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

Table 6 Psychometric validation: test–retest intraclass correlations of the ctGD1-PROM (n = 23)

Item	Observations	ICC	95% confidence interval
GD Education/job	22	0.509	0.118	0.763
GD Friends	23	0.682	0.384	0.851
GD Intimate	22	0.613	0.264	0.820
GD Hobbies leisure	23	0.757	0.508	0.889
GD Emotional burden	22	0.626	0.297	0.824
GD General-specific med	23	0.567	0.204	0.791
GD Concerns Gaucher	23	0.625	0.303	0.821
GD Current med. concerns	22	0.530	0.141	0.775
GD Dependent	22	0.500	0.107	0.757
GD Abdomen	22	0.974	0.939	0.989
GD Fatigued	22	0.816	0.606	0.919
GD Physical weakness	22	0.900	0.775	0.957
GD Bone pain	22	0.798	0.575	0.911
GD Depressed	22	0.482	0.075	0.749
GD Worried	22	0.512	0.123	0.764
GD Future	22	0.843	0.598	0.937
GD Satisfied	23	0.399	0.014	0.693
Average		0.893	0.750	0.954

ctGD1-PROM clinical trials (17-item) Gaucher disease type 1-specific patient-reported outcome measure questionnaire, GD Gaucher disease, ICC intraclass correlation coefficient

ISSN: 1750-1172