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Hypoparathyroidism, Familial Isolated

OMIM ID:

autosomal recessive
autosomal dominant

Hypoparathyroidism, Familial Isolated

Alternate Names

FIH
autosomal dominant hypoparathyroidism
autosomal recessive hypoparathyroidism

Defective Genes

PTH
GCMB

Clinical Characteristics

Ocular Features

Lens opacities may be present.

Systemic Features

The major signs and symptoms result from hypocalcemia. Neuromuscular irritability and various paresthesias may be present.  Some patients have  laryngeal spasm and latent tetany with grand mal seizures.  Alopecia, abnormal dentition and coarse brittle hair may be present.  Cognitive deficits and personality disorders are often a feature.  Brain imaging may show calcification of the basal ganglia.  Serum calcium levels are usually low while phosphorus levels are elevated.   Vitamin D precursor levels are usually low or low normal.

Genetics

Inheritance

Familial hypoparathyroidism may be due to mutations in the PTH gene (11p15.3) (either autosomal dominant or recessive inheritance) or in the GCMB gene (6p24.2) (autosomal dominant inheritance pattern).

There is also an X-linked form of hypoparathyroidism (307700) in which parathryroid tissue may be congenitally absent.

A family has been reported in which hypoparathryroidism was associated with lymphedema (247410) and progressive renal failure.  Ptosis, telecanthus, hypertrichosis, restrictive lung disease, and mitral valve prolapse may also be part of the disorder.

Pedigree

Autosomal dominant

Autosomal dominant disorders require only one mutation for the disease to be expressed.  Since an affected parent has two chromosomes, only one of which has the mutant gene, parents can expect that half (50%) of their children will receive that one and inherit the disease.  It is common for individuals that inherit the mutation, however, to not have evidence of the disease (nonpenetrance).

Image
Sample pedigree of autosomal dominant inheritance

Autosomal dominant inheritance leads to a vertical pattern of transmission

Autosomal recessive

In order for autosomal recessive disorders to be expressed, offspring generally must inherit two mutations, one from each carrier parent.  Carriers with only one mutation, such as the parents, do not have clinical disease.  Note that carrier parents can expect that 1 in 4 children (25%) will inherit both mutations and have the disorder, 2 in 4 children (50%) will be carriers like their parents, while 1 in 4 children (25%) inherit neither mutation.

Image
Sample pedigree of autosomal recessive inheritance

In order for autosomal recessive disorders to be expressed, offspring generally must inherit two mutations, one from each carrier parent. Carriers with only one mutation, such as the parents, do not have clinical disease. Note that carrier parents can expect that 1 in 4 children (25%) will inherit both mutations and have the disorder, 2 in 4 children (50%) will be carriers like their parents, while 1 in 4 children (25%) inherit neither mutation.

Treatment & Management

Normalization of calcium and phosphorus levels is a priority and this may result in some clearing of the lens opacities.  Cataract surgery may be indicated in selected individuals.

Selected Resources

Web Resources

Publications

Displaying 1 - 4 of 4

Autosomal or X‐linked recessive syndrome of congenital lymphedema, hypoparathyroidism, nephropathy, prolapsing mitral valve, and brachytelephalangy

PubMedID: 6638075

Familial isolated hypoparathyroidism: a molecular genetic analysis of 8 families with 23 affected persons

PubMedID: 3005800

Mutation of the signal peptide-encoding region of the preproparathyroid hormone gene in familial isolated hypoparathyroidism.

PubMedID: 2212001

The use of a diazonium salt for the determination of glutamic-oxalacetic transaminase in serum

PubMedID: 13863676