Dataset Information

The systemic management of cutaneous dermatomyositis: Results of a stepwise strategy.

ABSTRACT: Treatment of dermatomyositis (DM) is often achieved with a stepwise algorithm. However, the literature lacks quality evidence to support the use of this therapeutic strategy. The result of a stepwise therapeutic strategy in the management of skin-only DM is presented to better understand the clinical outcomes and allow for future studies. A cohort of 102 patients with DM, 41 of whom had skin-only disease, were seen between July 2009 and April 2013 at a referral-based connective tissue disease clinic. The Cutaneous Dermatomyositis Disease Area and Severity Index was used to prospectively assess disease severity and the outcomes in 41 adult patients with skin-only DM were analyzed. Of the 41 patients with skin-only DM, 23 patients (56.1%) received antimalarial medications alone and 18 patients (43.9%) received second- or third-line agents. Ten patients (24.4%) remained at the first level of the treatment algorithm and received only hydroxychloroquine. Prednisone was included in the treatment regimen for 11 patients with skin-only disease (26.8%). The results show that management of cutaneous DM often requires second-line agents because antimalarial medications alone are insufficient to treat most patients with skin-only disease.

SUBMITTER: Anyanwu CO

PROVIDER: S-EPMC5715209 | biostudies-literature | 2017 Dec

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

The systemic management of cutaneous dermatomyositis: Results of a stepwise strategy.

Anyanwu C O CO Chansky P B PB Feng R R Carr K K Okawa J J Werth V P VP

International journal of women's dermatology 20170613 4

Treatment of dermatomyositis (DM) is often achieved with a stepwise algorithm. However, the literature lacks quality evidence to support the use of this therapeutic strategy. The result of a stepwise therapeutic strategy in the management of skin-only DM is presented to better understand the clinical outcomes and allow for future studies. A cohort of 102 patients with DM, 41 of whom had skin-only disease, were seen between July 2009 and April 2013 at a referral-based connective tissue disease cl ...[more]

PMID: 29234712

Similar Datasets

Project description:ImportanceCutaneous disease in dermatomyositis has no standardized treatment approach and so presents a challenging task for patients and clinicians.ObjectiveTo study the efficacy and safety of apremilast as an add-on therapy in patients with recalcitrant cutaneous dermatomyositis.Design, setting, and participantsThis phase 2a, open-label, single-arm nonrandomized controlled trial was conducted at a single center from June 2018 to June 2021. Participants were 8 patients with recalcitrant cutaneous dermatomyositis, defined by a cutaneous disease activity severity index (CDASI) score greater than 5 despite treatment with steroids, steroid-sparing agents, or both. Data were analyzed from June 2018 to June 2021.InterventionsApremilast 30 mg orally twice daily was added to ongoing treatment regimens.Main outcomes and measuresThe primary outcome was the overall response rate (ORR) at 3 months. Key secondary outcomes were the safety and toxicity of apremilast and the durability of response at 6 months. The CDASI, muscle score, dermatology life quality index (DLQI), and depression assessments were performed at baseline and regularly until month 7. Skin biopsies were performed at baseline and 3 months after apremilast (defined as 3 months into active apremilast therapy) and tested for gene expression profiling and immunohistochemical stains. Adverse events were assessed using the Common Terminology Criteria for Adverse Events version 5.0.ResultsAmong 8 patients with recalcitrant cutaneous dermatomyositis (all women; mean [SD] age, 54 [15.9] years), a response was found at 3 months after apremilast among 7 patients (ORR, 87.5%). The mean (SD) decrease in CDASI was 12.9 (6.3) points at 3 months (P < .001). Apremilast was well tolerated, with no grade 3 or higher adverse events. Sequencing of RNA was performed on skin biopsies taken from 7 patients at baseline and at 3 months after therapy. Appropriate negative (ie, no primary antibody) and positive (ie, tonsil and spleen) controls were stained in parallel with each set of slides studied. Of 39 076 expressed genes, there were 195 whose expression changed 2-fold or more at P < .01 (123 downregulated and 72 upregulated genes). Gene set enrichment analysis identified 13 pathways in which apremilast was associated with downregulated expression, notably signal transducers and activators of transcription 1 (STAT1), STAT3, interleukin 4 (IL-4), IL-6, IL-12, IL-23, interferon γ (IFNγ), and tumor necrosis factor α (TNFα) pathways. In immunohistochemical staining, there was a mean (SD) decrease in phosphorylation levels STAT1 (22.3% [28.3%] positive cells) and STAT3 (13.4% [11.6%] positive cells) at the protein level, a downstream signaling pathway for the downregulated cytokines.Conclusions and relevanceThese findings suggest that apremilast was a safe and efficacious add-on treatment in recalcitrant dermatomyositis, with an overall response rate of 87.5% and associations with downregulation of multiple inflammatory pathways.Trial registrationClinicalTrials.gov Identifier: NCT03529955.

Dataset Information

The systemic management of cutaneous dermatomyositis: Results of a stepwise strategy.

Publications

The systemic management of cutaneous dermatomyositis: Results of a stepwise strategy.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets