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儿童急性淋巴细胞白血病异基因造血干细胞移植的清髓性预处理:全身照射还是化疗?——一项多中心欧洲血液与骨髓移植协会儿科数据工作组研究

Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?-A multicenter EBMT-PDWP study.

作者信息

Willasch Andre Manfred, Peters Christina, Sedláček Petr, Dalle Jean-Hugues, Kitra-Roussou Vassiliki, Yesilipek Akif, Wachowiak Jacek, Lankester Arjan, Prete Arcangelo, Hamidieh Amir Ali, Ifversen Marianne, Buechner Jochen, Kriván Gergely, Hamladji Rose-Marie, Diaz-de-Heredia Cristina, Skorobogatova Elena, Michel Gérard, Locatelli Franco, Bertaina Alice, Veys Paul, Dupont Sophie, Or Reuven, Güngör Tayfun, Aleinikova Olga, Sufliarska Sabina, Sundin Mikael, Rascon Jelena, Kaare Ain, Nemet Damir, Fagioli Franca, Klingebiel Thomas Erich, Styczynski Jan, Bierings Marc, Nagy Kálmán, Abecasis Manuel, Afanasyev Boris, Ansari Marc, Vettenranta Kim, Alseraihy Amal, Chybicka Alicja, Robinson Stephen, Bertrand Yves, Kupesiz Alphan, Ghavamzadeh Ardeshir, Campos Antonio, Pichler Herbert, Dalissier Arnaud, Labopin Myriam, Corbacioglu Selim, Balduzzi Adriana, Galimard Jacques-Emmanuel, Bader Peter

机构信息

Division for Stem Cell Transplantation and Immunology, Department for Children and Adolescents, University Hospital, Goethe University Frankfurt, Frankfurt, Germany.

St. Anna Children's Hospital, Department of Pediatrics, Medical University of Vienna, Vienna, Austria.

出版信息

Bone Marrow Transplant. 2020 Aug;55(8):1540-1551. doi: 10.1038/s41409-020-0854-0. Epub 2020 Mar 17.

Abstract

Although most children with acute lymphoblastic leukemia (ALL) receive fractionated total body irradiation (FTBI) as myeloablative conditioning (MAC) for allogeneic hematopoietic stem cell transplantation (allo-HSCT), it is an important matter of debate if chemotherapy can effectively replace FTBI. To compare outcomes after FTBI versus chemotherapy-based conditioning (CC), we performed a retrospective EBMT registry study. Children aged 2-18 years after MAC for first allo-HSCT of bone marrow (BM) or peripheral blood stem cells (PBSC) from matched-related (MRD) or unrelated donors (UD) in first (CR1) or second remission (CR2) between 2000 and 2012 were included. Propensity score weighting was used to control pretreatment imbalances of the observed variables. 3.054 patients were analyzed. CR1 (1.498): median follow-up (FU) after FTBI (1.285) and CC (213) was 6.8 and 6.1 years. Survivals were not significantly different. CR2 (1.556): median FU after FTBI (1.345) and CC (211) was 6.2 years. Outcomes after FTBI were superior as compared with CC with regard to overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and nonrelapse mortality (NRM). However, we must emphasize the preliminary character of the results of this retrospective "real-world-practice" study. These findings will be prospectively assessed in the ALL SCTped 2012 FORUM trial.

摘要

尽管大多数急性淋巴细胞白血病(ALL)患儿在接受异基因造血干细胞移植(allo-HSCT)时会接受分次全身照射(FTBI)作为清髓性预处理(MAC),但化疗能否有效替代FTBI仍是一个重要的争议问题。为了比较FTBI与基于化疗的预处理(CC)后的结局,我们进行了一项回顾性欧洲血液与骨髓移植协会(EBMT)注册研究。纳入了2000年至2012年间年龄在2至18岁、首次接受来自匹配相关(MRD)或无关供者(UD)的骨髓(BM)或外周血干细胞(PBSC)进行首次异基因造血干细胞移植(allo-HSCT)且处于首次完全缓解(CR1)或第二次缓解(CR2)状态的患儿,并采用倾向评分加权法来控制观察变量的预处理不平衡情况。共分析了3054例患者。CR1(1498例):FTBI组(1285例)和CC组(213例)的中位随访时间分别为6.8年和6.1年,生存率无显著差异。CR2(1556例):FTBI组(1345例)和CC组(211例)的中位随访时间为6.2年。在总生存(OS)、无白血病生存(LFS)、复发率(RI)和非复发死亡率(NRM)方面,FTBI后的结局优于CC。然而,我们必须强调这项回顾性“真实世界实践”研究结果的初步性质。这些发现将在ALL SCTped 2012 FORUM试验中进行前瞻性评估。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/5e61/8376634/7bc506123c88/41409_2020_854_Fig1_HTML.jpg

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