title: Efficacy and safety of ruxolitinib in Asian patients with myelofibrosis.
abstract: Myelofibrosis is characterized by progressive cytopenias, bone marrow fibrosis, splenomegaly and severe constitutional symptoms. In the phase 3 Controlled Myelofibrosis Study with Oral JAK Inhibitor Treatment (COMFORT) studies, ruxolitinib, a potent Janus kinase 1 (JAK1)/JAK2 inhibitor, provided substantial improvements in splenomegaly, symptoms, quality-of-life measures and overall survival compared with placebo or best available therapy. No assessments of the efficacy and safety of ruxolitinib have been conducted in Asian patients. Here, we describe results from an open-label, single-arm, phase 2 trial evaluating ruxolitinib in Asian patients with myelofibrosis (n = 120). The primary endpoint was met, with 31.7% of patients achieving a ≥ 35% reduction from baseline spleen volume at week 24. As measured by the 7-day Myelofibrosis Symptom Assessment Form v2.0, 49% of patients achieved a ≥ 50% reduction from baseline in total symptom score. Adverse events were consistent with those seen in the COMFORT studies. Ruxolitinib was well tolerated in Asian patients with myelofibrosis and provided substantial reductions in splenomegaly and improvements in symptoms.
authors.affiliation: Samsung Medical Center, Sungkyunkwan University School of Medicine , Seoul , Korea.
authors.forename: Chul Won
authors.initials: CW
authors.lastname: Jung
authors.forename: Lee-Yung
authors.initials: LY
authors.lastname: Shih
authors.forename: Zhijian
authors.initials: Z
authors.lastname: Xiao
authors.forename: Jin
authors.initials: J
authors.lastname: Jie
authors.forename: Hsin-An
authors.initials: HA
authors.lastname: Hou
authors.forename: Xin
authors.initials: X
authors.lastname: Du
authors.forename: Ming-Chung
authors.initials: MC
authors.lastname: Wang
authors.forename: Seonyang
authors.initials: S
authors.lastname: Park
authors.forename: Ki-Seong
authors.initials: KS
authors.lastname: Eom
authors.forename: Kenji
authors.initials: K
authors.lastname: Oritani
authors.forename: Shinichiro
authors.initials: S
authors.lastname: Okamoto
authors.forename: Tetsuzo
authors.initials: T
authors.lastname: Tauchi
authors.forename: Jin Seok
authors.initials: JS
authors.lastname: Kim
authors.forename: Daobin
authors.initials: D
authors.lastname: Zhou
authors.forename: Shigeki
authors.initials: S
authors.lastname: Saito
authors.forename: Junmin
authors.initials: J
authors.lastname: Li
authors.forename: Hiroshi
authors.initials: H
authors.lastname: Handa
authors.forename: Li
authors.initials: L
authors.lastname: Jianyong
authors.forename: Kohshi
authors.initials: K
authors.lastname: Ohishi
authors.forename: Ming
authors.initials: M
authors.lastname: Hou
authors.forename: Wu
authors.initials: W
authors.lastname: Depei
authors.forename: Katsuto
authors.initials: K
authors.lastname: Takenaka
authors.forename: Ting
authors.initials: T
authors.lastname: Liu
authors.forename: Yu
authors.initials: Y
authors.lastname: Hu
authors.forename: Taro
authors.initials: T
authors.lastname: Amagasaki
authors.forename: Kazuo
authors.initials: K
authors.lastname: Ito
authors.forename: Prashanth
authors.initials: P
authors.lastname: Gopalakrishna
authors.forename: Koichi
authors.initials: K
authors.lastname: Akashi
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D044466:Asian People; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D053613:Janus Kinase 1 / Q000037:antagonists & inhibitors*; D053614:Janus Kinase 2 / Q000037:antagonists & inhibitors*; D008297:Male; D008875:Middle Aged; D009367:Neoplasm Staging; D009570:Nitriles; D055728:Primary Myelofibrosis / Q000188:drug therapy* / Q000473:pathology; D011379:Prognosis; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*; D011720:Pyrazoles / Q000627:therapeutic use*; D011743:Pyrimidines; D011788:Quality of Life
chemical_list: D009570:Nitriles; D047428:Protein Kinase Inhibitors; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib; C507904:JAK1 protein, human; C507924:JAK2 protein, human; D053613:Janus Kinase 1; D053614:Janus Kinase 2
keywords: Asian patient population; JAK1/JAK2 inhibitor; Myelofibrosis; phase 2