title: Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy.
abstract: Patients with myelofibrosis (MF) have significant debilitating symptoms, physical disabilities, and poor health-related quality of life (HRQoL). Here, we report post-hoc analyses of the impact of ruxolitinib, a potent and selective JAK1 and JAK2 inhibitor, on disease-related symptoms and HRQoL in MF patients from the large phase 3 COMFORT-II study (N = 219). During the follow-up period of 48 weeks, HRQoL and MF-associated symptoms improved from baseline for ruxolitinib-treated patients but remained the same or worsened for best available therapy (BAT)-treated patients. Based on the European Organization for Research and Treatment of Cancer QoL Questionnaire core 30 items (EORTC QLQ-C30), treatment-induced differences in physical and role functioning, fatigue, and appetite loss significantly favoured ruxolitinib versus BAT from week 8 (P < 0·05) up to week 48 (P < 0·05). Ruxolitinib resulted in significantly higher response rates in global health status/QoL and Functional Assessment of Cancer Therapy-Lymphoma (FACT-Lym) summary scores versus BAT at most time points (P < 0·05). Significant improvements in the Lymphoma subscale (including symptoms of pain, fever, itching, fatigue, weight loss, loss of appetite, and other patient concerns), FACT-General, FACT-Lym trial outcome index, and FACT-Lym total were also observed with ruxolitinib versus BAT starting at week 8 and continuing thereafter. Overall, these data demonstrated that ruxolitinib improved HRQoL in MF patients and further support the use of ruxolitinib for the treatment of symptomatic MF.
authors.affiliation: Guy's and St. Thomas' NHS Foundation Trust, Guy's Hospital, London, UK. claire.harrison@gstt.nhs.uk
authors.forename: Claire N
authors.initials: CN
authors.lastname: Harrison
authors.forename: Ruben A
authors.initials: RA
authors.lastname: Mesa
authors.forename: Jean-Jacques
authors.initials: JJ
authors.lastname: Kiladjian
authors.forename: Haifa-Kathrin
authors.initials: HK
authors.lastname: Al-Ali
authors.forename: Heinz
authors.initials: H
authors.lastname: Gisslinger
authors.forename: Laurent
authors.initials: L
authors.lastname: Knoops
authors.forename: Margaret
authors.initials: M
authors.lastname: Squier
authors.forename: Andres
authors.initials: A
authors.lastname: Sirulnik
authors.forename: Estella
authors.initials: E
authors.lastname: Mendelson
authors.forename: Xiaolei
authors.initials: X
authors.lastname: Zhou
authors.forename: Catherine
authors.initials: C
authors.lastname: Copley-Merriman
authors.forename: Deborah S
authors.initials: DS
authors.lastname: Hunter
authors.forename: Richard S
authors.initials: RS
authors.lastname: Levy
authors.forename: Francisco
authors.initials: F
authors.lastname: Cervantes
authors.forename: Francesco
authors.initials: F
authors.lastname: Passamonti
authors.forename: Tiziano
authors.initials: T
authors.lastname: Barbui
authors.forename: Giovanni
authors.initials: G
authors.lastname: Barosi
authors.forename: Alessandro M
authors.initials: AM
authors.lastname: Vannucchi
mesh_terms: D000368:Aged; D005260:Female; D006801:Humans; D053613:Janus Kinase 1 / Q000037:antagonists & inhibitors; D053614:Janus Kinase 2 / Q000037:antagonists & inhibitors; D008297:Male; D009570:Nitriles; D055728:Primary Myelofibrosis / Q000188:drug therapy* / Q000201:enzymology / Q000473:pathology; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*; D011720:Pyrazoles / Q000627:therapeutic use*; D011743:Pyrimidines; D011788:Quality of Life; D011795:Surveys and Questionnaires; D016896:Treatment Outcome
chemical_list: D009570:Nitriles; D047428:Protein Kinase Inhibitors; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib; D053613:Janus Kinase 1; D053614:Janus Kinase 2
keywords: JAK1/JAK2 inhibitor; health-related quality of life; myelofibrosis; ruxolitinib