomide (analogs & derivatives, therapeutic use)
• Treatment Outcome