Mitsuyoshi Takaya, Arai Yasuyuki, Kondo Tadakazu, Kawata Takahito, Hirabayashi Shigeki, Tanaka Masatsugu, Mori Yasuo, Doki Noriko, Nishida Tetsuya, Kotani Takeharu, Ogata Masao, Tabayashi Takayuki, Eto Tetsuya, Sawa Masashi, Imada Kazunori, Kanda Junya, Ichinohe Tatsuo, Atsuta Yoshiko, Yanada Masamitsu
Department of Hematology Kyoto University Kyoto Japan.
Division of Precision Medicine Kyushu University Graduate School of Medical Science Fukuoka Japan.
EJHaem. 2025 Jan 21;6(1):e21088. doi: 10.1002/jha2.1088. eCollection 2025 Feb.
Understanding differences in clinical outcomes between PBSCT and BMT is important, and this study compared outcomes of HLA-matched related PBSCT and BMT using reduced-intensity conditioning (RIC) in adult acute myeloid leukemia (AML) patients.
Data from 402 patients who underwent either PBSCT ( = 294) or BMT ( = 108) between 2000 and 2022 were analyzed using the Japanese nationwide registry database. The primary endpoint was overall survival (OS), and secondary endpoints included disease-free survival (DFS), non-relapse mortality (NRM), and GVHD.
Results indicated no significant difference in 3-year OS (44.6% for PBSCT vs. 46.9% for BMT, HR 1.173, P = 0.299) and DFS (42.1% vs. 41.8%, HR 1.073, P = 0.639). PBSCT was more beneficial for avoiding relapse (20.3% vs. 12.4%, HR, 0.715, P = 0.059). However, PBSCT was associated with higher NRM (20.3% vs. 12.4%, HR 1.801, P = 0.025) due to more frequent, chronic GVHD (HR 1.889, P = 0.035). Subgroup analysis did not reveal specific patient groups that benefited more from PBSCT or BMT. Incidence of extensive chronic GVHD and NRM has improved in PBSCT recipients in recent years (2014-2022).
We conclude that related PBSCT with RIC regimens offers comparable prognosis to BMT for adult AML patients. Further optimization of prophylactic strategies for chronic GVHD is required to improve outcomes after PBSCT.
了解外周血干细胞移植（PBSCT）和骨髓移植（BMT）之间临床结果的差异很重要，本研究比较了采用减低强度预处理（RIC）的人类白细胞抗原（HLA）匹配的亲属PBSCT和BMT在成人急性髓系白血病（AML）患者中的结果。
使用日本全国登记数据库分析了2000年至2022年间接受PBSCT（n = 294）或BMT（n = 108）的402例患者的数据。主要终点是总生存期（OS），次要终点包括无病生存期（DFS）、非复发死亡率（NRM）和移植物抗宿主病（GVHD）。
结果表明，3年总生存期（PBSCT为44.6%，BMT为46.9%，风险比[HR]1.173，P = 0.299）和无病生存期（42.1%对41.8%，HR 1.073，P = 0.639）无显著差异。PBSCT在避免复发方面更有益（20.3%对12.4%，HR 0.715，P = 0.059）。然而，由于更频繁的慢性移植物抗宿主病（HR 1.889，P = 0.035），PBSCT与更高的非复发死亡率相关（20.3%对12.4%，HR 1.801，P = 0.025）。亚组分析未发现从PBSCT或BMT中获益更多的特定患者群体。近年来（2014 - 2022年），PBSCT受者广泛慢性移植物抗宿主病和非复发死亡率的发生率有所改善。
我们得出结论，对于成人AML患者，采用RIC方案的亲属PBSCT与BMT的预后相当。需要进一步优化慢性移植物抗宿主病的预防策略，以改善PBSCT后的结果。
