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A single-center, open-label, phase I/II trial (NCT03441958) assessed the safety and efficacy of UM171-expanded CB transplantation in 19 patients aged 18–65 years with high-risk or ultra-high-risk NDMM.1 UM171 is a potent hematopoietic stem cell self-renewal inducer that has been shown to expand CB stem cells and enhance multilineage blood cell recovery.1 Results from this trial were published in Transplantation and Cellular Therapy by Roy et al.1 |
Key learnings |
The median expansion of CD34+ cells after 7 days was 49.5-fold, with a median CD34+ cell dose of 4.62 × 106/kg. The median times to achieve ANC of 0.1 x 106/L and 0.5 x 106/L were 6 days and 10.5 days, while the median time to achieve a platelet count ≥20 x 106/L was 36 days. |
The 1-year cumulative incidences of Grade II–IV and Grade III–IV aGvHD were 68.4% and 5.3%, respectively, while the 2-year incidences of cGvHD and moderate/severe cGvHD were 15.8% and 10.5%, respectively. |
The 3-year CIR, PFS, OS, and NRM were 36.8%, 47.4%, 68.4%, and 15.8%, respectively. |
The safety findings were consistent with the known safety profile of CB transplantation. |
These results indicate that UM171-expanded CB transplantation is a feasible option in patients with high-risk and ultra-high-risk NDMM, showing the potential to reduce GvHD severity while maintaining rapid and effective engraftment. |
Abbreviations: aGvHD, acute graft-versus-host disease; ANC, absolute neutrophil count; CB, cord blood; cGvHD, chronic graft-versus-host disease; CIR, cumulative incidences of relapse; D, day; GvHD, graft-versus-host disease; NDMM, newly diagnosed multiple myeloma; NRM, non-relapse mortality; OS, overall survival; PFS, progression-free survival.
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