Journal
BRITISH JOURNAL OF HAEMATOLOGY
Volume 140, Issue 5, Pages 568-571Publisher
BLACKWELL PUBLISHING
DOI: 10.1111/j.1365-2141.2007.06890.x
Keywords
platelet genetic diseases; paediatric haemostasis; stem cell transplantation
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Funding
- Great Ormond Street Hospital Childrens Charity [V0904] Funding Source: researchfish
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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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