Steroid-refractory acute graft-versus-host disease (SR-aGVHD) is a serious complication that negatively affects the prognosis and quality of life of patients who receive allogeneic hematopoietic stem cell transplantation (allo-HSCT). Antithymocyte globulin (ATG) is one of the second-line treatments for SR-aGVHD. We retrospectively evaluated Karnofsky Performance Status (KPS) recovery and clinical response in 11 patients who received the response-guided low-dose ATG treatment for SR-aGVHD after allo-HSCT using alternative donors. The median dose of ATG per cycle was 1.0 mg/kg (range, 1.0–1.25 mg/kg) and the median number of cycles of ATG was 2 (range, 1–4). The overall response rate was 63.6%, and the estimated overall survival rate at 1 year was 63.6%. Two out of seven patients who survived 1 year after the response-guided ATG treatment had KPS of 80 or higher. The remaining 5 patients had KPS of lower than 80 due to moderate chronic GVHD (cGVHD) and/or ≥grade 3 infectious complications. Based on the poor prognosis of patients with SR-aGVHD, the response-guided ATG treatment represents one therapeutic option. The present results also suggest that chronic GVHD and infectious complications after the response-guided ATG treatment were associated with decreased KPS recovery and impaired social function.

类固醇难治性急性移植物抗宿主病 (SR-aGVHD) 是一种严重的并发症，会对接受异基因造血干细胞移植 (allo-HSCT) 的患者的预后和生活质量产生负面影响。抗胸腺细胞球蛋白 (ATG) 是 SR-aGVHD 的二线治疗之一。我们回顾性评估了 11 名患者的 Karnofsky 体能状态 (KPS) 恢复和临床反应，这些患者在使用替代供体的 allo-HSCT 后接受反应指导的低剂量 ATG 治疗 SR-aGVHD。每个周期 ATG 的中位剂量为 1.0 mg/kg（范围，1.0-1.25 mg/kg），ATG 的中位周期数为 2（范围，1-4）。总缓解率为 63.6%，1 年估计总生存率为 63.6%。在反应指导的 ATG 治疗后存活 1 年的 7 名患者中有 2 名 KPS 为 80 或更高。由于中度慢性 GVHD (cGVHD) 和/或≥3 级感染并发症，其余 5 名患者的 KPS 低于 80。基于 SR-aGVHD 患者的不良预后，响应引导的 ATG 治疗代表了一种治疗选择。目前的结果还表明，响应引导的 ATG 治疗后的慢性 GVHD 和感染并发症与 KPS 恢复减少和社会功能受损有关。