ESPE Abstracts

Introduction: Isolated growth hormone deficiency (GHD), which is idiopathic in most cases, is a rare disease. If left untreated, cases occurring in the prepubertal period will lead to permanent short stature. GHD was approved for recombinant human growth hormone (rhGH) replacement in 1985. In Vietnam, the diagnosis and treatment of growth disorders have been performed for 20 years, but only at a few hospitals in Ha Noi and Ho Chi Minh city. Furthermore, studies in our country of efficacy of rhGH treatment in GHD are still limited.

Objectives: To evaluate outcomes of rhGH replacement in improving height variables, such as height–standard deviation score (HT–SDS) and height velocity (ΔHT) in pediatric patients with isolated idiopathic GHD.

Methods: A case series study with 42 isolated idiopathic GHD patients treated at Nguyen Tri Phuong hospital from January 2018 to August 2023.

Results: We evaluated 42 cases of GHD patients, 76,2% are male. 21 patients had been treated over 2 years. Mean age at diagnosis is 8.2 ± 2.1 years old. HT–SDS pre-treatment was –1.8 ± 0.1 SD. Mean GH dose is 0.033 mg/kg/day.

Over 95% of patients had ΔHT reached at least 6 cm/year after treatment. In the first year, we saw better results with 60% of patients achieving 8–12 cm/year of height gain and 21% rose over 12 cm/year. Whereas in the second year, only 38% gain 8–12 cm/year, and no patient gains more than 12 cm/year. When comparing with target height (Mid parenteral height, MPH-SDS), first year HT–SDS reached MPH–SDS; and second year HT–SDS even exceeded MPH-SDS with statistical significance. rhGH replacement also had positive impacts on other parameters, such as improving bone age and IGF-1 levels. In details, IGF-1–SDS improved after 1 and 2 years of treatment, with an average of 1 SD and 1,3 SD respectively, compared to –0,7 SD pre-treatment.

Conclusion: GH replacement improved HT–SDS, height velocity and brought post treatment HT–SDS closer to the target height. GH treatment provides the opportunity for isolated idiopathic GHD children to achieve their expected genetic height.