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

A. Tefferi, H. K. Al-Ali, G. Barosi, T. Devos, H. Gisslinger, Q. Jiang, J. J. Kiladjian, R. Mesa, F. Passamonti, V. Ribrag, G. Schiller, A. M. Vannucchi, D. Zhou, D. Reiser, J. Zhong, R. P. Gale

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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 × 10 9 /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.

Original languageEnglish
Pages (from-to)896-902
Number of pages7
JournalLeukemia
Volume31
Issue number4
DOIs
Publication statusPublished - Apr 1 2017

ASJC Scopus subject areas

  • Hematology
  • Cancer Research
  • Anesthesiology and Pain Medicine

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    Tefferi, A., Al-Ali, H. K., Barosi, G., Devos, T., Gisslinger, H., Jiang, Q., Kiladjian, J. J., Mesa, R., Passamonti, F., Ribrag, V., Schiller, G., Vannucchi, A. M., Zhou, D., Reiser, D., Zhong, J., & Gale, R. P. (2017). A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence. Leukemia, 31(4), 896-902. https://doi.org/10.1038/leu.2016.300