Nearly all patients with recalcitrant cutaneous dermatomyositis who used apremilast as an add-on therapy showed a response to treatment after 3 months, according to recent research published in JAMA Dermatology.
“The findings of this proof-of-concept nonrandomized controlled trial suggest that apremilast may be a safe and efficacious option in the treatment of recalcitrant [dermatomyositis],” Carole Bitar, MD, of the department of dermatology at Tulane University School of Medicine, New Orleans, and colleagues wrote in their study. “To our knowledge, this is the first study to investigate the role of apremilast in [dermatomyositis], suggesting its mechanism of action in recalcitrant [dermatomyositis]. Larger clinical trials are needed to study apremilast in the treatment of naïve [dermatomyositis] and as a monotherapy.”
Bitar and colleagues conducted a phase 2a open-label trial between 2018 and 2021 involving 8 patients with recalcitrant cutaneous dermatomyositis who received twice-daily oral apremilast at a dose of 30 mg as an add-on therapy to their existing treatment of steroids and/or steroid-sparing agents. Participants included in the study were all women who were mean 54 years old with a cutaneous disease activity severity index (CDASI) higher than 5 points and maintained that score after treatment with steroids and/or steroid-sparing agents. The researchers continued to analyze CDASI, dermatology life quality index, muscle score, and depression up to 7 months, and performed skin biopsies at baseline and 3 months. They evaluated the overall response rate of patients at 3 months as a primary outcome, with secondary outcomes of durability of response at 6 months, safety, and toxicity.
The results at 3 months showed an overall response in 7 of 8 participants (87.5%), which was defined as a CDASI score decrease of 4 points or more within 3 months. The 1 participant who did not achieve this outcome initially had a favorable response within the first month but withdrew from the study after 3 months following disease progression. The researchers also found a significant decrease in mean CDASI score at 3 months from baseline of 12.9 points (P < .001). There were no grade 3 or higher adverse events in the study and the treatment was well tolerated.
Participants were also tested for gene expression profiling and immunohistochemical stains during the study, which identified 13 downregulated pathways after beginning apremilast add-on therapy, “notably JAK/STAT, IL-4, IL-6, IL-12, IL-23, IFN α and γ, and TNF α signaling pathways…These downregulated pathways represent key signaling pathways known to be altered in [dermatomyositis],” the researchers concluded.
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Disclosures: No authors declared financial ties to drugmakers. This study was supported in part by Amgen.
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