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Clinical Trial
. 2025 Oct;115(4):380-390.
doi: 10.1111/ejh.70005. Epub 2025 Jul 6.

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

Steffen Koschmieder  1   2 , Susanne Isfort  1   2   3 , Clemens Schulte  4 , Lutz Jacobasch  5 , Thomas Geer  6 , Marcel Reiser  7 , Michael Koenigsmann  8 , Bernhard Heinrich  9 , Jürgen Wehmeyer  10 , Eyck von der Heyde  11 , Hans Tesch  12 , Benedikt Gröschl  13 , Petra Bachhuber  14 , Susanne Großer  14 , Michael Koehler  15   16 , Heike L Pahl  17
Affiliations
Clinical Trial

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

Steffen Koschmieder et al. Eur J Haematol. 2025 Oct.

Abstract

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.

Keywords: clinical routine; myelofibrosis; real‐life analysis; ruxolitinib.

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Conflict of interest statement

S. Koschmieder reports research funding from Novartis, Bristol‐Myers Squibb, AOP Pharma, Janssen, Geron; advisory board honoraria from Pfizer, Incyte, Ariad, Novartis, AOP Pharma, BMS, Celgene, Geron, Janssen, CTI, Roche, Baxalta, Sanofi, Sierra Oncology, GSK, Abbvie, PharmaEssentia, Protagonist, and MSD; patent for BET inhibitor at RWTH Aachen University; honoraria from Novartis, BMS, Celgene, Geron, Janssen, Pfizer, Incyte, Ariad, Shire, Roche, AOP Pharma, GSK, Abbvie, MPN Hub, iOMEDICO, Astra Zeneca, and MSD; and other financial support (e.g., travel support) from Alexion, Novartis, BMS, Incyte, Ariad, AOP Pharma, Baxalta, CTI, Pfizer, Sanofi, Celgene, Shire, Janssen, Geron, Abbvie, Karthos, Sierra Oncology, Imago Bioscience, GSK, Abbvie, iOMEDICO, Protagonist, and MSD. S. Isfort reports advisory board honoraria from Pfizer, Incyte, GSK, Silence Therapeutics and Novartis; honoraria from Novartis, GSK, AOP Orphan, Abbvie, BMS, Pfizer, Incyte, and other financial support (e.g., travel support) from AOP Orphan, Alexion, Janssen, Novartis, Pfizer, Mundipharma, Roche, and Hexal. M. Reiser reports consultancy fees from Amgen, Janssen, Novartis, Stemline, Abbvie, Milteny, and travel support from Beigene. M. Koenigsmann reports honoraria from Pfizer, Abbvie, Bristol‐Myers Squibb, and AstraZeneca; travel support from Abbvie, Lilly, and Gilead Sciences. B. Heinrich reports consultancy fees from AstraZeneca, Daiichi Sankyo, and Boehringer Ingelheim; research funding from AstraZeneca, Jansen, Lilly, Novartis, and Roche; speakers bureau fees from AstraZeneca and Daiichi; and advisory fees from Gilead, Roche, and AstraZeneca. E. von der Heyde reports consultancy fees and honoraria from Novartis, Bristol‐Myers Squibb, and AstraZeneca. H. Tesch reports research funding from Lilly, and honoraria and travel support from Lilly, Novartis, Roche, GSK, Seagan, Pfizer, Lilly, AstraZeneca, Daiichi, Exact Science, and Vifor. B. Gröschl reports provided services for Novartis. P. Bachhuber reports employment with Novartis and holds equity in Novartis. S. Großer reports employment with Novartis and holds equity in Novartis. M. Koehler reports consultancy fees from Novartis. H.L. Pahl reports consultancy, honoraria, and advisory fees from Novartis and AOP Pharma; research funding from Novartis. The other authors declare no conflicts of interest.

Figures

FIGURE 1
FIGURE 1
Study efficacy outcomes. (A) Mean spleen length over time; (B, C) proportions experiencing constitutional symptom changes from baseline in Arms A and B.
FIGURE 2
FIGURE 2
Overall survival by (A) study arm and (B) RR6 risk category in Arm A (sonographic spleen assessment only).
FIGURE 3
FIGURE 3
Changes from BL over time in patient‐reported outcomes. MPN‐SAF TSS.
See this image and copyright information in PMC

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