Prognostic factors and long‐term follow‐up of basiliximab for steroid‐refractory acute graft‐versus‐host disease: Updated experience from a large‐scale study

Acute graft‐vs‐host disease (aGVHD) is one of the most important causes of early mortality after allogeneic hematopoietic stem cell transplantation (allo‐HSCT), particularly for those with steroid‐refractory (SR)‐aGVHD. We aimed to identify the prognostic factors and long‐term clinical outcomes of basiliximab treatment for SR‐aGVHD. Basiliximab was administered on days 1, 3, and 8, and repeated weekly until aGVHD was less than grade II, or patients showed no response after four doses. Out of 1498 patients receiving allo‐HSCT, 230 patients with SR‐aGVHD were enrolled. Grade III to IV aGVHD before basiliximab treatment significantly and independently predicted a poorer response to basiliximab in multivariate analysis. And, the cumulative incidence of overall response at 14 days, 28 days, and 56 days after treatment was 41.4% vs 23.1% (P = .023), 70.2% vs 43.6% (P = .002), and 80.1% vs 66.7% (P = .013), respectively. This was for those with grade II and grade III to IV aGVHD. Patients receiving more than four doses of basiliximab had higher rates of infections. The 4‐year cumulative incidence of total and severe chronic GVHD after basiliximab treatment was 44.8% (95% CI 38.3%‐51.3%) and 2.2% (95% CI 0.3%‐4.1%), respectively. The 4‐year cumulative incidence of relapse, non‐relapse mortality, disease‐free survival, and overall survival after basiliximab treatment was 11.3% (95% CI 7.2%‐15.4%), 30.0% (95% CI 24.1%‐35.9%), 58.7% (95% CI 52.3%‐65.1%), and 61.7% (95% CI 55.4%‐68.0%), respectively. Comorbidities before allo‐HSCT and refined Minnesota aGVHD risk score at diagnosis had significant influences on long‐term survival. Thus, basiliximab was a safe and effective treatment for patients with SR‐aGVHD.