Basal pharmacokinetic parameters of topically applied diacerein in pediatric patients with generalized severe epidermolysis bullosa simplex

Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes after minor mechanical friction. In a clinical phase 2/3 trial, diacerein has recently been...

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Published inOrphanet journal of rare diseases Vol. 13; no. 1; pp. 193 - 5
Main Authors Ablinger, Michael, Felder, Thomas K., Wimmer, Monika, Zauner, Roland, Hofbauer, Peter, Lettner, Thomas, Wolkersdorfer, Martin, Lagler, Florian B., Diem, Anja, Bauer, Johann W., Wally, Verena
Format Journal Article
LanguageEnglish
Published London BioMed Central 01.11.2018
BioMed Central Ltd
BMC
Subjects
Administration, Topical
Analysis
Anthraquinones - administration & dosage
Anthraquinones - chemistry
Anthraquinones - pharmacokinetics
Anthraquinones - therapeutic use
Anti-Inflammatory Agents - administration & dosage
Anti-Inflammatory Agents - chemistry
Anti-Inflammatory Agents - pharmacokinetics
Anti-Inflammatory Agents - therapeutic use
Clinical trials
Diacerein
Drug therapy
Epidermolysis bullosa
Epidermolysis Bullosa Simplex - drug therapy
Experiments
Genetic transformation
Human Genetics
Humans
Keratin
Letter to the Editor
Male
Medical research
Medicine
Medicine & Public Health
Molecular Structure
Mutation
Patient outcomes
Pediatric research
Pediatrics
Pharmacokinetics
Pharmacology/Toxicology
Rare diseases
Rare skin diseases
Skin
Topical application
Treatment outcome
Urine
Wounding
Keratin
Topical application
Epidermolysis bullosa
Pharmacokinetics
Diacerein
Online AccessGet full text
ISSN1750-1172
1750-1172
DOI10.1186/s13023-018-0940-1

