Outcome of Hematopoietic Stem Cell Transplantation in 813 Pediatric Patients with Fanconi Anemia

救世主兄弟 范科尼贫血 造血干细胞移植 医学 移植 干细胞 造血 肿瘤科 内科学 生物 遗传学 DNA修复 基因
作者
Su Han Lum,Diderik-Jan Eikema,Brian Piepenbroek,Robert Wynn,Sujith Samarasinghe,Arnaud Dalissier,Krzysztof Kałwak,Mouhab Ayas,Rose-Marie Hamladji,Akif Yeşilipek,Jean‐Hugues Dalle,Duygu Uçkan,Marc Bierings,Alphan Küpesiz,Khalid Halahleh,Е. В. Скоробогатова,Gülyüz Öztürk,Maura Faraci,C. Rénard,Pamela Evans,Selim Corbacioglu,Franco Locatelli,Carlo Dufour,Antonio M. Risitano,Régis Peffault de Latour
出处
期刊:Blood [American Society of Hematology]
标识
DOI:10.1182/blood.2023022751
摘要

Allogeneic hematopoietic stem cell transplantation (HSCT) is the only established curative option for Fanconi anemia (FA) associated bone marrow failure (BMF)/aplastic anemia (AA) and hematological malignancy. We performed a retrospective multicenter study on 813 FA children undergoing first HSCT between 2010 and 2018. Median duration of follow-up was 3.7 years (interquartile range, 3.4-4.0). Median age at transplant was 8.8 years (6.5-18.1). Overall survival (OS), event-free survival (EFS) and GvHD-free, relapse-free survival (GRFS) at 5 years were 83% (80-86%), 78% (75-81%) and 70% (67-74%) respectively. OS was comparable between matched family donor (MFD, n=441, 88%) and matched unrelated donor (MUD, n=162, 86%) and was superior to that of mismatched family or unrelated donor (MMFD/MMUD, n=144, 72%) and haploidentical donor (HID) (n=66, 70%, p<0.001). In multivariable analysis, a transplant indication of acute myeloid leukaemia/myelodysplastic syndrome compared to AA/BMF, use of MMFD/MMUD and HID compared to MFD, Fludarabine-Cyclophosphamide (FluCy) + other conditioning compared to FluCy independently predicted inferior OS, while alemtuzumab compared to ATG was associated with better OS. Age  10 years was associated with worse EFS and GRFS. Cumulative incidences (CIN) of primary and secondary graft failure were 2% (1-3%) and 3% (2-4%) respectively. CIN of grade II-IV acute GvHD, grade III-IV acute GvHD and chronic GvHD were 23% (20-26%), 12% (10-15%) and 8% (6-10%) respectively. The 5-year CIN of secondary malignancy was 2% (1-3%). These data suggest that HSCT should be offered to Fanconi Anemia patients with AA/BMF at a younger age in the presence of a well-matched donor.
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