Patient-Reported Outcomes and Provider Perceptions of Systemic Mastocytosis: Results From the PRISM 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.