Background/Aim: To validate prediction models for near final adult height (nFAH) by Ranke et al. [Horm Res Paediatr 2013;79:51-67]. Methods: Height data of 127 (82 male) idiopathic growth hormone (GH)-deficient children, treated with GH until nFAH, were retrieved from the database of the Belgian Society for Pediatric Endocrinology and Diabetology (BESPEED). nFAH was predicted after first-year GH treatment, applying prediction models by Ranke et al. Bland-Altman plots and Clarke error grid analyses were performed to assess clinical significance of the differences between observed and predicted nFAH. Results: In males, the predicted nFAH was higher than the observed nFAH (difference: 0.2 ± 0.7 SD; p < 0.01). In females, there was no significant difference. Bland-Altman analyses showed that the means of the differences between observed and predicted nFAH were close but not equal to zero, with overprediction for smaller heights and underprediction for taller heights. Clarke error grid analysis: in males, 59-61% of the predicted nFAH were within 0.5 SDS and 88% within 1.0 SDS from the observed nFAH; in females, 40-44% of the predicted nFAH were within 0.5 SDS and 76-78% within 1.0 SDS from the observed nFAH. Conclusion: Ranke's models accurately predicted nFAH in females and overpredicted nFAH in males by about 1.5 cm. In most individuals, the predicted nFAH was within 1 SDS of observed nFAH. These models can be of help in giving realistic expectations of adult height.
A sexual difference in serum inhibin B is already present at the end of gestation and changes in inhibin B during the 1st week of life are independent of follicle stimulating hormone changes and perinatal factors in both sexes. Our data suggest that neonatal inhibin B could be used to study whether the newborn has functional testes, i.e. in babies with ambiguous genitalia and/or bilateral cryptorchidism.
Context
Despite having normal GH secretion, individuals with Turner syndrome (TS) have short stature. Treatment with recombinant human GH is recommended for TS girls with short stature.
Objective
Evaluate the effectiveness and safety of Norditropin® (somatropin, Novo Nordisk) with up to 10 years of follow-up in children with TS.
Methods
Secondary analysis of Norditropin data from two non-interventional studies: NordiNet® IOS (NCT00960128) and the ANSWER Program (NCT01009905).
Results
2377 girls with TS were included in the safety analysis set (SAS), with 1513 in the treatment-naïve effectiveness analysis set (EAS). At the start of treatment, 1273 (84%) participants were prepubertal (EAS); mean (SD) age was 8.8 (3.9) years. Mean (SD) dose received at the start of GH treatment was 0.045 (0.011) mg/kg/day (EAS). Mean (SD) baseline IGF-I standard deviation score (SDS) was –0.86 (1.52), and mean (SD) duration of GH treatment (SAS) was 3.8 (2.8) years.
Height SDS (HSDS) increased throughout follow-up, with near-adult HSDS reached by 264 (17%) participants (mean [SD] –1.99 [0.94]; change from baseline +0.90 [0.85]). During the study, 695 (46%) participants (EAS) entered puberty at a mean (SD) age of 12.7 (1.9) years (whether puberty was spontaneous or induced was unknown). Within the SAS, mean IGF-I SDS (SD) at year 10 was 0.91 (1.69); change from baseline +1.48 (1.70). Serious adverse reactions were reported in 10 participants (epiphysiolysis [n = 3]).
Conclusions
Growth hormone-treated participants with TS responded well, without new safety concerns. Our real-world data are in agreement with previous studies.
Clinical trial registration
NordiNet® IOS (ClinicalTrials.gov, NCT00960128); the ANSWER Program (ClinicalTrials.gov, NCT01009905)
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