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Epidermolysis Bullosa

Epidermolysis Bullosa (EB) is a rare genetic disorder that affects the skin and mucous membranes, causing them to blister and tear easily. It is caused by a defect in the genes that control protein production that anchors the skin layers together. There are several types of EB, each with varying degrees of severity and different genetic mutations.

Epidermolysis Bullosa Simplex- This is the most common type of EB caused by mutations in genes that encode keratin proteins. It causes blisters to develop on the skin, which often occurs on the hands and feet.

Junctional Epidermolysis Bullosa- This type of EB is caused by gene mutations that encode laminin proteins. JEB is more severe than EBS and can cause blistering and erosion of the skin and mucous membranes and respiratory and digestive complications.

Dystrophic Epidermolysis Bullosa – A gene mutation that encodes collagen VII proteins causes DEB. The condition may lead to severe skin blistering, nail deformities, and blisters inside the mouth or throat.

Kindler Syndrome- This is a rare type of EB caused by mutations in the FERMT1 gene. Kindler Syndrome causes blistering, sensitivity to sunlight, and an increased risk of skin cancer.

Symptoms

The number of people developing EB in South Africa is rising. The symptoms of EB can range from mild blistering to life-threatening complications such as infections and systemic problems.

Some of the symptoms include:

  • Skin blisters: The most common symptom of EB is blistering of the skin, which can occur in response to minor trauma or friction.
  • Skin erosion: The blisters can burst and lead to skin erosion, leaving painful open wounds that can become infected.
  • Scarring: The repeated blistering and healing of the skin can lead to scarring and thickening.
  • Nail abnormalities: EB can cause nail dystrophy, or abnormal nail growth, including splitting, thickening, or loss of nails.
  • Dental problems: EB can affect the development of teeth, causing dental abnormalities such as cavities, gum disease, and missing or malformed teeth.

Causes

Genetic mutation is the primary cause of this skin problem; however, the specific mutations causing EB vary depending on the type. For example:

  • EBS is a result of mutations in genes encoding keratin proteins, which are important for maintaining the skin’s structural integrity.
  • JEB is a result of gene mutations encoding proteins that anchor the skin layers together, including laminin, collagen XVII, and integrin alpha6beta4.
  • Encoding in collagen VII leads to DEB. It is an important protein for anchoring the skin layers together.
  • Kindler Syndrome is caused by mutations in the FERMT1 gene. The absence of this protein disrupts many essential cell functions.

The primary risk factor for EB is having a family history of the condition, as it is an inherited disorder. However, in some cases, EB may occur spontaneously due to new mutations not inherited from parents.

Other risk factors for EB may include:

  • Recessive inheritance: Some types of EB, such as autosomal recessive DEB, require both parents to pass on a copy of the mutated gene for a child to develop the condition. Therefore, if both parents carry a mutated gene, their children may be at risk of developing the condition.
  • Consanguinity: Consanguinity, or the practice of marrying close relatives, can increase the risk of inherited genetic disorders, including EB. In communities where consanguinity is common, the prevalence of EB may be higher.
  • Prenatal diagnosis: If a family has a history of EB, it may be possible to diagnose the condition prenatally through genetic testing. This can help identify at-risk pregnancies and inform parents about their options.

Diagnosis & Complications

Your doctor may need to collect clinical, genetic, and family history tests to diagnose EB. Complications associated with EB can be severe and potentially life-threatening. Here are some of the common complications associated with EB and ways to prevent them:

  • Skin infections
  • Difficulty swallowing:
  • Anaemia
  • Contractures
  • Squamous cell carcinoma
  • Malnutrition

Treatment & Prevention

All types of EB can cause significant pain, discomfort, and disability, and there is currently no cure. Treatment focuses on managing symptoms, preventing complications, and improving quality of life. Treatment options include wound care, pain management, nutritional support, and surgical interventions. Individuals with EB require ongoing care and support from a team of healthcare professionals specialising in the condition.

 
 
 

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