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Harboyan Syndrome

OMIM ID:

autosomal recessive

Harboyan Syndrome

Alternate Names

CDPD
CDPD1
corneal dystrophy and sensorineural deafness

Defective Genes

SLC4A11

Clinical Characteristics

Ocular Features

The combination of congenital endothelial dystrophy and progressive neural deafness is known as Harboyan syndrome.  This disorder must be distinguished from another autosomal recessive disorder, congenital endothelial dystrophy 2 or CHED2 (217700), in which deafness does not occur.  While the corneal disease in Harboyan is present at birth, the deafness often does not become obvious until the second and third decades of life although audiometry can detect some hearing loss in the first decade.  The cornea is thickened and edematous resulting in various degrees of visual impairment, even to the level of counting fingers.  Electrophysiologic studies have been normal.

Systemic Features

No systemic abnormalities have been reported.

Genetics

Inheritance

This is an autosomal recessive disorder caused by a mutation in the SLC4A11 gene located on chromosome 20 (20p13-12).  It is allelic to simple, congenital endothelial corneal dystrophy (CHED2) (217700).  About half of reported cases occur sporadically and the rest have been reported in offspring of consanguineous matings.  Less than 30 cases have been reported worldwide.

Pedigree

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

Corneal transplantation is the treatment of choice and can result in substantial visual improvement.

Selected Resources

Publications

Displaying 1 - 3 of 3

Borate transporter SLC4A11 mutations cause both Harboyan syndrome and non-syndromic corneal endothelial dystrophy

PubMedID: 17220209

Congenital Corneal Dystrophy: Progressive Sensorineural Deafness in a Family

PubMedID: 5312820

Congenital hereditary endothelial dystrophy with progressive sensorineural deafness (Harboyan syndrome)

PubMedID: 18922146