Transcript Morquio A: Endurance (PPT)

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
Response to treatments
McDonald et al, J Pediatr Rehabil Med, 2010; Hendriksz et al, J Inherit Metab Dis, 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)
Image courtesy of Elizabeth Wright
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)
Harmatz et al, Mol Genet Metab, 2013; Harmatz et al, Pediatrics, 2005; McDonald et al, J Pediatr Rehabil Med, 2010;
Hendriksz et al, J Inherit Metab Dis, 2012
Endurance is impaired in Morquio A
Mean 6MWT
220
35
218
Steps/min
205
200
193.1
195
30
24.1
25
210
m
31.6
30
212.6
215
Mean 3MSCT
20
15
10
190
5
185
0
180
Age ≤ 18 years
n=248
Age > 18 years
n=68
All
n=316
Age ≤ 18 years
n=215
Age > 18 years
n=59
Mean 6MWT = 212.6 ±152.2 m (n = 316 subjects)
Mean 3MSCT = 30.0 ±24.0 stairs (n= 274 subjects)
MorCAP baseline data
Harmatz et al, Mol Genet Metab, 2013
All
n=274
Regular assessments are recommended
for optimal patient quality of life
At
diagnosis
Annually
As clinically
indicated
Evaluation by physiotherapist
X
X
X
6 MWT (if physically and developmentally able)
X
X
X
3 MSCT (if physically and developmentally able)
X
X
X
Assessment
Hendriksz et al, J Inherit Metab Dis, 2012