Abstract
RBC-transfusion dependence is common in persons with myeloproliferative neoplasm (MPN)-associated myelofibrosis. The objective of this study was to determine the rates of RBC-transfusion independence after therapy with pomalidomide vs placebo in persons with MPN-associated myelofibrosis and RBC-transfusion dependence. Two hundred and fifty-two subjects (intent-to-treat (ITT) population) including 229 subjects confirmed by central review (modified ITT population) were randomly assigned (2:1) to pomalidomide or placebo. Trialists and subjects were blinded to treatment allocation. Primary end point was proportion of subjects achieving RBC-transfusion independence within 6 months. One hundred and fifty-two subjects received pomalidomide and 77 placebo. Response rates were 16% (95% confidence interval (CI), 11, 23%) vs 16% (8, 26%; P=0.87). Response in the pomalidomide cohort was associated with ⩽4 U RBC/28 days (odds ratio (OR)=3.1; 0.9, 11.1), age ⩽65 (OR=2.3; 0.9, 5.5) and type of MPN-associated myelofibrosis (OR=2.6; 0.7, 9.5). Responses in the placebo cohort were associated with ⩽4 U RBC/28 days (OR=8.6; 0.9, 82.3), white blood cell at randomization >25 × 109/l (OR=4.9; 0.8, 28.9) and interval from diagnosis to randomization >2 years (OR=4.9; 1.1, 21.9). Pomalidomide was associated with increased rates of oedema and neutropenia but these adverse effects were manageable. Pomalidomide and placebo had similar RBC-transfusion-independence response rates in persons with MPN-associated RBC-transfusion dependence.
Highlights
Myeloproliferative neoplasm (MPN)-associated myelofibrosis is a clinical entity comprised of primary myelofibrosis, postpolycythaemia myelofibrosis and post-essential thrombocythaemia myelofibrosis.[1]
red blood cell (RBC) transfusions given for a haemoglobin concentration ⩽ 90 g/l were scored in determining eligibility. (For countries following Nordic guidelines, haemoglobin levels for RBC transfusions should not exceed 100 g/l haemoglobin.) RBC transfusions given because of bleeding or chemotherapy- or radiation-induced anaemia were excluded
We could not confirm the diagnosis of MPN-associated myelofibrosis at central histological review in 6 subjects nor RBC-transfusion dependence in 15
Summary
Myeloproliferative neoplasm (MPN)-associated myelofibrosis is a clinical entity comprised of primary myelofibrosis, postpolycythaemia myelofibrosis and post-essential thrombocythaemia myelofibrosis.[1] Anaemia is common in persons with MPNassociated myelofibrosis with frequencies of 30–100% in several large series (reviewed in references Cervantes et al.[2] and Passamonti et al.[3]). Anaemia and RBC-transfusion dependence are associated with poorer survival.[2,3,4,5,6] RBC transfusions are often given for severe anaemia. Some persons with MPN-associated myelofibrosis have decreased platelet levels. There is no US Food and Drug Administration- or European Medicines Agency-approved therapy for anaemia with or without
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