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Morquio A: Endurance. Endurance testing assesses functional capacity. Endurance tests measure the efficiency of performing a task and provide assessments of: Functional status of the cardiac, respiratory and musculoskeletal systems Overall disease progression Quality of life
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Endurance testing assesses functional capacity Endurance tests measure the efficiency of performing a task and provide assessments of: • Functional status of the cardiac, respiratory and musculoskeletal systems • Overall disease progression • Quality of life • Response to treatments McDonald et al, J PediatrRehabil Med, 2010; Hendriksz et al, J Inherit MetabDis, 2012
Submaximal intensity tests are sensitive indicators of endurance for MPS patients Recommended endurance tests for assessing submaximal functional capacity in Morquio A patients include: 6 minute walk test (6MWT) • measures how far a person can walk on a hard, flat surface in 6 minutes • a standardized test (American Thoracic Society, 2002) • used to assess endurance in patients with MPS VI (Harmatz et al, 2005) and Morquio A (Harmatz et al, 2013) 3 minute stair climb (3MSCT) • measures how many steps a person can ascend in 3 minutes, using rails and resting as needed • used to assess endurance in patients with MPS VI (Harmatz et al, 2005) and Morquio A (Harmatz et al, 2013) Image courtesy of Elizabeth Wright Harmatz et al, Mol Genet Metab, 2013; Harmatz et al, Pediatrics, 2005; McDonald et al, J PediatrRehabil Med, 2010; Hendriksz et al, J Inherit MetabDis, 2012
Endurance is impaired in Morquio A Mean 6MWT = 212.6 ±152.2 m (n = 316 subjects) Mean 3MSCT = 30.0 ±24.0 stairs (n= 274 subjects) Mean 6MWT Mean 3MSCT n=215 n=59 n=274 n=248 n=68 n=316 Harmatz et al, Mol Genet Metab, 2013 MorCAP baseline data
Regular assessments are recommended for optimal patient quality of life Hendriksz et al, J Inherit MetabDis, 2012