TY - JOUR
T1 - Unrelated bone marrow transplant for congenital amegakaryocytic thrombocytopenia
T2 - Report of two cases and review of the literature
AU - Frangoul, Haydar
AU - Keates-Baleeiro, Jennifer
AU - Calder, Cassie
AU - Manes, Becky
AU - Crossno, Christine
AU - Castaneda, Victoria L.
AU - Domm, Jennifer
PY - 2010/6
Y1 - 2010/6
N2 - 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.
AB - 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.
KW - Congenital amegakaryocytic thrombocytopenia
KW - Pediatrics
KW - Unrelated donor bone marrow transplantation
UR - http://www.scopus.com/inward/record.url?scp=77952276420&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=77952276420&partnerID=8YFLogxK
U2 - 10.1111/j.1399-3046.2009.01131.x
DO - 10.1111/j.1399-3046.2009.01131.x
M3 - Article
C2 - 19490489
AN - SCOPUS:77952276420
SN - 1397-3142
VL - 14
SP - E42-E45
JO - Pediatric transplantation
JF - Pediatric transplantation
IS - 4
ER -