دورية أكاديمية
Real-world effectiveness of burosumab in children with X-linked hypophosphatemic rickets.
| العنوان: | Real-world effectiveness of burosumab in children with X-linked hypophosphatemic rickets. |
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| المؤلفون: | Paloian NJ; Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, 600 Highland Ave, Madison, WI, 53792, USA. njpaloian@pediatrics.wisc.edu., Nemeth B; Department of Orthopedics and Rehabilitation, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA., Sharafinski M; Department of Radiology, Aurora Healthcare, Milwaukee, WI, USA., Modaff P; Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, 600 Highland Ave, Madison, WI, 53792, USA., Steiner RD; Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, 600 Highland Ave, Madison, WI, 53792, USA. |
| المصدر: | Pediatric nephrology (Berlin, Germany) [Pediatr Nephrol] 2022 Nov; Vol. 37 (11), pp. 2667-2677. Date of Electronic Publication: 2022 Feb 24. |
| نوع المنشور: | Journal Article |
| اللغة: | English |
| بيانات الدورية: | Publisher: Springer International Country of Publication: Germany NLM ID: 8708728 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1432-198X (Electronic) Linking ISSN: 0931041X NLM ISO Abbreviation: Pediatr Nephrol Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: Berlin : Springer International Original Publication: Berlin : Springer International, c1987- |
| مواضيع طبية MeSH: | Familial Hypophosphatemic Rickets*/drug therapy, Adolescent ; Antibodies, Monoclonal ; Antibodies, Monoclonal, Humanized ; Calcitriol/therapeutic use ; Child ; Child, Preschool ; Fibroblast Growth Factors ; Humans ; Infant ; Phosphates ; Retrospective Studies |
| مستخلص: | Background: X-linked hypophosphatemic rickets (XLH) is the most common cause of inherited rickets. Historically, XLH was treated with oral phosphate and calcitriol (conventional treatment). Burosumab, a fibroblast growth factor 23 (FGF-23) monoclonal antibody, was approved by the United States Food and Drug Administration (FDA) in 2018 for XLH treatment. Nevertheless, conventional treatment of XLH continues to be recommended by some specialists due to lack of published experience with burosumab in the clinical setting. We compared laboratory and radiographic changes observed following transition from conventional therapy to burosumab in pediatric XLH patients as part of routine care. Methods: This retrospective single-center study identified and retroactively studied twelve patients aged 1-18 years old with XLH previously treated with conventional therapy and transitioned to burosumab. Laboratory studies and radiographs were obtained routinely as standard of care during two treatment periods: (1) conventional therapy and (2) burosumab treatment. Laboratory values and radiologic rickets severity scores were compared between periods. Results: All laboratory values demonstrated improvement following 1 month of burosumab treatment, findings which were sustained over the 2-year study period. Rickets severity scores and height z-scores also improved with burosumab. There were no serious adverse events with burosumab, and adverse events overall were very infrequent and mild. One patient developed an asymptomatic mild elevation of serum phosphate while taking burosumab resulting in a temporary pause in therapy. Conclusions: Safety and effectiveness of burosumab in treatment of XLH were demonstrated as burosumab yielded statistically significant improvement in laboratory and radiographic markers of rickets and height compared to conventional therapy. A higher resolution version of the Graphical abstract is available as Supplementary information. (© 2022. The Author(s), under exclusive licence to International Pediatric Nephrology Association.) |
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| فهرسة مساهمة: | Keywords: Burosumab; FGF-23; Hypophosphatemia; Rickets; XLH |
| المشرفين على المادة: | 0 (Antibodies, Monoclonal) 0 (Antibodies, Monoclonal, Humanized) 0 (Phosphates) 62031-54-3 (Fibroblast Growth Factors) FXC9231JVH (Calcitriol) G9WJT6RD29 (burosumab) |
| تواريخ الأحداث: | Date Created: 20220225 Date Completed: 20220923 Latest Revision: 20221121 |
| رمز التحديث: | 20231215 |
| DOI: | 10.1007/s00467-022-05484-7 |
| PMID: | 35211790 |
| قاعدة البيانات: | MEDLINE |
Find this article in full text from Springer Verlag. 
Full Text Finder | تدمد: | 1432-198X |
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| DOI: | 10.1007/s00467-022-05484-7 |