ESPE Abstracts (2018) 89 RFC13.2

Growth Outcomes and Near Adult Height of Children with Congenital GH Deficiency (GHD) due to Abnormal Pituitary Development: Data from a Prospective, Multinational Observational Study

Christopher Childa, Juliane Legerb, Cheri Dealc, Imane Benabbadd, Nan Jiaa & Werner Blume

aLilly Diabetes, Eli Lilly and Company, Indianapolis, Indiana, USA; bPediatric Endocrinology Department & INSERM U 1141, Reference Center for Growth and Development Endocrine Diseases, Robert Debré University Hospital, Paris, France; cUniversity of Montreal and CHU Ste-Justine, Montreal, Canada; dEndocrinology and Diabetes Unit, Eli Lilly and Company, Paris, France; eUniversity Children’s Hospital, University of Giessen, Giessen, Germany

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). 1st-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).

Table 1
Proportion with MPHD52%75%30%8%
Baseline age (years)6.2 (5.6,6.9)4.5 (3.6,5.4)*8.9 (8.4,9.5)*10.2 (10.1,10.3)*
Height SDS−3.0 (−3.2,−2.8)−2.4 (−2.7,−2.1)*−2.8 (−3.0,−2.7)−2.4 (−2.4,−2.4)*
Maximum GH peak (ng/mL)3.9 (3.1,4.8)2.9 (2.3,3.5)5.0 (4.6,5.4)8.2 (8.0,8.4)*
1st-year height velocity SDS4.4 (3.8,4.9)3.4 (2.7,4.0)3.3 (3.0,3.7)*2.5 (2.4,2.5)*
1st-year Δheight SDS1.2 (1.1,1.3)1.1 (0.9,1.2)0.9 (0.8,0.9)*0.6 (0.6,0.6)*
NAH SDS−0.6 (−0.8,−0.3)−0.7 (−1.2,−0.3)−0.6 (−0.9,−0.4)−1.0 (−1.1,−1.0)*
NAH SDS gain2.5 (2.2,2.7)1.9 (1.4,2.4)2.1 (1.9,2.4)1.4 (1.3,1.4)*
GH duration (years)9.2 (8.3,10.0)11.2 (9.7,12.6)7.9 (7.0,8.9)5.6 (5.5,5.7)*
*significant difference to EPPMPHD=multiple pituitary hormone deficiencies

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 1st-year and NAH gain, but age at GH start and treatment duration varied.

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