Arai Yasuyuki, Brazauskas Ruta, He Naya, Al-Homsi A Samer, Chhabra Saurabh, Battiwalla Minoo, Yanada Masamitsu, Steinberg Amir, Diaz Perez Miguel Angel, Hong Sanghee, Kanda Junya, Bashey Asad, Frangoul Haydar A, Badawy Sherif M, Verdonck Leo F, Lazarus Hillard M, Yared Jean A, Hashem Hasan, Sharma Akshay, Aljurf Mahmoud, Dias Ajoy L, Abid Muhammad Bilal, Wirk Baldeep, Freytes César O, Zeidan Amer M, Gergis Usama, Beitinjaneh Amer, Askar Medhat, Pu Jeffrey J, Lehmann Leslie E, Rangarajan Hemalatha G, Wood William A, Hashmi Shahrukh, Yano Shingo, Kako Shinichi, Ozawa Yukiyasu, Doki Noriko, Kanda Yoshinobu, Fukuda Takahiro, Katayama Yuta, Ichinohe Tatsuo, Tanaka Junji, Teshima Takanori, Okamoto Shinichiro, Atsuta Yoshiko, Saber Wael
Department of Hematology Graduate School of Medicine Kyoto University Kyoto Japan.
Division of Biostatistics Institute for Health and Equity Medical College of Wisconsin Milwaukee Wisconsin USA.
EJHaem. 2025 May 28;6(3):e70061. doi: 10.1002/jha2.70061. eCollection 2025 Jun.
In this study, we compared outcomes of intensified myeloablative conditioning regimens using large registry data from Japan (Japanese Society for Transplantation and Cellular Therapy) and the United States (Center for International Blood and Marrow Transplant Research).
Adult patients who underwent their first myeloablative allogeneic hematopoietic stem cell transplantation (HSCT) for acute leukemia in remission between 2010 and 2018 using conditioning regimens of cyclophosphamide plus total-body irradiation (CY/TBI), CY/TBI+cytarabine (AraC), or CY/TBI+etoposide (VP16) were included.
The acute myeloid leukemia (AML) cohort ( = 480, 38.8%) indicated that overall survival (OS) was poorer in CY/TBI+AraC (hazard ratio [HR] 1.46, < 0.001) and CY/TBI+VP16 (HR 1.39, = 0.059) compared to CY/TBI. Relapse was not suppressed, while treatment-related mortality (TRM) was significantly higher (HR 1.78 and 1.74, < 0.001 and 0.018, respectively). In the acute lymphoblastic leukemia (ALL) cohort ( = 3901, 61.2%), OS was comparable among these regimens. With intensified regimens, relapse was significantly suppressed in CY/TBI+VP16 (HR 0.74, = 0.005), while TRM was higher (HR 1.21, = 0.077). No interactions were observed regarding the country.
In AML adding AraC and VP16 to CY/TBI had an adverse effect on OS. Conversely, in ALL, adding VP16 or AraC to CY/TBI did not affect survival, but the addition of VP16 reduced the risk of relapse.
The authors have confirmed clinical trial registration is not needed for this submission.
在本研究中，我们使用来自日本（日本移植与细胞治疗学会）和美国（国际血液与骨髓移植研究中心）的大型登记数据，比较了强化清髓预处理方案的疗效。
纳入2010年至2018年间接受首次清髓性异基因造血干细胞移植（HSCT）治疗处于缓解期急性白血病的成年患者，其预处理方案为环磷酰胺加全身照射（CY/TBI）、CY/TBI+阿糖胞苷（AraC）或CY/TBI+依托泊苷（VP16）。
急性髓系白血病（AML）队列（n = 480，38.8%）显示，与CY/TBI相比，CY/TBI+AraC（风险比[HR] 1.46，P < 0.001）和CY/TBI+VP16（HR 1.39，P = 0.059）的总生存期（OS）较差。复发未得到抑制，而治疗相关死亡率（TRM）显著更高（HR分别为1.78和1.74，P < 0.001和0.018）。在急性淋巴细胞白血病（ALL）队列（n = 3901，61.2%）中，这些方案的OS相当。对于强化方案，CY/TBI+VP16的复发显著降低（HR 0.74，P = 0.005），而TRM更高（HR 1.21，P = 0.077）。未观察到国家之间的交互作用。
在AML中，CY/TBI加用AraC和VP16对OS有不良影响。相反，在ALL中，CY/TBI加用VP16或AraC不影响生存，但加用VP16降低了复发风险。
作者已确认本提交不需要临床试验注册。
