Author(s)
Stander S1, Lim-Watson M2, Berger T3, Silverberg J4, Weisshaar E5, Friedman A6, Szepietowski JC7, Kim B8, Pariser D9, Ekanayake-Bohlig S10, Metz M11, Papandrikopoulou A12, Jiang GL2, Paolini J2, Martin D2, Yosipovitch G13
1University Hospital Münster, Münster, Germany, 2Kiniksa Pharmaceuticals, Lexington, MA, USA, 3University of California at San Francisco, San Francisco, CA, USA, 4George Washington University School of Medicine and Health Sciences, Washington, DC, USA, 5Ruprecht-Karls Hospital Heidelberg, Heidelberg, Germany, 6The George Washington University School of Medicine & Health Sciences, Washington, DC, USA, 7Wroclaw Medical University, Wroclaw, Poland, 8Washington University School of Medicine in St. Louis, St. Louis, MO, USA, 9Pariser Dermatology Specialists, Norfolk, VA, USA, 10MensingDerma Research GmbH, Hamburg, Germany, 11Charité - Universitätsmedizin Berlin, Berlin, Germany, 12Kiniksa Pharmaceuticals, Corp, Lexington, MA, USA, 13University of Miami, Miami, FL, USA
OBJECTIVES: To evaluate treatment utilization and patient burden in a multi-country, observational LOngitudinal Trial to Understand Symptomatology in Prurigo Nodularis (LOTUS-PN). METHODS: Prospective, multicenter, longitudinal study conducted in 11 sites in the United States and Europe to investigate PN pathophysiology. Patient demographics, medical conditions, treatments, and 6-months´s follow-up data for PN Intensity/Itch Severity, Patient-Reported Outcomes Measurement Information System (PROMIS 8a), Dermatology Life Quality Index (DLQI), and Pruritus Patient Benefit Index (PBI-P) were analyzed for 54 patients, with a physician-confirmed PN diagnosis with underlying conditions, who completed assessments from March 2017-September 2018. RESULTS: Patients (mean age 54 years [53.8+12.9] at diagnosis) were predominantly female (57%), and white (65%). Underlying medical conditions included depression (20%), hypothyroidism (19%), atopic dermatitis (17%), asthma (15%), allergic rhinitis (11%), and HIV/AIDS (6%). Participants reported treatment regimens of topical corticosteroids (39%), phototherapy (28%), antihistamines (22%), gabapentin (15%), oral and intralesional corticosteroids (9% each) and cyclosporine (7%); 15% had no treatment. At baseline, patients reported a mean score of 6.1+1.2 (numerical rating scale from “0” to “10”) for itch severity over the past 24 hours, and this average rating dropped to 4.0+0.6 by 6 months. For sleep quality, 24% indicated “very poor” and “poor” at baseline with similar responses observed at 6 months. Baseline total DLQI scores declined from 12.0±7.2 (“very large effect”) to 7.8±5.8 (“moderate effect”) at 6 months. PBI-P items most important to respondents (>70% of participants responding “very important” both at baseline and 6-month follow-up), included: 1) itch free, 2) healed of all skin defects, 3) clear diagnosis, and therapy and 4) confidence in the therapy. CONCLUSIONS: This observational longitudinal cohort demonstrated the substantial burden, undertreatment and unmet needs in PN patients despite conventional therapy. Targeted, safe and effective treatments that resolve PN symptoms and improve quality of life are needed for this difficult-to-treat disease.
Conference/Value in Health Info
2021-05, ISPOR 2021, Montreal, Canada
Value in Health, Volume 24, Issue 5, S1 (May 2021)
Code
PRO69
Topic
Health Service Delivery & Process of Care, Patient-Centered Research
Topic Subcategory
Disease Management, Patient-reported Outcomes & Quality of Life Outcomes, Treatment Patterns and Guidelines
Disease
Rare and Orphan Diseases, Sensory System Disorders
