ESPE Abstracts (2019) 92 P1-226

Response to Growth Hormone in Very Young Children (® International Outcome Study and ANSWER Program

Tilman R Rohrer1, Bradley Miller2, Vlady Ostrow3, Alberto Pietropoli4, Michel Polak5, Judith Ross6

1Department of Pediatric Endocrinology, University Children's Hospital, Saarland University Medical Center, Homburg, Germany. 2Pediatric Endocrinology, University of Minnesota Masonic Children's Hospital, Minneapolis, USA. 3Novo Nordisk Inc., Plainsboro, USA. 4Global Medical Affairs, Novo Nordisk Health Care AG, Zurich, Switzerland. 5Hôpital Universitaire Necker Enfants Malades, Assistance Publique-Hôpitaux de Paris Université Paris Descartes, INSERM U1016, Institut IMAGINE, Centre de Référence des Maladies Endocriniennes Rares de la Croissance et du Dévelopement, Paris, France. 6Department of Pediatrics, Thomas Jefferson University, Philadelphia, USA

Objectives: Limited information is available on how very young children with growth hormone deficiency (GHD) respond to growth hormone (GH) replacement. We compared response to 1 year of GH therapy in children aged <2 years and prepubertal children aged ≥2 years.

Methods: The two non-interventional, multicentre studies, NordiNet® International Outcome Study (IOS) (NCT00960128) and the ANSWER Program (NCT01009905), evaluated the effectiveness of Norditropin® (somatropin; Novo Nordisk A/S) as prescribed in a real-world clinical setting. Height standard deviation scores (SDS) and target height SDS were based on national standards (NordiNet® IOS) or the Centers for Disease Control standard.

Results: Baseline characteristics [% or mean (SD)] for patients <2 years (n=506) vs. ≥2 years (n=9810): male: 59.7% vs. 69.3%; age at treatment start (years): 1.11 (0.57) vs. 9.09 (3.40); bone age/chronological age: 0.86 (0.99) vs. 0.81 (0.18); height SDS: –2.55 (1.77) vs. –2.39 (1.01); target height SDS: –0.20 (0.98) vs. –0.57 (1.02); GH peak (ng/mL): 4.07 (4.78) vs. 5.45 (4.68); IGF–I SDS: –1.34 (1.07) vs. –1.71 (1.59); birth weight (g): 3033.3 (680.7) vs. 3032.3 (697.1); birth length (cm): 48.3 (3.8) vs. 48.9 (3.9); GH dose (mg/kg/day): 0.035 (0.014) vs. 0.037 (0.011). Isolated GHD was less common in patients aged <2 years (54.6%) vs. ≥2 years (91.4%). Numbers of additional pituitary deficiencies were as follows (<2 years vs. ≥2 years): one: 24.11% vs. 5.92%; two: 12.85% vs. 1.34%; ≥3: 8.50% vs. 1.32%. After 1 year's treatment, all growth-related measurements improved in both groups: [mean (SD)] values: height SDS: –1.17 (1.50) vs. –1.73 (1.00); IGF–I SDS: 0.76 (1.42) vs. 0.41 (1.61); height SDS change from baseline: 1.44 (1.22) vs. 0.67 (0.49). After 1 year mean GH dose (mg/kg/day) was 0.035 (0.011) vs. 0.038 (0.012); median dose [P10; P90] was 0.034 [0.023; 0.049] vs. 0.036 [0.026; 0.053]. Adverse reactions (considered possibly related to treatment) were reported in 1.78% vs. 1.83%, and serious adverse reactions in 0.79% vs. 0.45% (<2 years vs. ≥2 years, respectively).

Conclusion: Children aged <2 years who required GH replacement had more severe GHD, as indicated by lower mean GH peak levels in stimulation tests versus prepubertal children aged ≥2 years. The prevalence of multiple pituitary deficiency was higher in younger children; therefore, clinicians must test them for additional pituitary deficiencies. Severe GHDs should be fully investigated and treated early, which is demonstrated by greater height gain after 1 year's treatment.

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