Growth and Growth Hormone Use in Osteogenesis Imperfecta - Osteogenesis Imperfecta - page 249

nutritional and respiratory issues, it would also be
important to evaluate the effects on body mass index,
body composition and pulmonary function status with
rGH treatment, which is known to improve these indices
in other conditions.
In addition, it is well documented that there is a decel-
eration of linear growth in early childhood in patients
with OI. There have been few rGH treatment trials in
which therapy has been started at a young age. Many
FDA-approved indications for rGH involve its use from
an early age with better growth outcomes the earlier
the rGH is started, and based on the pattern of growth
observed in OI types III and IV, especially during the
early childhood years, 18,138 the use of rGH earlier may
help with catch-up growth at a critical period of time
and lead ultimately to better final adult heights. This is
substantiated by the improvement of linear growth in OI
with bisphosphonates when started young. 138
At this time, longer, properly controlled rGH treat-
ment trials are clearly needed in order to assess whether
rGH truly has effects on final adult height in patients
with OI, as well as whether it can improve secondary
outcomes such as body composition, body mass index,
muscle mass and pulmonary status. Most treatment tri-
als have been less than 2 years thus far, and therefore
long-term effects on height and other parameters are
unknown. Combined trials with bisphosphonates in
those children with the most severe growth failure seem
the most appropriate based on the effects of rGH alone
on bone. Careful studies over the past 25 years have
revealed rGH to be a relatively safe hormonal treatment
with the potential of multi-system benefit. However, fur-
ther studies on long-term effects not only in OI, but in all
rGH treatment studies, are necessary in order to assess
safety and efficacy fully.

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