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

Bone Marrow Transplantation volume 55, pages 376–383 (2020)Cite this article

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Abstract

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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Acknowledgements

List of contributing centers: IRRCS Ospedale Pediatrico Bambino Gesù Rome; Institute Curie Paris; University of Helsinki Hospital for Children & Adolescents; Clinica di Oncoematologia Pediatrica Dipartimento di Pediatria Padova; Our Lady’s Children’s Hospital Crumlin Dublin; Hebron hospital Barcelona; North Trent BMT Programme Sheffield Teaching Hospitals NHS Trust/Sheffield Children’s Hospital; Cape of Hope Wroclaw Medical University; Klinikum der Johann-Wolfgang Goethe Universitaet Frankfurt_am_Main; University Hospital Bern Paediatric Hematology/Oncology Bern; Inst. Português de Oncologia do Porto BMT Unit; Bristol Royal Hospital for Children Dept. of Paediatric Oncology/BMT; St. Anna Kinderspital Stem Cell Transplantation Unit Vienna; Edmond & Lily Safra Children’s Hospital Paediatric Hemato Oncology & BMT Tel-Hashomer; Pediatric University Teaching Hospital BMT Unit II Children’s Clinic Bratislava; Service d’Hématologie et d’Oncologie pédiatrique CHU Hautepierre Strasbourg; Turku University Hospital TD7 (Stem Cell Transplant Unit); Chu Estaing Service d’hématologie clinique Adulte et pédiatrie Clermont, University Hospital “Queen Johanna-Isul” Sofia, Poznan University of Medical Sciences Department of Pediatric Onology, Hematology & HSCT, The Children’s Hospital at Westmead Sydney; Clínica Universitaria de Navarra Pamplona; Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy; Istituto Giannina Gaslini, Genova, Italy.

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Authors and Affiliations

  1. Deptartment of Medical Oncology and Hematology, Pediatric Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy

    F. Spreafico

  2. EBMT Paris Study Office/CEREST-TC, Department of Haematology, Saint Antoine Hospital, INSERM UMR 938, Paris, France

    A. Dalissier

  3. St. Anna Kinderspital, Stem Cell Transplantation Unit, Vienna, Austria

    U. Pötschger & C. Peters

  4. Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome, Italy

    F. Locatelli

  5. Children, Adolescent and Young Adults Department, Institut Curie, Paris, France

    J. M. Michon

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

    P. Bader & A. Willasch

  7. Hematology Oncology Division, Department of Woman’s and Child’s Health, University of Padova, Padova, Italy

    G. Bisogno

  8. Sheffield Children’s Hospital Western Bank, Sheffield, UK

    D. Yeomanson

  9. Princess Máxima Centre for Pediatric Oncology, Utrecht, the Netherlands

    M. van den Heuvel Eibrink

  10. Department of Haematology and Oncology, Saarland University Hospital, Homburg, Germany

    N. Graf

  11. IRCCS Istituto Giannina Gaslini, Genova, Italy

    S. Dallorso

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  1. F. Spreafico
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Correspondence to F. Spreafico.

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Spreafico, F., Dalissier, A., Pötschger, U. et al. High dose chemotherapy and autologous hematopoietic cell transplantation for Wilms tumor: a study of the European Society for Blood and Marrow Transplantation. Bone Marrow Transplant 55, 376–383 (2020). https://doi.org/10.1038/s41409-019-0661-7

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