Background: GH treatment in severe GH deficiency during the transition period is a key matter.
Objective and hypotheses: To assess the outcome of 30 severe GH deficient (GHD) patients after a structured transition program, in a French Universitary Hospital between 1988 and 2014.
Method: 30 patients treated with GH until final height for congenital deficiency: 16/30 (12 ectopic posterior pituitary), brain tumors: 11/30 (5 Rathkes cleft cyst) and radiotherapy-induced GHD for leukemia: 3/30, persistent and severe GHD after pituitary reassessment (10 isolated and 20 multiple pituitary hormone deficiencies). Mean age at transfer was 17.4 (±1.9)y. Data in the last pediatric visit and 1, 2, 3 and 5 years later: GH doses, IGF1, and metabolic and bone status.
Results: Median follow-up was 3 years. 17/25 (68%) were still treated at one year, 14/19 (73.6%) at 2 years, 14/16 (87.5%) at 3 years and 8/11 (72.7%) at 5 years. Only 5/30(16.6%) dropped out of the follow-up after one year, and 2/25 (23.3%) after 2 years. IGF1 levels in the target (0.5±2DS) only for 5/25 (31.2%) at 1 year, decreasing to 2/19 (14.2%) at 2 years and 1/14(l7.1%) at 3 years, with average GH doses of 1.2, 0.9 and 0.8 mg/d. Decrease of BMI for men (24.8 kg/m2 vs 23.6 kg/m2), increase for women (26 kg/m2 vs 29.2 kg/m2). Bone status, glycaemia and lipids levels were stable after 5 years.
Conclusion: Most of GHD patients are still treated 5 years after transition, underlying an active collaboration between pediatric and adult teams. However, the insufficient initial IGF1 levels and its quick decrease afterwards suggest the need to emphasize patient education, through a motivational approach.
10 - 12 Sep 2016
European Society for Paediatric Endocrinology