Dandoy Christopher E, Davies Stella M, Woo Ahn Kwang, He Yizeng, Kolb Anders E, Levine John, Bo-Subait Stephanie, Abdel-Azim Hisham, Bhatt Neel, Chewning Joseph, Gadalla Shahinaz, Gloude Nicholas, Hayashi Robert, Lalefar Nahal R, Law Jason, MacMillan Margaret, O'Brien Tracy, Prestidge Timothy, Sharma Akshay, Shaw Peter, Winestone Lena, Eapen Mary
Cincinnati Children Hospital Medical Center, Cincinnati, OH, USA.
Division of Biostatics, Institute for Heath and Equity, Medical College of Wisconsin, Milwaukee, USA.
Haematologica. 2021 Jul 1;106(7):1839-1845. doi: 10.3324/haematol.2020.249458.
With limited data comparing hematopoietic cell transplant outcomes between myeloablative total body irradiation (TBI) containing and non-TBI regimens in children with de novo acute myeloid leukemia, the aim of this study was to compare transplant-outcomes between these regimens. Cox regression models were used to compare transplant-outcomes after TBI and non-TBI regimens in 624 children transplanted between 2008 and 2016. Thirty two percent (n=199) received TBI regimens whereas 68% (n=425) received non-TBI regimens. Five-year non-relapse mortality was higher with TBI regimens (22% vs. 11%, p<0.0001) but relapse was lower (23% vs. 37%, p<0.0001) compared to non-TBI regimens. Consequently, overall (62% vs. 60%, p=1.00) and leukemia-free survival (55% vs. 52%, p=0.42) did not differ between treatment groups. Grade II-IV acute GVHD was higher with TBI regimens (56% vs. 27%, p<0.0001) but not chronic GVHD. The 3-year incidence of gonadal or growth hormone deficiency was higher with TBI regimens (24% vs. 8%, p<0.001) but there were no differences in late pulmonary, cardiac or renal impairment. In the absence of a survival advantage, the choice of TBI or non-TBI regimen merits careful consideration with the data favoring non-TBI regimens to limit the burden of morbidity associated with endocrine dysfunction.
由于比较初治急性髓系白血病儿童含清髓性全身照射（TBI）方案与非TBI方案造血细胞移植结局的数据有限，本研究旨在比较这些方案的移植结局。采用Cox回归模型比较2008年至2016年间接受移植的624例儿童中TBI方案与非TBI方案后的移植结局。32%（n = 199）接受TBI方案，而68%（n = 425）接受非TBI方案。与非TBI方案相比，TBI方案的5年无复发生存率更高（22%对11%，p<0.0001），但复发率更低（23%对37%，p<0.0001）。因此，治疗组之间的总生存率（62%对60%，p = 1.00）和无白血病生存率（55%对52%，p = 0.42）没有差异。TBI方案的II-IV级急性移植物抗宿主病（GVHD）更高（56%对27%，p<0.0001），但慢性GVHD并非如此。TBI方案的性腺或生长激素缺乏3年发生率更高（24%对8%，p<0.001），但晚期肺部、心脏或肾脏损害没有差异。在没有生存优势的情况下，TBI或非TBI方案的选择值得仔细考虑，数据支持非TBI方案以限制与内分泌功能障碍相关的发病负担。
