Efficacy of Momelotinib in Myelofibrosis Patients: Results From a Multicenter Study
Momelotinib, a novel JAK1/2 inhibitor with inhibitory activities on activin A receptor type I, has shown breakthrough clinical efficacy in patients with myelofibrosis (MF) and anemia, a disease-related manifestation of challenging management. In this retrospective real-life multicenter Italian study, we investigated the safety and efficacy of momelotinib in a cohort of 39 consecutive MF patients, regardless of prior therapy. The median duration of treatment was 7 months, and the overall response rate was 56% in transfusion-dependent patients and 46% in the transfusion-independent group. At 24 weeks of treatment, a hemoglobin increase > 1.5 g/dL was observed in 26% of patients, and constitutional symptom improvement was reported in 51% of cases, with a spleen volume reduction > 35% in 28%. Therapy discontinuation occurred in 18% of patients, with only one leukemia progression and three deaths during follow-up. The safety profile was similar to that reported in clinical trials, with most toxicities of grade I-II. In conclusion, our real-life results support the use of momelotinib as an effective and safe therapeutic option for heavily pre-treated, cytopenic MF patients in real-world clinical practice.
Authors
- Assunta Lombardi (ORCID: https://orcid.org/0009-0002-0553-1655)
- Matteo Molica (ORCID: https://orcid.org/0000-0001-6391-4406)
- Nicola Di Renzo (ORCID: https://orcid.org/0000-0003-0604-6934)
- Massimo Gentile (ORCID: https://orcid.org/0000-0002-5256-0726)
- G. Tarantini (ORCID: https://orcid.org/0000-0001-8635-6902)
- Teresa Maria Santeramo
- Rosario Bianco (ORCID: https://orcid.org/0000-0002-4470-4670)
- Mario Annunziata (ORCID: https://orcid.org/0000-0001-9993-947X)
- Raffaele Fontana (ORCID: https://orcid.org/0009-0000-3990-120X)
- Ilaria Peluso (ORCID: https://orcid.org/0000-0002-6210-5241)
- Giuseppina Loglisci
- Valentina Giudice (ORCID: https://orcid.org/0000-0002-7492-6848)
- Roberto Guariglia (ORCID: https://orcid.org/0009-0009-4165-8646)
- Antonella Bruzzese (ORCID: https://orcid.org/0000-0002-9456-2404)
- Pellegrino Musto (ORCID: https://orcid.org/0000-0003-3277-6594)
- Fabrizio Pane (ORCID: https://orcid.org/0000-0003-2563-4125)
- Attilio Guarini (ORCID: https://orcid.org/0000-0002-9304-6651)
- Alex Malandrini (ORCID: https://orcid.org/0009-0005-7857-6768)
- Serena Luponio (ORCID: https://orcid.org/0009-0002-7063-970X)
- Francesco Mendicino (ORCID: https://orcid.org/0000-0001-6339-632X)
- Anna Maria Della Corte (ORCID: https://orcid.org/0009-0000-1972-6336)
- Laura De Fazio (ORCID: https://orcid.org/0009-0004-9280-2023)
- Carmine Selleri
- Ferdinando Frigeri (ORCID: https://orcid.org/0000-0002-9818-3073)
- Carolina Copia
- Catello Califano (ORCID: https://orcid.org/0000-0001-5633-4657)
- Maria Di Perna (ORCID: https://orcid.org/0000-0003-2977-0634)
- Novella Pugliese (ORCID: https://orcid.org/0000-0002-2714-5109)
- Marco Rossi (ORCID: https://orcid.org/0000-0002-1936-3819)
- Bianca Serio (ORCID: https://orcid.org/0000-0001-6613-2370)
- Danilo De Novellis (ORCID: https://orcid.org/0000-0002-2590-0529)
- Giuseppe Monaco (ORCID: https://orcid.org/0000-0001-9704-0671)
- V. Fazio
- Alessandra Ricco
- Maria Carmen Martorelli
Institutions
- University of Salerno (IT)
- Ospedale Antonio Cardarelli (IT)
- Centro Cardiologico Monzino (IT)
- Azienda Universitaria Ospedaliera Consorziale - Policlinico Bari (IT)
- Azienda Ospedaliera di Cosenza (IT)
- Fatebenefratelli Hospital (IT)
- Azienda Ospedaliera S.Giuseppe Moscati (IT)
- CTO Andrea Alesini (IT)
- Federico II University Hospital (IT)
- Istituto Tumori Bari (IT)
- Ospedali Riuniti San Giovanni di Dio e Ruggi d'Aragona (IT)
- Ospedale Sant'Anna (IT)
- Ospedale Vito Fazzi (IT)
- University of Naples Federico II (IT)
Publication Details
- Journal
- European Journal Of Haematology
- Published
- 2025-09-16
- DOI
- https://doi.org/10.1111/ejh.70034
- Primary Topic
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Università degli Studi di Salerno