Monocentric study of 112 consecutive patients with childhood onset GH deficiency around and after transition

Abstract

ObjectivesOur aim was to analyze a large cohort of childhood onset GH deficiency (CO-GHD) adults from a unique adult center, in order to analyze their clinical management and to study the metabolic and bone status in relation to GHD and to the other pituitary deficits, and to evaluate these parameters during the long-term follow-up.Design and methodsObservational retrospective cohort study on 112 consecutive CO-GHD adults transferred to our unit from 1st January 1994 to 1st March 2012. Evaluation of GHD in pediatrics and after transition was conducted following consensus guidelines. Data recorded from pediatric and adult files were GH doses, pituitary magnetic resonance imaging and function, and metabolic and bone status.ResultsMost patients presented with severe CO-GHD (64%) associated with other pituitary deficits (66%). CO-GHD was acquired in 56%, congenital in 33%, and idiopathic in 11% cases. Most patients (83%) stopped GH before transfer, at 16.3 years (median), despite persistence of GHD. Median age at transfer was 19.4 years. After transfer, GHD persisted in 101 patients and four of the 11 resolutive GHD were non idiopathic. IGF1 level was <−2 SDS in 70% of treated patients at transfer and in 34% of them after 3 years of treatment. Follow-up showed improvement in lipid profile and bone mineral density in severely persistent GHD patients under GH therapy. In multivariate analysis, the associated pituitary deficits seemed stronger determinant factors of metabolic and bone status than GHD.ConclusionsThis study raises concern about discontinuation of GH replacement therapy in pediatrics in severely persistent GHD patients and about the often insufficient dose of GH in the treatment of adult patients.

An audit of the management of childhood-onset growth hormone deficiency during young adulthood in Scotland

“…However, this prolonged period off rhGH may be associated with detrimental effects on somatic bone and body composition development during transition [ 23 , 24 ], with recommendations for prompt resumption of rhGH in individuals with clinical evidence of persistent GHD [ 25 ]. Furthermore, a longer interval off rhGH may increase the risk of being lost to follow up in these already vulnerable patients and continued follow up around this time is essential [ 26 ]. We recommend that in patients who are under the care of paediatric services, the evaluation of GHD in transition should be undertaken by the paediatric clinic, ideally in the context of a joint transition service to improve the follow up and smooth transfer of adolescents with chronic endocrine conditions to the adult services as previously suggested [ 27 ].…”

Managing Transition in Patients Treated with Growth Hormone

“…This is also important for children diagnosed as GH deficient at older age, even when already into the transition period ( 28 ). A significant proportion of patients who remain GH deficient at the end of pediatric treatment do not continue to receive GH during or after transition ( 29 ). Patients need a full explanation of the continued effects of GH on lipids, body composition, and quality of life.…”

Understanding Growth Hormone Secretion and Short Stature

“…Childhood growth disorders often have the following characteristics: short stature with a normal growth rate (growth and skeletal maturation rate within normal parameters, short family/genetic stature), short stature with normal growth rate and evidence of lower previous rate (delay in growth with retardation of bone maturation), abnormal growth rate combined with or without short stature (systemic disease or hormone deficiency), and growth acceleration. Is required for the following cases [15]: • Unexplained hypoglycemia, jaundice, or microphallus; • Early signs of potential hypopituitarism, including GHD; • Unexplained abnormally slow or fast growth; • Growth rate consistently below 10th or above 95th percentile than that expected for age; • Extremely short stature; • Stature lower than the 1st percentile for age (<−2.25 SD); • Pattern and prognosis of growth divergent from that of the family; • Estimation of stature (current stature percentile corrected for BA) differs significantly from PAH; • Abnormal body proportions.…”