Effectiveness of a modified doxorubicin-etoposide-methylprednisolone regimen for the treatment of adult refractory/relapsed macrophage activation syndrome

Effectiveness of a modified DEP regimen in adult refractory MAS 1 All patients underwent gene sequencing and were treated with the modified DEP regimen.The regimen included: liposomal doxorubicin 25 mg/m 2 on day 1; etoposide 100 mg/m 2 on day 1; methylprednisolone 2 mg/m 2 on days 1 to 3, 0.75 mg/m 2 on days 4 to 6, 0.25 mg/m 2 on days 7 to 9, and 0.1 mg/m 2 on days 10 to 21. 5 The clinical effectiveness and side effects were observed.The treatment assessment was carried out every 2 weeks.Details on the evaluation criteria are described in Supplementary material, Table S1.The survival time was calculated from the time of MAS diagnosis until death or May 2021.Statistical analysis Statistical analysis was performed using SPSS 26.0 (IBM Corp, Armonk, New York, United States).Nonnormally distributed variables were reported as medians and ranges.A survival analysis was performed using the Kaplan-Meier method.Results A total of 33 patients were enrolled, with a median follow -up period of 33 months (range, 1-71 months).Baseline patient characteristics are shown in Supplementary material, Table S2.Baseline laboratory values are presented in TAbLE 1. Gene sequencing revealed mutations in primary HLH -related genes in 15 patients (45.5%), but no pathogenic mutations were detected.Mutations in the UNC13D gene were the most common (5 patients [15.2%]).Details on the genetic variations are shown in Supplementary material, Table S3.After the first course of the modified DEP regimen, 27 patients (81.8%) achieved remission, with a CR rate of 15.5% (5 patients) and a PR rate of 66.7% (22 patients).Six patients (18.2%) showed