Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients
X-linked hypophosphataemia (XLH) is a life-long phosphate-wasting disorder that causes skeletal deformities, pain, stiffness, and fatigue and impairs quality of life. Burosumab was approved for use in adults in 2020. We describe three adults with persistent XLH symptoms who received burosumab treatm...
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| Language: | English |
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Elsevier
2024-12-01
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| Series: | Bone Reports |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2352187224000810 |
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| author | Julia Day Chandrin Jayatilleke Matthew Roy |
| author_facet | Julia Day Chandrin Jayatilleke Matthew Roy |
| author_sort | Julia Day |
| collection | DOAJ |
| description | X-linked hypophosphataemia (XLH) is a life-long phosphate-wasting disorder that causes skeletal deformities, pain, stiffness, and fatigue and impairs quality of life. Burosumab was approved for use in adults in 2020. We describe three adults with persistent XLH symptoms who received burosumab treatment in a real-world setting. Patients report improvements in pain, mobility, physical function, energy, fatigue, and mental wellbeing through patient-reported outcome measures, enriched with further detail from written testimonials. |
| format | Article |
| id | doaj-art-64547b4b26184dd7ac6d3097478f722a |
| institution | Kabale University |
| issn | 2352-1872 |
| language | English |
| publishDate | 2024-12-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Bone Reports |
| spelling | doaj-art-64547b4b26184dd7ac6d3097478f722a2024-11-27T05:02:48ZengElsevierBone Reports2352-18722024-12-0123101814Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patientsJulia Day0Chandrin Jayatilleke1Matthew Roy2Department of Rheumatology, Bristol Royal Infirmary, Bristol, BS2 8HW, UK; Corresponding author.Department of Rheumatology, Royal United Hospital, Bath BA1 3NG, UKDepartment of Rheumatology, Bristol Royal Infirmary, Bristol, BS2 8HW, UKX-linked hypophosphataemia (XLH) is a life-long phosphate-wasting disorder that causes skeletal deformities, pain, stiffness, and fatigue and impairs quality of life. Burosumab was approved for use in adults in 2020. We describe three adults with persistent XLH symptoms who received burosumab treatment in a real-world setting. Patients report improvements in pain, mobility, physical function, energy, fatigue, and mental wellbeing through patient-reported outcome measures, enriched with further detail from written testimonials.http://www.sciencedirect.com/science/article/pii/S2352187224000810BurosumabPatient-reported outcomeCase seriesEarly accessX-linked hypophosphatemia |
| spellingShingle | Julia Day Chandrin Jayatilleke Matthew Roy Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients Bone Reports Burosumab Patient-reported outcome Case series Early access X-linked hypophosphatemia |
| title | Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients |
| title_full | Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients |
| title_fullStr | Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients |
| title_full_unstemmed | Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients |
| title_short | Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients |
| title_sort | improvements with burosumab treatment in an early access programme for adults with x linked hypophosphataemia a case series of three patients |
| topic | Burosumab Patient-reported outcome Case series Early access X-linked hypophosphatemia |
| url | http://www.sciencedirect.com/science/article/pii/S2352187224000810 |
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