[Efficacy and safety of basiliximab in gastrointestinal chronic graft-versus-host disease].

Objective: To explore the efficacy and safety of basiliximab for gastrointestinal (GI) chronic graft-versus-host disease (cGVHD) . Methods: This was a retrospective case series. Patients who developed GI-cGVHD after transplantation at the Peking University Institute of Hematology between January 1, 2018, and December 31, 2023, were included. Outcomes included response rate, time to response, overall survival (OS) , and the incidence of neutropenia, anemia, thrombocytopenia, and infections. Results: A total of 41 patients with GI-cGVHD were included, with a median follow-up of 622 (range, 112-2 418) days. All patients received initial therapy including glucocorticoids, calcineurin inhibitors, and/or other immunosuppressive agents (eg, mycophenolate mofetil, mesenchymal stem cells, and sirolimus) . In the basiliximab group (n=25) , baseline diarrhea was grade 2-4 (grade 2, n=4; grade 3, n=18; grade 4, n=3) . Basiliximab was initiated a median of 6 days after initial therapy, and 12 patients (48.0% ) received >4 doses. In the basiliximab group, 22 patients (88.0% ) achieved complete response (CR) . Among patients who achieved CR, 15 (68.2% ) responded within ≤4 doses. In the non-basiliximab group (n=16) , baseline diarrhea was significantly milder than in the basiliximab group (P<0.001) and was grade 1-2 in all patients (grade 1, n=12; grade 2, n=4) . All patients in this group achieved CR. The response rate did not differ significantly between groups (88.0% vs 100.0% , P=0.150) . The 2-year OS rates were 75.4% and 86.7% (P=0.494) , the 2-year cumulative relapse rates were 16.9% and 14.3% (P=0.913) , and the 2-year non-relapse mortality rates were 14.2% and 6.7% (P=0.526) in the basiliximab and non-basiliximab groups, respectively. No new grade 3-4 hepatic or renal dysfunction or cytokine release syndrome was observed in the basiliximab group. In the basiliximab group, neutropenia, anemia, and thrombocytopenia occurred in 24.0% , 28.0% , and 28.0% of patients, respectively. Any infection, viral infection, bacterial infection, and fungal infection occurred in 52.0% , 36.0% , 32.0% , and 4.0% of patients, respectively. Conclusion: Basiliximab may be used to treat GI-cGVHD, with hematologic and infection-related adverse event rates comparable to those of other systemic immunosuppressants. These findings suggest a potential therapeutic option for GI-cGVHD; however, larger prospective studies are needed to further evaluate its safety.