Shimomura Yoshimitsu, Kitamura Tetsuhisa, Sugita Junichi, Terao Toshiki, Satake Atsushi, Hirakawa Tsuneaki, Uchida Naoyuki, Shimabukuro Masashi, Tanaka Masatsugu, Eto Tetsuya, Hiramoto Nobuhiro, Kataoka Keisuke, Nakamae Hirohisa, Takase Ken, Kawakita Toshiro, Arai Yasuyuki, Takeda Wataru, Ishimaru Fumihiko, Fukuda Takahiro, Atsuta Yoshiko, Nakasone Hideki, Kanda Junya
Department of Hematology, Kobe City Hospital Organization Kobe City Medical Center General Hospital, Kobe, Japan.
Department of Environmental Medicine and Population Science, Graduate School of Medicine, Osaka University, Suita, Japan.
Bone Marrow Transplant. 2024 Dec;59(12):1763-1772. doi: 10.1038/s41409-024-02424-x. Epub 2024 Sep 30.
Systemic corticosteroid therapy is a well-established first-line treatment for grades II-IV acute graft-versus-host disease (aGVHD). Recently, several developments have occurred, including the introduction of transplantation from human leukocyte antigen (HLA) haploidentical donors using post-transplant cyclophosphamide (PTCY-Haplo), and improvements in prognosis after cord blood transplantation (CBT) in Japan. This study aimed to analyze the association between donor sources and outcomes in patients with aGVHD. Our study included 2732 patients who developed grades II-IV aGVHD, and were treated with systemic corticosteroids. We compared HLA-matched related donors (MRD), HLA-matched unrelated donors (MUD), PTCY-Haplo, and CBT. We set endpoint as response rate, 1-year cumulative incidence of non-relapse mortality (NRM), and overall survival (OS). The adjusted odds ratios for a complete response (CR) were 0.99 (95% confidence interval [CI]: 0.74-1.31, P = 0.925) for MUD, 2.08 (95% CI: 1.35-3.25, P = 0.001) for PTCY-Haplo, and 1.08 (95% CI: 0.83-1.41, P = 0.550) for CBT compared with MRD. A significant increase in response rates for PTCY were only found in a single-organ involvement. No significant association was observed between the donor source and NRM or OS. In conclusion, PTCY-Haplo is associated with a high response rate in patients with a single-organ aGVHD; however, MUD and CBT were not associated with treatment response.
全身糖皮质激素治疗是公认的II-IV级急性移植物抗宿主病（aGVHD）的一线治疗方法。最近，出现了一些新进展，包括采用移植后环磷酰胺（PTCY-Haplo）进行人类白细胞抗原（HLA）单倍体相合供者移植，以及日本脐带血移植（CBT）预后的改善。本研究旨在分析aGVHD患者供者来源与预后之间的关联。我们的研究纳入了2732例发生II-IV级aGVHD并接受全身糖皮质激素治疗的患者。我们比较了HLA匹配的亲属供者（MRD）、HLA匹配的非亲属供者（MUD）、PTCY-Haplo和CBT。我们将终点设定为缓解率、非复发死亡率（NRM）的1年累积发生率和总生存期（OS）。与MRD相比，MUD达到完全缓解（CR）的校正比值比为0.99（95%置信区间[CI]：0.74-1.31，P = 0.925），PTCY-Haplo为2.08（95%CI：1.35-3.25，P = 0.001），CBT为1.08（95%CI：0.83-1.41，P = 0.550）。仅在单器官受累的情况下发现PTCY的缓解率显著增加。未观察到供者来源与NRM或OS之间存在显著关联。总之，PTCY-Haplo与单器官aGVHD患者的高缓解率相关；然而，MUD和CBT与治疗反应无关。
