Follow-up of five patients with FHHNC due to mutations in the Paracellin-1 gene

MT Wolf, J Dötsch, M Konrad, M Böswald… - Pediatric nephrology, 2002 - Springer
MT Wolf, J Dötsch, M Konrad, M Böswald, W Rascher
Pediatric nephrology, 2002Springer
Familial hypomagnesemia, hypercalciuria and nephrocalcinosis (FHHNC) is a rare
autosomal recessive inherited disorder that has recently been attributed to a defect in the
paracellin-1 (PCLN-1) gene, encoding for a protein responsible for the tubular reabsorption
of magnesium and calcium. Limited information is available on clinical course, therapy and
prognosis. We provide information on five patients with FHHNC and their follow-up at our
institution. Polyuria, nephrocalcinosis and hyperuricemia were the main clinical findings of a …
Abstract
Familial hypomagnesemia, hypercalciuria and nephrocalcinosis (FHHNC) is a rare autosomal recessive inherited disorder that has recently been attributed to a defect in the paracellin-1 (PCLN-1) gene, encoding for a protein responsible for the tubular reabsorption of magnesium and calcium. Limited information is available on clinical course, therapy and prognosis. We provide information on five patients with FHHNC and their follow-up at our institution. Polyuria, nephrocalcinosis and hyperuricemia were the main clinical findings of a diagnosis at a median age of 4.4 years. The clinical course of PCLN-1 mutations as presented in this study is highly variable, ranging from compensated renal failure to end-stage renal failure – as happened in two of our patients. The progression to renal failure cannot be deduced from the initial presentation. Medical treatment does not appear to influence the progression of the disease. Despite calcium and magnesium substitution, normal values could not be achieved in these patients. Early treatment with vitamin D and calcium was essential to maintain growth. Adequate treatment allows for a normal height and pubertal development.
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