People with epidermolysis bullosa (EB) often experience symptoms such as intense itching or severe pain. Unfortunately, these symptoms can be debilitating and may greatly affect overall quality-of-life. However, some burgeoning therapeutic options could change that. According to Epidermolysis Bullosa News, a clinical trial evaluating low-dose calcipotriol ointment showed efficacy in treating symptoms of dystrophic epidermolysis bullosa (DEB).
Interested in seeing the full study findings? Take a look at the findings published in Orphanet Journal of Rare Diseases.
About Epidermolysis Bullosa (EB)
Various gene mutations cause epidermolysis bullosa (EB), a group of rare connective tissue diseases that cause moderate-to-severe blistering on the skin and mucous membranes. Typically, EB is inherited in an autosomal recessive pattern, meaning patients must inherit one defective gene from each parent. However, it is possible to inherit EB in an autosomal dominant pattern. There are multiple forms of epidermolysis bullosa, including:
- Junctional (JEB) – this form is characterized by thin skin and large, ulcerative blisters.
- Epidermolysis bullosa simplex (EBS) – this form is characterized by thickened skin on the palms and soles, as well as widespread skin blistering.
- Dystrophic (DEB) – in this form, blistering mainly occurs on the hands and feet. This form also comes with an increased risk of skin cancer.
In the referenced study, researchers evaluated calcipotriol for DEB. Symptoms and clinical manifestations include:
- Fragile, easily blistered skin
- Widespread areas of missing or blistered skin at birth
- Milia (small, white spots present at healed but scarred skin)
- Cheilitis (inflammation of the lips)
- Poor fingernail and toenail formation
- Malnutrition or dehydration
- Fusion of the skin between fingers and toes
- Joint deformities
Calcipotriol
In the past, some research has suggested that Vitamin D3 could be beneficial in wound healing. For example, some research suggests that Vitamin D3 plays a role in both fighting infections and improving epidermal growth factors. So researchers questioned whether calcipotriol, a vitamin D3 analog, could help reduce itching and improve wound closure in those with DEB. Currently, calcipotriol is sometimes used to treat psoriasis, though the concentration used in the ointment within this study is significantly lower. Learn more about calcipotriol.
Altogether, six patients with DEB finished this clinical trial. At the trial’s onset, each patient had at least 2 wounds that were 6 square centimeters. During the trial, patients received either a placebo or calcipotriol for a 1-month period. Following this initial period, patients went two months without treatment before switching; those who had previously received calcipotriol received the placebo, and those who received the placebo got calcipotriol, for another month. Findings include:
- Calcipotriol improved pain within 2 weeks of treatment, though the pain scores were not significantly different following the 1-month treatment period between this and the placebo.
- Treatment improved wound closure and wound size compared to placebo. Calcipotriol also reduced itching associated with DEB.
- Altogether, the treatment was relatively safe and well-tolerated. Participants reported no side effects.
