Patient‐Reported Outcomes and Provider Perceptions of Systemic Mastocytosis: Results From the PRISM Study
Year: 2025
Type: article
Study design: Observational Study
Abstract: BACKGROUND: Systemic mastocytosis (SM) is a clonal mast cell disease primarily driven by the KIT D816V mutation and often characterised by unpredictable and debilitating symptoms. The Perceptions Realities and Insights on Systemic Mastocytosis (PRISM) survey queried patient and provider perceptions of SM in Europe. METHODS: PRISM (funded by Blueprint Medicines Corporation) was composed of two independent surveys: a 119-item patient survey on diagnosis, symptom burden, quality of life (QoL) and work impact; and a 103-item healthcare provider (HCP) survey on approaches to SM diagnosis and management. RESULTS: Data were evaluated from 540 patient respondents from Italy (n = 136), France (n = 112), the UK (n = 101), Germany (n = 83), Spain (n = 64), Austria (n = 26) and Switzerland (n = 18). Patients reported a high symptom burden and reduced physical functioning and mental health due to SM, despite taking, on average, seven medications. A majority of patients reported that SM impacted their ability to work (58.9%). A total of 618 HCP responses were collected from Italy (n = 203), Germany (n = 123), the UK (n = 110), Austria (n = 63), France (n = 56), Switzerland (n = 44) and Spain (n = 19). Various HCP types reported treating patients with SM; the majority were haematologists/oncologists (n = 167) and general practitioners (n = 178). HCPs perceived that SM impacted their patients' QoL, with 54% reporting SM affected patients' lives 'quite a bit'. The most frequently reported treatment goals were improving QoL and survival. CONCLUSIONS: PRISM is the largest international and first European survey to evaluate the burden of SM from both patient and HCP perspectives. Patients and HCPs perceived a high symptom burden, reduced QoL and reduced ability to work due to SM.
Source: Clinical & Experimental Allergy
DOI: 10.1111/cea.70101
Authors: Massimo Triggiani, Jessica Hobart, Iván Alvarez‐Twose, Cristina Bulai Livideanu, Franziska Ruëff, Stéphane Barete, Patrizia Bonadonna, Laurence Bouillet, Nauman M. Butt, Mark Drummond, Mar Guilarte, Karin Hartmann, Amélie Beaux, Celeste C. Finnerty, Nicole Hegmann, Patrizia Marcis, Eugenia Ribada, Waltraud Schinhofen, Gabriela Coletti, Andrew Dugdale, Cristian Perez, Dakota Powell, Teresa Green, Erin Sullivan, Deepti H. Radia, Ruben Mesa
Institutions: University of Salerno, Centre Hospitalier Universitaire de Toulouse, Ludwig-Maximilians-Universität München, Sorbonne Université, Pitié-Salpêtrière Hospital, Azienda Ospedaliera Universitaria Integrata Verona, Centre Hospitalier Universitaire de Grenoble, Beatson West of Scotland Cancer Centre, Vall d'Hebron Institut de Recerca, Vall d'Hebron Hospital Universitari, University of Basel, University Hospital of Basel, Mast Cell Disease Society, The University of Texas Medical Branch at Galveston, Blueprint Medicines (United States), Guy's and St Thomas' NHS Foundation Trust, Wake Forest Baptist Comprehensive Cancer Center
Language: English
Topic: Mast cells and histamine
Subfield: Immunology
Field: Immunology and Microbiology
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $4,760
OpenAlex ID: W4411722175