Yoshida Nao, Takahashi Yoshiyuki, Yabe Hiromasa, Kobayashi Ryoji, Watanabe Kenichiro, Kudo Kazuko, Yabe Miharu, Miyamura Takako, Koh Katsuyoshi, Kawaguchi Hiroshi, Goto Hiroaki, Fujita Naoto, Okada Keiko, Okamoto Yasuhiro, Kato Koji, Inoue Masami, Suzuki Ritsuro, Atsuta Yoshiko, Kojima Seiji
Department of Hematology and Oncology, Children's Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan.
Bone Marrow Transplant. 2020 Jul;55(7):1272-1281. doi: 10.1038/s41409-020-0948-8. Epub 2020 May 23.
Fludarabine/cyclophosphamide-based conditioning regimens are standard in bone marrow transplantation (BMT) for acquired bone marrow failure in children, however, graft failure may occur. Using the data from a nationwide transplantation registry, we compared the outcomes of children aged <16 years with acquired aplastic anemia and refractory cytopenia of childhood who underwent allogeneic BMT with either fludarabine/melphalan (n = 71) or fludarabine/cyclophosphamide (n = 296) between 2000 and 2016. The fludarabine/melphalan regimen provided excellent outcomes, with 3-year overall survival and failure-free survival rates of 98% and 97%, respectively. The 83% 3-year failure-free survival in the fludarabine/cyclophosphamide group was significantly inferior (P = 0.002), whereas the overall survival did not differ between the two groups. Late graft failure was the most common cause of treatment failure in the fludarabine/cyclophosphamide group, which experienced a significantly higher incidence of late graft failure than the fludarabine/melphalan group (11% vs. 3%; P = 0.035). Multivariate analyses showed that the fludarabine/melphalan regimen was associated with a better failure-free survival (hazard ratio [HR] 0.12; P = 0.005) and lower risk of late graft failure (HR 0.16; P = 0.037). Fludarabine/melphalan-based conditioning regimen can be a promising option for children with acquired bone marrow failure receiving BMT.
基于氟达拉滨/环磷酰胺的预处理方案是儿童获得性骨髓衰竭进行骨髓移植（BMT）的标准方案，然而，仍可能发生移植物失败。利用全国移植登记处的数据，我们比较了2000年至2016年间接受氟达拉滨/美法仑（n = 71）或氟达拉滨/环磷酰胺（n = 296）同种异体BMT的16岁以下获得性再生障碍性贫血和儿童难治性血细胞减少症患儿的结局。氟达拉滨/美法仑方案取得了优异的结局，3年总生存率和无失败生存率分别为98%和97%。氟达拉滨/环磷酰胺组83%的3年无失败生存率显著较低（P = 0.002），而两组的总生存率无差异。晚期移植物失败是氟达拉滨/环磷酰胺组治疗失败的最常见原因，该组晚期移植物失败的发生率显著高于氟达拉滨/美法仑组（11%对3%；P = 0.035）。多变量分析显示，氟达拉滨/美法仑方案与更好的无失败生存率（风险比[HR]0.12；P = 0.005）和更低的晚期移植物失败风险（HR 0.16；P = 0.037）相关。基于氟达拉滨/美法仑的预处理方案对于接受BMT的获得性骨髓衰竭儿童可能是一个有前景的选择。
