Safety, effectiveness and pharmacokinetics of high-dose propylene glycol-free melphalan (EVOMELA) with a prolonged infusion as myeloablative conditioning in Chinese multiple myeloma patients undergoing autologous stem cell transplantation: A prospective phase iv study.

In 30 seconds

This prospective, open-label, non-randomized phase IV study evaluated prolonged-infusion, high-dose BSES-melphalan conditioning in 67 Chinese patients with multiple myeloma undergoing autologous stem cell transplantation. Patients received 200 mg/m² in two doses before transplantation. All achieved myeloablation; median neutrophil and platelet engraftment times were 11 and 12 days, respectively. Among 65 evaluable patients, 27.7% achieved stringent complete response and 32.3% complete response. No treatment-related deaths or adverse events leading to withdrawal were reported. Prolonged infusion was associated with lower peak concentration but comparable AUCs.

Key findings

• All 67 patients achieved myeloablation, with a median time of 5 days.
• Median neutrophil and platelet engraftment occurred 11 and 12 days after transplantation, respectively.
• Among 65 evaluable patients, 18 (27.7%) achieved stringent complete response and 21 (32.3%) complete response.
• Prolonged infusion produced a lower Cmax with comparable AUCs; no treatment-related mortality was identified.

Why it matters

The findings provide prospective evidence on the use of prolonged-infusion BSES-melphalan conditioning in Chinese patients with multiple myeloma undergoing transplantation. Engraftment and response outcomes were reported alongside pharmacokinetic and safety data, helping characterize this regimen in the studied population. The lower peak concentration with comparable AUCs is relevant to evaluating infusion duration, although the study does not establish comparative benefit.

What to keep in mind

The study was open-label and non-randomized, with no comparator group; response outcomes were available for 65 of the 67 enrolled patients.