Ishita Gupta

United States

Evaluating the Current Landscape of Epidermolysis Bullosa Treatments

Ishita Gupta, MBBS1, Amritpal Kooner, MA2, Rahila Shaikh, MD3, George Hourany, BS4, Jarett Anderson, DO5

1. Department of Internal Medicine, St. Bernards Medical Center, Jonesboro, AR, USA
2. Chicago College of Osteopathic Medicine, Midwestern University, Downers Grove, IL, USA
3. Saba University School of Medicine, The Bottom, Saba, Dutch Caribbean
4. California Health Sciences University College of Osteopathic Medicine, California Health
Sciences University, Clovis, CA, USA
5. Chicago College of Osteopathic Medicine, Midwestern University, Downers Grove, IL, USA

Abstract

Background

Epidermolysis bullosa (EB) encompasses a group of rare blistering disorders that affect both the skin and mucous membranes. There is currently no cure for EB, and only supportive treatment is available to manage symptoms of pruritus and wounds. This paper analyzes the current treatment modalities of epidermolysis bullosa and provides a comprehensive review.

Methods

A comprehensive literature search was conducted in PubMed using relevant keywords and MeSH terms. Inclusion criteria included manuscripts published in the English language and must include a control group. Two independent reviewers will assess study eligibility based on predetermined criteria. Data extraction will cover study characteristics, participant demographics, treatment outcomes, and relevant findings.

Results

21 articles were identified which examined the effects of various interventions for the treatment of epidermolysis bullosa. Gene therapy studies demonstrated promising results, specifically those transducing the COL7A1 gene into patients showed significant wound healing. Topical therapies with bermagene geperpavec, diacerin, and b-vec showed promising results, while oleogel S-10 and SD-101 had mixed results. Oral therapies, including those with tetracycline, mycophenolate mofetil, serlopitant, and ECG showed limited results in improving patient outcomes.

Conclusions

The current literature shows a wide range of investigated therapies for the treatment and management of epidermolysis bullosa. Treatment modalities identified in this literature review were categorized into gene, oral, or topical therapies. While gene therapy stood out as an effective intervention in improving wound healing and patient reported outcomes, challenges were persistent amongst studies including small sample sizes and limited follow-up, warranting cautious optimism.