First Clinical Study on Long-Acting Growth Hormone Therapy in Children with Turner Sydrome

Abstract

Study on long-acting growth hormone (LAGH) therapy in Turner syndrome (TS). This 2-year retrospectively study included patients diagnosed with TS from 2018-2021. Patients were divided into four groups: Group 1 to 4 were low dose (0.1 mg/kg/ w), high-dose (0.2 mg/kg/w) LAGH, daily GH (0.38 mg/kg/w) and untreated control. The efficacy and safety data were analyzed. Seventy-five TS cases with the age 7.9±2.9 years and the bone age 6.8±2.8 years were recruited. In year 1:The change in height standard deviation score (∆HtSDS) and height velocity (HV) in Group 2 were comparable to Group 3, both of the two groups were higher than Group 1. ∆HtSDS and HV in all GH treatment group higher than untreated group. IGF1 increased in all treatment groups, only 4 cases had IGF1> 3SD. In year 2: ΔHtSDS and HV in Group 2 and 3 were comparable. 5 cases had IGF1> 3SD. Correlation analysis for LAGH efficacy at year 1 indicated that baseline variables correlated with ΔHtSDS included: GH dose, CA (chronological age) and bone age (BA). The HV was positively correlated with baseline GH dose, HtSDS, IGF-1SDS and negatively correlated with baseline CA, BA, and BMI. No GH-related serious adverse effects were observed. The high-dose LAGH treatment in TS patients is effective and safe as daily GH for 2 years. The favorable prognosis factors include sufficient GH dose, early treatment. IGF1 monitoring and weight control are important.