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Bone Marrow Transplant. 2019 Sep 18. doi: 10.1038/s41409-019-0661-7. [Epub ahead of print]

High dose chemotherapy and autologous hematopoietic cell transplantation for Wilms tumor: a study of the European Society for Blood and Marrow Transplantation.

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Deptartment of Medical Oncology and Hematology, Pediatric Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
EBMT Paris Study Office/CEREST-TC, Department of Haematology, Saint Antoine Hospital, INSERM UMR 938, Paris, France.
St. Anna Kinderspital, Stem Cell Transplantation Unit, Vienna, Austria.
Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome, Italy.
Children, Adolescent and Young Adults Department, Institut Curie, Paris, France.
Division for Stem Cell Transplantation and Immunology, Department for Children and Adolescents, University Hospital, Goethe University, Frankfurt/Main, Germany.
Hematology Oncology Division, Department of Woman's and Child's Health, University of Padova, Padova, Italy.
Sheffield Children's Hospital Western Bank, Sheffield, UK.
Princess Máxima Centre for Pediatric Oncology, Utrecht, the Netherlands.
Department of Haematology and Oncology, Saarland University Hospital, Homburg, Germany.
IRCCS Istituto Giannina Gaslini, Genova, Italy.


Survival for subgroups of patients with Wilms tumor (WT), such as those who suffer from relapse, is disappointing. Some patients' treatment plans include high-dose chemotherapy (HDT) with autologous hematopoietic cell transplantation (aHCT), although proof for its benefit is lacking. To increase the level of evidence regarding children with WT receiving aHCT as consolidation of first or second remission (after first relapse), we extracted relevant data from the European Blood and Marrow Transplantation Registry concerning 69 patients. Different HDT regimens were administered, mostly either melphalan-containing (n = 34) or thiotepa-containing (n = 14). For the whole population, 5-year overall survival (OS) and event-free survival (EFS) probabilities were 0.67 (±0.06) and 0.63 (±0.06), respectively (median observation time 7.8 years); for children transplanted in first remission, OS and EFS were 0.69 (±0.09) and 0.72 (±0.08). In univariate analysis, male gender and relapse in multiple sites were associated with lower OS probabilities. The use of a given pretransplant regimen (i.e. melphalan alone versus regimens with multiple drugs) did not seem to influence EFS/OS probability after aHCT, but significantly influenced platelet engraftment (more delayed with thiotepa). We here provide further data to improve the basis for future evidence-based clinical decision-making when using HDT and aHCT in relapsed/refractory WT.


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