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Impact of the primary aetiology upon the clinical outcome of adults with childhood-onset GH deficiency

Charlotte Hoybye, Peter Jönsson¹, John P Monson², Maria Kotowska-Häggström^1,3, Václav Hána⁴, Mitchell Geffner⁵ and Roger Abs⁶

Department of Endocrinology, Metabolism and Diabetology, Karolinska University Hospital, Solna, SE-171 76 Stockholm, Sweden
¹ KIGS/KIMS/ACROSTUDY Medical Outcomes, Pfizer Endocrine Care, Sollentuna, Sweden,
² Centre for Clinical Endocrinology, William Harvey Research Institute, St Bartholomew's Hospital, Queen Mary University of London, London, UK
³ Department of Pharmacy, Uppsala University, Uppsala, Sweden
⁴ Third Department of Internal Medicine, Charles University, Prague, Czech Republic
⁵ The Saban Research Institute of Childrens Hospital Los Angeles, Los Angeles, California, USA
⁶ University Hospital, Antwerp, Belgium

(Correspondence should be addressed to C Hoybye Email: charlotte.hoybye{at}karolinska.se)

Objective: The impact of the aetiology of childhood-onset GH deficiency (CO-GHD) on the clinical presentation during adulthood and the response to GH replacement has been poorly defined. Our study aims to characterize CO-GHD in adults due to different aetiologies and evaluate the effect of 2 years of GH replacement therapy.

Design and methods: Data from 353 adults with CO-GHD from Pfizer International Metabolic Database KIMS were retrospectively grouped according to GHD aetiology: non-organic disorder (n=147), organic pituitary disease (n=159), and brain tumour (n=47). Extent of pituitary dysfunction, IGF-I concentration, lipid concentrations and quality-of-life (QoL) were assessed at baseline and after 2 years of GH replacement.

Results: GHD was diagnosed at a later age in the organic pituitary group than in the other groups, resulting in a shorter duration of GH treatment during childhood. However, the final height was greater in the organic pituitary group. Panhypopituitarism was most common in the non-organic disorder and in the organic pituitary groups, while isolated GHD was more prominent in the brain tumour group. Serum IGF-I levels were the lowest in the non-organic group. QoL was the poorest in the brain tumour group. Lipid profile and QoL improved significantly during GH replacement.

Conclusion: The adverse consequences of CO-GHD in adulthood vary between aetiologies, but improve similarly with GH treatment. It is, therefore, important to consider retesting all patients with CO-GHD in early adulthood and, if persistent severe GHD is confirmed, recommence GH replacement.