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MoReLife - real-life data support the potential of momelotinib as a safe and effective treatment option for cytopenic myelofibrosis patients

Recurrent problems of patients with myelofibrosis (MF) are cytopenias, debiliating disease-related symptoms and splenomegaly. Whereas the latter are usually addressed by the JAK1/2 inhibitors ruxolitinib and fedratinib, cytopenias often remain critical. Momelotinib, a JAK1/2 inhibitor recently appro...

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Main Authors: Jilg, Stefanie (Author) , Schwaab, Juliana (Author) , Sockel, Katja (Author) , Crodel, Carl C. (Author) , Brueckl, Valeska (Author) , Stegelmann, Frank (Author) , Jentzsch, Madlen (Author) , Sasca, Daniel (Author) , Moyses, Margarete (Author) , Fuhrmann, Stephan (Author) , Gundel, Daniel (Author) , Caduc, Madlen (Author) , Teichmann, Lino L. (Author) , Heidel, Florian (Author) , Al-Ali, Haifa K. (Author) , Petrides, Petro E. (Author)
Format: Article (Journal)
Language:English
Published: October 2024
In: Annals of hematology
Year: 2024, Volume: 103, Issue: 10, Pages: 4065-4077
ISSN:1432-0584
DOI:10.1007/s00277-024-05908-4
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1007/s00277-024-05908-4
Verlag, lizenzpflichtig, Volltext: https://link.springer.com/article/10.1007/s00277-024-05908-4#citeas
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Author Notes:Stefanie Jilg, Juliana Schwaab, Katja Sockel, Carl C. Crodel, Valeska Brueckl, Frank Stegelmann, Madlen Jentzsch, Daniel Sasca, Margarete Moyses, Stephan Fuhrmann, Daniel Gundel, Madlen Caduc, Lino L. Teichmann, Florian Heidel, Haifa K. Al-Ali, Petro E. Petrides
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Summary:Recurrent problems of patients with myelofibrosis (MF) are cytopenias, debiliating disease-related symptoms and splenomegaly. Whereas the latter are usually addressed by the JAK1/2 inhibitors ruxolitinib and fedratinib, cytopenias often remain critical. Momelotinib, a JAK1/2 inhibitor recently approved for the treatment of anemic MF patients, was shown to improve anemia via a direct inhibition of activin A receptor type I. In this German-wide, multicenter, retrospective analysis the safety and efficacy profile of momelotinib was evaluated in a real world setting within a cohort of 60 MF patients independent of pre-treatment. The median duration of treatment was 12 weeks. As a new, but manageable safety finding, creatinine increase (CTC°1-2) was detected in 10/60 patients (17%). Interestingly, not only hemoglobin levels increased in 84% of patients, but also platelet values (67%). In the cohort of transfusion-dependent individuals (n = 38), transfusion requirement improved in 15 patients (39%) with 8 reaching transfusion independency (21%). Transfusion independency was achieved within a median of 4 weeks (range 2-12). Spleen size decreased in 13/53 individuals (25%) with a median response time of 6 weeks. Thereof, 11 patients had been pre-treated with JAK inhibitor(s) (85%). Clinical improvement was detected in 24/51 symptomatic individuals (47%) with a median response time of 4 weeks. 5 patients stopped treatment due to side effects (8%), 6 patients due to a worsening of clinical symptoms (10%). Taken together, the MoReLife analysis identifies momelotinib as potent and safe therapeutic option also for heavily pre-treated cytopenic MF patients under real world conditions.
Item Description:Online veröffentlicht: 29. Juli 2024
Gesehen am 18.11.2024
Physical Description:Online Resource
ISSN:1432-0584
DOI:10.1007/s00277-024-05908-4

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