| Title: | Better growth outcomes in GH-deficient children treated younger than 2 years of age |
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| Authors: | ID Rohrer, Tilman R. (Author)
ID Kotnik, Primož (Author)
ID Miller, Bradley S (Author)
ID Kelepouris, Nicky (Author)
ID Olsen, Anne Helene (Author)
ID Pietropoli, Alberto (Author)
ID Polak, Michel (Author)
ID Blair, Jo (Author) |
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| Files: |  PDF - Presentation file, download (782,51 KB)
MD5: 11C332415266207CD65BFA8B105A0212
 URL - Source URL, visit https://ec.bioscientifica.com/view/journals/ec/14/9/EC-25-0493.xml
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| Language: | English |
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| Typology: | 1.01 - Original Scientific Article |
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| Organization: |  UKC LJ - Ljubljana University Medical Centre
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| Abstract: | 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. |
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| Keywords: | growth factors, development/foetal nutrition, pituitary, paediatric endocrinology, growth hormone therapy, growth hormone deficiency, multiple pituitary hormone deficiency, clinical outcomes, real-world data |
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| Publication status: | Published |
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| Publication version: | Version of Record |
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| Year of publishing: | 2025 |
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| Number of pages: | str. 1-13 |
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| Numbering: | Vol. 14, iss. 9 |
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| PID: | 20.500.12556/DiRROS-28721  |
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| UDC: | 616.4 |
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| ISSN on article: | 2049-3614 |
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| DOI: | 10.1530/EC-25-0493 |
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| COBISS.SI-ID: | 273675011 |
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| Note: | Nasl. z nasl. zaslona;
Opis vira z dne 31. 3. 2026;
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| Publication date in DiRROS: | 31.03.2026 |
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| Views: | 176 |
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| Downloads: | 112 |
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