Division of Pediatric Nephrology, David Geffen School of Medicine and Mattel Children's Hospital, University of California Los Angeles, Los Angeles, California, USA.
Pediatr Transplant. 2024 Aug;28(5):e14803. doi: 10.1111/petr.14803.
Growth retardation and short final height is a common complication of chronic kidney disease (CKD) beginning in childhood, with profound deleterious effects on quality of life, mental health, and social achievement. Despite optimal treatments of causative factors for growth retardation in children with CKD, more than 50% of patients reach end-stage renal failure with a height >2 SD below the mean, and most do not demonstrate "catch-up" growth after receiving a kidney transplant. Four decades ago, recombinant human growth hormone (rhGH) treatment was introduced after studies showed increased growth velocity and improved height SDS in uremic subjects. Since then, an abundance of published data showed significant improvements in health-related quality of life, and most studies revealed no significant adverse effects. Clinical practice guidelines recommended rhGH treatment in CKD Stages 3-5D and after transplantation. Despite these guidelines, this therapy remained underutilized. Most commonly cited barriers to the implementation of rhGH treatment were the need for daily injections, financial challenges, physicians' unfamiliarity with guidelines, and fear of adverse events.
rhGH has been shown to improve growth and final height in short children with CKD, with minimal adverse effects. Despite data of its successful use generated over 3 decades, this treatment is underutilized. More judicious utilization of the treatment should emphasize educating patients, their care givers, and members of the multidisciplinary treating team. Additional studies are needed to assess the longer-term rhGH treatment in larger cohorts of patients, leading to additional supportive data and clearer recommendations.
生长迟缓及最终身高过矮是儿童时期起病的慢性肾脏病（CKD）的常见并发症，对生活质量、心理健康和社会成就均有深远的不良影响。尽管针对 CKD 患儿生长迟缓的病因采取了最佳治疗措施，但仍有超过 50%的患者在进入终末期肾衰竭时身高仍低于平均值 2 个标准差，且大多数患者在接受肾移植后并未表现出“追赶性”生长。四十年前，重组人生长激素（rhGH）治疗问世，相关研究表明尿毒症患者的生长速度加快，身高 SDS 得到改善。此后，大量已发表的数据显示，rhGH 治疗可显著改善健康相关生活质量，且大多数研究均未发现明显的不良影响。临床实践指南建议在 CKD 3-5D 期及移植后应用 rhGH 治疗。尽管有这些指南，但该疗法的应用仍不充分。rhGH 治疗实施受限最常被提及的原因包括需要每日注射、经济困难、医生对指南不熟悉以及担心不良事件。
rhGH 已被证实可改善 CKD 矮小儿童的生长和最终身高，且不良反应极小。尽管 30 多年来已积累了成功应用该治疗的数据，但该治疗的应用仍不充分。更合理地应用该治疗应强调对患者、其照顾者以及多学科治疗团队成员进行教育。需要进一步研究以评估更大患者队列中长期 rhGH 治疗的效果，从而提供更多支持性数据和更明确的建议。
