Single-agent Selinexor Versus Physician's Choice in Previously Treated Myelofibrosis: Results From the Phase 2 XPORT‑MF0035 Study

Rákkutatás · Myelofibrosis

Selinexor was generally well tolerated; common treatment‑emergent adverse events (TEAEs) included anemia, asthenia, and decreased weight.

Janus kinase inhibitors (JAKis) are the standard of care for myelofibrosis (MF) but responses are often incomplete and non‑durable. Selinexor, an oral selective inhibitor of Exportin 1 (XPO1), was evaluated versus physician’s choice (PC) in a Phase 2 randomized open‑label trial (XPORT‑MF0035) in patients with MF previously treated with a JAKi. The primary endpoint was spleen volume reduction ≥35% (SVR35). At 24 weeks, SVR35 was achieved by 8 % of patients in each arm; any‑time SVR35 was achieved by 17 % of selinexor patients and 8 % of PC patients. Selinexor was generally well tolerated with fewer grade ≥ 3 anemia events and a reduced transfusion burden. These results support further evaluation of selinexor in MF.

The study indicates that selinexor provides a modest spleen response and may improve cytopenia‑related safety compared with physician‑chosen therapies in patients with previously JAK‑inhibitor‑treated myelofibrosis, highlighting its potential as an additional therapeutic option.

Bizonyítékszint: Sejtvonalas. Laboratóriumi sejtekben vizsgálták.

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