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

Table 2 MDRI domains, MID thresholds, and study population from the Phase 3 Study of Laronidase

Domain	MID threshold	Study population with baseline deficiency in domain
6MWT	± 54 m	Approximately 50% < 350 m
FVC	± 11%	FVC < 80% was entry criterion; 100% qualify
Shoulder ROM	± 20°	Approximately 50% had ROM < 90°
AHI	± 10 events per hour	Approximately 50% AHI > 10
Visual acuity	± 2 lines	8 Patients (approximately 20%) > 20/200

MID minimally important difference, 6MWT six minute walk test. FVC forced vital capacity, ROM range of motion, AHI apnea hypopnea index

ISSN: 1750-1172