Connective Tissue Diseases

The Morphea Activity Measure (MAM) scoring tool is reliable and valid for measuring pediatric morphea disease activity, according to a study published online Feb. 8 in
 
 JAMA Dermatology
 
 .




 Maria Teresa García-Romero, M.D., M.P.H., from the National Institute of Pediatrics in Mexico City, and colleagues developed and validated the MAM scoring tool to evaluate morphea disease activity for use in clinical and research settings.




 Fourteen survey respondents who were experts in morphea were involved in the development phase. A final tool was developed after two rounds using a Delphi consensus method, which included 10 items that were indicative of morphea activity. The researchers conducted a validation phase among 14 patients, two dermatologists, and six pediatric dermatologists. Good interrater and intrarater agreement was seen for MAM total scores, with intraclass correlation coefficients of 0.844 and 0.856, respectively. Moderately strong correlations were seen between the MAM and the modified Localized Scleroderma Severity Index and Physician Global Assessment of Activity. In qualitative interviews, evaluators agreed that the tool was easy to use, measured morphea disease activity at a single time point, and should be responsive to changes in disease activity over time.




 "The MAM has potential as an outcome measure for use in both clinical and research settings," the authors write. "Further study, including a larger validation study and longitudinal testing to measure responsiveness to change, is warranted."




 Several authors disclosed financial ties to the biopharmaceutical and medical technology industries.




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Moderately strong correlations seen between MAM and modified Localized Scleroderma Severity Index, Physician Global Assessment of Activity

Morphea Activity Measure Reliable, Valid for Disease Activity

Lenabasum is well tolerated for the treatment of amyopathic dermatomyositis, according to a phase 2 study published online Oct. 1 in the
 
 Journal of Investigative Dermatology
 
 .




 Victoria P. Werth, M.D., from the University of Pennsylvania Perelman School of Medicine in Philadelphia, and colleagues evaluated the safety and efficacy of lenabasum in patients with refractory cutaneous dermatomyositis. The analysis included 22 participants randomly assigned to lenabasum (11 participants) or placebo (11 participants) for a regimen of lenabasum daily for 28 days (all participants) and then either lenabasum or placebo twice per day for 56 days.




 The researchers observed no serious or severe adverse events related to lenabasum, and no participants discontinued the study. In the lenabasum group, the adjusted least-squares mean for Cutaneous Dermatomyositis Disease Area and Severity Index activity decreased more, with the difference reaching significance on day 113.




 "Current treatments, which are limited and can involve frequent and costly intravenous infusions, are not always effective in treating its dermatological manifestations and can produce side effects," Werth said in a statement. "Our research showed that the cannabinoid receptor type 2 agonist lenabasum works to improve the skin of patients with amyopathic dermatomyositis in a short period of time. The identification of a well tolerated and effective oral medication greatly expands treatment options for patients with refractory skin disease."




 The study was partially funded by Corbus Pharmaceuticals, the developer of lenabasum, and several authors disclosed financial ties to the company.




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Results of phase 2 study suggest clinical benefit and favorable safety profile, warranting further study of lenabasum for DM

Lenabasum Studied for Refractory Cutaneous Dermatomyositis

For patients with cutaneous lupus erythematosus (CLE), normal-appearing skin is a type I interferon-rich, prelesional environment, according to a study published in the April 27 issue of
 
 Science Translational Medicine
 
 .




 Allison C. Billi, M.D., Ph.D., from the University of Michigan in Ann Arbor, and colleagues analyzed normal-appearing skin, lesional skin, and circulating immune cells from lupus patients via integrated single-cell RNA sequencing and spatial RNA sequencing to examine CLE immunopathogenesis.




 The researchers found that in patients with lupus, normal-appearing skin represented a type I interferon-rich, prelesional environment. Interferon-induced gene expression changes were seen in all major skin cell types and altered predicted cell-cell communication networks. In addition, lupus-enriched CD16 dendritic cells underwent interferon education in the skin, yielding proinflammatory phenotypes.




 "These interferon-educated immune cells seem to be priming many different cell types in the skin to overreact to stimuli with excessive inflammatory responses, manifesting as disfiguring skin lesions," Billi said in a statement. "We don't yet know all of the stimuli that can tip the balance and precipitate these rashes, but ultraviolet light certainly appears to be one of them."




 Several authors disclosed financial ties to the biopharmaceutical industry.




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Normal-appearing skin is type I interferon-rich environment; interferon-induced gene expression changes seen in all major skin type cells

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