Efficacy and influencing factors of recombinant human growth hormone therapy in children with Turner syndrome: a single-center retrospective cohort study

Background: Real-world evidence regarding the optimal initiation timing and safety of recombinant human growth hormone (rhGH) in children with Turner syndrome (TS) remains limited. This study aimed to evaluate the efficacy of rhGH therapy in children with TS and to identify clinically relevant factors influencing treatment outcomes, thereby providing evidence to inform individualized therapeutic strategies.

Methods: This retrospective cohort study included 50 pediatric patients with TS (3–14 years) who received rhGH therapy for ≥6 months. Eligibility criteria included a confirmed TS karyotype and height <−2 standard deviations (SDs). Baseline characteristics, laboratory parameters, and karyotypes were recorded. Patients were followed up every 6 months for up to 36 months, with height-related indicators [height standard deviation score (HtSDS), change in HtSDS (ΔHtSDS), growth velocity (GV)] and safety parameters [glycated hemoglobin (HbA1c), homeostatic model assessment for insulin resistance (HOMA-IR)] monitored. We used a generalized estimating equation (GEE) model to identify factors associated with rhGH treatment efficacy and then examined the interaction between follow-up duration and age at treatment initiation.

Results: Baseline characteristics showed a median age of 7 [5–11] years and a mean HtSDS of −3.18±0.93. After 36 months of treatment, the mean height increased from 114.50±14.90 cm at baseline to 132.82±11.77 cm, whereas HtSDS improved to −1.80±0.99 (mean ΔHtSDS 1.20±0.67). Safety evaluation in a stable 12-month cohort (n=33) showed stable HbA1c levels (P=0.87) and a physiological increase in HOMA-IR (P=0.049) within the normal clinical range. GEE analysis revealed that treatment duration was positively correlated with HtSDS and ΔHtSDS, but negatively correlated with GV (all P<0.05). Baseline HtSDS was positively associated with follow-up HtSDS (β=0.797, P<0.001). Interaction analysis indicated a significant interaction between initiation age and follow-up duration: patients who started therapy earlier exhibited more rapid increases in HtSDS and ΔHtSDS during the early phase (6–12 months), whereas those with later initiation showed a greater decline in GV at 36 months.

Conclusions: rhGH therapy appears to be effective and safe for enhancing linear growth in children with TS. The therapeutic response is time-dependent and appears to be optimized by earlier initiation and higher baseline height.