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Pediatr Transplant. 2014 Feb;18(1):E31-4. doi: 10.1111/petr.12175. Epub 2013 Oct 14.

Reduced intensity transplantation for congenital amegakaryocytic thrombocytopenia: report of a case and review of the literature.

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  • 1Department of Hematology/Oncology/BMT, Nationwide Children's Hospital, Ohio State University, Columbus, OH, USA.


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.

© 2013 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.


MPL gene mutation; congenital amegakaryocytic thrombocytopenia; hematopoietic stem cell transplant; matched-unrelated donor; reduced intensity conditioning

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