title: Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes.
abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) offers the potential to cure patients with an inherited bone marrow failure syndrome (IBMFS). However, the procedure involves the risk of treatment-related mortality and may be associated with significant early and late morbidity. For these reasons, the benefits should be carefully weighed against the risks. IBMFS are rare, whereas case reports and small series in the literature illustrate highly heterogeneous practices in terms of indications for HSCT, timing, stem cell source and conditioning regimens. A consensus meeting was therefore held in Vienna in September 2012 on behalf of the European Group for Blood and Marrow Transplantation to discuss HSCT in the setting of IBMFS. This report summarizes the recommendations from this expert panel, including indications for HSCT, timing, stem cell source and conditioning regimen.
authors.affiliation: Service d'Hématologie Greffe, Hôpital Saint-Louis, AP-HP, Paris, France.
authors.forename: R
authors.initials: R
authors.lastname: Peffault de Latour
authors.affiliation: Stem Cell Transplantation Unit, St Anna Children's Hospital, Vienna, Austria.
authors.forename: C
authors.identifier: http://orcid.org/0000-0003-0369-8515
authors.initials: C
authors.lastname: Peters
authors.affiliation: Royal Hospital for Sick Children, Glasgow, Scotland.
authors.forename: B
authors.initials: B
authors.lastname: Gibson
authors.affiliation: Department of Pediatrics and Adolescent Medicine, Pediatric Hematology and Oncology, University Medical Centre, Freiburg, Germany.
authors.forename: B
authors.initials: B
authors.lastname: Strahm
authors.affiliation: Department of Pediatrics, Division of Immuno-Hematology and Stem Cell Transplantation, Leiden University Medical Centre, Leiden, The Netherlands.
authors.forename: A
authors.initials: A
authors.lastname: Lankester
authors.affiliation: Servicio de Oncología y Hematología Pediátricas, Hospital Vall d'Hebron, Barcelona, Spain.
authors.forename: C D
authors.initials: CD
authors.lastname: de Heredia
authors.affiliation: Bone Marrow Transplantation Unit, San Gerardo Hospital, Monza, Italy.
authors.forename: D
authors.initials: D
authors.lastname: Longoni
authors.affiliation: Dipartimento di Emato Oncologia Pediatrica, Ematologia Clinica e di Laboratorio, IRCCS Istituto G. Gaslini, Genova, Italy.
authors.forename: F
authors.initials: F
authors.lastname: Fioredda
authors.affiliation: Dipartimento di Onco-Ematologia Pediatrica e Medicina Trasfusionale, U.O. di Oncologia Pediatrica, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy.
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
authors.affiliation: Pediatric Hematology Oncology and Stem Cell Transplantation Division, Schneider Children's Medical Centre of Israel, Sackler Faculty of Medicine Tel Aviv University, Petah Tikva, Israel.
authors.forename: I
authors.initials: I
authors.lastname: Yaniv
authors.affiliation: Department of Pediatric Oncology, Hematology and Transplantology, University of Medical Sciences, Poznan, Poland.
authors.forename: J
authors.initials: J
authors.lastname: Wachowiak
authors.affiliation: Service d'Hémato-Oncologie Pédiatrique, Hopital Trousseau AP-HP, Paris Registre des neutropénies, Centre de référence des déficits immunitaires héréditaires (neutropénies), Paris, France.
authors.forename: J
authors.initials: J
authors.lastname: Donadieu
authors.affiliation: Stem Cell Transplantation Unit, St Anna Children's Hospital, Vienna, Austria.
authors.forename: A
authors.initials: A
authors.lastname: Lawitschka
authors.affiliation: BMT-Unit, University Medical Centre Utrecht Pediatrics, Utrecht, The Netherlands.
authors.forename: M
authors.initials: M
authors.lastname: Bierings
authors.affiliation: Department of Pediatrics, Hematology and Oncology, University of Freiburg, Freiburg, Germany.
authors.forename: M
authors.initials: M
authors.lastname: Wlodarski
authors.affiliation: Pediatric Hematology, Oncology and Stem Cell Transplantation, Children's Hospital, University of Regensburg, Regensburg, Germany.
authors.forename: S
authors.initials: S
authors.lastname: Corbacioglu
authors.affiliation: Pediatric Hematology Unit, San Gerardo Hospital, Monza, Italy.
authors.forename: S
authors.initials: S
authors.lastname: Bonanomi
authors.affiliation: Pediatric Hematology, Great Ormond Street Hospital, London, UK.
authors.forename: S
authors.initials: S
authors.lastname: Samarasinghe
authors.affiliation: Service d'hémato-immunologie pédiatrique, GH Robert-Debré, AP-HP, Paris, France.
authors.forename: T
authors.initials: T
authors.lastname: Leblanc
authors.affiliation: Pediatric Hematology, G. Gaslini Institute, Genova, Italy.
authors.forename: C
authors.initials: C
authors.lastname: Dufour
authors.affiliation: Service d'hémato-immunologie pédiatrique, GH Robert-Debré, AP-HP, Paris, France.|Université Paris Diderot-Paris 7, Pres Paris-Cité Sorbonne, Paris, France.
authors.forename: J-H
authors.initials: JH
authors.lastname: Dalle
mesh_terms: D000293:Adolescent; D064591:Allografts; D000741:Anemia, Aplastic; D001855:Bone Marrow Diseases; D000080983:Bone Marrow Failure Disorders; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006457:Hemoglobinuria, Paroxysmal / Q000628:therapy*; D006801:Humans; D007223:Infant; D008297:Male; D019172:Transplantation Conditioning / Q000379:methods*