Novel Growth Hormone Treatment Beneficial Among Children With Turner Syndrome

A comparison of Growtropin (DA-3002, Dong-A ST. Co, Ltd), a new liquid recombinant human growth hormone (GH), and Genotropin (Pfizer) shows the former has comparable efficacy and safety to the latter among Korean children with Turner syndrome, reports BMC Endocrine Disorders.

Turner syndrome, caused by a missing or partially missing X chromosome, only affects female children. Problems that present with the condition include short stature, ovary nondevelopment, and heart defects.

“Short stature is the most consistent characteristic feature of Turner syndrome,” wrote the study authors. “To improve final heights of children with TS effectively, it is important to provide them with early and appropriate treatment using GH.”

Among the patients randomized almost 1:1 to the treatment group receiving DA-3002 (n = 28; mean [SD] age, 6.84 [2.62] years) or the Genotropin group (n = 30; mean age, 7.06 [2.96] years), both groups had younger bone age than their chronological age, at 6.03 (2.93) and 5.96 (2.63) years, respectively. Height, weight, and body mass index, as well as thyroid stimulating hormone, free thyroxine, insulin-like growth factor-1 (IGF-1), and 3 insulin-like growth factor binding protein-3 (IGFBP-3) levels were similar.

This year-long open-label, multicenter, randomized controlled trial included Korean girls with Turner syndrome, aged 2 to 12 years, whose height was among the 10th percentile or less of same-aged Korean individuals before the study. Prior treatment with GH excluded study participation. Eleven hospitals were covered in the study, which took place from February 2013 to May 2018. Overall close to three-fourths of the study participants had mosaicism.

The investigation produced the following results:

• Mean height velocity (HV) was 4.15 (0.30) cm/year in the DA-3002 group and 4.34 (0.29) cm/year in the Genotropin group
• Mean height standard deviation score (HtSDS) rose from 0.43 (0.22) to 0.70 (0.23) in the treatment group and 0.42 (0.24) to 0.66 (0.39) in the comparator group from 26 to 52 weeks
• Skeletal maturity (bone age) increased from baseline to 52 weeks in both groups, with the treatment group seeing mean changes of 1.44 (0.90) and 1.25 (0.58) at 26 and 52 weeks, and the comparator group seeing similar changes of 1.48 (0.90) and 1.47 (0.45), respectively
• The treatment group had mean increases of 208.33 (98.22) and 206.59 (105.76) ng/mL in IGF-1 levels and 1.18 (0.82) and 1.30 (0.93) mcg/mL in IGFBP-3 at 26 and 52 weeks, respectively
• The comparator group had mean increases of 226.65 (138.93) and 206.59 (105.76) ng/mL in IGF-1 levels and 1.22 (1.06) and 1.42 (1.31) mcg/mL in IGFBP-3 at 26 and 52 weeks, respectively
• HV, HtSDS, bone age, IGF-1, and IGFBP-3 all showed statistically significant increases from baseline in both groups over the course of the study
• Only 1 adverse drug reaction (injection-site erythema) was seen, and it occurred in the treatment group
• Adverse events happened in 3 subjects in each group

In addition to the noted height benefits, the authors highlighted that lipid profiles improve and arterial hypertension prevalence drops following GH treatment. This finding holds particular importance because there is a higher risk of cardiovascular (CV) disease and congenital CV malformation among women with Turner syndrome, the authors noted.

“This clinical study demonstrated an excellent growth promoting effect of DA-3002 in children with short stature due to TS and confirmed its safety,” the authors concluded. “DA-3002 is another effective option for patients with TS considering GH treatment without any safety concern.”

Still, they recommend a long-term study to validate their findings.

Reference

Kim J, Kim M-S, Suh B-K, et al. Recombinant growth hormone therapy in children with Turner Syndrome in Korea: a phase III Randomized Trial. BMC Endocr Disord. Published online December 10, 2021. doi:10.1186/s12902-021-00904-5