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

Area of Science:

• Hematology
• Oncology
• Public Health

Background:

• Systemic mastocytosis (SM) is a rare clonal mast cell disease, primarily driven by the KIT D816V mutation.
• SM is characterized by unpredictable and debilitating symptoms impacting patients' daily lives.

Purpose of the Study:

• To evaluate the perceptions and burden of systemic mastocytosis (SM) in Europe.
• To gather insights from both patients and healthcare providers (HCPs) on SM diagnosis, symptom burden, quality of life (QoL), and work impact.

Main Methods:

• The Perceptions Realities and Insights on Systemic Mastocytosis (PRISM) survey involved 540 patient respondents and 618 HCP respondents across seven European countries.
• Two independent surveys were conducted: a 119-item patient survey and a 103-item HCP survey.

Main Results:

• Patients reported a high symptom burden, reduced physical and mental health, and significant impact on work ability (58.9%).
• Despite multiple medications, patients experienced substantial QoL reduction.
• HCPs also perceived a significant negative impact of SM on patients' QoL, with improving QoL and survival as primary treatment goals.

Conclusions:

• The PRISM survey is the largest international and first European study to assess SM burden from patient and HCP perspectives.
• Both patients and HCPs perceive a high symptom burden, reduced quality of life, and impaired work ability associated with SM.