Department of Hematology, Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
Department of Hematology, Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China; Peking-Tsinghua Center for Life Sciences, Beijing, China.
Biol Blood Marrow Transplant. 2020 Feb;26(2):351-357. doi: 10.1016/j.bbmt.2019.10.031. Epub 2019 Nov 5.
Basiliximab has been used successfully as a second-line treatment for steroid-refractory (SR) acute graft-versus-host disease (aGVHD) in adult patients after haploidentical hematopoietic stem cell transplant (haplo-HSCT) but has not been studied separately in the pediatric setting. We retrospectively reviewed 100 pediatric patients after haplo-HSCT receiving basiliximab for grades II (57%), III (27%), and IV (16%) SR aGVHD between January 2015 and December 2017. The median number of basiliximab doses was 4 (range, 2 to 9). The day 28 overall response rate was 85%, with complete response in 74% of patients, partial response in 11% of patients, and no response in 15% of patients. The day 28 overall response rates were 94.6% in skin SR aGVHD, 81.6% in gut SR aGVHD, and 66.7% in liver SR aGVHD. Infectious complications included bacterial infection (11%), presumed or documented fungal infections (7%), cytomegalovirus viremia (53%), Epstein-Barr virus viremia (11%), human herpesvirus-6 viremia (7%), and herpes simplex virus viremia (1%). The 3-year overall survival, disease-free survival, nonrelapse mortality, and relapse rates between responders and nonresponders were 81.3% versus 46.7% (P < .001), 79.0% versus 46.7% (P = .001), 6.1% versus 33.3% (P < .001), and 14.9% versus 20.0% (P = .46), respectively. We conclude that basiliximab is an effective second-line agent for pediatric patients with SR aGVHD after haplo-HSCT, particularly for skin SR aGVHD.
巴利昔单抗已成功用于单倍体造血干细胞移植（haplo-HSCT）后成人患者类固醇难治性（SR）急性移植物抗宿主病（aGVHD）的二线治疗，但尚未在儿科环境中单独研究。我们回顾性分析了 2015 年 1 月至 2017 年 12 月期间接受巴利昔单抗治疗的 100 例 haplo-HSCT 后发生 II 级（57%）、III 级（27%）和 IV 级（16%）SR aGVHD 的儿科患者。巴利昔单抗剂量中位数为 4 剂（范围 2 至 9 剂）。第 28 天总缓解率为 85%，完全缓解率为 74%，部分缓解率为 11%，无反应率为 15%。皮肤 SR aGVHD 的第 28 天总缓解率为 94.6%，肠道 SR aGVHD 为 81.6%，肝脏 SR aGVHD 为 66.7%。感染并发症包括细菌感染（11%）、推定或确诊真菌感染（7%）、巨细胞病毒血症（53%）、EB 病毒血症（11%）、人疱疹病毒 6 血症（7%）和单纯疱疹病毒血症（1%）。缓解组和未缓解组的 3 年总生存率、无病生存率、非复发死亡率和复发率分别为 81.3%比 46.7%（P<0.001）、79.0%比 46.7%（P=0.001）、6.1%比 33.3%（P<0.001）和 14.9%比 20.0%（P=0.46）。我们的结论是，巴利昔单抗是 haplo-HSCT 后发生 SR aGVHD 的儿科患者的有效二线治疗药物，特别是皮肤 SR aGVHD。
