The clinical benefit of ruxolitinib across patient subgroups: analysis of a placebo-controlled, Phase III study in patients with myelofibrosis.

Journal Article (Clinical Trial, Phase III;Journal Article)

Myelofibrosis (MF) patients can present with a wide spectrum of disease characteristics. We analysed the consistency of ruxolitinib efficacy across patient subgroups in the COntrolled MyeloFibrosis Study With ORal JAK Inhibitor Treatment (COMFORT-I,) a double-blind trial, where patients with intermediate-2 or high-risk MF were randomized to twice-daily oral ruxolitinib (n = 155) or placebo (n = 154). Subgroups analysed included MF subtype (primary, post-polycythaemia vera, post-essential thrombocythaemia), age (≤65, > 65 years), International Prognostic Scoring System risk group, baseline Eastern Cooperative Oncology Group performance status (0, 1, ≥2), JAK2 V617F mutation (positive, negative), baseline haemoglobin level (≥100, <100 g/l), baseline platelet count (100-200 × 10(9)/l, >200 × 10(9)/l), baseline palpable spleen size (≤10, >10 cm), and baseline quartile of spleen volume and Total Symptom Score (TSS; Q1 = lowest, Q4 = highest). Mean percentage change from baseline to week 24 in spleen volume and TSS were calculated for ruxolitinib and placebo in each subgroup. Overall survival was estimated by Kaplan-Meier method according to original randomization group. In ruxolitinib-treated patients, reductions in spleen volume and TSS and evidence of improved survival relative to placebo across subgroups were consistent with those seen in the COMFORT-I population, confirming that ruxolitinib is an effective therapy for the spectrum of MF patients studied in COMFORT-I.

Full Text

Duke Authors

Cited Authors

  • Verstovsek, S; Mesa, RA; Gotlib, J; Levy, RS; Gupta, V; DiPersio, JF; Catalano, JV; Deininger, M; Miller, C; Silver, RT; Talpaz, M; Winton, EF; Harvey, JH; Arcasoy, MO; Hexner, E; Lyons, RM; Paquette, R; Raza, A; Vaddi, K; Erickson-Viitanen, S; Sun, W; Sandor, V; Kantarjian, HM

Published Date

  • May 2013

Published In

Volume / Issue

  • 161 / 4

Start / End Page

  • 508 - 516

PubMed ID

  • 23480528

Pubmed Central ID

  • PMC4055021

Electronic International Standard Serial Number (EISSN)

  • 1365-2141

Digital Object Identifier (DOI)

  • 10.1111/bjh.12274


  • eng

Conference Location

  • England