Topical cholesterol/lovastatin for the treatment of porokeratosis: A pathogenesis-directed therapy - 11/12/19
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Abstract |
Background |
Porokeratosis is associated with mevalonate pathway gene mutations. Therapeutic options are few and often limited in efficacy. We hypothesized that topical therapy that aims to replenish cholesterol, an essential mevalonate pathway end-product, and block the accumulation of mevalonate pathway toxic metabolites could alleviate porokeratosis.
Objective |
To study the efficacy of topical cholesterol/lovastatin in different variants of porokeratosis.
Methods |
We enrolled a series of 5 porokeratosis patients,1 with disseminated superficial actinic porokeratosis, 2 with porokeratosis palmaris et plantaris disseminata, and 2 with linear porokeratosis. Patients were genotyped before initiation of therapy. Patients then applied topical cholesterol/lovastatin twice daily to a unilaterally defined treatment area for up to 3 months. The response was evaluated and patients photographed at every visit.
Results |
Three patients had MVD mutations, and 2 patients had PMVK mutations. Treatment with topical cholesterol/lovastatin (but not cholesterol alone) resulted in near complete clearance of disseminated superficial actinic porokeratosis lesions after 4 weeks of therapy and moderate improvement of porokeratosis palmaris et plantaris disseminata lesions and linear porokeratosis lesions. There were no adverse events.
Limitations |
Case series design with a small number of patients.
Conclusion |
Topical cholesterol/lovastatin is an effective and well-tolerated therapy for porokeratosis that underscores the utility of a pathogenesis-based therapy that replaces deficient end products and prevents accumulation of potentially toxic precursors.
Le texte complet de cet article est disponible en PDF.Key words : cholesterol, disseminated superficial actinic porokeratosis, genetics, genetic skin diseases, linear porokeratosis, medical dermatology, mevalonate pathway, pediatric dermatology, porokeratosis, statins, therapy, topical therapy
Abbreviations used : CHILD, DSAP, LP, PPPD
Plan
Funding sources: Supported in part by the National Institutes of Health (R01 AR071491 to Dr Choate) and the Yale Center for Mendelian Genomics (U54 HG006504). Dr Atzmony was supported by Davidoff Foundation. |
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Conflicts of interest: None disclosed. |
Vol 82 - N° 1
P. 123-131 - janvier 2020 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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