[First-line treatment for chronic graft-versus-host disease: a real-world study].

Abstract

Objective: To systematically evaluate the efficacy, safety, quality of life, and related influencing factors of first-line treatment in patients with chronic graft-versus-host disease (cGVHD) and to provide evidence-based support for optimizing treatment strategies. Methods: This single-center retrospective analysis involved 243 patients diagnosed as having cGVHD who received first-line treatment at the Second Affiliated Hospital of Army Medical University between January 2017 and December 2023. Of the patients, 159 (65.4% ) received the standard regimen (corticosteroids with or without calcineurin inhibitors), 51 (21.0% ) received corticosteroids plus a second-line agent (ruxolitinib or sirolimus), and 33 (13.6% ) did not receive corticosteroids as first-line therapy due to contraindications (ruxolitinib alone). The primary endpoints were the overall response rate (ORR) and failure-free survival (FFS) at 6 months after treatment. The secondary endpoints comprised organ-specific responses, survival rates, nonrelapse mortality (NRM), recurrence rates, and quality of life [assessed using the Lee Symptom Scale (LSS), the 36-Item Short Form Health Survey (SF-36), and the EuroQol 5-Dimension 5-Level instrument (EQ-5D-5L) ]. Results: At 6 months after treatment, 132 patients achieved complete or partial response to first-line therapy, yielding an ORR of 60% and an FFS of 73.6%. ORRs were higher in the joint/fascia (68.2% ) and the skin (66.4% ) and the lowest in the lungs (30.0% ). The median follow-up was 43.9 months, the 5-year overall survival rate was 80.0%, and the 5-year NRM was 7.5%. Both overall survival and nonrelapse mortality were significantly better in responders than in nonresponders (P<0.01 and P=0.01, respectively). Multivariate analysis identified liver involvement as an independent risk factor for adverse treatment reactions (OR=0.506, P=0.021). Quality-of-life surveys indicated that the symptom burden was significantly lower in responders than in nonresponders (7.3±5.6 vs 12.3±7.8, P=0.01), whereas no significant between-group differences were observed in SF-36 physical and mental component scores or the EQ-5D-5L utility index. Conclusion: Glucocorticoid-based first-line treatment showed acceptable efficacy and reduced symptom burden in some patients with cGVHD. However, significant heterogeneity was observed in treatment responses among different organs, and liver involvement indicated poor prognosis.