Efficacy of Momelotinib in Myelofibrosis Patients: Results From a Multicenter Study.
СтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод пока не подготовлен
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Martorelli Maria Carmen, Pugliese Novella, Di Perna Maria, De Novellis Danilo, Lombardi Assunta, De Fazio Laura, Loglisci Giuseppina, Peluso Ilaria, Molica Matteo, Copia Carolina, Ricco Alessandra, Bruzzese Antonella, Mendicino Francesco, De Fazio Vincenza, Santeramo Teresa Maria, Bianco Rosario, Monaco Giuseppe, Fontana Raffaele, Guariglia Roberto, Serio Bianca, Luponio Serena, Della Corte Anna Maria, Malandrini Antonella, Giudice Valentina, Frigeri Ferdinando, Tarantini Giuseppe, Guarini Attilio, Gentile Massimo, Musto Pellegrino, Annunziata Mario, Di Renzo Nicola, Rossi Marco, Califano Catello, Pane Fabrizio, Selleri Carmine
Аннотация
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.