ESPE Abstracts

Background: Children with structural hypothalamic-pituitary anomalies, e.g. ectopic posterior pituitary (EPP) with/without pituitary stalk interruption syndrome, septo-optic dysplasia (SOD), and isolated anterior pituitary aplasia/hypoplasia (AP/HP) usually have more severe GHD and better auxological outcomes with GH therapy than those with normal hypothalamic-pituitary magnetic resonance imaging findings. However, adult height data is limited.

Objective: To characterize growth and near-adult height (NAH) outcomes in GH-treated patients with EPP, SOD, AP/HP or ‘Other’-GHD, using Genetics and Neuroendocrinology of Short Stature International Study (GeNeSIS) data.

Methods: Patients were grouped by investigator-provided diagnoses: i) EPP (including interrupted pituitary stalk); ii) SOD; iii) AP/HP; and iv) Other-GHD (non-acquired/non-pituitary abnormality associated). Statistical significance was assessed by non-overlap of 95% confidence limits (CI). Height standard deviation score (SDS) was calculated using US age/sex-adjusted CDC data. NAH was defined by ≥1 of closed epiphyses, height velocity <2 cm/year, bone age >14 years (girls)/>16 years (boys).

Results: Patients with EPP were younger at baseline than AP/HP or Other-GHD, but older than SOD, with significantly shorter stature than SOD or Other-GHD (Table 1). 1^st-year height velocity SDS and Δheight SDS were greatest for EPP, significantly different from AP/HP and Other-GHD (Table 1). Height SDS gain from baseline to NAH was greatest for EPP, with significant difference from Other-GHD, albeit after longer GH treatment duration (Table 1).

Conclusion: Patients with structural hypothalamic-pituitary abnormalities had more severe GHD and greater height deficit than those without such abnormalities. They appeared to have better outcomes of GH treatment, with EPP having the best 1^st-year and NAH gain, but age at GH start and treatment duration varied.