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Better growth outcomes in GH-deficient children treated younger than 2 years of age
Tilman Robert Rohrer1, Primož Kotnik2,3, Bradley S Miller4
1Division of Pediatric Endocrinology, Department of Pediatrics and Neonatology, University Children's Hospital, Saarland University Medical Center, Homburg, Germany.
Insights
Starting growth hormone (GH) treatment for GH deficiency (GHD) before age 2 improves height outcomes but increases serious adverse events. Early diagnosis and monitoring are crucial for young GHD patients.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Clinical Outcomes Research
Background:
- Limited data exist on growth hormone (GH) treatment response in very young children with GH deficiency (GHD).
- This study compares clinical outcomes in GHD children based on age at GH treatment initiation (<2 years vs. ≥2 years).
Purpose of the Study:
- To evaluate the efficacy and safety of GH treatment in pediatric GHD patients based on age at treatment start.
- To compare height standard deviation score (SDS) changes and adverse events between early (<2 years) and later (≥2 years) treatment initiation.
Main Methods:
- Pooled data from two observational studies (NordiNet® IOS and ANSWER Program) of pediatric patients receiving Norditropin®.
- Patients with GHD, remaining pre-pubertal after 1 year, were grouped by age at treatment start.
- Primary outcome: change in height SDS at 1 and 10 years; secondary outcomes: adverse drug reactions (ADRs) and serious adverse events (SAEs).
Main Results:
- Children initiating GH treatment <2 years (n=507) showed greater height SDS improvement at 1 year (1.4 vs. 0.75) and 10 years (3.2 vs. 2.2) compared to those ≥2 years (n=7,486).
- Serious adverse events (SAEs) were more frequent in the <2 years group (3.3%) versus the ≥2 years group (0.67%).
Conclusions:
- Initiating GH treatment before age 2 in children with GHD leads to superior height outcomes.
- Early GH treatment initiation is associated with a higher incidence of SAEs, underscoring the need for careful monitoring.
Background:
Limited data are available on the growth response to growth hormone (GH) treatment in very young children with GH deficiency (GHD). In the present analysis, we compared clinical outcomes after GH treatment in children with GHD aged <2 and ≥2 years at the start of GH treatment.
Methods:
We analysed pooled data from two observational studies of paediatric patients who received Norditropin® treatment: NordiNet® IOS (NCT00960128) and the ANSWER Program (NCT01009905). Patients with GHD, who remained pre-pubertal after 1 year of treatment, were grouped by age at treatment start (<2 years; ≥2 years). The primary effectiveness outcome was change in height standard deviation score (SDS) after 1 and 10 years. We also investigated the frequency of non-serious adverse drug reactions (ADRs), serious ADRs and serious adverse events (SAEs).
Results:
In total, 507 and 7,486 children initiated treatment at <2 and ≥2 years of age, respectively. Height SDS (mean change (SD) from baseline) improved after 1 year of treatment in both groups and was greater in children initiating treatment at <2 years than in those initiating treatment at ≥2 years (1.4 (1.2) and 0.75 (0.5), respectively); these findings were sustained after 10 years of treatment (3.2 (1.7) and 2.2 (1.3), respectively). SAEs were more frequent in children initiating treatment at <2 years vs ≥ 2 years (3.3 vs 0.67%, respectively).
Conclusions:
Children aged <2 years at GH treatment initiation had better height outcomes, but more SAEs, after 1 and 10 years of GH treatment compared to children starting GH at age ≥2 years.
Trial Registration:
NordiNet® IOS, ClinicalTrials.gov NCT00960128; ANSWER Program, ClinicalTrials.gov NCT01009905.
Plain Language Summary:
Data from two large studies showed that children with growth hormone deficiency (GHD) who began treatment with Norditropin® under 2 years of age had better growth than those first treated at or above 2 years of age, but also had more side effects. This highlights the value of early diagnosis, treatment and close monitoring of children with GHD.
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