ESPE2022 Poster Category 1 GH and IGFs (27 abstracts)
A real life evaluation of rhGH efficacy in children born SGA
Giulia Tattesi 1,2 , Annalisa Deodati 3,1 , Elena Inzaghi 4 , Maria Elisa Amodeo 1 , Carla Bizzarri 5 , Marco Cappa 5 & Stefano Cianfarani 3,1,6
1Diabetology and Growth Disorders-Bambino Gesù Children's Hospital, Rome, Italy; 2San Gerardo Hospital-Milano Bicocca University, Monza, Italy; 3Department of Systems Medicine-Tor Vergata University, Rome, Italy; 4Pediatrics Department-Bambino Gesù Children's Hospital, Rome, Italy; 5Endocrinology Unit-Bambino Gesù Children's Hospital, Rome, Italy; 6Karolinska Institutet Department of Women's and Children's Health, Stockholm, Sweden
Background: Children born SGA are at risk of becoming short adults. Although most children born SGA show catch-up growth in the first 24 months of life, approximately 10% remain below the 3rd centile throughout childhood and adolescence. The available evidence shows that GH therapy can increase adult height in short children born SGA although long-term response is variable and closely related to the 1st year response.
Aims: The aims of this study were: 1) to evaluate the response to 1st rhGH therapy and 2) the efficacy of long-term rhGH therapy on near adult height in SGA children.
Methods and Results: We conducted a retrospective case-control study in a cohort of 54 children born SGA treated with rhGH (mean age 6.7 ± 2.5; 16F/35M) compared with 47 untreated SGA (mean age 6.3 ± 2.9; 30F/21M) after 1 year of follow-up. GH dose was 0.23 ± 0.09 mg/kg/week. Mean duration of therapy was 5.7 ± 2.62 years. The mean height at first evaluation in SGA treated children was -3.1± 0.8 SDS vs -2.9 ± 1.22 SDS in untreated children. After 1 year of follow-up mean height in treated SGA was -2.6 ± 0.8 SDS vs -2.2 ± 0.63 SDS. Mean height gain was 0.5 ± 0.8 SDS (about 3.1 cm) in treated SGA vs 0.7 ± 0.9 SDS (about 4.3 cm) in untreated subjects (P=0.1). Mean near adult height was -1.9 ± 0.6 SDS in 17 treated patients with mean height gain from baseline to near adult height of 0.55 ± 0.8 SDS (about 3.4 cm).
Conclusion: Our study show that the response to rhGH therapy is on average dissatisfying in the first year of treatment, with wide individual variability. The overall height gain from baseline to near adult height overlaps the same magnitude in SDS observed in the 1st year of therapy
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