The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases

Table 1 Pearson correlation with CHAQ/HAQ for individual and O’Brien rank score of three clinical endpoints

Assessment	Pearson correlation with CHAQ/HAQ^a, N = 76	p value
6MWT	− 0.24	0.032
FVC	− 0.19	0.091
Shoulder ROM	− 0.23	0.051
O’Brien rank score (6MWT, FVC, Shoulder ROM)	− 0.50	< 0.001

6MWT six minute walk test, FVC forced vital capacity, ROM range of motion
^aPatient assessments pooled from treatment and placebo arms. Higher CHAQ/HAQ scores indicate greater impairment

ISSN: 1750-1172