Topical Diacerein Ointment for Epidermolysis Bullosa Simplex

A Review

Allison L. Limmer, BA, BS; Crystal E. Nwannunu, BS; Radhika Shah, BS, MS; Kendall Coleman, BSA; Ravi R. Patel, MD; Uyen Ngoc Mui, MD; Stephen K. Tyring, MD, PhD

Disclosures

Skin Therapy Letter. 2019;24(3):7-9. 

In This Article

Abstract and Introduction

Abstract

Epidermolysis bullosa (EB) is a group of rare mucocutaneous fragility disorders often presenting in infancy and early childhood with painful blistering of the skin and mucous membranes. The severity of EB blister burden varies by disease subtype. Studies have shown that patients with generalized severe epidermolysis bullosa simplex (EBS), a variant characterized by extreme fragility, develop blisters in the setting of overproduced, mutated K14 protein, a component of the intermediate filament integral in keratinocyte stability, and constitutive activation of interleukin (IL)-1 , a pro-inflammatory cytokine that promotes the hyperproliferation of keratinocytes. Diacerein, a rhein prodrug and anthraquinone, has been shown to reduce expression of K14 and inhibit IL-1 converting enzyme. In clinical trials, topical 1% diacerein was shown to be an effective and safe, non-invasive treatment for patients suffering from EBS. This review examines the clinical trials of topical diacerein and its role in EBS. Diacerein ointment was granted US FDA Rare Pediatric Disease designation in May 2018 and Fast Track development designation in August 2018.

Introduction

Epidermolysis bullosa (EB) is a group of rare genetic diseases that causes fragile blistering of the skin.[1] EB presents most commonly in infancy and early childhood, but in some cases it can present later in adolescence.[2] Painful skin blisters often manifest spontaneously or secondary to friction against the skin, minor injury, or even from heat.[3] Patients with EB are often called "Butterfly Children," as their skin is as fragile as the wings of a butterfly.[4]This inherited disease not only causes blister formation of the skin, but also can occur within mucosal membranes such as the oral cavity and the respiratory, gastrointestinal, and genitourinary tracts.[5,6] The severity of EB varies with the type and inheritance pattern of the disease.[4] Milder forms commonly resolve with time, but severe forms are associated with more painful and often disabling blistering, scarring, and deformation that can lead to life-threatening health complications.[1]

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