title: A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome.
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.
authors.affiliation: Hôpital Saint Louis, Eurocord, Paris, France zhanna.shekhovtsova@fccho-moscow.ru.|Dmitry Rogachev National Research Centre of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation.
authors.forename: Zhanna
authors.initials: Z
authors.lastname: Shekhovtsova
authors.affiliation: Bone Marrow Transplantation Service, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Brazil.
authors.forename: Carmem
authors.initials: C
authors.lastname: Bonfim
authors.affiliation: Hôpital Saint Louis, Eurocord, Paris, France.|Service d'Hematologie et Therapie Cellulaire, Hôpital Saint Antoine, Paris, France.
authors.forename: Annalisa
authors.initials: A
authors.lastname: Ruggeri
authors.affiliation: Bone Marrow Transplantation Service, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Brazil.
authors.forename: Samantha
authors.initials: S
authors.lastname: Nichele
authors.affiliation: Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC, USA.
authors.forename: Kristin
authors.initials: K
authors.lastname: Page
authors.affiliation: Section of Pediatric SCT, King Faisal Specialist Hospital & Research Centre-Riyadh, Saudi Arabia.
authors.forename: Amal
authors.initials: A
authors.lastname: AlSeraihy
authors.affiliation: Programa de Hematologia Oncologia Departamento de Pediatria, Pontificia Universidad Catolica de Chile, Santiago, Chile.
authors.forename: Francisco
authors.initials: F
authors.lastname: Barriga
authors.affiliation: Servicio de Hematologia y Oncologia Pediatrica, Hospital Vall d'Hebron, Barcelona, Spain.
authors.forename: José Sánchez
authors.initials: JS
authors.lastname: de Toledo Codina
authors.affiliation: Great Ormond Street Hospital Children's Charity, London, UK.
authors.forename: Paul
authors.initials: P
authors.lastname: Veys
authors.affiliation: Pediatric Blood and Marrow Transplantation Program, University Hospital Utrecht, the Netherlands.
authors.forename: Jaap Jan
authors.initials: JJ
authors.lastname: Boelens
authors.affiliation: Department of Oncology, Hematology and Stem Cell Transplantation, The Queen Silvia Children's Hospital Gothenburg, Sweden.
authors.forename: Karin
authors.initials: K
authors.lastname: Mellgren
authors.affiliation: Hematology-Oncology Division, Centre Hospitalier Universitaire Sainte-Justine, Montréal, QC, Canada.
authors.forename: Henrique
authors.initials: H
authors.lastname: Bittencourt
authors.affiliation: Sydney Children's Hospital Kids Cancer Centre, Randwick, Australia.
authors.forename: Tracey
authors.initials: T
authors.lastname: O'Brien
authors.affiliation: The Children's Hospital at Westmead, Sydney, Australia.
authors.forename: Peter J
authors.initials: PJ
authors.lastname: Shaw
authors.affiliation: Wroclaw Medical University, Poland.
authors.forename: Alicja
authors.initials: A
authors.lastname: Chybicka
authors.affiliation: Hôpital Saint Louis, Eurocord, Paris, France.
authors.forename: Fernanda
authors.initials: F
authors.lastname: Volt
authors.affiliation: Hôpital Saint Louis, Eurocord, Paris, France.|Service d'Hematologie et Therapie Cellulaire, Hôpital Saint Antoine, Paris, France.
authors.forename: Federica
authors.initials: F
authors.lastname: Giannotti
authors.affiliation: Hôpital Saint Louis, Eurocord, Paris, France.|Centre Scientifique de Monaco, Monaco.
authors.forename: Eliane
authors.initials: E
authors.lastname: Gluckman
authors.affiliation: Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC, USA.
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.affiliation: Institute of Cellular Medicine, Newcastle University, Newcastle-Upon-Tyne, UK.
authors.forename: Andrew R
authors.initials: AR
authors.lastname: Gennery
authors.affiliation: Hôpital Saint Louis, Eurocord, Paris, France.|Oxford University Hospitals NHS Trust, UK.
authors.forename: Vanderson
authors.initials: V
authors.lastname: Rocha
mesh_terms: D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods* / Q000592:standards; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology / Q000517:prevention & control; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D018570:Risk Assessment; D016896:Treatment Outcome; D061349:Unrelated Donors; D014923:Wiskott-Aldrich Syndrome / Q000401:mortality / Q000628:therapy*