title: 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.affiliation: Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Naval
authors.initials: N
authors.lastname: Daver
authors.forename: Aditi
authors.initials: A
authors.lastname: Shastri
authors.forename: Tapan
authors.initials: T
authors.lastname: Kadia
authors.forename: Alfonso
authors.initials: A
authors.lastname: Quintas-Cardama
authors.forename: Elias
authors.initials: E
authors.lastname: Jabbour
authors.forename: Marina
authors.initials: M
authors.lastname: Konopleva
authors.forename: Susan
authors.initials: S
authors.lastname: O'Brien
authors.forename: Sherry
authors.initials: S
authors.lastname: Pierce
authors.forename: Lingsha
authors.initials: L
authors.lastname: Zhou
authors.forename: Jorge
authors.initials: J
authors.lastname: Cortes
authors.forename: Hagop
authors.initials: H
authors.lastname: Kantarjian
authors.forename: Srdan
authors.initials: S
authors.lastname: Verstovsek
mesh_terms: D000368:Aged; D000740:Anemia / Q000139:chemically induced / Q000188:drug therapy*; D020533:Angiogenesis Inhibitors / Q000627:therapeutic use*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D055728:Primary Myelofibrosis / Q000188:drug therapy*; D011446:Prospective Studies; D013792:Thalidomide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D020533:Angiogenesis Inhibitors; D013792:Thalidomide; C467566:pomalidomide
keywords: Anemia; Myelofibrosis; Pomalidomide
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