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- Title
Mini-dose methotrexate combined with methylprednisolone for the initial treatment of acute GVHD: a multicentre, randomized trial.
- Authors
Wang, Yu; Liu, Qi-Fa; Wu, De-Pei; Xu, Zheng-Li; Han, Ting-Ting; Sun, Yu-Qian; Huang, Fen; Fan, Zhi-Ping; Xu, Na; Chen, Feng; Zhao, Ye; Kong, Yuan; Mo, Xiao-Dong; Xu, Lan-Ping; Zhang, Xiao-Hui; Liu, Kai-Yan; Huang, Xiao-Jun
- Abstract
Background: There is an urgent unmet need for effective initial treatment for acute graft-versus-host disease (aGVHD) adding to the standard first-line therapy with corticosteroids after allogeneic haematopoietic stem cell transplantation (allo-HSCT). Methods: We performed a multicentre, open-label, randomized, phase 3 study. Eligible patients (aged 15 years or older, had received allo-HSCT for a haematological malignancy, developed aGVHD, and received no previous therapies for aGVHD) were randomly assigned (1:1) to receive either 5 mg/m2 MTX on Days 1, 3, or 8 and then combined with corticosteroids or corticosteroids alone weekly. Results: The primary endpoint was the overall response rate (ORR) on Day 10. A total of 157 patients were randomly assigned to receive either MTX plus corticosteroids (n = 78; MTX group) or corticosteroids alone (n = 79; control group). The Day 10 ORR was 97% for the MTX group and 81% for the control group (p =.005). Among patients with mild aGVHD, the Day 10 ORR was 100% for the MTX group and 86% for the control group (p =.001). The 1-year estimated failure-free survival was 69% for the MTX group and 41% for the control group (p =.002). There were no differences in treatment-related adverse events between the two groups. Conclusions: In conclusion, mini-dose MTX combined with corticosteroids can significantly improve the ORR in patients with aGVHD and is well tolerated, although it did not achieve the prespecified 20% improvement with the addition of MTX. Trial registration: The trial was registered with clinicaltrials.gov (NCT04960644).
- Subjects
STEM cell transplantation; HEMATOPOIETIC stem cell transplantation; METHOTREXATE; ADVERSE health care events; METHYLPREDNISOLONE; GRAFT versus host disease
- Publication
BMC Medicine, 2024, Vol 22, Issue 1, p1
- ISSN
1741-7015
- Publication type
Article
- DOI
10.1186/s12916-024-03395-y