Department of Pediatric Endocrinology and Diabetology, Centre Hospitalier de Luxembourg, Luxembourg, Luxembourg.
The BElgian Society for PEdiatric Endocrinology and Diabetology (BESPEED), Brussels, Belgium.
Front Endocrinol (Lausanne). 2023 Jun 2;14:1112938. doi: 10.3389/fendo.2023.1112938. eCollection 2023.
A substantial proportion of SGA patients present with a syndrome underlying their growth restriction. Most SGA cohorts comprise both syndromic and non-syndromic patients impeding delineation of the recombinant human growth hormone (rhGH) response. We present a detailed characterization of a SGA cohort and analyze rhGH response based on adult height (AH).
Clinical and auxological data of SGA patients treated with rhGH, who had reached AH, were retrieved from BELGROW, a national database of all rhGH treated patients held by BESPEED (BElgian Society for PEdiatric Endocrinology and Diabetology). SGA patients were categorized in syndromic or non-syndromic patients.
272 patients were included, 42 classified as syndromic (most frequent diagnosis (n=6): fetal alcohol syndrome and Silver-Russell syndrome). Compared with non-syndromic patients, syndromic were younger [years (median (P10/P90)] 7.43 (4.3/12.37) vs 10.21 (5.43/14.03), p=0.0005), shorter (height SDS -3.39 (-5.6/-2.62) vs -3.07 (-3.74/-2.62), p=0.0253) and thinner (BMI -1.70 (-3.67/0.04) vs -1.14 (-2.47/0.27) SDS, p=0.0054) at start of rhGH treatment. First year rhGH response was comparable (delta height SDS +0.54 (0.24/0.94) vs +0.56 (0.26/0.92), p=0.94). Growth pattern differed with syndromic patients having a higher prepubertal (SDS +1.26 vs +0.83, p=0.0048), but a lower pubertal height gain compared to the non-syndromic group (SDS -0.28 vs 0.44, p=0.0001). Mean rhGH dose was higher in syndromic SGA patients (mg/kg body weight/day 0.047 (0.039/0.064) vs 0.043 (0.035/0.056), p=0.0042). AH SDS was lower in syndromic SGA patients (-2.59 (-4.99/-1.57) vs -2.32 (-3.3/-1.2), p=0.0107). The majority in both groups remained short (<-2 SDS: syndromic 71%, non-syndromic 63%). Total height gain was comparable in both groups (delta height SDS +0.76 (-0.70/1.48) vs +0.86 (-0.12/1.86), p=0.41).
Compared to non-syndromic SGA patients, syndromic SGA patients were shorter when starting rhGH therapy, started rhGH therapy earlier, and received a higher dose of rhGH. At AH, syndromic SGA patients were shorter than non-syndromic ones, but their height gain under rhGH therapy was comparable.
相当一部分 SGA 患者的生长受限与潜在的综合征有关。大多数 SGA 队列包括综合征和非综合征患者，这阻碍了对重组人生长激素（rhGH）反应的明确界定。我们对 SGA 队列进行了详细的描述，并根据成年身高（AH）分析了 rhGH 反应。
从 BELGROW 中检索了接受 rhGH 治疗且已达到 AH 的 SGA 患者的临床和生长数据，BELGROW 是由 BESPEED（比利时儿科内分泌学和糖尿病学会）持有的所有接受 rhGH 治疗的患者的国家数据库。将 SGA 患者分为综合征或非综合征患者。
共纳入 272 名患者，其中 42 名被诊断为综合征（最常见的诊断（n=6）：胎儿酒精综合征和 Silver-Russell 综合征）。与非综合征患者相比，综合征患者年龄更小[年龄（中位数（P10/P90）] 7.43（4.3/12.37）vs 10.21（5.43/14.03），p=0.0005），更矮（身高 SDS-3.39（-5.6/-2.62）vs -3.07（-3.74/-2.62），p=0.0253）和更瘦（BMI SDS-1.70（-3.67/0.04）vs -1.14（-2.47/0.27），p=0.0054）在开始 rhGH 治疗时。第一年 rhGH 反应相当（身高 SDS 增加 0.54（0.24/0.94）vs 0.56（0.26/0.92），p=0.94）。生长模式不同，综合征患者的青春期前生长（SDS+1.26 vs+0.83，p=0.0048）更高，但青春期身高增长低于非综合征组（SDS-0.28 vs 0.44，p=0.0001）。综合征 SGA 患者的 rhGH 剂量更高（mg/kg 体重/天 0.047（0.039/0.064）vs 0.043（0.035/0.056），p=0.0042）。综合征 SGA 患者的 AH SDS 较低（-2.59（-4.99/-1.57）vs-2.32（-3.3/-1.2），p=0.0107）。两组中大多数患者仍较矮（< -2 SDS：综合征患者 71%，非综合征患者 63%）。两组的总身高增长相当（身高 SDS 增加 0.76（-0.70/1.48）vs 0.86（-0.12/1.86），p=0.41）。
与非综合征 SGA 患者相比，综合征 SGA 患者在开始 rhGH 治疗时更矮，开始 rhGH 治疗的时间更早，接受的 rhGH 剂量更高。在 AH 时，综合征 SGA 患者比非综合征 SGA 患者更矮，但他们在 rhGH 治疗下的身高增长相当。
