Real-World Efficacy of Weekly Somatrogon on Growth and Bone Health in Pediatric Growth Hormone Deficiency: A 12-Month Retrospective Cohort Study.

IF 1.5 4区 医学 Q4 ENDOCRINOLOGY & METABOLISM
Mohammad Hosny Awad, Reham Ghanim, Rania Eladl, Zulf Mughal, Manal Mustafa
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Abstract

Background: Growth hormone deficiency (GHD) in children results in short stature and impaired bone health. While daily growth hormone (GH) injections are effective, they are associated with adherence challenges. Somatrogon, a long-acting recombinant human GH, allows weekly administration, potentially improving treatment compliance.

Methods: This retrospective cohort study included 39 prepubertal children with GHD treated with weekly Somatrogon at Al Jalila Children's Hospital, Dubai. Diagnosis was based on clinical, biochemical, and radiological criteria, including height standard deviation score (SDS) < -2.0, subnormal growth velocity, and subnormal peak GH in one stimulation test (<10 ng/mL) supported by low IGF-1 and/or abnormal MRI. Growth outcomes and bone health indices were assessed over 12 months using auxology, IGF-1 levels, and BoneXpert-derived Bone Health Index (BHI) SDS and Metacarpal Index (MCI) SDS.

Results: After 12 months of therapy, mean height SDS improved significantly from -2.16 ± 0.80 to -1.65 ± 0.71 (p < 0.001). IGF-1 SDS rose from -1.38 ± 1.02 to 0.88 ± 1.57 (p < 0.001). Adult predicted height and BHI SDS also improved significantly (p = 0.005 and p < 0.001, respectively). No significant changes were observed in bone age SDS or MCI SDS.

Conclusions: Weekly Somatrogon significantly improved linear growth, IGF-1 levels, and cortical bone health without advancing bone age in children with GHD. These findings support the efficacy of long-acting GH therapy and its potential to optimize growth and skeletal outcomes in clinical practice.

每周使用生长激素对生长激素缺乏症儿童生长和骨骼健康的实际疗效:一项12个月的回顾性队列研究。
背景:儿童生长激素缺乏症(GHD)导致身材矮小和骨骼健康受损。虽然每天注射生长激素(GH)是有效的,但它们与坚持挑战有关。生长激素是一种长效重组人生长激素,允许每周给药,可能提高治疗依从性。方法:本回顾性队列研究纳入了39名在迪拜Al Jalila儿童医院接受每周一次生长激素治疗的青春期前GHD儿童。诊断依据临床、生化和放射学标准,包括一次刺激试验中身高标准差评分(SDS) < -2.0,生长速度亚正常,GH峰值亚正常(结果:治疗12个月后,平均身高SDS从-2.16±0.80显著改善至-1.65±0.71 (p < 0.001)。IGF-1 SDS从-1.38 ± 1.02上升到0.88 ± 1.57 (p )结论:每周一次的生长激素显著改善了GHD儿童的线性生长、IGF-1水平和皮质骨健康,而没有提前骨龄。这些发现支持长效生长激素治疗的疗效及其在临床实践中优化生长和骨骼结果的潜力。
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来源期刊
Journal of Clinical Research in Pediatric Endocrinology
Journal of Clinical Research in Pediatric Endocrinology ENDOCRINOLOGY & METABOLISM-PEDIATRICS
CiteScore
3.60
自引率
5.30%
发文量
73
审稿时长
20 weeks
期刊介绍: The Journal of Clinical Research in Pediatric Endocrinology (JCRPE) publishes original research articles, reviews, short communications, letters, case reports and other special features related to the field of pediatric endocrinology. JCRPE is published in English by the Turkish Pediatric Endocrinology and Diabetes Society quarterly (March, June, September, December). The target audience is physicians, researchers and other healthcare professionals in all areas of pediatric endocrinology.
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