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Blood. 2015 Oct 15;126(16):1885-92; quiz 1970. doi: 10.1182/blood-2015-02-628859. Epub 2015 Jul 16.

Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation.

Author information

1
Unit of Clinical and Experimental Haematology, Istituto Giannina Gaslini, Genova, Italy;
2
Centro Interdipartimentale di Biostatistica e Bioinformatica, Università di Roma Tor Vergata, Rome, Italy;
3
Department of Medical Statistics and Bioinformatics, Leiden University Medical Centre, Leiden, The Netherlands;
4
Molecular Immunology Unit, University College London, London, United Kingdom;
5
Haematology and Oncology Department, Great Ormond Street Hospital Children's Charity, London, United Kingdom;
6
Pediatric Hematology Oncology, Hospital Trousseau, Paris, France;
7
Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia;
8
Stem Cell Transplantation Unit, St. Anna Children's Hospital, Vienna, Austria;
9
Bone Marrow Transplantation Unit, Istituto Giannina Gaslini, Genova, Italy;
10
Department of Pediatric Hematology, Akdeniz University Medical School, Antalya, Turkey;
11
Hospital Necker Hematology and Immunology Unit, Hospital Necker, Paris, France;
12
Pediatric Blood and Bone Marrow Transplantation Program, University Medical Center Utrecht, Utrecht, The Netherlands;
13
Pediatric Hematology, Oncology and Bone Marrow Transplantation, Istanbul University, Istanbul, Turkey;
14
Department of Pediatric Hematology, Our Lady's Children Hospital, Dublin, Ireland;
15
Bone Marrow Transplant Unit, Great Ormond Street Hospital for Children, London, United Kingdom;
16
Department of Hematology, Karolinska University Hospital, Stockholm, Sweden;
17
Service d'Hematologie - Transplantation, Centre Hospitalier Universitaire Saint - Louis - Assistance Publique - Hôpitaux de Paris, Paris, France;
18
Servicio de Hematologia y Oncologia Pediétrica, Hospital Vall d'Hebron, Barcelona, Spain;
19
Department of Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel;
20
Department of Hematology, Hannover Medical School, Hannover, Germany;
21
Hematology and Transplantology, Saint Petersburg State Medical Pavlov University, Ratsa Gorbacheva Memorial Children's Institute, St. Petersburg, Russia;
22
Department of Paediatric Haematology Bone Marrow Unit, Royal Manchester Children's Hospital Manchester, United Kingdom;
23
Department and Clinic of Pediatric Oncology, Hematology and Bone Marrow Transplantation, Wroclaw Medical University, Poland; and.
24
Department of Hematological Medicine, King's College Hospital/King's College London, London, United Kingdom.

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.

PMID:
26185129
DOI:
10.1182/blood-2015-02-628859
[Indexed for MEDLINE]
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