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Reduced intensity transplantation for congenital amegakaryocytic thrombocytopenia: Report of a case and review of the literature
Author(s) -
Woods Gary,
Bajwa Rajinder P. S.,
Rose Melissa J.
Publication year - 2014
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.12175
Subject(s) - medicine , alemtuzumab , fludarabine , melphalan , regimen , pediatrics , bone marrow transplant , transplantation , surgery , bone marrow transplantation , chemotherapy , cyclophosphamide
CAMT is a bone marrow failure syndrome that usually presents with isolated thrombocytopenia soon after birth. HSCT is curative, and MAC is associated with increased transplant‐related morbidity and mortality, especially in the unrelated setting. We used a RIC regimen with alemtuzumab, fludarabine, and melphalan in a seven‐month‐old patient with CAMT who underwent a MUD HSCT . The transplant was well tolerated with few complications. Neutrophil and platelet engraftment occurred on day +12 and +29, respectively, and she had 100% donor chimerisms on days +19.