ESPE Abstracts (2022) 95 FC10.5

ESPE2022 Free Communications GH and IGFs (6 abstracts)

Features and outcomes of syndromic vs non-syndromic children born small for gestational age (SGA) under growth hormone therapy in a Belgian cohort

Marianne Becker 1 , Muriel Thomas 2 , Cécile Brachet 3 , Claudine Heinrichs 3 , Hilde Dotremont 4 , Karl Logghe 5 , Kristina Casteels 6,7 , Anne Rochtus 6,7 , Daniel Klink 8 , Martine Cools 9 , Kathleen De Waele 9 , Anne-Simone Parent 10 , Guy Massa 11 , Willem Staels 12 , Inge Gies 12 , Philippe Lysy 13 & Dominique Beckers 14

1Centre hospitalier de Luxembourg, Luxembourg, Luxembourg; 2The BElgian Society for PEdiatric Endocrinology and Diabetology (BESPEED), Brussels, Belgium; 3University Children’s Hospital HUDE, Brussels, Belgium; 4University Hospital Antwerp, Edegem, Belgium; 5AZdelta Hospital, Roeselare, Belgium; 6University Hospitals Leuven, Leuven, Belgium; 7KU Leuven, Leuven, Belgium; 8ZNA Queen Paola Children´s Hospital Antwerp, Antwerp, Belgium; 9Universitair Ziekenhuis Gent, Gent, Belgium; 10University Hospital Liège, Liège, Belgium; 11Jessa Hospital, Hasselt, Belgium; 12University Hospital Brussels, Brussels, Belgium; 13Université Catholique de Louvain, Brussels, Belgium; 14Université Catholique de Louvain, CHU UCL Namur, Yvoir, Belgium

Background and Aim: A substantial proportion of SGA patients have a syndrome underlying their growth restriction. Most SGA cohorts comprise both syndromic (S-SGA) and non-syndromic patients (nonS-SGA) impeding delineation of the recombinant human growth hormone (rhGH) response. We present a detailed characterization of the Belgian SGA cohort and analyze rhGH response based on adult height (AH).

Patients & Methods: Clinical and auxological data of SGA patients treated with rhGH that reached AH were retrieved from BELGROW, a national database of all rhGH treated patients held by BESPEED (Belgian Society for Pediatric Endocrinology and Diabetology). Z-score calculation: Niklasson 1991 / Intergrowth (< 28.5 weeks) for birth parameters; Roelants 2004 for follow-up data. AH SDS was calculated using SDS for 21 years. Patients were classified as S-SGA if they had: genetically confirmed syndrome, fetal alcohol syndrome (FAS), or minimum 2 additional symptoms suggesting a syndromic origin (congenital heart defects, mental retardation, dysmorphic features). Subjects with an underlying chronic disease known to impair growth were excluded.

Results: 276 patients were included, 42 were classified as syndromic (most frequent diagnosis: 10 FAS, 8 Silver-Russel syndrome, 3 3M syndrome). Compared with nonS-SGA, S-SGA were more frequently male (74% vs 54%), had lower birth weight (values expressed as median, (P10/P90): -2.79 SDS, (-4.26/-1.34) vs -2.26 (-3.46/-1.41), P=0.0018), lower birth length (-3.09 SDS, (-4.72/-1.2) vs -2.47 (-3.72/-1.63), P=0.0308), were younger (7.58 years (4.3/12.71) vs 10.24 (5.42/13.95), P=0.0022), shorter (-3.37 SDS (-5.42/-2.62) vs -3.07 (-3.74/-2.62), P=0.0438) and thinner (BMI SDS -1.61 (-3.65/0.25) vs -1.13 (-2.47/0.28), P=0.0198) at start of rhGH. Midparental height was lower in nonS-SGA (-1.38 SDS (-2.25/-0.49) vs -1.23 (-2.18/-0.02), P=0.0416). First year rhGH response was comparable (delta height SDS +0.5 (0.24/0.94) vs +0.56 (0.26/0.91), P=0.92). Mean dose rhGH was higher in S-SGA (0.047 mg/kg/day (0.039/0.064) vs 0.043 (0.035/0.056), P=0.0052). AH was lower in S-SGA (-2.59 SDS (-4.95/-1.57) vs -2.32 (-3.3/-1.2), P=0.0136; (mean±SD) male 162.1±8.2 / female 147.6±11.2 cm vs male 165.9±6.4 / female 152.6±5.1 cm). The majority in both groups remained short (syndromic 71%, non-syndromic 63%). Total height gain was comparable in both groups (delta height SDS +0.73 (-0.95/1.48) vs +0.85 (-0.11/1.84), NS P=0.25).

Conclusions: Compared to non-syndromic SGA patients, syndromic patients have lower birth weight and length and shorter stature when starting rhGH therapy, started rhGH therapy earlier, and received a higher rhGH doses. At AH, syndromic patients were shorter than non-syndromic patients, but their height gain under rhGH therapy was comparable.

Volume 95

60th Annual ESPE (ESPE 2022)

Rome, Italy
15 Sep 2022 - 17 Sep 2022

European Society for Paediatric Endocrinology 

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