Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial
Language English Country United States Media print-electronic
Document type Clinical Trial, Phase II, Clinical Trial, Phase III, Journal Article, Randomized Controlled Trial
PubMed
29410318
DOI
10.1016/j.jaad.2018.01.019
PII: S0190-9622(18)30130-0
Knihovny.cz E-resources
- Keywords
- diacerein, epidermolysis bullosa, keratin, rare genodermatosis,
- MeSH
- Patient Compliance MeSH
- Anthraquinones therapeutic use MeSH
- Anti-Inflammatory Agents MeSH
- Administration, Topical MeSH
- Child MeSH
- Double-Blind Method MeSH
- Epidermolysis Bullosa Simplex diagnosis drug therapy MeSH
- Risk Assessment MeSH
- Humans MeSH
- Follow-Up Studies MeSH
- Child, Preschool MeSH
- Drug Administration Schedule MeSH
- Severity of Illness Index MeSH
- Orphan Drug Production * MeSH
- Treatment Outcome MeSH
- Dose-Response Relationship, Drug MeSH
- Check Tag
- Child MeSH
- Humans MeSH
- Male MeSH
- Child, Preschool MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Clinical Trial, Phase II MeSH
- Clinical Trial, Phase III MeSH
- Randomized Controlled Trial MeSH
- Names of Substances
- Anthraquinones MeSH
- Anti-Inflammatory Agents MeSH
- diacerein MeSH Browser
BACKGROUND: Epidermolysis bullosa simplex (EBS) is a rare genetic, blistering skin disease for which there is no cure. Treatments that address the pathophysiology of EBS are needed. OBJECTIVE: Compare the impact of 1% diacerein cream with placebo in reducing the number of blisters in EBS. METHODS: In a randomized, placebo-controlled, phase 2/3 trial we used a 1% diacerein topical formulation to treat defined skin areas in 17 patients. In a 2-period crossover trial, patients were randomized to either placebo or diacerein for a 4-week treatment and a 3-month follow-up in period 1. After a washout, patients were crossed over during period 2. The prespecified primary end point was the proportion of patients with a reduction of number of blisters by more than 40% from baseline in selected areas over the treatment episode. RESULTS: Of the patients receiving diacerein, 86% in episode 1 and 37.5% in episode 2 met the primary end point (vs 14% and 17% with placebo, respectively). This effect was still significant after the follow-up. Changes in absolute blister numbers were significant for the diacerein group only. No adverse effects were observed. LIMITATIONS: Low patient numbers and no invasive data acquisition because of clinical burden in children. CONCLUSION: This trial provides evidence of the impact of 1% diacerein cream in the treatment of EBS.
Department of Laboratory Medicine Paracelsus Medical University Salzburg Austria
Department of Pediatric Dermatology Children's Hospital Brno Czech Republic
Landesapotheke Salzburg Department of Production Hospital Pharmacy Salzburg Austria
Research Office Biostatistics Paracelsus Medical University Salzburg Austria
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