Different long-term response to growth hormone therapy in small-versus appropriate-for-gestational-age children with growth hormone deficiency

Background/Aims: The role of birth weight on growth hormone (GH) therapy response in GH-deficient (GHD) children has not been fully elucidated. Therefore, we examined the growth of 23 small-for-gestational-age GHD children (SGA-GHD, 11 females and 12 males), 26 appropriate-for-gestational-age GHD children (AGA-GHD, 11 females and 15 males) during the first 5 years of GH therapy and that of 22 non-GH-treated SGA children (12 females and 10 males). Methods: We collected height and height velocity measurements yearly. Results: In AGA-GHD children, height was always greater than in the SGA groups and significantly increased from the fourth year of treatment. Height velocity was higher (SGA-GHD: 1.72 ± 0.30 standard deviation score, SDS, AGA-GHD: 2.67 ± 0.21 SDS; p = 0.039) in AGA-GHD children during the first year of treatment. The AGA-GHD group showed the highest percentage (52.4%) of subjects surpassing mid-parental height and the greatest height gain after 5 years of follow-up. Conclusion: Our results show that birth size is an important factor affecting the response to GH therapy in GHD children during the first 5 years of treatment. The paediatric endocrinologist should be aware of this factor when planning the management of GHD children born SGA.