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STAT3 Dominant Negative
Disease (STAT3DN)

STAT3 Dominant Negative Disease (STAT3DN) is a rare genetic immune-deficiency disorder that leads to facial deformities, skin and teeth problems, and frequent colds and lung infections. STAT3DN is also known as Job Syndrome or Hyper-IgE syndrome, because it is caused by a mutation in white blood cells (the disease-fighting immune cells of the body) that leads to the overproduction of IgE and an inability to recruit neutrophils to the site of an infection. IgE is an antibody (immunoglobulin) that gets secreted by plasma cells (a type of white blood cell), and neutrophils are important white blood cells that are part of the first line of defence against new infections.

The lack of neutrophils in STAT3DN leads to many of its most notable features, including the pneumonia and lung infections it causes, and the cold Staphylococcal abscesses it causes on the skin. Staphylococcus aureus is a type of bacteria that usually causes painful, red, warm abscesses full of pus when it affects the skin, but in STAT3DN, the Staphylococcus abscesses are painless, and not red or hot.

Symptoms

The symptoms of STAT3DN can be remembered with the acronym ABCDEF:

  • A – abscesses on skin
  • B – baby teeth never fall out, leading to two sets of teeth in adulthood
  • C – coarse facial features, with a prominent forehead and fleshy nose, lips, and gums
  • D – damaged lungs due to frequent bouts of pneumonia
  • E – eczema
  • F – fractures of bones from only minor trauma

Causes

STAT3DN is caused by a genetic mutation in a gene called STAT3. This mutation leads to reduced production of Helper T cells, which are an important type of white blood cell. Helper T cells normally help to attract neutrophils to the site of an infection. With low Helper T cells, neutrophils don’t act as they should, and infections like abscesses and pneumonia are very common in STAT3DN.

The exact cause of the hypersecretion of IgE is not known, but this is also a feature of STAT3DN.

 Risk Factors

There are no known risk factors for STAT3DN other than family history. This is because STAT3DN is an autosomal dominant genetic disorder, which means that for a couple where one parent has the disease and the other does not, each child of theirs will have a 50% chance of getting STAT3DN.

Diagnosis

One of the most important diagnostic markers in STAT3DN is high levels of IgE. This can be found by taking a blood sample. In combination with this, the classic features of STAT3DN like eczema, cold abscesses, and retained baby teeth are noted clinically by a doctor. These classic features are scored on a special scale called the HIES scoring system. If enough of these features exist, the diagnosis of STAT3DN is supported.

Treatment & Management

Unfortunately, STAT3DN cannot be cured. Typically, STAT3DN is managed supportively, which means it is treated with measures and interventions that help improve the day-to-day lives of affected people.

Dermatologists and infectious-disease specialist doctors may help to manage eczema and skin abscesses. Dentists and otolaryngologists (ear, nose, and throat doctors) may manage retained baby teeth and any problematic facial abnormalities. Some of the management of STAT3DN may include medicine for infections, while other problems may require surgery. The exact treatment depends on the specifications of the treating doctor. People with STAT3DN usually require the care of multiple doctors in different specialties, and it is important to follow their directions closely.

 
 
 

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