Table 5.

Psychometric validation: internal consistency reliability statistics for the ctGD1-PROM

Item	Item-test correlation	Item-rest correlation	Inter-item correlation	Cronbach’s alpha
GD Education/job	0.658	0.605	0.434	0.925
GD Friends	0.783	0.745	0.422	0.921
GD Intimate	0.576	0.514	0.442	0.927
GD Hobbies leisure	0.741	0.696	0.426	0.922
GD Emotional burden	0.848	0.821	0.415	0.919
GD General-specific med	0.356	0.276	0.464	0.933
GD Concerns Gaucher	0.208	0.118	0.476	0.936
GD Current med. concerns	0.364	0.286	0.463	0.932
GD Dependent	0.594	0.533	0.439	0.926
GD Abdomen	0.616	0.553	0.438	0.926
GD Fatigued	0.802	0.767	0.420	0.921
GD Physical weakness	0.868	0.844	0.413	0.919
GD Bone pain	0.868	0.843	0.413	0.919
GD Depressed	0.834	0.805	0.417	0.920
GD Worried	0.870	0.846	0.413	0.919
GD Future	0.792	0.757	0.421	0.921
GD Satisfied	0.826	0.795	0.417	0.920
Overall			0.431	0.928

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