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Pediatr Transplant. 2012 May;16(3):E69-73. doi: 10.1111/j.1399-3046.2010.01414.x. Epub 2010 Nov 26.

Unrelated hematopoietic stem cell transplantation in a patient with congenital dyserythropoietic anemia and iron overload.

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Division of Hematology, CHOC Children's Hospital and UC Irvine Medical Center, Orange, CA 92868, USA.


CDA is a heterogeneous group of disorders that result in morphologically abnormal erythroid maturation and ineffective erythropoiesis. Curative therapy for CDA focuses on the use of HSCT using fully matched sibling donors. This is the first report of a Type II CDA patient with severe iron overload who was successfully treated with HSCT using a HLA-matched unrelated donor after aggressive chelation therapy. Given the challenges of HSCT in any patient with CDA and severe iron overload, the role of novel approaches to iron chelation and HSCT is discussed.

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