期刊
PEDIATRIC BLOOD & CANCER
卷 45, 期 2, 页码 212-216出版社
WILEY
DOI: 10.1002/pbc.20332
关键词
congenital amegakaryocytic thrombocytopenia; marrow failure; monosomy 7; reduced intensity; transplantation
Congenital amegalkaryocytic thrombocytopenia (CAMT) is a rare inherited bone marrow failure syndrome that has the potential to progress to pancytopenia and acute myeloid leukemia. Hematopoietic stem-cell transplantation (HSCT) is presently the only curative treatment approach. We used a reduced intensity transplantation regimen in a CAMT patient with aplastic anemia and monosomy 7 who had no matched related donor. The patient had rapid and durable engraftment with minimal complications and is well 24 months post-transplantation. Thus, reduced intensity conditioning might be a feasible approach to stem-cell transplantation in patients with CAMT who do not have a related donor and who are at increased risk of toxicity from standard conditioning regimens. (c) 2005 Wiley-Liss, Inc.
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