- Daver, Naval;
- Shastri, Aditi;
- Kadia, Tapan;
- Quintas-Cardama, Alfonso;
- Jabbour, Elias;
- Konopleva, Marina;
- O’Brien, Susan;
- Pierce, Sherry;
- Zhou, Lingsha;
- Cortes, Jorge;
- Kantarjian, Hagop;
- Verstovsek, Srdan
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.