Background: NordiNet® IOS (NCT00960128), a non-interventional study, collects long-term effectiveness and safety data of GH (Norditropin®, Novo Nordisk) treatment in everyday clinical practice.
Objective and hypotheses: Identify paediatric patients more likely to experience a second adverse event (AE).
Method: Based on diagnosis at GH treatment start and associated risk for mortality, patients were classified into: low-risk (idiopathic GH deficiency, idiopathic short stature, small for gestational age), intermediate-risk (multiple pituitary hormone deficiency, clinically defined syndromes) or high-risk (malignancy, craniopharyngioma, chronic renal failure) groups. Incidence rates (IRs)/1000 patient-years of exposure (PYE), for adverse drug reactions (ADRs), serious ADRs (SADRs) and serious AEs (SAEs) were calculated. AEs were classified using System Organ Class/MedDRA Preferred Terms.
Results: Data were analysed for 16 359 patients (mean (S.D.) age 8.7 (3.9) years; treatment duration 4.1 (3.0) years; GH dose 34.2 (10.0) μg/kg per day; low-risk, 62.1%; intermediate-risk, 32.6%; high-risk, 5.3%). 372 patients reported 434 AEs: one AE, 89.8% (n=334); two AEs, 8.1% (n=30); ≥3 AEs 2.2% (n=8). IRs/1000 PYE were: ADRs, 3.78; SAEs, 3.84; SADRs 1.11. IRs were significantly higher in the intermediate- and high- versus low-risk group (P<0.05). Post-AE, GH dose was unchanged in 54.5 and 37.9% of the low- and high-risk groups, respectively; treatment discontinuation was more common in the high- (39.4%) vs low-risk group (17.4%). Proportions of patients with one AE were: low-risk, 92.6% (n=138); intermediate-risk, 88.0% (n=147); high-risk, 87.5% (n=49). Higher proportions of patients in the intermediate 10.2% (n=17) and high- 8.9% (n=5) vs low-risk group, 5.4% (n=8) reported two AEs; ≥3 AEs occurred in <4% in any risk group.
Conclusion: These results are consistent with previous reports from NordiNet IOS and reconfirm the overall favourable safety profile of GH treatment. Patients in the intermediate- and high-risk groups were more likely to have a second AE than those in the low-risk group. A similar low rate for ≥3 AEs was reported across all risk groups.
10 - 12 Sep 2016
European Society for Paediatric Endocrinology