Yoon Ji-Hee, Choi Yunha, Lee Yena, Yoo Han-Wook, Choi Jin-Ho
Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
Ann Pediatr Endocrinol Metab. 2021 Jun;26(2):105-111. doi: 10.6065/apem.2040150.075. Epub 2021 Jun 30.
Osteoporosis is a skeletal disorder characterized by reduced bone mass that results in increased risk of fractures. Pediatric osteoporosis can be caused by monogenic diseases, chronic diseases, and/or their treatment. This study was performed to investigate the effect of pamidronate infusion on osteoporosis in children and adolescents.
This study included 13 unrelated pediatric patients (10 males and 3 females) whose bone mineral density (BMD) z-score was <-2.0. Pamidronate was administered intravenously at a dosage of 1 mg/kg for 3 consecutive days every 4 months. Clinical and biochemical findings were reviewed retrospectively. The BMD values of the lumbar spine and femoral neck were assessed by dual energy x-ray absorptiometry at baseline and annually.
The underlying diseases were immobilization (62%), inflammatory bowel disease (23%), protein-losing enteropathy (8%), and idiopathic juvenile osteoporosis (8%). The mean age at the start of treatment was 12.7±4.3 years. Duration of treatment ranged from 12-50 months. The baseline height-standard deviation score (SDS) and weight-SDS were -2.01±2.08 and -2.60±1.62, respectively. The lumbar spine BMD z-scores improved significantly after 1 year of pamidronate treatment, but the femoral neck BMD z-scores did not. However, both z-scores had significantly increased by the end of treatment.
This study demonstrated that pamidronate treatment increased BMD in pediatric patients with osteoporosis with no significant adverse events. Further studies are required to better define the long-term efficacy and safety of pamidronate therapy in a large number of pediatric patients.
骨质疏松症是一种以骨量减少为特征的骨骼疾病，会导致骨折风险增加。儿童骨质疏松症可由单基因疾病、慢性疾病和/或其治疗引起。本研究旨在调查帕米膦酸输注对儿童和青少年骨质疏松症的影响。
本研究纳入了13名无亲缘关系的儿科患者（10名男性和3名女性），其骨密度（BMD）z评分＜-2.0。每4个月连续3天静脉注射帕米膦酸，剂量为1mg/kg。回顾性分析临床和生化检查结果。在基线时和每年通过双能X线吸收法评估腰椎和股骨颈的BMD值。
潜在疾病包括制动（62%）、炎症性肠病（23%）、蛋白丢失性肠病（8%）和特发性青少年骨质疏松症（8%）。治疗开始时的平均年龄为12.7±4.3岁。治疗持续时间为12 - 50个月。基线身高标准差评分（SDS）和体重SDS分别为-2.01±2.08和-2.60±1.62。帕米膦酸治疗1年后，腰椎BMD z评分显著改善，但股骨颈BMD z评分未改善。然而，到治疗结束时，两个z评分均显著增加。
本研究表明，帕米膦酸治疗可增加儿童骨质疏松症患者的骨密度，且无明显不良事件。需要进一步研究以更好地确定帕米膦酸治疗在大量儿科患者中的长期疗效和安全性。
