Abstract：
BACKGROUND: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an important therapeutic option for patients with hematological malignancies. However, the development of graft-versus-host disease (GVHD) after allo-HSCT remains a challenge. Although systemic steroid therapy is the established first-line therapy for acute GVHD (aGVHD) and chronic GVHD (cGVHD), many patients are unresponsive or resistant to corticosteroid therapy, and the response is insufficient.
OBJECTIVE: To evaluate the clinical characteristics of patients who developed aGVHD and cGVHD after allo-HSCT.
METHODS: This noninterventional, retrospective study used large national registry data from the Transplant Registry Unified Management Program. The study included 29,690 patients with hematological diseases who underwent their first allo-HSCT between January 2010 and December 2019. The primary endpoints of this study were the cumulative incidence of aGVHD and cGVHD. The secondary endpoints were overall survival (OS) and non-relapse mortality (NRM) of patients with aGVHD and cGVHD and OS and NRM of patients who received second-line therapy for aGVHD.
RESULTS: Of 29,690 patients who underwent allo-HSCT, 2,807, 6,167, 10,556, 774, and 9,339 patients received related bone marrow (RBM), related peripheral blood (RPB), unrelated bone marrow, unrelated peripheral blood (UPB), and unrelated cord blood, respectively. The cumulative incidence of aGVHD (grades II-IV) at 100 days was high after the related and unrelated mismatched transplantation. Furthermore, response rate for the first- and second-line therapy for aGVHD was low in the RBM/RPB-mismatched (59.6%/61.6%) and UPB-mismatched subgroup (45.5%), respectively. The 3-year NRM in patients with aGVHD was high in the RPB and UPB mismatched subgroups (37.9% and 31.2%, respectively).
CONCLUSIONS: Developing a novel treatment for steroid-refractory aGVHD is necessary to improve transplant outcomes, particularly for patients undergoing HLA-mismatched transplantation.
OBJECTIVE: To evaluate the clinical characteristics of patients who developed aGVHD and cGVHD after allo-HSCT.
METHODS: This noninterventional, retrospective study used large national registry data from the Transplant Registry Unified Management Program. The study included 29,690 patients with hematological diseases who underwent their first allo-HSCT between January 2010 and December 2019. The primary endpoints of this study were the cumulative incidence of aGVHD and cGVHD. The secondary endpoints were overall survival (OS) and non-relapse mortality (NRM) of patients with aGVHD and cGVHD and OS and NRM of patients who received second-line therapy for aGVHD.
RESULTS: Of 29,690 patients who underwent allo-HSCT, 2,807, 6,167, 10,556, 774, and 9,339 patients received related bone marrow (RBM), related peripheral blood (RPB), unrelated bone marrow, unrelated peripheral blood (UPB), and unrelated cord blood, respectively. The cumulative incidence of aGVHD (grades II-IV) at 100 days was high after the related and unrelated mismatched transplantation. Furthermore, response rate for the first- and second-line therapy for aGVHD was low in the RBM/RPB-mismatched (59.6%/61.6%) and UPB-mismatched subgroup (45.5%), respectively. The 3-year NRM in patients with aGVHD was high in the RPB and UPB mismatched subgroups (37.9% and 31.2%, respectively).
CONCLUSIONS: Developing a novel treatment for steroid-refractory aGVHD is necessary to improve transplant outcomes, particularly for patients undergoing HLA-mismatched transplantation.
摘要：
背景：异基因造血干细胞移植（allo-HSCT）是血液系统恶性肿瘤患者的重要治疗选择。然而,allo-HSCT后移植物抗宿主病(GVHD)的发展仍然是一个挑战.尽管全身性类固醇治疗是急性GVHD（aGVHD）和慢性GVHD（cGVHD）的既定一线治疗，许多患者对皮质类固醇治疗无反应或耐药，反应不足。
目的：评估allo-HSCT后发生aGVHD和cGVHD患者的临床特征。
方法：这种非干预性，回顾性研究使用来自移植注册中心统一管理计划的大型国家注册中心数据.该研究包括29,690名血液病患者，他们在2010年1月至2019年12月期间接受了首次allo-HSCT。这项研究的主要终点是aGVHD和cGVHD的累积发病率。次要终点是aGVHD和cGVHD患者的总生存期（OS）和非复发死亡率（NRM），以及接受aGVHD二线治疗的患者的OS和NRM。
结果：在29,690名接受allo-HSCT的患者中，2,807,6,167,10,556,774和9,339名患者接受相关骨髓（RBM），相关外周血（RPB），无关骨髓,无关外周血（UPB），和无关的脐带血，分别。相关和无关的错配移植后100天时aGVHD（II-IV级）的累积发生率很高。此外，在RBM/RPB不匹配（59.6％/61.6％）和UPB不匹配（45.5％）的亚组中，aGVHD的一线和二线治疗的反应率较低，分别。在RPB和UPB不匹配的亚组中，aGVHD患者的3年NRM较高（37.9%和31.2%，分别)。
结论：开发一种新的治疗类固醇难治性aGVHD的方法对于改善移植结果是必要的，特别是对于接受HLA不匹配移植的患者。
目的：评估allo-HSCT后发生aGVHD和cGVHD患者的临床特征。
方法：这种非干预性，回顾性研究使用来自移植注册中心统一管理计划的大型国家注册中心数据.该研究包括29,690名血液病患者，他们在2010年1月至2019年12月期间接受了首次allo-HSCT。这项研究的主要终点是aGVHD和cGVHD的累积发病率。次要终点是aGVHD和cGVHD患者的总生存期（OS）和非复发死亡率（NRM），以及接受aGVHD二线治疗的患者的OS和NRM。
结果：在29,690名接受allo-HSCT的患者中，2,807,6,167,10,556,774和9,339名患者接受相关骨髓（RBM），相关外周血（RPB），无关骨髓,无关外周血（UPB），和无关的脐带血，分别。相关和无关的错配移植后100天时aGVHD（II-IV级）的累积发生率很高。此外，在RBM/RPB不匹配（59.6％/61.6％）和UPB不匹配（45.5％）的亚组中，aGVHD的一线和二线治疗的反应率较低，分别。在RPB和UPB不匹配的亚组中，aGVHD患者的3年NRM较高（37.9%和31.2%，分别)。
结论：开发一种新的治疗类固醇难治性aGVHD的方法对于改善移植结果是必要的，特别是对于接受HLA不匹配移植的患者。
