Evidence map›Paper›PMID 31449901›Full record

ArticleJournal of the American Academy of Dermatology2020

Topical cholesterol/lovastatin for the treatment of porokeratosis: A pathogenesis-directed therapy.

Lihi Atzmony, Young H Lim, Claire Hamilton, Jonathan S Leventhal, Annette Wagner, Amy S Paller, Keith A Choate

Abstract read
In one paragraph

Article in Journal of the American Academy of Dermatology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 57 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
57citing papers in PubMed, 2 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

57 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Pooled it
  2. Pooled it
  3. Application of mevalonolactone prevents deterioration of epidermal barrier function by accelerating the lamellar granule lipid transport system.Skin research and technology : official journal of International Society for Bioengineering and the Skin (ISBS) [and] International Society for Digital Imaging of Skin (ISDIS) [and] International Society for Skin Imaging (ISSI) · 2022
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  7. Long-term disease control of disseminated superficial actinic porokeratosis (DSAP) with simvastatin 2%/ cholesterol 2% cream.Journal der Deutschen Dermatologischen Gesellschaft = Journal of the German Society of Dermatology : JDDG · 2026
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  12. Identification of two novelFrontiers in medicine · 2026
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors.

Lihi AtzmonyDepartment of Dermatology, Yale University School of Medicine, New Haven, Connecticut; Department of Genetics, Yale University School of Medicine, New Haven, Connecticut; Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
Young H LimDepartment of Dermatology, Yale University School of Medicine, New Haven, Connecticut; Department of Genetics, Yale University School of Medicine, New Haven, Connecticut; Department of Pathology, Yale University School of Medicine, New Haven, Connecticut.
Claire HamiltonDepartment of Dermatology, Yale University School of Medicine, New Haven, Connecticut.
Jonathan S LeventhalDepartment of Dermatology, Yale University School of Medicine, New Haven, Connecticut.
Annette WagnerDepartment of Dermatology, Northwestern University Feinberg School of Medicine, Chicago, Illinois; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
Amy S PallerDepartment of Dermatology, Northwestern University Feinberg School of Medicine, Chicago, Illinois; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
Keith A ChoateDepartment of Dermatology, Yale University School of Medicine, New Haven, Connecticut; Department of Genetics, Yale University School of Medicine, New Haven, Connecticut; Department of Pathology, Yale University School of Medicine, New Haven, Connecticut. Electronic address: keith.choate@yale.edu.

Funding

Yale Clinical and Translational Science Award (U Component)UL1TR001863 · NCATS · YALE UNIVERSITY · PI John H. Krystal, LUCILA OHNO-MACHADO · 2016 to 2026
$102.9M
MEDICAL SCIENTIST TRAINING PROGRAMT32GM007205 · NIGMS · YALE UNIVERSITY · PI KAZMIERCZAK, BARBARA I · 1985 to 2019
$42.9M
Yale Center for Mendelian DisordersU54HG006504 · NHGRI · YALE UNIVERSITY · PI GERSTEIN, MARK BENDER, GUNEL, MURAT · 2012 to 2015
$11.0M
Training in Investigative DermatologyT32AR007016 · NIAMS · YALE UNIVERSITY · PI KEITH A CHOATE, MICHAEL GIRARDI · 1986 to 2026
$7.8M
Genetics and Pathobiology of Cutaneous Mosaic DisordersR01AR071491 · NIAMS · YALE UNIVERSITY · PI KEITH A CHOATE · 2018 to 2026
$4.2M
NCATS NIH HHS UL1 TR001863NHGRI NIH HHS U54 HG006504NIAMS NIH HHS R01 AR071491NIAMS NIH HHS T32 AR007016NIGMS NIH HHS T32 GM007205
6 · The paper itself

Abstract

backgroundPorokeratosis 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.

objectiveTo study the efficacy of topical cholesterol/lovastatin in different variants of porokeratosis.

methodsWe 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.

resultsThree 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.

conclusionTopical 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.

Indexed as

Administration, CutaneousAdultAnticholesteremic AgentsCarboxy-LyasesChild, PreschoolCholesterolDrug CombinationsGenotypeHumansLovastatinMiddle AgedMutationOintmentsPhosphotransferases (Phosphate Group Acceptor)PorokeratosisYoung AdultAnticholesteremic AgentsCarboxy-LyasesCholesterolDrug CombinationsLovastatinOintmentsphosphomevalonate kinasePhosphotransferases (Phosphate Group Acceptor)pyrophosphomevalonate decarboxylasecholesteroldisseminated superficial actinic porokeratosisgeneticsgenetic skin diseaseslinear porokeratosismedical dermatologymevalonate pathwaypediatric dermatologyporokeratosisstatinstherapytopical therapy

Identifiers

PMID31449901
PMCPMC7039698

What OpenQuestion holds

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Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.