Harada Kaito, Kanda Junya, Hirayama Masahiro, Wada Fumiya, Uchida Naoyuki, Tanaka Masatsugu, Nakamae Hirohisa, Tokunaga Masahito, Ishiwata Kazuya, Onizuka Makoto, Hasegawa Yuta, Fukuda Takahiro, Eto Tetsuya, Kurita Naoki, Kawakita Toshiro, Jinguji Atsushi, Ishimaru Fumihiko, Atsuta Yoshiko, Nakasone Hideki
Department of Hematology and Oncology, Tokai University School of Medicine, Isehara, Japan.
Department of Hematology and Oncology, Graduate School of Medicine, Kyoto University, Kyoto, Japan.
Transplant Cell Ther. 2025 Feb;31(2):103.e1-103.e13. doi: 10.1016/j.jtct.2024.11.011. Epub 2024 Nov 26.
Unrelated cord blood transplantation (UCBT) and haploidentical transplantation using posttransplant cyclophosphamide (PTCy-haplo) are alternatives for patients lacking a human leukocyte antigen-matched donor. CD34 cell counts in cord blood affect transplantation outcomes, particularly nonrelapse mortality (NRM). The primary objective of this study was to compare the transplantation outcomes after UCBT and PTCy-haplo focusing on CD34 cell counts in cord blood. This retrospective study used data from 2014 to 2020 from a Japanese nationwide database. UCBT cases were divided into those with UCBT with higher (UCB-H; ≥.84 × 10/kg) and lower (UCB-L; <.84 × 10/kg) CD34 cell counts, depending on the median CD34 cell count. The study cohort comprised cases of PTCy-haplo (n = 1142), UCB-H (n = 3185), and UCB-L (n = 3172). In the multivariate analysis, neutrophil engraftment was significantly better in the PTCy-haplo than in the UCB-H (hazard ratio [HR], .64; 95% confidence interval [CI], .57 to .70; P < .001) and UCB-L groups (HR, .45; 95% CI, .41 to .50; P < .001). The UCB-H group showed similar NRM (HR, 1.19, 95% CI, 1.00 to 1.43, P = .051) and OS (HR, 1.05, 95% CI, .94 to 1.18, P = .38) compared with PTCy-haplo, whereas UCB-L was significantly associated with poor NRM (HR, 1.35, 95% CI, 1.13 to 1.61, P = .001) and OS (HR, 1.13, 95% CI, 1.01 to 1.26, P = .038). In contrast, the UCB-H (HR, .86; 95% CI, .75 to .98; P = .027) and UCB-L groups (HR, .80; 95% CI, .70 to .92; P = .001) were associated with lower relapse rate. Regarding the graft-versus-host disease (GVHD), the UCB-H and UCB-L groups were identified as significant risk factors for the development of grade II-IV acute GVHD (UCB-H: HR, 1.73; 95% CI, 1.51 to 1.99; P < .001; UCB-L: HR, 1.55; 95% CI, 1.35 to 1.78; P < .001) and grade III-IV acute GVHD (UCB-H: HR, 2.28; 95% CI, 1.78 to 2.91; P < .001; UCB-L: HR, 1.85; 95% CI, 1.44 to 2.37; P < .001), but neither were associated with the incidence of all-grade GVHD (UCB-H: HR, 1.12; 95% CI, .95 to 1.32; P = .16; UCB-L: HR, 1.08; 95% CI, .91 to 1.27; P = .37) or extensive chronic GVHD (UCB-H: HR, .86; 95% CI, .68 to 1.09; P = .21; UCB-L: HR, .88; 95% CI, .69 to 1.12; P = .31). Furthermore, higher NRM in UCB-L was attributed to higher infection-related mortality (HR, 1.50; 95% CI, 1.15 to 1.95; P = .003) but not GVHD-related mortality (HR, 1.15; 95% CI, .82 to 1.62; P = .43), whereas UCB-H was not a significant risk factor for both infection-related mortality (HR, 1.29; 95% CI, .99 to 1.69; P = .06) and GVHD-related mortality (HR, 1.28; 95% CI, .90 to 1.80; P = .16). UCB-H offered similar NRM and OS to PTCy-haplo, whereas UCB-L had worse outcomes. Our results can provide useful information for optimal donor selection.
无关供者脐血移植（UCBT）和使用移植后环磷酰胺的单倍体相合移植（PTCy-haplo）是缺乏人类白细胞抗原匹配供者患者的替代方案。脐血中的CD34细胞计数会影响移植结果，尤其是非复发死亡率（NRM）。本研究的主要目的是比较UCBT和PTCy-haplo后的移植结果，重点关注脐血中的CD34细胞计数。这项回顾性研究使用了来自日本全国数据库2014年至2020年的数据。根据CD34细胞计数中位数，UCBT病例分为CD34细胞计数较高（UCB-H；≥0.84×10⁶/kg）和较低（UCB-L；<0.84×10⁶/kg）的两组。研究队列包括PTCy-haplo病例（n = 1142）、UCB-H病例（n = 3185）和UCB-L病例（n = 3172）。在多变量分析中，PTCy-haplo组的中性粒细胞植入明显优于UCB-H组（风险比[HR]，0.64；95%置信区间[CI]，0.57至0.70；P < 0.001）和UCB-L组（HR，0.45；95% CI，0.41至0.50；P < 0.001）。与PTCy-haplo相比，UCB-H组的NRM（HR，1.19，95% CI，1.00至1.43，P = 0.051）和总生存期（OS；HR，1.05，95% CI，0.94至l.18，P = 0.38）相似，而UCB-L组与较差的NRM（HR，1.35，95% CI，1.13至1.61，P = 0.001）和OS（HR，1.13，95% CI，1.01至1.26，P = 0.038）显著相关。相比之下，UCB-H组（HR，0.86；95% CI，0.75至0.98；P = 0.027）和UCB-L组（HR，0.80；95% CI，0.70至0.92；P = 0.001）的复发率较低。关于移植物抗宿主病（GVHD），UCB-H组和UCB-L组被确定为发生II-IV级急性GVHD（UCB-H：HR，1.73；95% CI，1.51至1.99；P < 0.001；UCB-L：HR，1.55；95% CI，1.35至1.78；P < 0.001）和III-IV级急性GVHD（UCB-H：HR，2.28；95% CI，1.78至2.91；P < 0.001；UCB-L：HR，1.85；95% CI，1.44至2.37；P < 0.001）的显著风险因素，但两者均与所有级别的GVHD发生率（UCB-H：HR，1.12；95% CI，0.95至1.32；P = 0.16；UCB-L：HR，1.08；95% CI，0.91至1.27；P = 0.37）或广泛慢性GVHD（UCB-H：HR，0.86；95% CI，0.68至1.09；P = 0.21；UCB-L：HR，0.88；95% CI，0.69至1.12；P = 0.31）无关。此外，UCB-L组较高的NRM归因于较高的感染相关死亡率（HR，1.50；95% CI，1.15至1.95；P = 0.003），而非GVHD相关死亡率（HR，1.15；95% CI，0.82至1.62；P = 0.43），而UCB-H组对于感染相关死亡率（HR，1.29；95% CI，0.99至1.69；P = 0.06）和GVHD相关死亡率（HR，1.28；95% CI，0.90至1.80；P = 0.16）均不是显著风险因素。UCB-H组的NRM和OS与PTCy-haplo相似，而UCB-L组的结果较差。我们的结果可为最佳供者选择提供有用信息。
