Context Recombinant human insulin-like growth factor-I (rhIGF-I) is now available to treat children with short stature resulting from severe primary IGF-I deficiency. This review from the Drug and Therapeutics Committee of the Lawson Wilkins Pediatric Endocrine Society discusses different aspects of rhIGF-I therapy, particularly with regards to potential advantages and disadvantages in comparison to the traditional use of recombinant human growth hormone (rhGH) for treatment of short stature.

Evidence acquisition We used the Entrez-PubMed search engine to conduct a review of publications addressing IGF-I deficiency, the use of rhIGF-I and treatment for short stature.

Evidence synthesis rhIGF-I, as a twice daily subcutaneous injection, is now approved for treatment of short stature in children with severe primary IGF-I deficiency, which may occur as a consequence of mutations in the GH receptor (GHR), defects in the post-GHR signaling pathway, and IGF-I gene defects. It is also approved for children with GH deficiency who develop neutralizing antibodies to GH. RhIGF-I significantly improves growth in these conditions. However, adult height may still be suboptimal, possibly due to lack of direct GH effects. Dosing regimes for rhIGF-I administration are under investigation, as are other indications for use of rhIGF-I.

Conclusion The use of rhIGF-I is justified in conditions approved by the FDA. Until more substantial data become available, the use of rhIGF-I outside FDA recommendations should only be investigational.