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Understanding motivational drivers and barriers to patient participation in diabetes research are important to ensure research is relevant and valuable. Young adults with type 1 diabetes (T1D) completed a 31-question qualitative survey evaluating participant experience, understanding, and motivators and barriers to research involvement. A total of 35 participants, 19–28 years of age, 60% female, completed the survey. Motivating factors included personal benefit, relationship with the study team, curiosity, financial compensation, altruism, and nostalgia. Older participants (>22 years) reported higher levels of trust in the study team (p = 0.02) and their relationship with the study team positively influenced their decision to participate (p = 0.03). Financial compensation was a strong motivator for participants with higher education (p = 0.02). Age, sex, education level, and trust in the study team influenced participants’ understanding. Barriers included logistics and lack of familial support. Important motivational drivers and barriers to participation in research by young adults with T1D must be considered to increase research engagement and facilitate the discovery of new knowledge.

Purpose: Physical activity (PA) is an important determinant of skeletal health. In young adults with type 1 diabetes (T1D) fracture risk is increased, yet few studies have examined the PA and bone health relationship. Therefore, this pilot cross-sectional study characterized PA levels and their association with bone parameters measured by high resolution peripheral quantitative computed tomography (HR-pQCT) in young adults with T1D., Methods: HR-pQCT (Xtreme CTII) was used to measure bone outcomes at the distal tibia and radius, and accelerometery (ActiGraph GT3X) recorded daily minutes of light and moderate-vigorous physical activity (MVPA). Quadratic regression analyses were conducted with a p -value ≤ 0.05 considered significant., Results: PA data from 19 young adults (23.1 ± 1.9 years) with T1D was analyzed. Over half (63 %) of participants completed ≥150 min of MVPA per week, however, most measured activity time per day (57 %) was spent in sedentary pursuits. Significant non-linear associations were found between the duration of MVPA and several trabecular bone parameters at the tibia., Conclusions: In young adults with T1D, MVPA may have site specific (tibia) and compartment specific (trabecular) non-linear associations with bone. Further studies should confirm these findings, which may help inform evidence-based exercise recommendations to optimize bone health in young adults with T1D., Competing Interests: The authors (Sarah L West, Michelle Furman, Rahim Moineddin, and Etienne Sochett) have no conflicts of interest, or competing interest to declare., (© 2024 The Authors. Published by Elsevier Inc.)

Context: In an open-label, randomized, controlled, phase 3 trial in 61 children aged 1 to 12 years with X-linked hypophosphatemia (XLH), burosumab improved rickets vs continuing conventional therapy with active vitamin D and phosphate., Objective: We conducted an analysis to determine whether skeletal responses differed when switching to burosumab vs continuing higher or lower doses of conventional therapy., Methods: Conventional therapy dose groups were defined as higher-dose phosphate [greater than 40 mg/kg] (HPi), lower-dose phosphate [40 mg/kg or less] (LPi), higher-dose alfacalcidol [greater than 60 ng/kg] or calcitriol [greater than 30 ng/kg] (HD), and lower-dose alfacalcidol [60 ng/kg or less] or calcitriol [30 ng/kg or less] (LD)., Results: At week 64, the Radiographic Global Impression of Change (RGI-C) for rickets was higher (better) in children randomly assigned to burosumab vs conventional therapy for all prebaseline dose groups: HPi (+1.72 vs +0.67), LPi (+2.14 vs +1.08), HD (+1.90 vs +0.94), LD (+2.11 vs +1.06). At week 64, the RGI-C for rickets was also higher in children randomly assigned to burosumab (+2.06) vs conventional therapy for all on-study dose groups: HPi (+1.03), LPi (+1.05), HD (+1.45), LD (+0.72). Serum alkaline phosphatase (ALP) also decreased in the burosumab-treated patients more than in the conventional therapy group, regardless of on-study phosphate and active vitamin D doses., Conclusion: Prior phosphate or active vitamin D doses did not influence treatment response after switching to burosumab among children with XLH and active radiographic rickets. Switching from conventional therapy to burosumab improved rickets and serum ALP more than continuing either higher or lower doses of phosphate or active vitamin D., (© The Author(s) 2023. Published by Oxford University Press on behalf of the Endocrine Society.)

