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.

Clinic of Pediatric Hematology and Oncology University Medical Centre Hamburg Germany

Department of Pediatric Hematology and Oncology 2nd Faculty of Medicine Charles University and University Hospital Motol Prague Czech Republic

Department of Pediatrics and Adolescent Medicine St Anna Children's Hospital Medical University of Vienna Vienna Austria

Division of Hematology Oncology Toronto Hospital for Sick Children Toronto Ontario Canada

Division of Leukemia and Lymphoma Children's Cancer Centre National Centre for Child Health and Development Tokyo Japan

Fred Hutchinson Cancer Research Centre Seattle Washington USA

Hematology Oncology Department of Pediatrics Aarhus University Hospital Aarhus Denmark

Hôpital d'enfants Armand Trousseau Paris France

Human Genetics Hannover Medical School Hannover Germany

Independent Laboratory of Genetic Diagnostics Medical University of Lublin Lublin Poland

Nemours Children's Hospital Wilmington Delaware USA

Newcastle University Centre for Cancer Newcastle UK

Princess Maxima Centre for Pediatric Oncology Utrecht The Netherlands

Rigshospitalet University Hospital Copenhagen Denmark

Royal Hospital for Sick Children Glasgow Scotland UK

Skane University Hospital Lund Sweden

St Anna Children's Cancer Research Institute Vienna Austria

Universitats Klinikum Essen Germany

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