Basiliximab for steroid‐refractory acute graft‐versus‐host disease: A real‐world analysis

Abstract

Steroid‐refractory (SR) acute graft‐versus‐host disease (aGVHD) is one of the leading causes of early mortality after allogeneic hematopoietic stem cell transplantation (allo‐HSCT). We investigated the efficacy, safety, prognostic factors, and optimal therapeutic protocol for SR‐aGVHD patients treated with basiliximab in a real‐world setting. Nine hundred and forty SR‐aGVHD patients were recruited from 36 hospitals in China, and 3683 doses of basiliximab were administered. Basiliximab was used as monotherapy (n = 642) or in combination with other second‐line treatments (n = 298). The cumulative incidence of overall response rate (ORR) at day 28 after basiliximab treatment was 79.4% (95% confidence interval [CI] 76.5%–82.3%). The probabilities of nonrelapse mortality and overall survival at 3 years after basiliximab treatment were 26.8% (95% CI 24.0%–29.6%) and 64.3% (95% CI 61.2%–67.4%), respectively. A 1:1 propensity score matching was performed to compare the efficacy and safety between the monotherapy and combined therapy groups. Combined therapy did not increase the ORR; conversely, it increased the infection rates compared with monotherapy. The multivariate analysis showed that combined therapy, grade III–IV aGVHD, and high‐risk refined Minnesota aGVHD risk score before basiliximab treatment were independently associated with the therapeutic response. Hence, we created a prognostic scoring system that could predict the risk of having a decreased likelihood of response after basiliximab treatment. Machine learning was used to develop a protocol that maximized the efficacy of basiliximab while maintaining acceptable levels of infection risk. Thus, real‐world data suggest that basiliximab is safe and effective for treating SR‐aGVHD.

Efficacy and safety of ruxolitinib in steroid-refractory graft-versus-host disease: A meta-analysis

“…In recently real-world studies, the ORR and CRR of basiliximab at day 28 were 0.66–0.79 and 0.52–0.61, respectively in patients with SR-aGVHD ( 65 , 66 ). Compared with this study, the efficacy of basiliximab seemed to be compared with ruxolitinib.…”

Salvage therapy with basiliximab and etanercept for severe steroid‐refractory acute graft‐versus‐host disease

“…While such cases tend to be less responsive to basiliximab monotherapy and other salvage approaches, we were encouraged by an ORR >70% reported with basiliximab + etanercept when used earlier in the course of SRaGVHD management. 5 The ORR was lower and the incidence of severe infections was higher than observed in the aforementioned studies when used earlier in the management of SRaGVHD, [2][3][4][5] which is expected given these patients received basiliximab + etanercept as third-or fourth-line treatment, and all had grade 3-4 SRaGVHD. We found basiliximab + etanercept salvage therapy to result in a high ORR in severe SRaGVHD in this small, single-center, "real world" analysis, including responses in patients with inadequate response to novel agents such as tocilizumab and Rux.…”

“…Severe SRaGVHD was present in 48.1% at the time of basiliximab initiation. 4 Patients started basiliximab early, at a median of 5 days from aGVHD occurrence. The ORR was 79.4%, with CR observed in 52.2%.…”

A Predicted Model for Refractory/Recurrent Cytomegalovirus Infection in Acute Leukemia Patients After Haploidentical Hematopoietic Stem Cell Transplantation

“…ATG, cyclosporine A (CSA), mycophenolate mofetil, and short-term methotrexate were administered to prevent GVHD ( Supplementary Methods ) ( Wang et al., 2019 ). Protocol for GVHD and minimal residual disease therapy had been reported in detail ( Liu et al., 2020 ; Zhao et al., 2021 ; Fan et al., 2021 ; Shen et al., 2021 ; Mo et al., 2022 ; Shen et al., 2022 ; Shen et al., 2022 ).…”