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Haematologica. 2017 Jun;102(6):1112-1119. doi: 10.3324/haematol.2016.158808. Epub 2017 Mar 2.

A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome.

Author information

1
Hôpital Saint Louis, Eurocord, Paris, France zhanna.shekhovtsova@fccho-moscow.ru.
2
Dmitry Rogachev National Research Centre of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation.
3
Bone Marrow Transplantation Service, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Brazil.
4
Hôpital Saint Louis, Eurocord, Paris, France.
5
Service d'Hematologie et Therapie Cellulaire, Hôpital Saint Antoine, Paris, France.
6
Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC, USA.
7
Section of Pediatric SCT, King Faisal Specialist Hospital & Research Centre-Riyadh, Saudi Arabia.
8
Programa de Hematologia Oncologia Departamento de Pediatria, Pontificia Universidad Catolica de Chile, Santiago, Chile.
9
Servicio de Hematologia y Oncologia Pediatrica, Hospital Vall d'Hebron, Barcelona, Spain.
10
Great Ormond Street Hospital Children's Charity, London, UK.
11
Pediatric Blood and Marrow Transplantation Program, University Hospital Utrecht, the Netherlands.
12
Department of Oncology, Hematology and Stem Cell Transplantation, The Queen Silvia Children's Hospital Gothenburg, Sweden.
13
Hematology-Oncology Division, Centre Hospitalier Universitaire Sainte-Justine, Montréal, QC, Canada.
14
Sydney Children's Hospital Kids Cancer Centre, Randwick, Australia.
15
The Children's Hospital at Westmead, Sydney, Australia.
16
Wroclaw Medical University, Poland.
17
Centre Scientifique de Monaco, Monaco.
18
Institute of Cellular Medicine, Newcastle University, Newcastle-Upon-Tyne, UK.
19
Oxford University Hospitals NHS Trust, UK.

Abstract

Wiskott-Aldrich syndrome is a severe X-linked recessive immune deficiency disorder. A scoring system of Wiskott-Aldrich syndrome severity (0.5-5) distinguishes two phenotypes: X-linked thrombocytopenia and classic Wiskott-Aldrich syndrome. Hematopoietic cell transplantation is curative for Wiskott-Aldrich syndrome; however, the use of unrelated umbilical cord blood transplantation has seldom been described. We analyzed umbilical cord blood transplantation outcomes for 90 patients. The median age at umbilical cord blood transplantation was 1.5 years. Patients were classified according to clinical scores [2 (23%), 3 (30%), 4 (23%) and 5 (19%)]. Most patients underwent HLA-mismatched umbilical cord blood transplantation and myeloablative conditioning with anti-thymocyte globulin. The cumulative incidence of neutrophil recovery at day 60 was 89% and that of grade II-IV acute graft-versus-host disease at day 100 was 38%. The use of methotrexate for graft-versus-host disease prophylaxis delayed engraftment (P=0.02), but decreased acute graft-versus-host disease (P=0.03). At 5 years, overall survival and event-free survival rates were 75% and 70%, respectively. The estimated 5-year event-free survival rates were 83%, 73% and 55% for patients with a clinical score of 2, 4-5 and 3, respectively. In multivariate analysis, age <2 years at the time of the umbilical cord blood transplant and a clinical phenotype of X-linked thrombocytopenia were associated with improved event-free survival. Overall survival tended to be better in patients transplanted after 2007 (P=0.09). In conclusion, umbilical cord blood transplantation is a good alternative option for young children with Wiskott-Aldrich syndrome lacking an HLA identical stem cell donor.

PMID:
28255019
PMCID:
PMC5451344
DOI:
10.3324/haematol.2016.158808
[Indexed for MEDLINE]
Free PMC Article

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