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.

• Authors: Xiao-Dong Mo, Shen-Da Hong, Yan-Li Zhao, Er-Lie Jiang, Jing Chen, Yang Xu, Zi-Min Sun, Wei-Jie Zhang, Qi-Fa Liu, Dai-Hong Liu, Ding-Ming Wan, Wen-Jian Mo, Han-Yun Ren, Ting Yang, He Huang, Xi Zhang, Xiao-Ning Wang, Xian-Min Song, Su-Jun Gao, Xin Wang, Yi Chen, Bing Xu, Ming Jiang, Xiao-Bing Huang, Xin Li, Hong-Yu Zhang, Hong-Tao Wang, Zhao Wang, Ting Niu, Ji-Shi Wang, Ling-Hui Xia, Xiao-Dan Liu, Fei Li, Fang Zhou, Tao Lang, Jiong Hu, Sui-Jing Wu, Xiao-Jun Huang
• Journal: American journal of hematology (Am J Hematol) Vol. 97 Issue 4 Pg. 458-469 (04 2022) ISSN: 1096-8652 [Electronic] United States
• PMID: 35064928 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2022 Wiley Periodicals LLC.

Chemical References

• Steroids
• Basiliximab

Topics

• Acute Disease
• Basiliximab (therapeutic use)
• Graft vs Host Disease (drug therapy, etiology)
• Hematopoietic Stem Cell Transplantation (adverse effects, methods)
• Humans
• Retrospective Studies
• Steroids (therapeutic use)