The physical and emotional impact of cutaneous dermatomyositis: a qualitative study.

Dermatomyositis (DM) is a rare autoimmune disease characterized by distinctive cutaneous manifestations, often accompanied by muscle inflammation and interstitial lung disease. DM has a significant impact on quality of life (QoL) in patients, due to the physical and emotional symptoms caused by their disease. Despite this known emotional impact, there is no published literature capturing how adults with DM feel about their disease, from their perspective. We seek to better understand how cutaneous DM impacts patients in their daily lives. Seventeen patients with cutaneous DM presenting to an autoimmune dermatology clinic were interviewed about how their cutaneous findings have impacted their life. Patients were asked three questions: what troubles you the most about your cutaneous/skin DM, how much bother does the skin DM cause, and what about your skin disease most impacts your daily life. Responses were scribed by a second researcher. Themes and subthemes from the interviews were generated. Of 17 patients, 17 (100%) were female, 7 (41%) had amyopathic DM, median age was 65 years (IQR 48-68), and median Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) activity score was 12 (IQR 6-17.5) at the time of interview. Seven themes emerged. Most reported physical signs included: itchiness (n = 10, 59%) and physical pain/uncomfortableness (n = 6, 35%). Our study demonstrates that patients are burdened by the physical, emotional and social aspects of their disease, and struggle to manage it. This better understanding of how patients feel will help guide management and allow clinicians to address patient needs. Additionally, these insights may help in the development of QoL tools that address the concerns of patients with severe and chronic skin conditions, like DM.

Safety, Tolerability, and Activity of ALXN1830 Targeting the Neonatal Fc Receptor in Chronic Pemphigus.

Pemphigus is a debilitating IgG-mediated autoimmune disease requiring better tolerated, more targeted, and rapid onset therapies. ALXN1830 is a humanized IgG4 antibody that blocks neonatal Fc receptor interactions with IgG. A multicenter, open-label safety and tolerability phase 1b/2 trial (NCT03075904) was conducted in North America from July 2017 to January 2019 and included patients aged ≥18 years with a confirmed diagnosis of pemphigus (vulgaris or foliaceus) and active disease. Dosing included five weekly intravenous doses of ALXN1830 (10 mg/kg) and follow-up through day 112 (study termination). Pharmacokinetics, pharmacodynamics, safety, and efficacy, as evaluated by determining the change in the median pemphigus disease area index, were determined. In this pilot study of eight patients, five weekly infusions of ALXN1830 produced a rapid improvement in the pemphigus disease area index score within 14 days of the first dose. Pemphigus disease area index improvement increased further together with reductions in IgG, circulating immune complexes of IgG, and anti-desmoglein antibodies without affecting albumin, IgM, IgA, or C-reactive protein levels. ALXN1830 was well-tolerated, with headache as the most common adverse event. This study reveals the importance of neonatal Fc receptor in the biology of pemphigus and the potential for use of ALXN1830 in pemphigus treatment.