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Unrelated bone marrow transplant for congenital amegakaryocytic thrombocytopenia: Report of two cases and review of the literature
Author(s) -
Frangoul Haydar,
KeatesBaleeiro Jennifer,
Calder Cassie,
Manes Becky,
Crossno Christine,
Castaneda Victoria L.,
Domm Jennifer
Publication year - 2010
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/j.1399-3046.2009.01131.x
Subject(s) - medicine , bone marrow transplant , bone marrow , bone transplantation , bone marrow transplantation , surgery , pediatrics , transplantation
Frangoul H, Keates‐Baleeiro J, Calder C, Manes B, Crossno C, Castaneda VL, Domm J. Unrelated bone marrow transplant for congenital amegakaryocytic thrombocytopenia: Report of two cases and review of the literature.
Pediatr Transplantation 2010: 14:E42–E45. © 2009 John Wiley & Sons A/S. Abstract:  CAMT is a very rare cause of thrombocytopenia in infants. Most of the patients will progress to marrow failure. Allogeneic stem cell transplant remains the only curative therapy. We present two patients with CAMT who underwent an unrelated donor bone marrow transplant, one after developing marrow failure and another early in the course of the disease. Both patients tolerated the transplant with minimal toxicity and durable engraftment. We also present a comprehensive review of the literature for unrelated donor transplant for this condition.

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