title: 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: Division of Hematology, Department of Internal Medicine, Mayo Clinic College of Medicine, Rochester, MN, USA.
authors: A
authors: A
authors: Tefferi
authors: Universitätsklinikum Leipzig, Leipzig, Germany.
authors: H K
authors: HK
authors: Al-Ali
authors: Fondazione IRCCS Policlinico S Matteo, Pavia, Italy.
authors: G
authors: G
authors: Barosi
authors: Universitaire Ziekenhuizen, Leuven, Belgium.
authors: T
authors: T
authors: Devos
authors: Medizinische Universität Wien, Vienna, Austria.
authors: H
authors: H
authors: Gisslinger
authors: Peking University People's Hospital, Beijing, China.
authors: Q
authors: Q
authors: Jiang
authors: Hôpital Saint-Louis, Paris, France.
authors: J-J
authors: JJ
authors: Kiladjian
authors: Mayo Clinic, Scottsdale, AZ, USA.
authors: R
authors: R
authors: Mesa
authors: University of Insubria, Varese, Italy.
authors: F
authors: F
authors: Passamonti
authors: Department of Haematology, Queen’s University, Belfast, UK.
authors: M F
authors: MF
authors: McMullin
authors: Gustave Roussy, Université Paris-Saclay, Saint-Aubin, France.
authors: V
authors: V
authors: Ribrag
authors: Division of Hematology and Oncology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
authors: G
authors: G
authors: Schiller
authors: Centro Ricerca e Innovazione Malattie Mieloproliferative-CRIMM, Azienda Ospedaliera Universitaria Careggi, Florence, Italy.|Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy.
authors: A M
authors: AM
authors: Vannucchi
authors: Peking Union Medical College Hospital, Beijing, China.
authors: D
authors: D
authors: Zhou
authors: Celgene Corporation, Summit, NJ, USA.
authors: D
authors: D
authors: Reiser
authors: Celgene Corporation, Summit, NJ, USA.
authors: J
authors: J
authors: Zhong
authors: Celgene Corporation, Summit, NJ, USA.|Haematology Research Centre, Imperial College London, London, UK.
authors: R P
authors: RP
authors: Gale
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D015415:Biomarkers; D017707:Erythrocyte Transfusion / Q000379:methods; D005260:Female; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009196:Myeloproliferative Disorders / Q000150:complications* / Q000175:diagnosis; D010641:Phenotype; D055728:Primary Myelofibrosis / Q000175:diagnosis / Q000209:etiology* / Q000628:therapy*; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome; D057188:Workflow
chemical_list: D015415:Biomarkers; D007155:Immunologic Factors; D013792:Thalidomide; C467566:pomalidomide