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Table 2 Overview of the rmGD1-PROM and ctGD1-PROM questions and structure

From: Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials

Part No. of questions Topics Scale
ctGD1-PROM and rmGD1-PROM
 1A 4 Restriction of activities, education, and work 6-point verbal response scale
  1 Concern about emotional burden
 1B 2 Context of GD concerns relating to general health and medication 5-point verbal response scale
  1 Context of GD concerns relating to other medical concerns
 2 1 Dependence on others 0–10 numeric rating scale
  6 Presence/severity of symptoms
  1 Satisfaction with treatment
  1 Concern about the future
rmGD1-PROM only
  6 Concern about comorbidities, disease burden, and cost 6-point verbal response scale
  1 Context of GD concerns relating to other medical concerns 5-point verbal response scale
  1. ctGD1-PROM clinical trials (17-item) Gaucher disease type 1-specific patient-reported outcome measure questionnaire, GD Gaucher disease, rmGD1-PROM routine monitoring (24-item) GD1-specific patient-reported outcome measure questionnaire