Faculty

Mohamad Maghnie

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Impact of GH treatment in terms of efficacy and safety: Lessons from registries

In 1985, recombinant human GH (rhGH) was approved by the US Food and Drug Administration (FDA) and the availability of an unlimited supply of rhGH improved access to therapy for children with GHD. The increased supply of rhGH allowed investigation into treatment of multiple conditions associated with short stature not associated with GHD leading to FDA approval for treatment of children with growth failure associated with chronic renal insufficiency, Turner syndrome, Prader-Willi Syndrome, small for gestational age without adequate catch-up growth, idiopathic short stature, short stature homeobox (SHOX) deficiency, and Noonan syndrome.

Since then GH registries (postmarketing studies), originally mandated by the FDA in 1985, have been an invaluable resource for determining the safety and efficacy of GH treatment. Huge amounts of data were generated and the volume of real-world data overcomes several limitations. A variety of disease states were monitored helping to the understanding and management of pediatric GHD and other conditions treated with rhGH. The data were highly generalizable to the clinical practice setting.

The goals of the presentation are to provide a summary of the main published data with relevant impact on efficacy and safety findings from all patients, and to illustrate the value and utility of long-term registry for future studies. GH resulted to be efficacious with a good safety profile. A state of the art about the results of SAGHE studies will be given.

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