Clinical characteristics and symptom progression of dermatomyositis subtypes: A retrospective analysis of a prospective database.

BACKGROUND: Disease characteristics of classic dermatomyositis (DM) and clinically amyopathic DM (CADM) are well established, but there exists limited knowledge on the disease progression of these subtypes. OBJECTIVE: The objective of this study was to longitudinally track and characterize classic DM and CADM patients who experience changes in disease presentation. METHODS: We conducted a retrospective review of prospectively collected data on 269 DM patients from a longitudinal database. RESULTS: A total of 51% of the patients had classic DM and 49% had CADM. Forty percent of the classic DM patients became postmyopathic (PmDM). Median Cutaneous Dermatomyositis Disease Area and Severity Index activity (CDASI-A) score was lower in PmDM patients than in classic DM patients (13.0 vs 16.0), but 45% of the PmDM patients had CDASI-A scores > 14. Five percent of the CADM patients developed muscle involvement. Compared with CADM patients, those who developed muscle symptoms had milder skin disease before subtype conversion (median CDASI-A 12.0 vs 16.0) and at subtype conversion (median CDASI-A 9.0 vs 16.0). LIMITATIONS: This was a retrospective study conducted at a single tertiary-care dermatology clinic. CONCLUSIONS: Forty percent of the classic DM patients became PmDM. The majority continue with muscle disease, and many continue to have moderate/severe skin disease. CADM has a low risk of progressing to muscle disease, with the extent of skin disease as a potential predictive factor.

A fatal case of hemophagocytic lymphohistiocytosis due to neonatal lupus erythematosus.

Neonatal lupus erythematosus (NLE) is an autoimmune disease caused by the passive transfer of autoantibodies from mother to child during pregnancy. A rare complication of NLE is hemophagocytic lymphohistiocytosis (HLH), a potentially life-threatening hyperinflammatory state more commonly associated with other rheumatologic disorders. Herein, we describe a fatal case of NLE-associated HLH.

Emerging immunotherapeutic strategies for cutaneous lupus erythematosus: an overview of recent phase 2 and 3 clinical trials.

INTRODUCTION: Cutaneous lupus erythematosus (CLE) is an autoimmune disease that is clinically heterogenous and may occur with or without the presence of systemic lupus erythematosus (SLE). While existing on a spectrum, CLE and SLE present differences in their underlying pathogenesis and therapeutic responses. No new therapies have been approved in recent decades by the U.S. Food and Drug Administration for CLE, although frequently refractory to conventional therapies. There is an unmet need to develop effective drugs for CLE as it significantly impacts patients' quality of life and may leave irreversible disfiguring damage. AREAS COVERED: This review provides an update on the latest phase 2 and 3 clinical trials performed in CLE or SLE using skin-specific outcome measures. Emergent therapies are presented alongside their mechanism of action as recent translational studies have permitted identification of critical targets among immune cells and/or pathways involved in CLE. EXPERT OPINION: While the recent literature has few trials for CLE, drugs targeting type I interferon, its downstream signaling and plasmacytoid dendritic cells have shown promising results. Further research is required to develop long-awaited effective therapies, and this review highlights the importance of implementing trials dedicated to CLE to fill the current gap in CLE therapeutics.

Periorbital hypopigmentation and telangiectasias: Clues to diagnosing neonatal lupus in skin of color.

Neonatal lupus erythematosus (NLE) is an autoimmune disease characterized by a periorbital erythematous rash. Although post-inflammatory hypopigmentation and telangiectasias are known possible sequelae, these features may be particularly noticeable in skin of color. Herein, we describe two infants with skin of color in whom periorbital hypopigmentation and telangiectasias were clues to the diagnosis of NLE.

Validation of Cutaneous Dermatomyositis Disease Area and Severity Index Activity Score and Other Efficacy Outcomes as Measures of Skin Disease in Dermatomyositis in the Lenabasum Phase 3 Trial.

In the past decade, there have been six industry-sponsored phase 3 trials in adult patients with dermatomyositis (DM), primarily focusing on improving muscle weakness. However, skin disease is a cardinal manifestation of DM. This study evaluated the sensitivity of Cutaneous Dermatomyositis Disease Area and Severity Index Activity score, Cutaneous Dermatomyositis Activity Investigator Global Assessment, Total Improvement Score, and other outcome measures used in DM clinical trials to detect improvement in DM skin disease activity. Data analyzed from the lenabasum phase 3 trial in DM showed that improvement in Cutaneous Dermatomyositis Disease Area and Severity Index Activity score increased proportionately with the degree of patient- or physician-reported improvement in skin disease, consistently measuring improvement when clinically meaningful improvement was reported at weeks 16-52. In contrast, Cutaneous Dermatomyositis Activity Investigator Global Assessment measured little change from baseline with reported no improvement in skin disease but also a similar change from baseline with slight improvement. No Skindex-29+3 subscale performed well at reflecting increasing degrees of improvement in skin disease. Extramuscular Global Assessment and Total Improvement Score generally showed increasing levels of improvement as the degree of patient- and physician-reported improvement in skin disease increased, but these are composite measures and are not specific to improvement in DM skin disease. To measure clinically meaningful improvement in skin disease in a DM trial, Cutaneous Dermatomyositis Disease Area and Severity Index Activity score is the more sensitive outcome measure across time points.

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.

Interhospital Transfer of Intracerebral Hemorrhage Patients Undergoing Minimally Invasive Surgery: The Experience of a New York City Hospital System.

OBJECTIVE: The impact of interhospital transfer (IHT) on outcomes of patients with intracerebral hemorrhage (ICH) has not been well studied. We seek to describe the protocolized IHT and systems of care approach of a New York City hospital system, where ICH patients undergoing minimally invasive surgery (MIS) are transferred to a dedicated ICH center. METHODS: We retrospectively reviewed 100 consecutively admitted patients with spontaneous ICH. We gathered information on demographics, variables related to IHT, clinical and radiographic characteristics, and details about the clinical course and outpatient follow-up. We grouped patients into 2 cohorts: those admitted through IHT and those directly admitted through the emergency department. Primary outcome was good functional outcome at 6 months, defined as modified Rankin Scale score 0-3. RESULTS: Of 100 patients, 89 underwent IHT and 11 were directly admitted. On multivariable analysis, there were no significant differences in 6-month functional outcome between the 2 cohorts. All transfers were managed by a system-wide transfer center and 24/7 hotline for neuroemergencies. An ICH-specific IHT protocol was followed, in which a neurointensivist provided recommendations for stabilizing patients for transfer. Average transfer time was 199.7 minutes and average distance travelled was 13.6 kilometers. CONCLUSIONS: In our hospital system, a centralized approach to ICH management and a dedicated ICH center increased access to specialist services, including MIS. Most patients undergoing MIS were transferred from outside hospitals, which highlights the need for additional studies and descriptions of experiences to further elucidate the impact of and best protocols for the IHT of ICH patients.