The Burden of Systemic Mastocytosis in Select European Countries: Evidence from the Prism Patient Survey
Author(s)
Mesa R1, Triggiani M2, Hobart J3, Green T4, Powell D4, Gressier-Sayag C4, Baisley W5, Lahue B5, Rueff F6, Schinhofen W7, Hegmann N8, Livideanu C9, Beaux A10, Marcis P11, Alvarez-Twose I12, Ribada E13, Radia D14, Finnerty C15
1Atrium Health Wake Forest Baptist Comprehensive Cancer Center, Winston-Salem, NC, USA, 2Università degli Studi di Salerno Dipartimento di Medicina, Chirurgia e Odontoiatria “Scuola Medica Salernitana”/DIPMED, Fisciano, Italy, 3The UK Mastocytosis Support Group, London, UK, 4Blueprint Medicines, Cambridge, MA, USA, 5Alkemi, Manchester Center, VT, USA, 6Clinic and Polyclinic for Dermatology and Allergology, Munich, Germany, 7Association Mastozytose e.V., Tönisvorst, Germany, 8Patient Advocacy Group (Selbsthilfe Mastozytose e.V.), Odenthal, Germany, 9CHU de Toulouse - Hôpital Larrey, Toulouse, France, 10Patient Advocacy Group (ASSOMAST), Paris, France, 11Patient Advocacy Group (ASIMAS), Castello d'argile (BO), Italy, 12Hospital Virgen Del Valle, Toledo, Spain, 13Patient Advocacy Group (AEDM), Madrid, Spain, 14Guy's and St. Thomas' NHS Foundation Trust, London, UK, 15The Mastocytosis Society, Sterling, MA, USA
Presentation Documents
OBJECTIVES: Systemic Mastocytosis (SM) is a rare disease associated with anaphylaxis, GI disruptions, fatigue, and other symptoms. One objective of PRISM, a multi-country research program, is to evaluate SM disease burden for patients.
METHODS: Patients reporting an SM diagnosis were eligible to participate in the PRISM Survey. The 119-item online survey queried SM type [advanced (ASM), non-advanced (non-ASM), subtypes, if known], symptoms, healthcare patterns and quality of life. The survey included the 12-item Short-Form Health Survey (SF-12), Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) which generates a Total Symptom Score (TSS, higher=worse, ≥42 indicates severe disease), and the Work Productivity and Activity Impairment Questionnaire (WPAI). This analysis reports PRISM patient survey results from the UK, Germany, and Austria.
RESULTS: Respondents included 210 patients (70% female) from Germany (n=83), UK (n=101), and Austria (n=26). Over half (129/210) reported non-ASM (98 ISM), 35 reported ASM, and 46 reported ‘unknown’ type. Allergy/anaphylaxis symptoms were reported by 11% as ‘most bothersome’ followed by itching (10%) and GI symptoms (7%). Patients reported 2.9 mean current medications (most common non-ASM medications: H1-antihistamines=31%, epinephrine=26%), high symptom severity scores (mean TSS non-ASM=42.6, ASM=52.0, ISM=41.2) and impaired quality of life (SF-12 median PCS=37.6, MCS=40.5). Reported recent anaphylaxis events varied by country (Germany=39%, UK=57%, Austria=69% reported ≥1 anaphylaxis event in the last year) with over half (51%) of all patients reporting an anaphylactic event in last year. SM impacts on productivity increased with symptom severity reported. Overall, 11% reported being unable to work, 34% reduced work hours and among patients reporting severe SM (TSS ≥42), 18% reported being unable to work and 48% reduced work hours.
CONCLUSIONS: Patients enrolled in PRISM reported high rates of anaphylaxis, use of multiple treatments, and negative impacts on quality of life and productivity. Disease-modifying therapies for SM can fill this unmet need.
Conference/Value in Health Info
Value in Health, Volume 26, Issue 11, S2 (December 2023)
Code
PCR136
Topic
Patient-Centered Research, Study Approaches
Topic Subcategory
Health State Utilities, Patient-reported Outcomes & Quality of Life Outcomes, Surveys & Expert Panels
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)