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Abstract Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes after minor mechanical friction. In a clinical phase 2/3 trial, diacerein has recently been shown to significantly reduce blister numbers upon topical application. In this study we addressed basic pharmacokinetic parameters of locally applied diacerein in vitro and in vivo. Ex vivo experiments using a Franz diffusion cell confirmed the uptake and bio-transformation of diacerein to rhein in a porcine skin model. Rhein, the active metabolite of diacerein, was also detected in both urine and serum samples of two EBS-gen sev patients who topically applied a 1% diacerein ointment over a period of 4 weeks. The accumulated systemic levels of rhein in EBS-gen sev patients were lower than reported levels after oral application. These preliminary findings point towards the uptake and prolonged persistance of diacerein / rhein within the intended target organ - the skin. Further, they imply an acceptable safety profile at the systemic level. Trial registration DRKS. DRKS00005412 . Registered 6 November 2013.
AbstractList Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes after minor mechanical friction. In a clinical phase 2/3 trial, diacerein has recently been shown to significantly reduce blister numbers upon topical application. In this study we addressed basic pharmacokinetic parameters of locally applied diacerein in vitro and in vivo.
Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes after minor mechanical friction. In a clinical phase 2/3 trial, diacerein has recently been shown to significantly reduce blister numbers upon topical application. In this study we addressed basic pharmacokinetic parameters of locally applied diacerein in vitro and in vivo. Ex vivo experiments using a Franz diffusion cell confirmed the uptake and bio-transformation of diacerein to rhein in a porcine skin model. Rhein, the active metabolite of diacerein, was also detected in both urine and serum samples of two EBS-gen sev patients who topically applied a 1% diacerein ointment over a period of 4 weeks. The accumulated systemic levels of rhein in EBS-gen sev patients were lower than reported levels after oral application. These preliminary findings point towards the uptake and prolonged persistance of diacerein / rhein within the intended target organ - the skin. Further, they imply an acceptable safety profile at the systemic level. Trial registration DRKS. DRKS00005412 . Registered 6 November 2013.
Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes after minor mechanical friction. In a clinical phase 2/3 trial, diacerein has recently been shown to significantly reduce blister numbers upon topical application. In this study we addressed basic pharmacokinetic parameters of locally applied diacerein in vitro and in vivo. Ex vivo experiments using a Franz diffusion cell confirmed the uptake and bio-transformation of diacerein to rhein in a porcine skin model. Rhein, the active metabolite of diacerein, was also detected in both urine and serum samples of two EBS-gen sev patients who topically applied a 1% diacerein ointment over a period of 4 weeks. The accumulated systemic levels of rhein in EBS-gen sev patients were lower than reported levels after oral application. These preliminary findings point towards the uptake and prolonged persistance of diacerein / rhein within the intended target organ - the skin. Further, they imply an acceptable safety profile at the systemic level. Trial registration DRKS. DRKS00005412. Registered 6 November 2013. Keywords: Epidermolysis bullosa, Diacerein, Topical application, Pharmacokinetics, Keratin
Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes after minor mechanical friction. In a clinical phase 2/3 trial, diacerein has recently been shown to significantly reduce blister numbers upon topical application. In this study we addressed basic pharmacokinetic parameters of locally applied diacerein in vitro and in vivo. Ex vivo experiments using a Franz diffusion cell confirmed the uptake and bio-transformation of diacerein to rhein in a porcine skin model. Rhein, the active metabolite of diacerein, was also detected in both urine and serum samples of two EBS-gen sev patients who topically applied a 1% diacerein ointment over a period of 4 weeks. The accumulated systemic levels of rhein in EBS-gen sev patients were lower than reported levels after oral application. These preliminary findings point towards the uptake and prolonged persistance of diacerein / rhein within the intended target organ - the skin. Further, they imply an acceptable safety profile at the systemic level. Trial registration DRKS. DRKS00005412. Registered 6 November 2013.
Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes after minor mechanical friction. In a clinical phase 2/3 trial, diacerein has recently been shown to significantly reduce blister numbers upon topical application. In this study we addressed basic pharmacokinetic parameters of locally applied diacerein in vitro and in vivo. Ex vivo experiments using a Franz diffusion cell confirmed the uptake and bio-transformation of diacerein to rhein in a porcine skin model. Rhein, the active metabolite of diacerein, was also detected in both urine and serum samples of two EBS-gen sev patients who topically applied a 1% diacerein ointment over a period of 4 weeks. The accumulated systemic levels of rhein in EBS-gen sev patients were lower than reported levels after oral application. These preliminary findings point towards the uptake and prolonged persistance of diacerein / rhein within the intended target organ - the skin. Further, they imply an acceptable safety profile at the systemic level. TRIAL REGISTRATION: DRKS. DRKS00005412 . Registered 6 November 2013.Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes after minor mechanical friction. In a clinical phase 2/3 trial, diacerein has recently been shown to significantly reduce blister numbers upon topical application. In this study we addressed basic pharmacokinetic parameters of locally applied diacerein in vitro and in vivo. Ex vivo experiments using a Franz diffusion cell confirmed the uptake and bio-transformation of diacerein to rhein in a porcine skin model. Rhein, the active metabolite of diacerein, was also detected in both urine and serum samples of two EBS-gen sev patients who topically applied a 1% diacerein ointment over a period of 4 weeks. The accumulated systemic levels of rhein in EBS-gen sev patients were lower than reported levels after oral application. These preliminary findings point towards the uptake and prolonged persistance of diacerein / rhein within the intended target organ - the skin. Further, they imply an acceptable safety profile at the systemic level. TRIAL REGISTRATION: DRKS. DRKS00005412 . Registered 6 November 2013.
Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes after minor mechanical friction. In a clinical phase 2/3 trial, diacerein has recently been shown to significantly reduce blister numbers upon topical application. In this study we addressed basic pharmacokinetic parameters of locally applied diacerein in vitro and in vivo. Ex vivo experiments using a Franz diffusion cell confirmed the uptake and bio-transformation of diacerein to rhein in a porcine skin model. Rhein, the active metabolite of diacerein, was also detected in both urine and serum samples of two EBS-gen sev patients who topically applied a 1% diacerein ointment over a period of 4 weeks. The accumulated systemic levels of rhein in EBS-gen sev patients were lower than reported levels after oral application. These preliminary findings point towards the uptake and prolonged persistance of diacerein / rhein within the intended target organ - the skin. Further, they imply an acceptable safety profile at the systemic level. TRIAL REGISTRATION: DRKS. DRKS00005412 . Registered 6 November 2013.
Abstract Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in blistering and wounding of the skin and mucous membranes after minor mechanical friction. In a clinical phase 2/3 trial, diacerein has recently been shown to significantly reduce blister numbers upon topical application. In this study we addressed basic pharmacokinetic parameters of locally applied diacerein in vitro and in vivo. Ex vivo experiments using a Franz diffusion cell confirmed the uptake and bio-transformation of diacerein to rhein in a porcine skin model. Rhein, the active metabolite of diacerein, was also detected in both urine and serum samples of two EBS-gen sev patients who topically applied a 1% diacerein ointment over a period of 4 weeks. The accumulated systemic levels of rhein in EBS-gen sev patients were lower than reported levels after oral application. These preliminary findings point towards the uptake and prolonged persistance of diacerein / rhein within the intended target organ - the skin. Further, they imply an acceptable safety profile at the systemic level. Trial registration DRKS. DRKS00005412. Registered 6 November 2013.
ArticleNumber 193
Audience Academic
Author Ablinger, Michael
Lagler, Florian B.
Lettner, Thomas
Wolkersdorfer, Martin
Wimmer, Monika
Zauner, Roland
Felder, Thomas K.
Hofbauer, Peter
Bauer, Johann W.
Diem, Anja
Wally, Verena
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Issue 1
Keywords Keratin
Topical application
Epidermolysis bullosa
Pharmacokinetics
Diacerein
Language English
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SSID ssj0045308
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Snippet Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in...
Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting in...
Abstract Generalized severe epidermolysis bullosa simplex (EBS-gen sev) is caused by mutations within either the KRT5 or KRT14 gene, phenotypically resulting...
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StartPage 193
SubjectTerms Administration, Topical
Analysis
Anthraquinones - administration & dosage
Anthraquinones - chemistry
Anthraquinones - pharmacokinetics
Anthraquinones - therapeutic use
Anti-Inflammatory Agents - administration & dosage
Anti-Inflammatory Agents - chemistry
Anti-Inflammatory Agents - pharmacokinetics
Anti-Inflammatory Agents - therapeutic use
Clinical trials
Diacerein
Drug therapy
Epidermolysis bullosa
Epidermolysis Bullosa Simplex - drug therapy
Experiments
Genetic transformation
Human Genetics
Humans
Keratin
Letter to the Editor
Male
Medical research
Medicine
Medicine & Public Health
Molecular Structure
Mutation
Patient outcomes
Pediatric research
Pediatrics
Pharmacokinetics
Pharmacology/Toxicology
Rare diseases
Rare skin diseases
Skin
Topical application
Treatment outcome
Urine
Wounding
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Title Basal pharmacokinetic parameters of topically applied diacerein in pediatric patients with generalized severe epidermolysis bullosa simplex
URI https://link.springer.com/article/10.1186/s13023-018-0940-1
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Volume 13
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