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

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

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	6-point verbal response scale
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	5-point verbal response scale
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

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

ISSN: 1750-1172