Abstract IA05: Fanconi's anemia: Emerging therapeutic opportunities
摘要:
Fanconi's anemia (FA) is a genetically heterogeneous inherited disorder characterized by bone marrow failure, birth defects and cancer susceptibility. At the cellular level, FA features hypersensitivity to DNA cross-linking agents, cell cycle abnormalities and abnormal responses to cytokines. Therapy for the hematopoietic defects of FA has consisted of supportive care, stem cell transplantation and administration of androgens such as oxymetholone. Unfortunately, even successfully transplanted FA patients have a very high risk for subsequent solid tumors, especially squamous cell carcinomas of oropharyngeal and urogenital areas.
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DOI:
10.1158/1538-7445.CANSUSC14-IA05
被引量:
年份:
2014
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