Patients with dermatomyositis and refractory skin disease on steroids and one steroid-sparing agent.

Dermatomyositis, Adult Type

Patients with refractory cutaneous dermatomyositis will be started on apremilast 30 mg twice a day in addition to a stable dose of their treatment regimen (including steroids and steroid sparing agents).

Apremilast 30mg

Tulane University

Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis

A Phase 2, Open Label Single Arm Study for Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis

Cutaneous dermatomyositis disease area and severity index (CDASI) activity score is a validated tool to measure skin disease activity in dermatomyositis. The overall response rate (ORR) includes partial and complete responses. Complete response is defined by a CDASI activity score of zero. Partial response is defined by a decrease of CDASI activity score of at least 4 points. Calculation is performed as the CDASI activity score at 3 month(s) minus the score at baseline. Missing data will be handled using the last observation carried forward approach (LOCF).
CDASI activity score: Units on a scale from 0-100. Higher scores represent worse outcome.

The proportion of participants experiencing adverse events and serious adverse events will be measured over 7 months period (6 months during the study and 1 month follow up).

The durability of response will be measured using the CDASI activity score at 6 months minus CDASI activity score at 3 months. Complete response durability is defined as zero or minus difference between CDASI activity score at 6 months and CDASI activity score at 3 months. Partial response durability is defined as >4 points difference between CDASI activity score at 6 months and CDASI activity score at 3 months. Missing data will be handled using the last observation carried forward approach (LOCF).
CDASI activity score: Units on a scale from 0-100. Higher scores represent worse outcome.

Complete delayed response is defined by a CDASI activity score of zero at 6 months. Partial delayed response is defined by a decrease of CDASI activity score of at least 4 points at 6 months compared to baseline. Calculation is performed as the CDASI activity score at 6 months minus the score at baseline. Missing data will be handled using the last observation carried forward approach (LOCF).
CDASI activity score: Units on a scale from 0-100. Higher scores represent worse outcome.

Dermatology Life Quality Index (DLQI) is a validated tool to measure quality of life in patients with skin disease. Complete response is defined by a DLQI of zero at 3, and 6 months. Partial response is defined by a decrease of DLQI of at least 5 points at 3, and 6 months compared to baseline. Calculation is performed as the DLQI at 3, and 6 months minus the score at baseline. Missing data will be handled using the last observation carried forward approach (LOCF).
Units : Units on a scale from 0-30, higher scores represent worse outcome.

Calculate the mean change in muscle enzymes value creatine kinase and aldolase at 3 and 6 month(s) compared to baseline in patients with muscle disease.
Units: U/L, higher numbers represent worse outcomes

MMT-8 (Manual Muscle Testing-8) score is a validated tool to assess muscle strength. Calculate the mean change in MMT-8 score at 3 and 6 month(s) compared to baseline in patients with muscle disease.
Units: Units on a scale. Scale goes from 0-150. 150 is perfect strength.

Skin biopsies from lesional skin will be performed before treatment with apremilast and after 3 months of treatment to assess changes in gene expression profiling and immunohistochemistry stain. Gene expression profiling will be analyzed using inferential statistics with a False Discovery Rate (FDR) of < 0.05.

Data and research resources generated by this study will be made available to the research community and to the public at large. This strategy includes, but not limited to, presentations (poster or oral) at local, national, and international meetings; published abstracts, and journal articles (peer reviewed). Contact information will be provided in publications for direct inquiries of researchers. Data generated from sequencing and gene expression profiling will be deposited into the Gene Expression Omnibus (GEO), NCBI, to be accessible by general public. The study protocol will be shared with other researchers on the clinical trial.gov website. No identified personal patient informations will be shared.

aldolase/misselijkheid

Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis

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