The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosis

Table 3 Clinical domains, endpoints, and MCID thresholds for the GTR

Domain	Endpoint	Established MCID^a
Pharmacodynamic	Serum oligosaccharides	Last serum oligosaccharide value ≤ 4 µmol/L
Functional	3MSCT	Improvement ≥ 7 steps/min [14]
	6MWT	Improvement ≥ 30 m [16]
	FVC%	Improvement ≥ 10% [15]
Quality of life	CHAQ DI	Improvement ≤ –0.130 [17]
Quality of life	CHAQ Pain VAS	Improvement ≤ –0.246 [17]

3MSCT 3-min stair-climb test, 6MWT 6-min walk test, CHAQ DI Child Health Assessment Questionnaire Disability Index, CHAQ Pain VAS Child Health Assessment Questionnaire Pain Visual Analog Scale, FVC% forced volume capacity, percentage of predicted, GTR Global Treatment Response, MCID minimal clinically important difference, MPS IV A mucopolysaccharidosis type IV A
^aMCID were derived from proxy diseases (3MSCT from MPS IV A clinical trials; 6MWT and FVC% from Pompe; CHAQ DI and CHAQ Pain VAS from juvenile arthritis)

ISSN: 1750-1172