Impact of ruxolitinib on the natural history of primary myelofibrosis: a comparison of the DIPSS and the COMFORT-2 cohorts

• Published in: Blood Vol. 123; no. 12; pp. 1833 - 1835
• Main Authors: Passamonti, Francesco, Maffioli, Margherita, Cervantes, Francisco, Vannucchi, Alessandro Maria, Morra, Enrica, Barbui, Tiziano, Caramazza, Domenica, Pieri, Lisa, Rumi, Elisa, Gisslinger, Heinz, Knoops, Laurent, Kiladjian, Jean Jaques, Mora, Barbara, Hollaender, Norbert, Pascutto, Cristiana, Harrison, Claire, Cazzola, Mario
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 20.03.2014
• Subjects: Adult; Aged; Aged, 80 and over; Cohort Studies; Female; Humans; Janus Kinases - antagonists & inhibitors; Kaplan-Meier Estimate; Male; Middle Aged; Primary Myelofibrosis - drug therapy; Primary Myelofibrosis - mortality; Prognosis; Protein Kinase Inhibitors - therapeutic use; Pyrazoles - therapeutic use
• ISSN: 0006-4971; 1528-0020; 1528-0020
• DOI: 10.1182/blood-2013-12-544411

The international prognostic scoring system (IPSS) provides reliable risk assessment in patients with primary myelofibrosis (PMF). Recent clinical trials in PMF patients with intermediate-2 or high IPSS risk have shown a survival advantage of ruxolitinib over placebo (COMFORT-1) or best available therapy (COMFORT-2). Because crossover was allowed in these studies, we analyzed the cohort of ruxolitinib-naive patients used for developing the dynamic IPSS (DIPSS). By adopting ad hoc statistical analyses, we compared survival from diagnosis of 100 PMF patients receiving ruxolitinib within COMFORT-2 with that of 350 patients of the DIPSS study. Subjects were properly matched, and both left-truncation and right-censoring were accounted in order to compare higher IPSS risks exclusively. Patients receiving ruxolitinib had longer survival (5 years, 95% confidence interval [CI]: 2.9-7.8 vs 3.5 years, 95% CI: 3.0-3.9) with a hazard ratio of 0.61 (95% CI: 0.41-0.91; P = .0148). This observation suggests that ruxolitinib may modify the natural history of PMF. •Patients with primary myelofibrosis and intermediate-2 or high IPSS risk have a median life expectancy of 4 years or less.•PMF patients with higher IPSS risks who receive ruxolitinib treatment have longer survival than those who receive conventional therapy.