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Transpl Int. 2020 Mar 4. doi: 10.1111/tri.13601. [Epub ahead of print]

Pediatric acute graft-versus-host disease prophylaxis and treatment: surveyed real-life approach reveals dissimilarities compared to published recommendations.

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SCT-Unit, St. Anna Children's Hospital, Medical University Vienna, Vienna, Austria.
Stem Cell Transplantation Department, Great Ormond Street Hospital, London, UK.
Division of Pediatric Hematology, and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Centre, University of Freiburg, Freiburg im Breisgau, Germany.
Hemato-Immunology Department, Robert Debré Hospital, Paris, France.
Immuno-Hematology Unit, Robert Debré Hospital, Paris 7 - Paris Diderot University, Paris, France.
Clinica Pediátrica, Università degli Studi di Milano Bicocca, Monza, Italy.
Brenda E.S. Gibson, Royal Hospital for Sick Children, Glasgow, UK.
Servicio de Hematologia y Oncologia Pediátrica, Hospital Vall d'Hebron, Barcelona, Spain.
Department of Pediatric Oncology, Hematology and Transplantology, University of Medical Sciences, Poznan, Poland.
EBMT Pediatric Diseases Working Party Office, Hematology and Cell Therapy Department, Saint-Antoine Teaching Hospital, Paris, France.
Hospital for Children & Adolescents, University of Helsinki, Helsinki, Finland.
Department of Pediatric Haematology Oncology and BMT Unit, Sackler Faculty of Medicine, Schneider Children's Medical Center of Israel, Tel Aviv University, Tel Aviv-Yafo, Israel.
Pediatric Hematology, Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium.
Division for Stem Cell Transplantation, University Children's Hospital, Frankfurt/Main, Germany.
Division of Pediatric Stem Cell Therapy, Clinic for Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich-Heine-University, Duesseldorf, Germany.
Department of Pediatric Hematology, Oncology and Stem Cell Transplantation, University of Hospital Regensburg, Regensburg, Germany.


Pediatric allogeneic hematopoietic cell transplantation (HCT) practices differ from those of adults, particularly the heterogeneity of transplantable nonmalignant diseases and the lower incidence of graft-versus-host disease (GVHD). Several guidelines regarding the management of acute (a) GVHD in adult HCT have been published. We aimed to capture the real-life approaches for pediatric aGVHD prophylaxis/treatment, and data from 75/193 (response rate 39%) EBMT centers (26 countries) were included, representing half (48%) of the pediatric EBMT-HCT activity. Results with ≥75% approval from respondents (74/75) for GVHD prophylaxis after myeloablative HCT for malignancies partially contradict published guidelines: Single-agent cyclosporine A (CsA) was used for matched sibling donor HCT in 47%; blood CsA levels were reported lower; the relapse risk in malignant diseases influenced GVHD prophylaxis with early withdrawal of CsA; distinct longer duration of CsA was employed in nonmalignant diseases. Most centers used additional anti-thymocyte globulin for matched unrelated and mismatched donor HCT, but not for matched siblings. Regarding prophylaxis in nonmyeloablative conditioning (mainly for nonmalignant diseases), responses showed broad heterogeneity. High conformity was found for first-line treatment; however, results regarding steroid-refractory aGVHD indicate an earlier diagnosis in children. Our findings highlight the need for standardized pediatric approaches toward aGVHD prophylaxis/treatment differentiated for malignant and nonmalignant underlying diseases.


acute GVHD; hematopoietic cell transplantation; pediatrics; prophylaxis; treatment


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