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, Ruben Mesa, F. Passamonti, V. Ribrag, G. Schiller, A. M. Vannucchi, D. Zhou, D. Reiser, J. Zhong, R. P. Gale

Research output: Contribution to journalArticle

19 Citations (Scopus)

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 (US)
Pages (from-to)896-902
Number of pages7
JournalLeukemia
Volume31
Issue number4
DOIs
StatePublished - Apr 1 2017
Externally publishedYes

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Primary Myelofibrosis
Odds Ratio
Placebos
Neoplasms
Random Allocation
Neutropenia
Population
pomalidomide
Edema
Leukocytes
Confidence Intervals
Therapeutics

ASJC Scopus subject areas

  • Hematology
  • Cancer Research
  • Anesthesiology and Pain Medicine

Cite this

A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence. / Tefferi, A.; Al-Ali, H. K.; Barosi, G.; Devos, T.; Gisslinger, H.; Jiang, Q.; Kiladjian, J. J.; Mesa, Ruben; Passamonti, F.; Ribrag, V.; Schiller, G.; Vannucchi, A. M.; Zhou, D.; Reiser, D.; Zhong, J.; Gale, R. P.

In: Leukemia, Vol. 31, No. 4, 01.04.2017, p. 896-902.

Research output: Contribution to journalArticle

Tefferi, A, Al-Ali, HK, Barosi, G, Devos, T, Gisslinger, H, Jiang, Q, Kiladjian, JJ, Mesa, R, Passamonti, F, Ribrag, V, Schiller, G, Vannucchi, AM, Zhou, D, Reiser, D, Zhong, J & Gale, RP 2017, 'A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence', Leukemia, vol. 31, no. 4, pp. 896-902. https://doi.org/10.1038/leu.2016.300
Tefferi, A. ; Al-Ali, H. K. ; Barosi, G. ; Devos, T. ; Gisslinger, H. ; Jiang, Q. ; Kiladjian, J. J. ; Mesa, Ruben ; Passamonti, F. ; Ribrag, V. ; Schiller, G. ; Vannucchi, A. M. ; Zhou, D. ; Reiser, D. ; Zhong, J. ; Gale, R. P. / A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence. In: Leukemia. 2017 ; Vol. 31, No. 4. pp. 896-902.
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T1 - A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence

AU - Tefferi, A.

AU - Al-Ali, H. K.

AU - Barosi, G.

AU - Devos, T.

AU - Gisslinger, H.

AU - Jiang, Q.

AU - Kiladjian, J. J.

AU - Mesa, Ruben

AU - Passamonti, F.

AU - Ribrag, V.

AU - Schiller, G.

AU - Vannucchi, A. M.

AU - Zhou, D.

AU - Reiser, D.

AU - Zhong, J.

AU - Gale, R. P.

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