Division of Endocrinology, Department of Pediatrics, University of Ottawa, Ottawa, Ontario, Canada.
Division of Endocrinology, Department of Pediatrics, Nationwide Children's Hospital, The Ohio State University College of Medicine, Columbus, Ohio, USA.
Horm Res Paediatr. 2022;95(4):354-362. doi: 10.1159/000524994. Epub 2022 May 13.
Pediatric metabolic bone and mineral disorders encompass a wide variety of disorders that can be challenging to diagnose and treat because of inadequate physician training about optimal management.
As practice variation and confidence levels may impact clinical outcome, we sought to assess physician confidence in managing pediatric metabolic bone and mineral disorders and the spectrum of treatment practices among members of the Pediatric Endocrine Society (PES) and the Canadian Pediatric Endocrine Group (CPEG). Questionnaires were distributed via e-mail to all members of the PES and CPEG and 244 were completed. Responses were summarized using descriptive statistics, and proportions were compared using χ2 or Fisher's exact tests, as appropriate.
Variations were observed among the respondents' confidence in the management of bone disorders and in the criteria used to initiate/discontinue intravenous bisphosphonates or prescribe burosumab therapy. Respondents felt confident with the management of 4 out of 20 pediatric bone conditions (confidence was defined as >90% of respondents reporting feeling "somewhat confident" or "very confident"). Physicians working in a bone clinic were more confident in prescribing burosumab for the treatment of X-linked hypophosphatemic rickets compared to those not working in a bone clinic (65% vs. 47%, p = 0.03). Most respondents (52%) reported having received inadequate training in pediatric metabolic bone and mineral disorders.
DISCUSSION/CONCLUSION: Dedicated training, knowledge acquisition, and education resources are needed to increase confidence and standardize the use of bone-targeted therapies.
儿科代谢性骨和矿物质疾病涵盖了广泛的疾病，由于医生对最佳管理的培训不足，这些疾病的诊断和治疗可能具有挑战性。
由于实践差异和信心水平可能会影响临床结果，我们试图评估儿科内分泌学会（PES）和加拿大儿科内分泌组（CPEG）成员管理儿科代谢性骨和矿物质疾病的信心水平以及治疗实践的范围。调查问卷通过电子邮件分发给 PES 和 CPEG 的所有成员，共完成了 244 份。使用描述性统计数据总结回答，使用 χ2 或 Fisher 精确检验比较比例，视情况而定。
在管理骨骼疾病的信心和启动/停止静脉双膦酸盐或开布鲁索马治疗的标准方面，受访者之间存在差异。受访者对 20 种儿科骨骼疾病中的 4 种疾病的管理有信心（信心定义为> 90%的受访者报告感到“有些自信”或“非常自信”）。在骨诊所工作的医生比不在骨诊所工作的医生更有信心开布鲁索马治疗 X 连锁低磷血症性佝偻病（65%比 47%，p = 0.03）。大多数受访者（52%）报告说他们在儿科代谢性骨和矿物质疾病方面接受的培训不足。
讨论/结论：需要专门的培训、知识获取和教育资源，以提高信心并标准化使用骨靶向治疗。
