期刊
BRITISH JOURNAL OF HAEMATOLOGY
卷 140, 期 5, 页码 568-571出版社
BLACKWELL PUBLISHING
DOI: 10.1111/j.1365-2141.2007.06890.x
关键词
platelet genetic diseases; paediatric haemostasis; stem cell transplantation
类别
资金
- Great Ormond Street Hospital Childrens Charity [V0904] Funding Source: researchfish
Glanzmann thrombasthenia (GT) is a rare autosomal recessive platelet function disorder. Stem cell transplantation (SCT) is curative, but it is only indicated in selected patients with a severe clinical phenotype or who develop anti-platelet antibodies. SCT have previously been limited to full intensity myeloablative conditioning regimens. This study details the successful outcome of SCT in five consecutive patients with GT, three of whom received reduced intensity conditioning (RIC) with stem cells from non-sibling donors. This is the first time RIC SCT has been reported in GT, and offers the possibility of curative therapy with reduced late effects.
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