ResearchPad - new-frontiers-in-bone-and-mineral-metabolism Default RSS Feed en-us © 2020 Newgen KnowledgeWorks <![CDATA[OR29-03 The Effects of Hormone Therapy on Premature Ovarian Failure Following Allogenic Hematopoietic Stem Cell Transplantation: A Single-Center Experience]]> Background: With increasing survival rates after hematopoietic stem cell transplantation (HSCT), it has become important to evaluate methods of improving patients’ quality of life. Most female patients of childbearing age experience premature ovarian failure after transplantation, which results in decreased quality of life and an increase in fracture risk due to rapid bone loss. We analyzed the effects of hormone therapy (HT) on serum follicle stimulating hormone (FSH), serum estradiol, and bone mineral density (BMD) in young female HSCT recipients. Methods: This retrospective cohort study included 234 female patients who underwent allogenic HSCT between April 2009 and April 2018 at our center. The maximum age at the time of transplantation was 40 years, and patients were followed up for at least 3 years. Of the 734 patients who were initially screened, 360 patients aged <18 years and 8 who were transferred to another institution after transplantation were excluded from the study. There were 93 patients who died within 3 years of transplantation, while 30 were lost to follow-up, and 9 were followed-up for less than 3 years. Changes in hormone levels and BMD, according to HT regimen, were evaluated in 234 patients. Results: The mean age at transplantation was 30.47 ± 6.55 years. Out of 234 patients, 170 (72.6%) patients received HT, starting treatment at a mean of 15.1 ± 8.2 months after transplantation. A significant increase in estradiol level was observed in patients receiving HT (p < 0.001); no difference was observed between the 3 different types of HT regimen (p = 0.534). After 2 years of HT, BMD was significantly increased at all measurement sites: lumbar spine 5.8 ± 6.26% (p < 0.001), femoral neck 3.4 ± 17.78% (p = 0.037), total hip 2.1 ± 7.15% (p = 0.001). Again, there was no difference in changes between the HT regimens (p = 0.646 for lumbar spine, p = 0.840 for femoral neck, and p = 0.855 for total hip). These changes were significant even in patients with graft versus host disease (GVHD) or steroid exposure. Conclusion: In patients with premature ovarian failure following allogenic HSCT, HT effectively lowered serum FSH and increased serum estradiol levels. HT significantly increased BMD regardless of the history of GVHD or steroid exposure. These changes in hormones and BMD were independent of the HT regimen.

<![CDATA[OR29-05 A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP): 12-Month Outcomes]]> 90% ankylosed across 15 major joints; total score range 0 to 30 [higher scores indicate more severe mobility limitations]) and the FOP Physical Function Questionnaire (FOP-PFQ; percent total score). Changes from Baseline at Month 12 were evaluated for new HO volume, CAJIS, and FOP-PFQ. Results: Of 114 participants (pts) with Baseline data, 99 (4 to 56 years at enrollment; mean 17 years of age; 56% male) also had a Month 12 assessment. A total of 93 pts had evaluable WBCT scans at Baseline and Month 12 and were included in the HO analysis.In total, 40% (37/93) of pts had new HO over 12 months; the mean volume of new HO in these pts was 57,706 mm3 (SD=100,079 mm3; median=20,753 mm3; range: 522 to 438,826 mm3). Of the pts with new HO, 65% (24/37) reported at least one flare-up (mean rate of 2.3 flare-ups/year).Over 12 months, 60% (56/93) of pts did not have new HO; 43% (24/56) of them reported at least one flare-up (mean rate of 1.8 flare-ups/year).Mean changes from Baseline in CAJIS and FOP-PFQ were minimal: CAJIS: 0.6 (SD=2.4; median=1.0; n=99) and FOP-PFQ: 4.4% (SD=11.2; median=3.7%; n=90); and were similar across pts with or without new HO. Conclusions: In participants with FOP, although deterioration of physical function is expected over a patient’s lifetime, CAJIS and FOP-PFQ scores did not worsen significantly in the relative short-term of this study. However, HO volume, quantified by WBCT, increased over the course of 12 months. These results show that measuring HO may be a viable way to monitor changes in FOP over short periods of time. ]]> <![CDATA[OR29-01 Long-Term Safety in Adults with X-Linked Hypophosphatemia (XLH) Treated with Burosumab, a Fully Human Monoclonal Antibody Against FGF23: Final Results of a Phase 3 Trial]]> <![CDATA[OR29-07 A Novel Estimate of Creatinine Excretion to Determine Adequacy of 24-Hr Urine Collection]]> 10% between the two consecutive collections were excluded. In the initial 115 pairs of inpatient 24-hour urine collections (50 female, 65 male) participants, creatinine excretion/BW fell outside the currently accepted reference ranges in >50% of collections. The proportion below the reference range increased with higher BMI. In this derivation dataset, linear regression models were then constructed to predict 24-hr urine creatinine excretion from race, sex, age, weight and height. Reliable prediction of observed 24-hr urine creatinine excretion was confirmed in a validation dataset that included 50 pairs of 24-hour urine samples similarly collected in an inpatient research setting.This new prediction model performed significantly better than the currently used reference ranges in a large outpatient dataset including 1,399 pairs of 24-hour urine collections. In women, actual creatinine excretion fell within the 95% prediction intervals for our derived equation in 90% of cases using the new interval vs 46% using the current reference range. The corresponding values were 90% and 33% in men. In both genders, the superiority of the new prediction over the current reference range was more pronounced at higher BMI.We therefore propose revision of currently used criteria to define adequacy of 24-hour urine collection to account for the impact of obesity. The proposed equation incorporates readily available demographic parameters to predict urine creatinine excretion. These findings have wide implications on patient care and research studies, and future studies should test this equation in different settings, diets, and populations. ]]> <![CDATA[OR29-06 Burosumab Improves Biochemical, Skeletal, and Clinical Features of Tumor-Induced Osteomalacia Syndrome]]> <![CDATA[OR29-02 Natural Language Processing of Radiology Reports Improves Identification of Patients with Fracture]]> 50y; bone involved; etc). At the development site, XRAIT was used to search the emergency patient presentations of people over 50 years of age and compared to referrals to FLS (usual care) during the same 3-month period. XRAIT analyzed all plain radiographs and CT scans (n = 5089) while n = 224 were referred to FLS for usual care. External validation: XRAIT was used to analyze digitally readable radiology reports in an untrained cohort from DOES (n = 327) to calculate sensitivity and specificity.Results: XRAIT identified a 5-fold higher number of potential significant fractures (349/5089) compared to manual case finding (70/224). 339/349 were confirmed fractures (97.1%). Only 29% of those eligible were started or recommended anti-resorptive therapy, including those seen by the fracture liaison service. XRAIT unadjusted for the local radiology reporting styles in DOES had a sensitivity of 69.6% and specificity of 95%. Conclusion: XRAIT identifies clinically significant fractures efficiently with minimal additional human resources. Its high specificity in an untrained cohort suggests it could be used at other sites. Automated methods of patient identification may assist fracture liaison services to identify fractures that still remain largely untreated. ]]> <![CDATA[OR29-04 TGFβ Regulates Bone Perilacunar/Canalicular Remodeling in a Sexually Dimorphic Manner]]>