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      Burosumab for the Treatment of Tumor‐Induced Osteomalacia

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          ABSTRACT

          Tumor‐induced osteomalacia (TIO) is caused by phosphaturic mesenchymal tumors producing fibroblast growth factor 23 (FGF23) and is characterized by impaired phosphate metabolism, skeletal health, and quality of life. UX023T‐CL201 is an ongoing, open‐label, phase 2 study investigating the safety and efficacy of burosumab, a fully human monoclonal antibody that inhibits FGF23, in adults with TIO or cutaneous skeletal hypophosphatemia syndrome (CSHS). Key endpoints were changes in serum phosphorus and osteomalacia assessed by transiliac bone biopsies at week 48. This report focuses on 14 patients with TIO, excluding two diagnosed with X‐linked hypophosphatemia post‐enrollment and one with CSHS. Serum phosphorus increased from baseline (0.52 mmol/L) and was maintained after dose titration from week 22 (0.91 mmol/L) to week 144 (0.82 mmol/L, p < 0.0001). Most measures of osteomalacia were improved at week 48: osteoid volume/bone, osteoid thickness, and mineralization lag time decreased; osteoid surface/bone surface showed no change. Of 249 fractures/pseudofractures detected across 14 patients at baseline, 33% were fully healed and 13% were partially healed at week 144. Patients reported a reduction in pain and fatigue and an increase in physical health. Two patients discontinued: one to treat an adverse event (AE) of neoplasm progression and one failed to meet dosing criteria (receiving minimal burosumab). Sixteen serious AEs occurred in seven patients, and there was one death; all serious AEs were considered unrelated to treatment. Nine patients had 16 treatment‐related AEs; all were mild to moderate in severity. In adults with TIO, burosumab exhibited an acceptable safety profile and was associated with improvements in phosphate metabolism and osteomalacia. © 2020 The Authors. Journal of Bone and Mineral Research published by American Society for Bone and Mineral Research..

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          Poorly controlled cancer pain is a significant public health problem throughout the world. There are many barriers that lead to undertreatment of cancer pain. One important barrier is inadequate measurement and assessment of pain. To address this problem, the Pain Research Group of the WHO Collaborating Centre for Symptom Evaluation in Cancer Care has developed the Brief Pain Inventory (BPI), a pain assessment tool for use with cancer patients. The BPI measures both the intensity of pain (sensory dimension) and interference of pain in the patient's life (reactive dimension). It also queries the patient about pain relief, pain quality, and patient perception of the cause of pain. This paper describes the development of the Brief Pain Inventory and the various applications to which the BPI is suited. The BPI is a powerful tool and, having demonstrated both reliability and validity across cultures and languages, is being adopted in many countries for clinical pain assessment, epidemiological studies, and in studies of the effectiveness of pain treatment.
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                Author and article information

                Contributors
                sjandebe@jhmi.edu
                Journal
                J Bone Miner Res
                J Bone Miner Res
                10.1002/(ISSN)1523-4681
                JBMR
                Journal of Bone and Mineral Research
                John Wiley & Sons, Inc. (Hoboken, USA )
                0884-0431
                1523-4681
                12 January 2021
                April 2021
                : 36
                : 4 ( doiID: 10.1002/jbmr.v36.4 )
                : 627-635
                Affiliations
                [ 1 ] Johns Hopkins University School of Medicine Baltimore MD USA
                [ 2 ] Colorado Center for Bone Research Lakewood CO USA
                [ 3 ] Duke University Durham NC USA
                [ 4 ] Indiana University School of Medicine Indianapolis IN USA
                [ 5 ] Yale University School of Medicine New Haven CT USA
                [ 6 ] Mayo Clinic College of Medicine Rochester MN USA
                [ 7 ] McGill University Montreal Canada
                [ 8 ] Ultragenyx Pharmaceutical Inc. Novato CA USA
                Author notes
                [*] [* ] Address correspondence to: Suzanne M Jan de Beur, MD, 5501 Hopkins Bayview Circle JHAAC 3B.75 Baltimore, MD 21224. E‐mail: sjandebe@ 123456jhmi.edu

                Author information
                https://orcid.org/0000-0001-6973-5679
                Article
                JBMR4233
                10.1002/jbmr.4233
                8247961
                33338281
                d370d006-c528-41c5-b31e-21209576804f
                © 2020 The Authors. Journal of Bone and Mineral Research published by American Society for Bone and Mineral Research.

                This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.

                History
                : 01 December 2020
                : 30 June 2020
                : 10 December 2020
                Page count
                Figures: 2, Tables: 3, Pages: 9, Words: 6952
                Funding
                Funded by: Kyowa Kirin Co. LDT
                Funded by: Ultragenyx Pharmaceutical , open-funder-registry 10.13039/100013220;
                Categories
                Clinical Trial
                Clinical Trial
                Custom metadata
                2.0
                April 2021
                Converter:WILEY_ML3GV2_TO_JATSPMC version:6.0.2 mode:remove_FC converted:01.07.2021

                Human biology
                bone histomorphometry,tumor‐induced bone disease,clinical trials,osteomalacia and rickets,pth/vit d/fgf23

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