An Observational Study on Response to Growth Hormone Therapy in Indian Patients of Short Stature with Special Emphasis on Biochemical Parameters and Bone Biomarkers.

Introduction: There is a lack of Indian data on short stature treatment using recombinant human growth hormone (rhGH). We explored the effects of such treatment in eastern Indian patients, with emphasis on biochemical parameters and bone biomarkers in addition to basic anthropometry. Methods: Our descriptive study covered 50 short stature patients of varied aetiology attending endocrine outpatient department (OPD) of a tertiary care teaching hospital. Patients were followed up for 1 year after the index visit, and prospective data were reconciled with past medical records. A dose of rhGH used was 0.18-0.375 mg/kg as standard, starting dose mostly being 0.2 mg/kg. Dosing was adjusted if the physician judged the clinical outcome to be less favourable than expected. Anthropometric parameters (height, weight, body mass index (BMI) and skeletal age) were recorded clinically, and various biochemical parameters and bone biomarkers were estimated from blood. Results: Among 50 subjects, 60% had idiopathic growth hormone (GH) deficiency and 26% had Turner's syndrome. The median age at treatment start was 10 years, and the median treatment duration was 25.5 months. The height increased more in the first year of therapy. In the last 6 months, the height velocity was approximately 0.5 cm/month. Although the weight increased significantly, the increment slowed down in the last 6 months. Both remained less than age- and gender-matched references throughout. The skeletal age was on average 2 years behind chronological age (CA)-being 8.7, 9.6 and 11.3 years, respectively, at therapy start, after one year and at study end. Fasting blood glucose (FBG), total cholesterol and calcium level changes were not statistically significant. Serum cortisol and phosphate showed a modest but statistically significant rise, while thyroid-stimulating hormone (TSH) level declined. Insulin-like growth factor 1 (IGF-1) increase was relatively pronounced. Among bone biomarkers, a decrease in CTx and an increase in vitamin D were significant. Dual-energy X-ray absorptiometry (DEXA) data indicated that bone mineral density was less than that of age-matched controls despite treatment. The therapy was well tolerated. Conclusions: rhGH treatment leads to significant improvement in anthropometry in Indian children comparable with Western data. Bone biomarker changes indicate decreased bone resorption and increased bone formation although bone mineral density still lags behind age-matched controls.