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Watson Syndrome

OMIM ID:

autosomal dominant

Watson Syndrome

Alternate Names

pulmonic stenosis with cafe-au-lait spots

Defective Genes

NF1

Clinical Characteristics

Ocular Features

Iris nodules similar to those seen in neurofibromatosis are found in some but not all patients with Watson syndrome.

Systemic Features

Short stature and low normal intelligence are the most consistent features.  Pulmonic stenosis and cafe-au-lait spots are also common.   The macrocephaly is relative and not striking.  Neurofibromas have been seen in a minority of patients.

Genetics

Inheritance

Mutations in the NF1(17q11.2) gene have been identified in members of several large pedigrees with an apparent autosomal dominant pattern.

It remains uncertain if this condition is allelic to neurofibromatosis I(162200) or if Watson syndrome is the result of mutations in contiguous genes.

The LEOPARD syndrome(151100) shares some clinical similarities such as short stature, pulmonic stenosis, cognitive deficits and cafe-au-lait spots but is caused by mutations in PTPN11.   The phenotype also resembles Noonan syndrome in some aspects.

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

Treatment & Management

There is no known treatment for this condition but multidisciplinary management is recommended for isolated problems.

Publications

Displaying 1 - 1 of 1

Watson syndrome: is it a subtype of type 1 neurofibromatosis?

PubMedID: 1770531