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

Artikel i vetenskaplig tidskrift
Författare Zhanna Shekhovtsova
Carmem Bonfim
Annalisa Ruggeri
Samantha Nichele
Kristin Page
Amal AlSeraihy
Francisco Barriga
José Sánchez de Toledo Codina
Paul Veys
Jaap Jan Boelens
Karin Mellgren
Henrique Bittencourt
Tracey O'Brien
Peter J Shaw
Alicja Chybicka
Fernanda Volt
Federica Giannotti
Eliane Gluckman
Joanne Kurtzberg
Andrew R Gennery
Vanderson Rocha
Publicerad i Haematologica
Volym 102
Nummer/häfte 6
Sidor 1112-1119
ISSN 1592-8721
Publiceringsår 2017
Publicerad vid
Sidor 1112-1119
Språk en
Länkar dx.doi.org/10.3324/haematol.2016.15...
www.ncbi.nlm.nih.gov/entrez/query.f...
Ämneskategorier Pediatrik

Sammanfattning

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.

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