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Joyce M. Lee; Matthew M. Davis; Sarah J. Clark; Timothy P. Hofer; Alex
R. Kemper
Arch Pediatr Adolesc Med. 2006;160(3):263-269.
The
purpose of this study was to estimate the cost-effectiveness of growth hormone
(GH) therapy for idiopathic short stature (ISS). Children with ISS represent the
shortest 1.2 percent of all children based on age and gender. Since they are
otherwise healthy children, there is considerable debate about the costs and
benefits for a 1- to 2-inch height gain from hormone therapy. It is estimated
that at the time of this study there were 400,000 children with ISS, ages 4 to
15, eligible for growth hormone therapy in the US.
The study compared the
height gain by adulthood for children treated for five years with growth hormone
therapy to that of children who received no medical intervention. The children
had all been diagnosed with ISS with no evidence of growth hormone deficiency or
other known medical conditions that might compromise their growth. The study
measured incremental cost per child, incremental growth per child, and
incremental cost per inch of final height gain. The study did not evaluate the
impact of growth hormone therapy on improvements to quality of life, or the
possible long-term effects on salary and future earnings.
The estimated
incremental cost-effectiveness ratio of GH therapy for ISS compared with no
therapy was $52,634 per inch (per 2.54 cm), or $99,959 per child, reflecting an
incremental growth of 1.9 in (4.8 cm). Alternate treatment strategies such as
increased duration of GH treatment and high pubertal dosing of GH did not
substantially improve the cost-effectiveness ratio. Growth variability in
response to GH had the greatest impact on the cost-effectiveness of GH therapy.
The cost of the medication was a major factor in the cost-effectiveness
estimates.
The study concluded that due to variability in the growth
response, targeted growth hormone treatment of children with ISS who have the
greatest potential for growth, appears critical for maximizing
cost-effectiveness of GH treatment. However, the significance of the cost per
inch is difficult to judge until the utility gains associated with height gain
after GH therapy for ISS can be ascertained.
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