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Leukemia. 2017 Apr;31(4):896-902. doi: 10.1038/leu.2016.300. Epub 2016 Oct 24.

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

Author information

1
Division of Hematology, Department of Internal Medicine, Mayo Clinic College of Medicine, Rochester, MN, USA.
2
Universitätsklinikum Leipzig, Leipzig, Germany.
3
Fondazione IRCCS Policlinico S Matteo, Pavia, Italy.
4
Universitaire Ziekenhuizen, Leuven, Belgium.
5
Medizinische Universität Wien, Vienna, Austria.
6
Peking University People's Hospital, Beijing, China.
7
Hôpital Saint-Louis, Paris, France.
8
Mayo Clinic, Scottsdale, AZ, USA.
9
University of Insubria, Varese, Italy.
10
Department of Haematology, Queen’s University, Belfast, UK.
11
Gustave Roussy, Université Paris-Saclay, Saint-Aubin, France.
12
Division of Hematology and Oncology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
13
Centro Ricerca e Innovazione Malattie Mieloproliferative-CRIMM, Azienda Ospedaliera Universitaria Careggi, Florence, Italy.
14
Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy.
15
Peking Union Medical College Hospital, Beijing, China.
16
Celgene Corporation, Summit, NJ, USA.
17
Haematology Research Centre, Imperial College London, London, UK.

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.

TRIAL REGISTRATION:

ClinicalTrials.gov NCT01178281.

PMID:
27773929
PMCID:
PMC5383927
DOI:
10.1038/leu.2016.300
[Indexed for MEDLINE]
Free PMC Article

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