Kyowa Kirin Presents Real-World Evidence Demonstrating Clinically Meaningful Impact of Burosumab Treatment in Adults with X-linked Hypophosphatemia
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Findings from
UK burosumab early access program spotlighted in oral presentation at ASBMR annual meeting - Statistically significant improvements seen in patient-reported measures of pain, stiffness, physical function, and health-related quality of life
GALASHIELS & MARLOW,
“The improvements we observed in patient-reported symptoms and health-related quality of life provide additional evidence of the real-world impact of treatment on outcomes relevant to patients and clinicians,” said Judith Bubbear, MD, at the
XLH is a rare genetic, progressive, metabolic bone disease that causes skeletal abnormalities, stiffness, pain, and impaired physical function.1 In this retrospective, longitudinal, real-world study, researchers assessed the experience of 136 burosumab-naïve adults with XLH enrolled in the
Investigators observed statistically significant (p<0.05) and clinically relevant improvements in mean patient-reported outcomes after six and 12 months of burosumab treatment, including:
- Brief Pain Inventory-Short Form (BPI-SF) domains: Worst Pain, Pain Severity, and Pain Interference.
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Western Ontario and McMaster Universities Arthritis Index (WOMAC) for Stiffness, Physical Function, Pain, and Total Score. - EuroQol 5-Dimension (EQ-5D) 5-Level utility and visual analogue scale scores, measures of health-related quality of life.
“XLH is a lifelong, progressive disease, and
About X-linked hypophosphataemia (XLH)
XLH is caused by a genetic mutation which leads to overexpression of the protein FGF23, a protein involved in the regulation of phosphate concentration in the blood. In XLH, FGF23 is produced in excess leading to depletion of phosphate in the blood, known as hypophosphataemia.1
Individuals living with the disease may display a multitude of symptoms including short stature, limb deformities, bone and joint pain, oral abscesses, and hearing loss.1 To manage this wide variety of symptoms, the disease is managed through multi-disciplinary teams.2
About CRYSVITA (burosumab)
CRYSVITA (burosumab) is a recombinant human monoclonal antibody (mAb) that binds to the protein fibroblast growth factor 23 (FGF23). This has the impact of inhibiting the action of FGF23, allowing phosphate regulation in the body to be restored.3
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References
1Beck-Nielsen SS, et al. 2019. FGF23 and its role in X-linked hypophosphatemia-related morbidity.
2Kubota T. X-linked hypophosphatemia transition and team management. Endocrines. 2022;3(3):411-418.
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Stacey.Minton@kyowakirin.com
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