This protocol is specific to patients with primary myelofibrosis who present with anemia, absence of del(5q), and serum erythropoietin ≥500 mU/mL. These laboratory findings define a distinct sub-population in which anemia-directed treatment selection is guided by both the chromosomal profile and the measured erythropoietin level.
For this specific presentation, particular pharmacological agents are recommended as a first-line approach to managing anemia. The choice between available options depends on the individual clinical picture.
The primary endpoint is an anemia response: transfusion cessation for patients who are transfusion-dependent, or a hemoglobin increase of 2 g/dL or greater for those who are transfusion-independent.
DOI: 10.1182/blood.2022017423
Anemia response with danazol was reported in 30% of 50 patients (18% in patients with TD, 43% in patients with TI).
Thalidomide or lenalidomide have limited activity (TI rates of 11% and 16%, respectively).
Anemia response is usually defined according to the International Working Group on Myeloproliferative Neoplasms Research and treatment (IWGMRT) criteria: transfusion cessation if previous RBC transfusion dependency (TD), or hemoglobin increase ≥2 g/dL (modifiable in 1.5 g/dL) in case of RBC transfusion independency (TI).
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