Diacerein 1% Ointment for the Treatment of Epidermolysis Bullosa Simplex: A Randomized, Controlled Trial
June 2023 | Volume 22 | Issue 6 | 599 | Copyright © June 2023
Published online May 16, 2023
Joyce Teng , Amy S. Paller , Anna L. Bruckner , Dedee F. Murrell MD PhDd, Jemima E. Mellerio MDe, Christine Bodemer MD PhDf, Anna E. Martinez MDg, Aida Lugo-Somolinos MDh, Eli Sprecher MD PhDi, Martin Laimer MDj, Verena Wally PhDk, Yiumo Michael Chan PhDl, Sandy Y. Lin PhDl, Mary Spellman MDm, Johann W. Bauer MDj, k
aStanford University, School of Medicine, Palo Alto, CA
bNorthwestern University, Feinberg School of Medicine, Chicago, IL
cUniversity of Colorado School of Medicine and Children’s Hospital Colorado, Aurora, CO
dSt. George Hospital, UNSW Sydney, Sydney, Australia
eSt John’s Institute of Dermatology, Guy’s and St Thomas’ NHS Foundation Trust, London, United Kingdom
fDepartment of Dermatology, Expert Centre for Epidermolysis Bullosa (MAGEC), Necker Enfants Malades Hospital, Paris, France
gGreat Ormond Street Hospital, London, England
hUniversity of North Carolina (UNC) - Chapel Hill, Chapel Hill, NC
iDivision of Dermatology, Tel Aviv Sourasky Medical Center, Department of Human Molecular Genetics & Biochemistry,
Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
jDepartment of Dermatology and EB House Austria, University Hospital of the Paracelsus Medical University, Salzburg, Austria
kEB House Austria, Research Program for Molecular Therapy of Genodermatoses,
Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University, Salzburg, Austria
lTWi Biotechnology, Inc., Taipei, Taiwan
mCastle Creek Biosciences, Exton, PA
Abstract
Background: In epidermolysis bullosa simplex (EBS), epithelial structural fragility results in blisters and erosions. Diacerein 1% ointment has been shown to reduce this blistering.
Objective: To evaluate the efficacy and safety of diacerein 1% ointment in the treatment of EBS.
Methods: A double-blind study of 54 patients with EBS were randomized to diacerein 1% or vehicle ointment once daily. The primary endpoint ( ≥60% reduction in body surface area of EBS) and the key secondary endpoint ( ≥2-point reduction in the Investigator’s Global Assessment) were evaluated at 8 weeks.
Results: There was no difference in the proportion of patients achieving either key efficacy endpoint between the diacerein 1% and vehicle groups (P>0.05). No difference in treatment emergent adverse events were noted between the groups. In post hoc analysis stratified by EBS subtypes, an IGA score of 0 or 1 was reported in 6 of 13 patients with severe EBS in the diacerein group (46.2%), compared with 2 of 13 patients with severe EBS in the vehicle group (15.4%); (relative risk= 3.08, 95% CI = 0.71, 13.4).
Conclusions: Although there was no significant difference in outcomes between the groups, further study may elucidate the effects of diacerein on EBS lesions, especially in patients with severe EBS.
Teng J, Paller AS, Bruckner AL, et al. Diacerein 1% ointment for the treatment of epidermolysis bullosa simplex: a randomized, controlled trial. J Drugs Dermatol. 2023;22(6):599-604. doi:10.36849/JDD.7108
INTRODUCTION
Epidermolysis bullosa simplex (EBS), the most common type of epidermolysis bullosa, is a rare genetic skin condition, diagnosed in approximately 8 per 1 million births.
1 Characterized by mechanical fragility and inflammation of the epidermis, the clinical phenotype of EBS is extremely heterogenous and varies from mild blistering of the palms and soles to a severe disease involving widespread blistering, hyperkeratosis, nail dystrophy, skin pigmentation changes, and infection.
2,3
Autosomal dominant mutations in KRT5 or KRT14, encoding keratins 5 and 14, respectively, cause more than 70% of EBS cases. The resultant dysfunctional and disorganized keratins impair basal epidermal cell structural integrity and signaling,3-5 and lead to aberrant expression of inflammatory markers,
including interleukin-1β (IL-1β ).6
There are no approved treatments for EBS. Therapy is currently limited to local wound care, pain and itch management, and
