OBJECTIVE:

To estimate the cost-effectiveness of growth hormone (GH) therapy for idiopathic short stature (ISS).

DESIGN:

Cost-effectiveness analysis.

SETTING:

Decision model.

PATIENTS:

A cohort of 10-year-old prepubertal boys with ISS treated with GH.

INTERVENTIONS:

Comparison of children treated for 5 years with GH therapy vs children receiving no intervention.

MAIN OUTCOME MEASURES:

Incremental cost per child, incremental growth per child, and incremental cost per inch of final height gain.

RESULTS:

The estimated incremental cost-effectiveness ratio of GH therapy for ISS in the base case analysis compared with no therapy was 52,634 dollars per inch (per 2.54 cm), or 99,959 dollars 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. Probabilistic sensitivity analyses showed that growth variability in response to GH had the greatest impact on the cost-effectiveness of GH therapy.

CONCLUSIONS:

Targeted treatment of children with ISS with 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.