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Treatment Option Overview for PMF
Source: Myeloproliferative Neoplasms Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: September 27, 2024 · Captured 2026-09-09.
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Context: Treatment of Primary Myelofibrosis
Asymptomatic low-risk patients (based on the aforementioned prognostic systems) should be monitored with a watchful waiting approach. The development of symptomatic anemia, marked leukocytosis, drenching night sweats, weight loss, fever, or symptomatic splenomegaly warrants therapeutic intervention.
The profound anemia that develops in this disease usually requires red blood cell transfusion. Red blood cell survival is markedly decreased in some patients; this can sometimes be treated with glucocorticoids. Disease-associated anemia may occasionally respond to:[7,27-29]
Erythropoietic growth factors. Erythropoietin and darbepoetin are less likely to help when patients are transfusion dependent or manifest a serum erythropoietin level greater than 125 U/L.[30,31]
Prednisone (40–80 mg/day).
Danazol (600 mg/day).
Thalidomide (50 mg/day) with or without prednisone.[32] Patients on thalidomide require prophylaxis for avoiding thrombosis and careful monitoring for hematologic toxicity.
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Lenalidomide (10 mg/day) with or without prednisone.[33-35] In the presence of del(5q), lenalidomide with or without prednisone, can reverse anemia and splenomegaly in most patients.[33-35] However, patients receiving lenalidomide require prophylaxis for avoiding thrombosis and careful monitoring for hematologic toxicity.
Pomalidomide.[36] Patients on pomalidomide require prophylaxis for avoiding thrombosis and careful monitoring for hematologic toxicity.
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