ESPE Yearbook of Paediatric Endocrinology

Brief summary: In this study the authors reported the adult height and timing of puberty of a cohort of Danish children born small for gestational age (SGA) and treated with rhGH in comparison with national growth standards. rhGH treatment significantly increased height SDS in adulthood as compared to the height SDS at treatment start. Age at peak height velocity did not differ compared with the reference cohort, although peak height velocity was reduced in SGA subjects. SGA boys had an earlier onset of puberty compared with the reference cohort.

Approximately 10% of children born small for gestational age (SGA) fail to catch-up their growth during postnatal life, ending up with a shorter adult height (1, 2). Short stature is an approved indication for rhGH treatment in Europe since 2003. Previous studies have reported the overall efficacy of rhGH therapy in increasing adult height, though a considerable variability in the individual responses to therapy exists (3, 4). In this prospective longitudinal multicenter study, 102 short children born SGA treated with rhGH were evaluated for final height (n=47), peak height velocity, age at peak height velocity and puberty onset.

Height gain SDS from start of treatment up to adult height was on average 0.9 SDS in girls and 1.6 SDS in boys with wide individual variability. 57% of the patients reached an adult height within the normal range (above –2 SDS) and 53% achieved an adult height within their genetic target. A prediction model was applied to the dataset and adult height (SDS) was compared with the predicted adult height (SDS). In girls gain in height compared with the prediction was 0.71 SDS, which is equal to 4.5 cm, and in boys 0.61 SDS, which is equal to 4.0 cm. In both genders, peak height velocity was lower than the reference cohort, with no difference in age at peak height velocity. Age at puberty onset was earlier in SGA boys compared with the reference cohort but not in girls.

These data confirm the moderate efficacy of rhGH treatment in short SGA children and the large individual variability in the response to treatment, due to the heterogeneity of the conditions underlying the ‘diagnosis’ of SGA.

References: 1. Hokken-Koelega AC, De Ridder MA, Lemmen RJ, Den Hartog H, De Muinck Keizer-Schrama SM, Drop SL. Children born small for gestational age: do they catch up? Pediatr Res. 1995;38(2):267–271. 2. Albertsson-Wikland K, Wennergren G, Wennergren M, Vilbergsson G, Rosberg S. Longitudinal follow-up of growth in children born small for gestational. 3. Van Pareren Y, Mulder P, Houdijk M, Jansen M, Reeser M, Hokken-Koelega A. Adult height after long-term, continuous growthhormone (GH) treatment in short children born small for gestational age: results of a randomized, double-blind, dose-response GH trial. J Clin Endocrinol Metab. 2003;88(8):3584–3590. 4. Adler E, Lambert A-S, Bouvattier C, et al. Determinants of final height in patients born small for gestational age treated with recombinant growth hormone. Horm Res Paediatr. 2021;94(1–2):52–62.