PICALM::MLLT10在儿童白血病中的预后影响尚未明确描述。本多中心回顾性研究由十个国际柏林-法兰克福-明斯特（International Berlin Frankfurt Münster）附属研究团体和儿童肿瘤学会（Children's Oncology Group）合作完成。参与单位通过荧光原位杂交和/或RNA测序确认了PICALM::MLLT10融合基因的存在。共有98名儿童符合研究标准。T细胞急性淋巴细胞白血病（T-ALL）和急性髓细胞白血病（AML）占据优势，分别为55例（56%）和39例（40%）。大多数患者根据疾病表型接受化疗方案：58%接受了ALL方案，40%接受了AML方案，1%接受了混合方案。对于PICALM::MLLT10 ALL的儿童，预后良好：5年无事件生存率（EFS）为67%，5年总生存率（OS）为76%，但PICALM::MLLT10 AML的儿童预后较差：5年EFS为22%，5年OS为26%。造血干细胞移植（HSCT）对于PICALM::MLLT10 AML患者的预后并没有明显改善：接受HSCT的患者5年EFS为20%，未接受HSCT的患者为23%（p=0.6），5年OS分别为37%和36%（p=0.7）。总之，本研究证实了PICALM::MLLT10 AML与不良预后相关，无法通过HSCT挽救患者；需要探索新的治疗选择。© 2023英国血液病学学会和约翰威利出版社有限公司。

The prognostic impact of PICALM::MLLT10 status in childhood leukaemia is not well described. Ten International Berlin Frankfurt Münster-affiliated study groups and the Children's Oncology Group collaborated in this multicentre retrospective study. The presence of the PICALM::MLLT10 fusion gene was confirmed by fluorescence in situ hybridization and/or RNA sequencing at participating sites. Ninety-eight children met the study criteria. T-cell acute lymphoblastic leukaemia (T-ALL) and acute myeloid leukaemia (AML) predominated 55 (56%) and 39 (40%) patients, respectively. Most patients received a chemotherapy regimen per their disease phenotype: 58% received an ALL regimen, 40% an AML regimen and 1% a hybrid regimen. Outcomes for children with PICALM::MLLT10 ALL were reasonable: 5-year event-free survival (EFS) 67% and 5-year overall survival (OS) 76%, but children with PICALM::MLLT10 AML had poor outcomes: 5-year EFS 22% and 5-year OS 26%. Haematopoietic stem cell transplant (HSCT) did not result in a significant improvement in outcomes for PICALM::MLLT10 AML: 5-year EFS 20% for those who received HSCT versus 23% for those who did not (p = 0.6) and 5-year OS 37% versus 36% (p = 0.7). In summary, this study confirms that PICALM::MLLT10 AML is associated with a dismal prognosis and patients cannot be salvaged with HSCT; exploration of novel therapeutic options is warranted.© 2023 British Society for Haematology and John Wiley & Sons Ltd.