what-when-how
In Depth Tutorials and Information
the self-care and social function domains. This study
also indicates that only the type of OI and total muscle
strength could be considered as significant predictors
for the level of ambulation. Furthermore, bodily weight
was significantly lower in the group that progressed in
level of ambulation, whereas children with a decline
in level of ambulation had significantly higher bodily
weight, speculating that increased weight influenced
negatively the anti-gravity activities. The authors con-
cluded that functional ability improved significantly
over time; however, it did not reach normative values
(particularly in types III and IV) probably due to a pla-
teau phase in functional ability.
10
The study of Sakkers
et al. reported on the skeletal effects and functional
outcome with olpadronate in 34 children with OI type
I, III and IV in a 2-year randomized placebo-controlled
study. Primary outcomes were incident fractures of
long bones and changes in bone mineral content (BMC),
bone mineral density (BMD) and functional outcome.
The results indicated that olpadronate treatment was
associated with a 31% reduction in relative risk of frac-
ture of long bones whereas there were no detectable
effects on functional outcome, anthropometrics or verte-
bral height.
35
The lack of beneficial effects on functional
outcome contrasted with previously reported uncon-
trolled studies with pamidronate infusions. Vertebral
growth and increases in seated height, longitudinal
growth, arm span and body weight were seen in both
groups, but catch-up growth as a result of olpadronate
treatment was not observed. Longer follow-up might be
necessary for demonstrating possible catch-up growth.
The study of Land et al. evaluated the functional
abilities and the level of ambulation during pamidro-
nate therapy in children with moderate to severe OI.
Functional abilities, ambulation and grip force were
assessed in 59 patients (mean age: 6.1 years) during
3 years of pamidronate treatment. Functional skills
(mobility and self-care) were both assessed by using the
PEDI. Ambulation level was assessed by using the mod-
ified Bleck score. Although mobility and self-care scores
increased during the study period (+43% and +30%,
respectively), no normative values were reached. The
average ambulation score changed from 0.8 to 1.9. The
difference in self-care scores did not reach significance.17
17
Recently, Montpetit et al. compared the activities
and participation in the domains of mobility, self-care,
domestic life and social functioning in young adults
according to OI type (
n
= 54, 24 completed files; mean
age (SD): 25.0 (2.6) years).
36
Participants with OI type I
reported full independence, and only a few respon-
dents with OI types IV and V reported some limitations
in mobility and domestic life activities. Young adults
with OI type III had significantly lower activity scores
in aspects of mobility and domestic life and lower levels
of participation in employment, sporting activities and
transportation. Participation in leisure and social inter-
actions were not different across OI types. Young adults
with more severe types of OI had greater activity limita-
tions and participation restrictions.
36
Perceived Competence and Quality of Life
Perceived competence (using the Harter Self-
Perception Profile for Children) has been compared to
range of motion, muscle strength and functional ability
in 40 children with OI (mean age (SD): 12.6 (3.2) years)
who were not treated with medication.
13
This study of
Engelbert et al. reported fair to strong positively per-
ceived competence in OI types I, III and IV, except for
fairly negative perceived competence in the athletic
performance subscale and the romance subscale in OI
type I and OI type III, respectively.
13
No correlations
were found between ROM and athletic performance
and physical appearance, muscle strength and athletic
performance or physical appearance, or the functional
skills, concerning self-care and mobility, with the sub-
scales of the perceived competence. The authors, there-
fore, concluded that although ROM, muscle strength,
and functional and walking ability were related to
the severity of the disease and differed significantly
between the OI types I, III and IV, overall perceived
competence in children with OI was fairly to strongly
positive, without significant differences between the
different types of OI.
13
This latter indicated that clini-
cal characteristics in the body function and structure
domain of the ICF are no prerequisites for perceived
competence and quality of life.
The study of Seikaly et al. measured aspects of quality
of life using total mobility (PEDI), self-care (WEEFIM),
well-being, pain and analgesic scores. All quality of
life markers, except for mobility score, improved in
response to alendronate therapy.
30
Furthermore, Kok
et al. performed a double-blind randomized placebo-
controlled trial to investigate whether oral bisphospho-
nates influence the quality of life in children with OI
during a 2-year follow-up. Thirty-four children with OI,
aged 3-18 years of age, with a restricted level of ambula-
tion, were included. Quality of life was measured using
self-perception profile for children (SPPC) and health-
utility index (HUI). The results of this study indicated
that only slight differences in quality of life in favor of
the bisphosphonate group were found.
28
OUTCOME MEASURES AND UTILIZED
INSTRUMENTS
Based on the ICF-CY model, outcome measures and
utilized instruments should cover all domains of func-
tioning (body structure and function, and activities and