Idiopathic Infantile Hypercalcemia (IIH) is characterized by hypercalcemia and hypercalciuria owing to PTH-independent increases in circulating concentrations of 1,25(OH)2D. At least 3 forms of IHH can be distinguished genetically and mechanistically: infantile hypercalcemia-1 (Hypercalcemia, Infantile, 1; HCINF1) due to CYP24A1 mutations results in decreased inactivation of 1,25(OH)2D; HCINF2 due to SLC34A1 mutations results in excessive 1,25(OH)2D production; and HCINF3 in which a variety of gene variants of uncertain significance (VUS) have been identified and where the mechanism for increased 1,25 (OH)2D is unclear. Conventional management with dietary calcium and vitamin D restriction has only limited success. Induction of the P450 enzyme CYP3A4 by rifampin can provide an alternate pathway for inactivation of 1,25(OH)2D that is useful in HCINF1 and may be effective in other forms of IIH. We sought to assess the efficacy of rifampin to decrease levels of serum 1,25(OH)2D and calcium, and urinary calcium concentrations in subjects with HCINF3, and to compare the response to a control subject with HCINF1. Four subjects with HCINF3 and the control subject with HCINF1 completed the study using rifampin 5 mg/kg/day and 10 mg/kg/day each for 2 months separated by a 2-month washout period. Patients had age-appropriate intake of dietary calcium plus 200 IU vitamin D/day. Primary outcome was efficacy of rifampin to lower serum concentrations of 1,25(OH)2D. The secondary outcomes included the reduction of serum calcium, urinary calcium excretion (as random urine calcium: creatinine (ca:cr) ratio) and serum 1,25(OH)2D/PTH ratio. Rifampin was well tolerated and induced CYP3A4 at both doses in all subjects. The control subject with HCINF1 showed significant response to both rifampin doses with decreases in the serum concentration of 1,25(OH)2D and the 1,25(OH)2D/PTH ratio while the serum and urine ca:cr levels were unchanged. The four patients with HCINF3 showed reductions in 1,25(OH)2D and urinary ca:cr after 10 mg/kg/d, but hypercalcemia did not improve and there were variable responses in 1,25(OH)2D/PTH ratios. These results support further longer-term studies to clarify the usefulness of rifampin as a medical therapy for IIH., Competing Interests: Declaration of competing interest None., (Copyright © 2023 The Authors. Published by Elsevier Ltd.. All rights reserved.)

Understanding motivational drivers and barriers to patient participation in diabetes research are important to ensure research is relevant and valuable. Young adults with type 1 diabetes (T1D) completed a 31-question qualitative survey evaluating participant experience, understanding, and motivators and barriers to research involvement. A total of 35 participants, 19-28 years of age, 60% female, completed the survey. Motivating factors included personal benefit, relationship with the study team, curiosity, financial compensation, altruism, and nostalgia. Older participants (>22 years) reported higher levels of trust in the study team (p = 0.02) and their relationship with the study team positively influenced their decision to participate (p = 0.03). Financial compensation was a strong motivator for participants with higher education (p = 0.02). Age, sex, education level, and trust in the study team influenced participants' understanding. Barriers included logistics and lack of familial support. Important motivational drivers and barriers to participation in research by young adults with T1D must be considered to increase research engagement and facilitate the discovery of new knowledge., Competing Interests: The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article., (© The Author(s) 2022.)

Osteosarcoma is the most common pediatric malignant bone tumor. Concomitant osteoporosis has typically been attributed to oncologic therapy. The present case series is aimed to describe 3 patients who presented with osteoporosis or osteopenia before, or early in, their oncology treatment. In our patients, bone health and its complications had significant impacts including pain, reduced mobility, prolonged admission, and delays in recovery. Our patients experienced improvement with resection of their primary tumor and with bisphosphonate infusion. Future studies are required to determine the prevalence osteoporosis at presentation of osteosarcoma and the role of bisphosphonates., Competing Interests: The authors declare no conflict of interest., (Copyright © 2022 Wolters Kluwer Health, Inc. All rights reserved.)