What question did this study set out to answer?

This trial aimed to assess the efficacy and safety of selinexor combined with VRD in patients with newly diagnosed multiple myeloma and extramedullary disease.

January 24, 2026

Efficacy and Safety of Selinexor Combined with VRD in Newly Diagnosed Multiple Myeloma with EMD: A Phase 2 Trial.

Puntos clave

This trial aimed to assess the efficacy and safety of selinexor combined with VRD in patients with newly diagnosed multiple myeloma and extramedullary disease.
Multicenter, open-label, single-arm phase II trial
30 patients enrolled, with 29 treated forming modified intention-to-treat cohort
Induction treatment of four 28-day cycles with selinexor, bortezomib, lenalidomide, and dexamethasone
Primary endpoint was overall response rate during induction
Overall response rate achieved was 89.7%, with 58.6% achieving stringently complete response
Imaging showed regression of extramedullary disease in 89.7% of patients
12-month progression-free survival rate was 87.9% and overall survival rate was 96.3%
Grade ≥3 adverse events occurred in 37.3%, primarily thrombocytopenia and neutropenia

Resumen

Extramedullary disease (EMD) at diagnosis confers a poor prognosis in newly diagnosed multiple myeloma (NDMM). This multicenter, open-label, single-arm phase II investigator-initiated trial evaluated selinexor combined with bortezomib, lenalidomide, and dexamethasone (SVRD) in NDMM with EMD. Between October 17, 2022 and November 27, 2025, 30 patients were enrolled; 29 treated patients formed the modified intention-to-treat (mITT) and safety populations (median age 60 years). Induction comprised four 28-day SVRD cycles with protocol-specified consolidation/maintenance and optional ASCT. The primary endpoint was best overall response rate (ORR) during induction per IMWG; patients without a post-baseline assessment were imputed non-responders. In the mITT cohort, ORR was 89.7% (sCR 58.6%, CR 3.4%, VGPR 10.3%, PR 17.2%). Imaging documented EMD regression in 89.7% (complete resolution 79.3%, partial 10.3%); 12-month PFS and OS rates were 87.9% and 96.3%, respectively (medians not reached; median follow-up 18 months). High-risk cytogenetics were present in 31.0%, and 27.6% met ultra-high-risk ("double-hit") criteria. Grade ≥3 treatment-emergent adverse events occurred in 37.3% patients, most commonly thrombocytopenia (24.1%), neutropenia (6.9%), and pneumonia (10.3%); no treatment-related deaths occurred. SVRD produced deep hematologic responses and high EMD clearance with manageable toxicity, enabling ASCT in 51.7%. These findings support SVRD as a rational frontline option for EMD-positive NDMM and justify randomized studies to confirm durability and benchmark against contemporary quadruplets and cellular therapies. NCT# NCT05900882.

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