Dayal Devi, Gupta Atul, Gupta Saniya, Dutta Aditya
Endocrinology and Diabetes Unit, Department of Pediatrics, Postgraduate Institute of Medical Educa-tion and Research, Chandigarh, India.
Department of Endocrinology, Postgraduate Institute of Medical Education and Research, Chandigarh, India.
Pediatr Endocrinol Diabetes Metab. 2019;25(4):194-201. doi: 10.5114/pedm.2019.89642.
The conventional management of hypoparathyroidism in children involves the use of calcium and vitamin D analogs. This therapy effec-tively increases the serum calcium levels but worsens hypercalciuria and its consequences such as nephrocalcinosis and renal insuffi-ciency. Although replacement with the missing parathyroid hormone (PTH) is ideal and available for more than 2 decades, the reported concerns of osteosarcoma prohibited its use in children with open epiphyses. Nevertheless, the data accumulated over the past several years suggests that the fears of bone malignancies were probably overstated. With an aim to review the available data on recombinant PTH (rhPTH) use, we performed a literature search using international databases and identified 15 studies involving approximately 70 children with hypoparathyroidism due to various etiologies who received rhPTH1-34 for durations between 1 day and 13.5 years. All the studies appear to indicate that rhPTH1-34 therapy is an effective short and long-term strategy for treatment of hypoparathyroidism with better metabolic control, lesser effects on renal function and improved quality of life as compared to conventional therapy. A more significant conclusion is the safety of long-term use of rhPTH1-34 with no observed adverse skeletal effects so far. However, all studies mention the importance of a continued surveillance for adverse effects in the treated patients. This narrative review discusses the experi-ence of rhPTH1-34 use exclusively in children.
儿童甲状旁腺功能减退症的传统治疗方法包括使用钙和维生素D类似物。这种疗法能有效提高血清钙水平，但会加重高钙尿症及其后果，如肾钙质沉着症和肾功能不全。尽管补充缺失的甲状旁腺激素（PTH）是理想的治疗方法，且已有二十多年的应用历史，但由于有报道称担心骨肉瘤的发生，因此禁止在骨骺未闭合的儿童中使用。然而，过去几年积累的数据表明，对骨恶性肿瘤的担忧可能被夸大了。为了回顾重组PTH（rhPTH）使用的现有数据，我们使用国际数据库进行了文献检索，确定了15项研究，涉及约70名因各种病因导致甲状旁腺功能减退症的儿童，他们接受rhPTH1-34治疗的时间为1天至13.5年。所有研究似乎都表明，与传统疗法相比，rhPTH1-34治疗是治疗甲状旁腺功能减退症的一种有效的短期和长期策略，具有更好的代谢控制、对肾功能影响较小以及生活质量改善等优点。一个更重要的结论是rhPTH1-34长期使用的安全性，目前尚未观察到不良骨骼影响。然而，所有研究都提到了对接受治疗的患者持续监测不良反应的重要性。本叙述性综述专门讨论了rhPTH1-34在儿童中的使用经验。
