title: Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation.
abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment of severe congenital neutropenia (SCN), but data on outcome are scarce. We report on the outcome of 136 SCN patients who underwent HSCT between 1990 and 2012 in European and Middle East centers. The 3-year overall survival (OS) was 82%, and transplant-related mortality (TRM) was 17%. In multivariate analysis, transplants performed under the age of 10 years, in recent years, and from HLA-matched related or unrelated donors were associated with a significantly better OS. Frequency of graft failure was 10%. Cumulative incidence (day +90) of acute graft-versus-host disease (GVHD) grade 2-4 was 21%. In multivariate analysis, HLA-matched related donor and prophylaxis with cyclosporine A and methotrexate were associated with lower occurrence of acute GVHD. Cumulative incidence (1 year) of chronic GVHD was 20%. No secondary malignancies occurred after a median follow-up of 4.6 years. These data show that the outcome of HSCT for SCN from HLA-matched donors, performed in recent years, in patients younger than 10 years is acceptable. Nevertheless, given the TRM, a careful selection of HSCT candidates should be undertaken.
authors.affiliation: Unit of Clinical and Experimental Haematology, Istituto Giannina Gaslini, Genova, Italy;
authors.forename: Francesca
authors.identifier: http://orcid.org/0000-0003-1504-5511
authors.initials: F
authors.lastname: Fioredda
authors.affiliation: Centro Interdipartimentale di Biostatistica e Bioinformatica, Università di Roma Tor Vergata, Rome, Italy;
authors.forename: Simona
authors.initials: S
authors.lastname: Iacobelli
authors.affiliation: Department of Medical Statistics and Bioinformatics, Leiden University Medical Centre, Leiden, The Netherlands;
authors.forename: Anja
authors.initials: A
authors.lastname: van Biezen
authors.affiliation: Molecular Immunology Unit, University College London, London, United Kingdom;
authors.forename: Bobby
authors.initials: B
authors.lastname: Gaspar
authors.affiliation: Haematology and Oncology Department, Great Ormond Street Hospital Children's Charity, London, United Kingdom;
authors.forename: Phil
authors.initials: P
authors.lastname: Ancliff
authors.affiliation: Pediatric Hematology Oncology, Hospital Trousseau, Paris, France;
authors.forename: Jean
authors.initials: J
authors.lastname: Donadieu
authors.affiliation: Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia;
authors.forename: Mahmoud
authors.initials: M
authors.lastname: Aljurf
authors.affiliation: Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, Austria;
authors.forename: Christina
authors.initials: C
authors.lastname: Peters
authors.affiliation: Unit of Clinical and Experimental Haematology, Istituto Giannina Gaslini, Genova, Italy;
authors.forename: Michaela
authors.initials: M
authors.lastname: Calvillo
authors.affiliation: Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, Austria;
authors.forename: Susanne
authors.initials: S
authors.lastname: Matthes-Martin
authors.affiliation: Bone Marrow Transplantation Unit, Istituto Giannina Gaslini, Genova, Italy;
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Morreale
authors.affiliation: Department of Medical Statistics and Bioinformatics, Leiden University Medical Centre, Leiden, The Netherlands;
authors.forename: Nelleke
authors.initials: N
authors.lastname: van 't Veer-Tazelaar
authors.affiliation: Department of Medical Statistics and Bioinformatics, Leiden University Medical Centre, Leiden, The Netherlands;
authors.forename: Liesbeth
authors.initials: L
authors.lastname: de Wreede
authors.affiliation: Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia;
authors.forename: Amal
authors.initials: A
authors.lastname: Al Seraihy
authors.affiliation: Department of Pediatric Hematology, Akdeniz University Medical School, Antalya, Turkey;
authors.forename: Akif
authors.initials: A
authors.lastname: Yesilipek
authors.affiliation: Hospital Necker Hematology and Immunology Unit, Hospital Necker, Paris, France;
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
authors.affiliation: Pediatric Blood and Bone Marrow Transplantation Program, University Medical Center Utrecht, Utrecht, The Netherlands;
authors.forename: Marc
authors.initials: M
authors.lastname: Bierings
authors.affiliation: Pediatric Hematology, Oncology and Bone Marrow Transplantation, Istanbul University, Istanbul, Turkey;
authors.forename: Gulyuz
authors.initials: G
authors.lastname: Ozturk
authors.affiliation: Department of Pediatric Hematology, Our Lady's Children Hospital, Dublin, Ireland;
authors.forename: Owen
authors.initials: O
authors.lastname: Smith
authors.affiliation: Bone Marrow Transplant Unit, Great Ormond Street Hospital for Children, London, United Kingdom;
authors.forename: Paul
authors.initials: P
authors.lastname: Veys
authors.affiliation: Department of Hematology, Karolinska University Hospital, Stockholm, Sweden;
authors.forename: Per
authors.initials: P
authors.lastname: Ljungman
authors.affiliation: Service d'Hematologie - Transplantation, Centre Hospitalier Universitaire Saint - Louis - Assistance Publique - Hôpitaux de Paris, Paris, France;
authors.forename: Régis
authors.initials: R
authors.lastname: Peffault de Latour
authors.affiliation: Servicio de Hematologia y Oncologia Pediétrica, Hospital Vall d'Hebron, Barcelona, Spain;
authors.forename: José
authors.initials: J
authors.lastname: Sánchez de Toledo Codina
authors.affiliation: Department of Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel;
authors.forename: Reuven
authors.initials: R
authors.lastname: Or
authors.affiliation: Department of Hematology, Hannover Medical School, Hannover, Germany;
authors.forename: Arnold
authors.initials: A
authors.lastname: Ganser
authors.affiliation: Hematology and Transplantology, Saint Petersburg State Medical Pavlov University, Ratsa Gorbacheva Memorial Children's Institute, St. Petersburg, Russia;
authors.forename: Boris
authors.initials: B
authors.lastname: Afanasyev
authors.affiliation: Department of Paediatric Haematology Bone Marrow Unit, Royal Manchester Children's Hospital Manchester, United Kingdom;
authors.forename: Robert
authors.initials: R
authors.lastname: Wynn
authors.affiliation: Department and Clinic of Pediatric Oncology, Hematology and Bone Marrow Transplantation, Wroclaw Medical University, Poland; and.
authors.forename: Krzysztof
authors.initials: K
authors.lastname: Kalwak
authors.affiliation: Department of Hematological Medicine, King's College Hospital/King's College London, London, United Kingdom.
authors.forename: Judith
authors.initials: J
authors.lastname: Marsh
authors.affiliation: Unit of Clinical and Experimental Haematology, Istituto Giannina Gaslini, Genova, Italy;
authors.forename: Carlo
authors.initials: C
authors.lastname: Dufour
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D064591:Allografts; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000008:administration & dosage*; D005060:Europe / Q000453:epidemiology; D005260:Female; D006084:Graft Rejection / Q000453:epidemiology / Q000517:prevention & control; D006086:Graft vs Host Disease / Q000453:epidemiology / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation; D006650:Histocompatibility Testing; D006801:Humans; D015994:Incidence; D008297:Male; D008727:Methotrexate / Q000008:administration & dosage*; D008877:Middle East; D009503:Neutropenia / Q000151:congenital / Q000453:epidemiology / Q000628:therapy; D012189:Retrospective Studies; D012955:Societies, Medical; D061349:Unrelated Donors
chemical_list: D016572:Cyclosporine; D008727:Methotrexate