Background: Growth hormone (GH) therapy has been available for more than 60 years to treat short stature due to GH deficiency (GHD). GHD is the most frequent pituitary hormone deficiency in childhood, with an incidence of 1 in 4,000–10,000 children. It may be congenital or acquired, but most cases are idiopathic.Method: We aimed to analyse the response to GH therapy in children with GHD and to compare outcomes between isolated GHD (IGHD) and multiple pituitary hormone deficiency (MPHD), and between transient and persistent GHD.We conducted a retrospective study including all children treated for GHD in our paediatric tertiary center who achieved final height between October 2010 and June 2025. Patients were divided into two groups: IGHD and MPHD. Persistency of GHD was assessed after achievement of adult height, and patients were further classified as having persistent or transient GHD. Statistical significance was set at p<0.05. Results are presented as median [P25–P75]. Chi-squared and Mann-Whitney tests were used for categorical and continuous variables, respectively.Results: Sixty-four patients were included (53.1% male). Median age at GH therapy initiation was 11.45 years [8.47–13.14]. Height gain was 1.30 z-scores [0.81–2.00] over 5.33 years of treatment [3.63–7.16], resulting in a median final height z-score of –1.59 [–2.20 to –0.86]. Median BMI z-score variation during treatment was –0.14 [–0.54 to 0.62], showing a tendency towards reduction but without statistical significance (p=0.96). MRI was performed in 62 patients, revealing hypothalamic–pituitary abnormalities in 41.9%.In the IGHD group (n=56), therapy was initiated at 11.51 years [9.95–13.38], versus 5.60 years [2.04–8.15] in MPHD (n=8). Baseline height z-score was –2.94 [–3.46 to –2.41] and –3.97 [–4.96 to –2.97], respectively (p=0.09). Final height was –1.62 [–2.20 to –0.86] vs. –0.90 [–3.03 to 1.23] (p=0.14). Height gain was significantly higher in MPHD: Δz-score 2.80 [1.04–3.52] vs. 1.20 [0.81–1.79] in IGHD (p=0.02).Comparing the transient (n=38) with the persistent GHD group (n=21), GH therapy was started at 11.48 years [8.29–13.48] versus 7.58 years [2.65–9.91]. Baseline height z-score was –2.84 [–3.60 to –2.58] vs. –3.47 [–4.53 to –2.70] (p=0.009). Final height z-score was –1.55 [–3.17 to –1.55] vs. –1.64 [–2.60 to –0.95] (p=0.14). Height gain was 1.18 [0.81–1.85] vs. 1.59 [0.72–2.61] (p=0.40).Children with transient GHD presented a significantly higher proportion of isolated GHD compared with those with persistent GHD (97.4% vs. 71.4%, p=0.006).Hypothalamic–pituitary MRI abnormalities were observed in 32.4% of patients with transient GHD and 61.9% of those with persistent GHD, without a statistically significant difference between groups.Conclusion: GH therapy significantly improved height (median Δz-score +1.30). Height gain was greater in patients with MPHD compared with IGHD. No significant differences were observed between transient and persistent GHD. Despite the known association between hypothalamic–pituitary MRI abnormalities and persistent GHD, 32% of patients with transient GHD also presented abnormalities, highlighting the importance of retesting these patients during the transition phase.
