GH continues to be produced after cessation of growth regulating the metabolic process and the functional integrity of many tissues in adult life. Adults who lack GH develop a characteristic clinical picture of metabolic and body compositional abnormalities. The clinical manifestations of increased cardiovascular risk, impaired physical function, psychological well being and diminished quality of life (QoL) are reversed by GH replacement therapy. There is strong evidence from over 25 years of global experience that GH replacement therapy is safe with no increased risk of pituitary tumour recurrence or of de novo malignancies.
Patients with organic pituitary disease should be investigated for GH deficiency with an intention to treat using validated diagnostic tests for GH deficiency(1). A blood IGF-I measurement is not a sensitive diagnostic test but is valuable for guiding the dose of GH replacement which is higher in women. The benefits on physical and psychological function occur over several months in parallel with an improvement in QoL.
PBAC twice rejected (in 2001 and 2011) the subsidizing of GH for adults with GH deficiency on the basis of uncertain cost-effectiveness of treatment. Following a joint application by the ESA and APEG supported by GH Pharmas, the PBAC has recommended the listing of GH as a pharmaceutical benefit for patients with severe GH deficiency and a poor QoL(2). QoL is quantified by an Assessment of Growth Hormone Deficiency in Adults (AGHDA) Questionaire. The scores determines eligibility for initiation and continuation of GH replacement therapy. The impending Section 100 listing is expected to benefit about 1000 patients in Australia.