title: Response to ruxolitinib in patients with intermediate-1-, intermediate-2-, and high-risk myelofibrosis: results of the UK ROBUST Trial.
abstract: Myelofibrosis is characterized by splenomegaly and debilitating constitutional symptoms that negatively impact patients' quality of life. ROBUST, a UK, open-label, phase II study, evaluated the safety and efficacy of ruxolitinib in patients with myelofibrosis (N = 48), including intermediate-1 risk patients. The primary composite endpoint was the proportion of patients achieving treatment success [≥ 50% reduction in palpable spleen length and/or a ≥ 50% decrease in Myelofibrosis Symptom Assessment Form Total Symptom Score (MF-SAF TSS)] at 48 weeks. This was the first time that efficacy of ruxolitinib in myelofibrosis has been evaluated based on these criteria and the first time the MF-SAF was used in a population of patients solely from the United Kingdom. Overall, 50% of patients and 57% of intermediate-1 risk patients, achieved treatment success; reductions in spleen length and symptoms were observed in all risk groups. The majority of patients (66.7%) experienced ≥ 50% reductions from baseline in spleen length at any time. Improvements in MF-SAF TSS were seen in 80.0%, 72.7%, and 72.2% of intermediate-1, intermediate-2, and high-risk patients, respectively. Consistent with other studies of ruxolitinib, the most common haematological adverse events were anaemia and thrombocytopenia. Results indicate that most patients with myelofibrosis, including intermediate-1 risk patients, may benefit from ruxolitinib treatment.
authors.affiliation: MRC Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, UK.
authors.forename: Adam J
authors.initials: AJ
authors.lastname: Mead
authors.affiliation: Imperial College Healthcare NHS Trust, Hammersmith Hospital, London, UK.
authors.forename: Dragana
authors.initials: D
authors.lastname: Milojkovic
authors.affiliation: Department of Haematology, Cardiff University, Heath Park, Cardiff, UK.
authors.forename: Steven
authors.initials: S
authors.lastname: Knapper
authors.affiliation: Leicester Royal Infirmary, Leicester, UK.
authors.forename: Mamta
authors.initials: M
authors.lastname: Garg
authors.affiliation: The Royal Bournemouth and Christchurch Hospitals NHS Foundation Trust, Bournemouth, UK.
authors.forename: Joseph
authors.initials: J
authors.lastname: Chacko
authors.affiliation: Department of Haematology, Western General Hospital, Edinburgh, UK.
authors.forename: Mira
authors.initials: M
authors.lastname: Farquharson
authors.affiliation: Department of Haematology, Manchester Royal Infirmary, Manchester, UK.
authors.forename: John
authors.initials: J
authors.lastname: Yin
authors.affiliation: Hull and East Yorkshire Hospitals NHS Trust, Hull, UK.
authors.forename: Sahra
authors.initials: S
authors.lastname: Ali
authors.affiliation: Department of Haematology, Royal Liverpool University Hospital, Liverpool, UK.
authors.forename: Richard E
authors.initials: RE
authors.lastname: Clark
authors.affiliation: Novartis Pharmaceuticals UK, Frimley, UK.
authors.forename: Chris
authors.initials: C
authors.lastname: Andrews
authors.affiliation: Novartis Pharmaceuticals UK, Frimley, UK.
authors.forename: Meryem Ktiouet
authors.initials: MK
authors.lastname: Dawson
authors.affiliation: Guy's and St. Thomas' NHS Foundation Trust, London, UK.
authors.forename: Claire
authors.initials: C
authors.lastname: Harrison
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D006801:Humans; D008875:Middle Aged; D009570:Nitriles; D055728:Primary Myelofibrosis / Q000188:drug therapy* / Q000473:pathology; D011720:Pyrazoles / Q000009:adverse effects / Q000627:therapeutic use*; D011743:Pyrimidines; D012307:Risk Factors
chemical_list: D009570:Nitriles; D011720:Pyrazoles; D011743:Pyrimidines; C540383:ruxolitinib
keywords: Janus kinase inhibitor; Myelofibrosis Symptom Assessment Form; myelofibrosis; ruxolitinib; symptoms