title: Real-World Assessment of Clinical Outcomes in Patients with Lower-Risk Myelofibrosis Receiving Treatment with Ruxolitinib.
abstract: Few trial-based assessments of ruxolitinib in patients with lower-risk myelofibrosis (MF) have been conducted, and no studies have made such assessments in a real-world population. We assessed changes in spleen size and constitutional symptoms during ruxolitinib treatment using a retrospective, observational review of anonymized US medical record data of patients diagnosed with IPSS low-risk (n = 25) or intermediate-1-risk (n = 83) MF. The majority of patients were male (low risk, 60%; intermediate-1 risk, 69%). Most patients (92% and 77%) were still receiving ruxolitinib at the medical record abstraction date (median observation/exposure time, 8 months). The proportion of patients with moderate or severe palpable splenomegaly (≥10 cm) decreased from diagnosis (56%) to best response (12%). Fatigue was reported in 47% of patients and was the most common constitutional symptom. For most symptoms in both risk groups, shifts in the distribution of severity from more to less severe from diagnosis to best response were observed. Both patients with low-risk and intermediate-1-risk MF experienced a substantial decrease in spleen size with ruxolitinib treatment in real-world settings. For most symptoms examined, there were distinct improvements in the distribution of severity during ruxolitinib treatment. These findings suggest that patients with lower-risk MF may benefit clinically from ruxolitinib treatment.
authors.affiliation: RTI Health Solutions, Research Triangle Park, NC 27709, USA.
authors.forename: Keith L
authors.initials: KL
authors.lastname: Davis
authors.affiliation: Novartis Pharmaceuticals Corporation, East Hanover, NJ 07936, USA.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Côté
authors.affiliation: RTI Health Solutions, Waltham, MA 02451, USA.
authors.forename: James A
authors.initials: JA
authors.lastname: Kaye
authors.affiliation: Novartis Pharmaceuticals Corporation, East Hanover, NJ 07936, USA.
authors.forename: Estella
authors.initials: E
authors.lastname: Mendelson
authors.affiliation: Novartis Pharmaceuticals Corporation, East Hanover, NJ 07936, USA.
authors.forename: Haitao
authors.initials: H
authors.lastname: Gao
authors.affiliation: Novartis Pharma AG, 4056 Basel, Switzerland.
authors.forename: Julian
authors.initials: J
authors.lastname: Perez Ronco