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Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study.


ABSTRACT: In this retrospective collaborative study, we have analyzed long-term outcome and donor cell engraftment in 194 patients with Wiskott-Aldrich syndrome (WAS) who have been treated by hematopoietic cell transplantation (HCT) in the period 1980- 2009. Overall survival was 84.0% and was even higher (89.1% 5-year survival) for those who received HCT since the year 2000, reflecting recent improvement of outcome after transplantation from mismatched family donors and for patients who received HCT from an unrelated donor at older than 5 years. Patients who went to transplantation in better clinical conditions had a lower rate of post-HCT complications. Retrospective analysis of lineage-specific donor cell engraftment showed that stable full donor chimerism was attained by 72.3% of the patients who survived for at least 1 year after HCT. Mixed chimerism was associated with an increased risk of incomplete reconstitution of lymphocyte count and post-HCT autoimmunity, and myeloid donor cell chimerism < 50% was associated with persistent thrombocytopenia. These observations indicate continuous improvement of outcome after HCT for WAS and may have important implications for the development of novel protocols aiming to obtain full correction of the disease and reduce post-HCT complications.

SUBMITTER: Moratto D 

PROVIDER: S-EPMC3156052 | biostudies-literature | 2011 Aug

REPOSITORIES: biostudies-literature

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Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study.

Moratto Daniele D   Giliani Silvia S   Bonfim Carmem C   Mazzolari Evelina E   Fischer Alain A   Ochs Hans D HD   Cant Andrew J AJ   Thrasher Adrian J AJ   Cowan Morton J MJ   Albert Michael H MH   Small Trudy T   Pai Sung-Yun SY   Haddad Elie E   Lisa Antonella A   Hambleton Sophie S   Slatter Mary M   Cavazzana-Calvo Marina M   Mahlaoui Nizar N   Picard Capucine C   Torgerson Troy R TR   Burroughs Lauri L   Koliski Adriana A   Neto Jose Zanis JZ   Porta Fulvio F   Qasim Waseem W   Veys Paul P   Kavanau Kristina K   Hönig Manfred M   Schulz Ansgar A   Friedrich Wilhelm W   Notarangelo Luigi D LD  

Blood 20110609 6


In this retrospective collaborative study, we have analyzed long-term outcome and donor cell engraftment in 194 patients with Wiskott-Aldrich syndrome (WAS) who have been treated by hematopoietic cell transplantation (HCT) in the period 1980- 2009. Overall survival was 84.0% and was even higher (89.1% 5-year survival) for those who received HCT since the year 2000, reflecting recent improvement of outcome after transplantation from mismatched family donors and for patients who received HCT from  ...[more]

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