Cockaynes syndrom

Cockaynes syndrom är en sjukdom som är medfödd. Symptomen är bland mycket annat, småväxta, Hörselnedsättning dövhet, Synnedsättning, ataxi(skakningar) o.s.v.

Syndromet är mycket ovanligt. I Sverige finns, för oss kända, endast 4 personer varav Linda och Claes är 2 av dem.

Och här en beskrivning på engelska, and in english.

Cockayne´s Syndrome

(Cockayne-Neill Dwarfism)

 

Cockayne in 1936 [1] and again in 1946 [2] described a brother and sister with dwarfism, microphaly, mental retardation, peculier retinal pigmentation, optic atrophy, exposure dermatitis, and progressive neurologic deterioration. Neill and Dingwall [3] described two brothers with similar features. More than 20 pations have subsequently been described [4-9].

 

Physical features

 

  Head:  All of the reported patiens have been microcephalic.

  Face:  At birth the appearance is normal. A loss of subcutaneous fat gradually develops during childhood. Older patiens usually have senile appearance with sunken eyes and prominence of the maxilla and nose.

  Eyes:  The vision is initially normal. Pigmentary retinal degeneration, optic atrophy, and arterial narrowing gradually develop during childhood. Other common but less frequent eye findings are cataracs, loss of the macular reflex, nystagmus, lack of tearing, and poor response to mydriatic drugs.

  Ears:  The eares appear prominent because of the loss of subcutaneous fat in the face.

  Chest:  The trunk is short and sometimes barrel-shaped.

  Breasts:  At the age of expected puberty the females do not have normal breast development.

  Genitalia:  Several of the reported males have had cryptorchidism. At puberty the penis is of normal size, but the testes remain small and the pubic hair growth often has a feminiue pattern.

  Back:  Some patiens have a stooped posture and dorsal kyphosis.

  Limbs:  The arms and legs appear to be disproportionately long in comparison to the trunk. The hands seem disproportionately large. The range of motion of the hips, knees, and ankles becomes prgressively more restricted.

  Skin:  A scaly, erythematous dermatitis develops after exposure to the sun, usually on the hands, legs, face, and ears. The dermatitis of the face may have a “butterfly” distribution. Older patiens have thickned, wrinkled skin, especially on the face.

  Height:  The length at birth is normal. Shortness of stature is evident in childhood. Adults are usually less then 4 feet (123 cm) tall.

  Weight:  These pations are usually thin.

 

Nervous Systems

 

Early psychomotor development is slow. The mental deficiency is marked, with most IQ estimates blow 50. Evidence of proressive neurologic deterioration is usually apperent in the second decade. The muscle power deminishes and the gait becomes awkward. Nystagmus, ataxia, an  intention tremor, and incoordination develop. Initially the deep tendon reflexes are hyperactive, later hypoactive. Most patiens become blind and deaf.

 

Pathology

 

The brain is small and the cerbral cortex and cerebellum appear atrophic. Ther is a marked reduction in the cerebral white matter. Myelin stains show a patchy demyelinization that is often severe in the subcortical white matter. Calcification may be marked; it is symmetrically distributed in the cortex, basal ganglia, and cerebellum. Segmental demyelination in peripheral nerves has also been reported. Renal biopsis in two patients showed thickening of the mesangium and the basement membrane of the glomerulus, some collapsed capillary loops, hyalinized glomeruli, and tubular atrophy with interstitial fibrosis.

 

Laboratory studies

 

The cerebrospinal fluid protein is usually increased. Three reported patients had diminished glomerular function; one also had azotemia and hyperchloremic acidosis. This later patients also had type II hyperlipoproteinemia and fasting hyperinsulinemia. Another patient had hyperglycemia and an increased plasma insulin level. The skeletal maturation is normal. A thickned calvarium and anterior notching of the thoracic vertebrae are sometimes present. Intracranial calcifications are often visible on the skull x-ray, especially in the region of the basal ganglia.

 

Treatment and prognosis

 

By 20 years of age these patients are usually unable to care for themselves. Death usually occurs in late childhood or early adulthood from inanition and respiratory infections.

 

Genetics

 

Autosomal recessive.

 

Åter hemsidan