Neoplasia in familial dysautonomia: a 20-year review in a young patient population.

Gold-von Simson, Gabrielle; Romanos-Sirakis, Eleny; Maayan, Channa; Axelrod, Felicia B · J Pediatr · 2009

retrospective_cohort · Level III

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Abstract

We reviewed the charts of all patients with familial dysautonomia (n = 631) and found that 2% had been diagnosed with tumors. We hypothesize that the IkappaB Kinase-associated protein gene mutation, which causes aberrant RNA splicing in patients with familial dysautonomia, may contribute to tumorigenesis in this genetically homogenous patient population.

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