Final Results from a Large, Non-Interventional, Phase 4 Study of Ruxolitinib for the Treatment of Myelofibrosis in Clinical Routine.

JAKoMo was a long-term, multicenter, non-interventional study observing the efficacy, safety, and quality of life (QOL) effects of ruxolitinib (RUX), managed per clinical routine at investigator discretion, for treatment of 943 patients with myelofibrosis (MF) in 122 German centers. Patients ≥ 18 years with a diagnosis of PMF or PPV-MF or PET-MF, who were suitable for in-label treatment with RUX were eligible and could be included either before (479 previously RUX-naïve [Arm A]) or after the start of treatment (464 RUX-experienced patients [Arm B]) and were followed over 36 months. Arm A showed rapid (≤ 6 months), sustained improvements from baseline in all efficacy outcomes and most QOL measures. Both arms showed an ~17% increase in the proportion of patients experiencing a normal German QOL during follow-up. Arm B entered the study with better outcomes and QOL than Arm A, with outcomes generally remaining stable over time. Adverse events were less common than in registrational trials, possibly due, in part, to lower real-world RUX dosing. Survival was comparable to published data. The JAKoMo study demonstrates that real-world RUX treatment of MF promotes significant and sustained clinical and QOL benefits, including improvement of general health and alleviation of MF-associated fatigue. Maximum sustained responses were generally achieved within 6 months and associated with fewer adverse events than in published randomized trials, which may reflect more conservative and personalized real-world dosing. Trial Registration: The JAKOMO trial: http://clinicaltrials.gov/show/NCT05044026.