A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence.

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.

• Authors: A Tefferi, H K Al-Ali, G Barosi, T Devos, H Gisslinger, Q Jiang, J-J Kiladjian, R Mesa, F Passamonti, M F McMullin, V Ribrag, G Schiller, A M Vannucchi, D Zhou, D Reiser, J Zhong, R P Gale
• Journal: Leukemia (Leukemia) Vol. 31 Issue 4 Pg. 896-902 (04 2017) ISSN: 1476-5551 [Electronic] England
• PMID: 27773929 (Publication Type: Clinical Trial, Phase III, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Chemical References

• Biomarkers
• Immunologic Factors
• Thalidomide
• pomalidomide

Topics

• Adult
• Aged
• Aged, 80 and over
• Biomarkers
• Erythrocyte Transfusion (methods)
• Female
• Humans
• Immunologic Factors (administration & dosage, adverse effects, therapeutic use)
• Male
• Middle Aged
• Myeloproliferative Disorders (complications, diagnosis)
• Phenotype
• Primary Myelofibrosis (diagnosis, etiology, therapy)
• Thalidomide (administration & dosage, adverse effects, analogs & derivatives, therapeutic use)
• Treatment Outcome
• Workflow