Natural history of adolescent-onset cystinosis

JP Midgley, R El-Kares, F Mathieu, P Goodyer - Pediatric nephrology, 2011 - Springer
JP Midgley, R El-Kares, F Mathieu, P Goodyer
Pediatric nephrology, 2011Springer
Cystinosis is a rare autosomal recessive disease caused by mutations of the CTNS gene in
which cystine accumulates throughout the body as a result of a defective efflux of cystine
from lysosomes. Three phenotypic forms have been described according to the age of onset
and the severity of the clinical symptoms: infantile, intermediate, and ocular non-
nephropathic cystinosis. Here we report the natural history of cystinosis in a 55-year-old man
with intermediate nephropathic cystinosis diagnosed at 9 years of age. Although tubulopathy …
Abstract
Cystinosis is a rare autosomal recessive disease caused by mutations of the CTNS gene in which cystine accumulates throughout the body as a result of a defective efflux of cystine from lysosomes. Three phenotypic forms have been described according to the age of onset and the severity of the clinical symptoms: infantile, intermediate, and ocular non-nephropathic cystinosis. Here we report the natural history of cystinosis in a 55-year-old man with intermediate nephropathic cystinosis diagnosed at 9 years of age. Although tubulopathy was unnoticed in the early years, he required transplantation at age 16. Sequencing analysis of all the CTNS exons revealed that the proband is homozygous for a 21-bp in-frame deletion in exon 5 (c. 198_218del21), resulting in an in-frame deletion of 7 amino acids from the N-terminal domain of the cystinosin protein. Our patient has had relatively mild extra-renal disease despite lack of early cysteamine therapy. He has been able to attend university and pursue a professional career into the 6th decade.
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