Treatment Practices and Confidence in the Management of Pediatric Metabolic Bone Disorders

Abstract

<b><i>Introduction:</i></b> 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. <b><i>Methods:</i></b> 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. <b><i>Results:</i></b> 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 &#x3e;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%, <i>p</i> = 0.03). Most respondents (52%) reported having received inadequate training in pediatric metabolic bone and mineral disorders. <b><i>Discussion/Conclusion:</i></b> Dedicated training, knowledge acquisition, and education resources are needed to increase confidence and standardize the use of bone-targeted therapies.