Topics in Pediatric Leukemia -- Fanconi's Anemia: New Insights

Noah Federman, MD; Kathleen M. Sakamoto, MD, PhD

In This Article


The inherited bone marrow failure syndromes in children comprise a number of rare congenital disorders in which the bone marrow is unable to produce blood cells; this results in a deficiency of 1 or more cell lines. Studies on the pathogenesis of bone marrow failure syndromes have helped to elucidate the regulation of cellular growth and differentiation in the hematopoietic environment.[1]

Over the last decade, we have achieved a greater understanding of the genetic and molecular aspects of these rare disorders. Genes have been identified for many of the inherited syndromes. This article reviews Fanconi's anemia (FA), the most common of the rare inherited bone marrow failure syndromes, and discusses new insights on the pathophysiology of this disease.


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