Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial.
J Am Acad Dermatol
; 78(5): 892-901.e7, 2018 05.
Article
en En
| MEDLINE
| ID: mdl-29410318
ABSTRACT
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.Palabras clave
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Producción de Medicamentos sin Interés Comercial
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Epidermólisis Ampollosa Simple
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Antraquinonas
Tipo de estudio:
Clinical_trials
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Etiology_studies
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Observational_studies
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Prognostic_studies
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Risk_factors_studies
Límite:
Child
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Child, preschool
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Female
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Humans
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Male
Idioma:
En
Revista:
J Am Acad Dermatol
Año:
2018
Tipo del documento:
Article