Mini-dose methotrexate plus methylprednisolone for patients with aGvHD: Results from a phase III trial

Results from a phase III trial (NCT04960644) evaluating the safety and efficacy of mini-dose methotrexate combined with corticosteroids for the treatment of patients with acute graft-versus-host disease (aGvHD) were published in BMC Medicine by Wang, et al.¹ Patients aged 15–65 years with previously untreated aGvHD were randomized 1:1 to receive either mini-dose methotrexate (MTX) plus methylprednisolone (MTX group, n = 78) or methylprednisolone alone (control group, n = 79).¹

The study met its primary endpoint of overall response rate on Day 10 (97% vs 81% in the MTX vs control group; p = 0.005), suggesting that mini-dose MTX plus methylprednisolone could be a viable option to enhance early treatment responses in patients with aGvHD.

Additionally, the 1-year estimated failure-free survival rate was improved in the MTX vs control group (69% vs 41%; p = 0.002).

Mini-dose MTX plus methylprednisolone was well tolerated, and incidences of cytopenias and mucositis were similar between groups. 

These findings, of improved efficacy and a tolerable safety profile compared with corticosteroids alone, support the integration of mini-dose MTX plus corticosteroids into clinical practice for the management of aGVHD and highlight a potential pathogenesis-oriented therapeutic approach.