2018
DOI: 10.1016/j.jaad.2018.01.019
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Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial

Abstract: This trial provides evidence of the impact of 1% diacerein cream in the treatment of EBS.

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Cited by 51 publications
(68 citation statements)
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“…We further showed that this results in increased transcriptional levels of AP‐1‐responsive genes, among them KRT14 and IL1B , in a positive feedback loop, which exacerbates the phenotype due to accumulation of keratin 14 (wild‐type and mutated) in the cytoplasm. We also demonstrated that downregulation of KRT14 expression ameliorates EBS‐gen sev hallmarks in vitro and in vivo …”
Section: Resultsmentioning
confidence: 61%
See 1 more Smart Citation
“…We further showed that this results in increased transcriptional levels of AP‐1‐responsive genes, among them KRT14 and IL1B , in a positive feedback loop, which exacerbates the phenotype due to accumulation of keratin 14 (wild‐type and mutated) in the cytoplasm. We also demonstrated that downregulation of KRT14 expression ameliorates EBS‐gen sev hallmarks in vitro and in vivo …”
Section: Resultsmentioning
confidence: 61%
“…However, their potentially short‐term availability currently renders such approaches an attractive option for increasing patients’ quality of life. Especially for causal therapies, which aim to correct genetic mutations, several hurdles remain …”
mentioning
confidence: 99%
“…No adverse effect was reported. Confirmative (monocentric and international multi‐centre) phase II/III studies to assess the potential of topical diacerein to downregulate the activity of IL‐1 and reduce the auto‐inflammatory effects in the skin of patients with EBS have been recently published . In this 2‐period crossover trial, 17 patients were randomized to either placebo or diacerein for a 4‐week treatment and a 3‐month follow‐up in period 1.…”
Section: Clinical Researchmentioning
confidence: 99%
“…A topical treatment, Oleogel S-10, consisting of a plant extract that reduces inflammation and promotes keratinocyte migration, has advanced to Phase 3 trials [25]. A topical anti-inflammatory, Diacerein, has completed a small 15patient Phase 2 trial with efficacy for EB simplex [26], and is recruiting by invitation for its long-term safety trial (NCT03389308). Another topical treatment, Zorblisa, unfortunately failed to show significant efficacy in a recent Phase 3 trial (clinicaltrials.gov, NCT02384460), and oral polyphenon E failed to show efficacy in a Phase 2 trial [27].…”
Section: Clinical Studies On Ebmentioning
confidence: 99%