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Primary analysis of JUMP, a phase 3b, expanded‐access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis, including those with low platelet counts
Author(s) -
AlAli Haifa Kathrin,
Griesshammer Martin,
Foltz Lynda,
Palumbo Giuseppe A.,
Martino Bruno,
Palandri Francesca,
Liberati Anna Marina,
Coutre Philipp,
GarcíaHernández Carmen,
Zaritskey Andrey,
Tavares Renato,
Gupta Vikas,
Raanani Pia,
Giraldo Pilar,
Hänel Mathias,
Damiani Daniela,
Sacha Tomasz,
Bouard Catherine,
Paley Carole,
Tiwari Ranjan,
Mannelli Francesco,
Vannucchi Alessandro M.
Publication year - 2020
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.16462
Subject(s) - ruxolitinib , medicine , myelofibrosis , discontinuation , cohort , adverse effect , surgery , bone marrow
Summary Ruxolitinib is a potent Janus kinase (JAK) 1/JAK2 inhibitor approved for the treatment of myelofibrosis (MF). Ruxolitinib was assessed in JUMP, a large ( N = 2233), phase 3b, expanded‐access study in MF in countries without access to ruxolitinib outside a clinical trial, which included patients with low platelet counts (<100 × 10 9 /l) and patients without splenomegaly – populations that have not been extensively studied. The most common adverse events (AEs) were anaemia and thrombocytopenia, but they rarely led to discontinuation (overall, 5·4%; low‐platelet cohort, 12·3%). As expected, rates of worsening thrombocytopenia were higher in the low‐platelet cohort (all grades, 73·2% vs. 53·5% overall); rates of anaemia were similar (all grades, 52·9% vs. 59·5%). Non‐haematologic AEs, including infections, were mainly grade 1/2. Overall, ruxolitinib led to meaningful reductions in spleen length and symptoms, including in patients with low platelet counts, and symptom improvements in patients without splenomegaly. In this trial, the largest study of ruxolitinib in patients with MF to date, the safety profile was consistent with previous reports, with no new safety concerns identified. This study confirms findings from the COMFORT studies and supports the use of ruxolitinib in patients with platelet counts of 50–100 × 10 9 /l. (ClinicalTrials.gov identifier NCT01493414).