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

Table 3 MDRI domains and MID thresholds from the Phase 3 study of Vestronidase Alfa [17]

Domain	MID threshold
6MWT	23 m and ≥ 10% change from baseline [2, 3, 5, 6, 18,19,20,21]
FVC_%pred	5% absolute change or ≥ 10% relative change from baseline in FVC_%pred [2, 6]
Shoulder flexion ROM	≥ 20 degree change of passive shoulder range of motion [2, 3, 5, 22]
Visual acuity	≥ 3 lines (corrected, both eyes) [23,24,25]
BOT-2 fine motor	Fine motor precision: ≥ change of 0.72
BOT-2 fine motor	Manual dexterity: ≥ change of 1.47 [26]
BOT-2 gross motor	Balance: ≥ change of 0.57
BOT-2 gross motor	Running speed and agility: ≥ change of 0.59 [26]

6MWT six minute walk test, FVC_%pred forced vital capacity percentage of predicted, ROM range of motion, BOT-2 Bruininks–Oseretsky Test

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