Aim: Data on response to GH treatment in the very young children with GH deficiency is scarce. The aim of this study was to evaluate the growth response in such children in a national multicentre study and to analyse the factors affecting the growth response.
Materials and methods: In this study, we retrospectively evaluated the files of GH deficiency patients who had started GH treatment between 03 years of age who were being followed in 14 different centres from different regions of Turkey between 19 February 2014 and 23 October 2014. The study was approved by the Clinical Studies Ethics Committee. All collected data were obtained from patient hospital records. An electronic case recording form (CRF) was created. The CRF covered demographic features, as well as clinical and laboratory findings of the patients. The CRF was uploaded to the website of FAVOR Web Registry System (www.favorsci.org). Data entered in the registry were also checked for consistency by one of the authors (SÇ). The time given for patient enrolment was eight months. By the end of the deadline the collected patient record data were entered to Microsoft Excel database and subsequently transferred to SPSS for Windows statistical software for statistical analysis. The duration of GH treatment was accepted to be at least 12 mo. The patients were further subdivided according to isolated vs multiple pituitary hormone deficiency (MPHD) and age at onset of therapy: 012 months vs 1236 months. Patients with MPHD received appropriate replacement therapy.
Results: There were 42 patients with GH deficiency (23 males, 19 females) with a peak GH response (after GH stimulation test or at hypoglycaemia) of 0.69±0.14 ng/ml. 30 had MPHD and 12 had isolated GH deficiency. The mean age at onset of GH therapy was 11.2±1.03 mo. Mean GH dose used was 31.7±1.4 μg/kg per day. Results of GH therapy over 1 year are shown on the Table. There was a significant increase in length SDS (P=0.000), weight SDS (P=0.000) and BMI SDS (P=0.02) over 1-year of therapy. Height velocity over one year showed positive correlation with weight increment (r=0.38), but did not show correlation with birth weight, peak GH level, GH dose and BMI. In MPHD Group 1st year response was significantly higher (16.5±4.2 cm) than in the isolated GH deficiency group (12.8±3.3 cm) (P=0.014). In the group started GH between 012 months the response (18.0±4.2 cm) was higher than in the ones started between 1236 months (13.3±3.1 cm) (P=0.013). There was no difference between girls and boys with respect to the growth response. Neither was a difference in growth response between those with minipuberty or not. Multiple regression analysis did not reveal a significant parameter to explain the differences in growth response.
Conclusion: Among children with GH deficiency, young children with MPHD respond better than isolated GH deficiency and those children aged between 012 months at onset of therapy respond better than 1236 months children. The most significant factor in growth response was weight gain.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology