Single agent selinexor is active in patients with myelofibrosis refractory or intolerant to JAK inhibitors

Rákkutatás · Myelofibrosis

Selinexor provides a measurable reduction in spleen volume and symptom score in JAK‑inhibitor–refractory myelofibrosis patients.

Phase II study of selinexor in patients with myelofibrosis refractory or intolerant to JAK inhibitors (ESSENTIAL). 17 patients (median age 66) were enrolled; median duration of JAK‑inhibitor therapy 13 months. Overall, 17% of patients achieved ≥35% reduction in spleen volume at week 24 (SVR₃₅). No complete responses were observed. The most common grade ≥ 3 adverse events were nausea, fatigue, anorexia, and diarrhea; the drug was well tolerated at the dose of 60 mg weekly. Median progression‑free survival was 28 months; median overall survival 35.6 months. Thus, selinexor shows modest clinical activity and acceptable safety in JAK‑inhibitor–refractory myelofibrosis.

A new therapeutic option for patients who fail standard JAK‑inhibitor treatment, offering a clinically relevant spleen response and acceptable toxicity profile.

Bizonyítékszint: Állatkísérletes. Állatmodellben vizsgálták.

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