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Effect of Burosumab Compared With Conventional Therapy on Younger vs Older Children With X-linked Hypophosphatemia
DC Field | Value | Language |
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dc.contributor.author | Ward, Leanne M. | - |
dc.contributor.author | Glorieux, Francis H. | - |
dc.contributor.author | Whyte, Michael P. | - |
dc.contributor.author | Munns, Craig F. | - |
dc.contributor.author | Portale, Anthony A. | - |
dc.contributor.author | Hoegler, Wolfgang | - |
dc.contributor.author | Simmons, Jill H. | - |
dc.contributor.author | Gottesman, Gary S. | - |
dc.contributor.author | Padidela, Raja | - |
dc.contributor.author | Namba, Noriyuki | - |
dc.contributor.author | Cheong, Hae Il | - |
dc.contributor.author | Nilsson, Ola | - |
dc.contributor.author | Mao, Meng | - |
dc.contributor.author | Chen, Angel | - |
dc.contributor.author | Skrinar, Alison | - |
dc.contributor.author | Roberts, Mary Scott | - |
dc.contributor.author | Imel, Erik A. | - |
dc.date.accessioned | 2022-10-12T00:54:31Z | - |
dc.date.available | 2022-10-12T00:54:31Z | - |
dc.date.created | 2022-08-17 | - |
dc.date.issued | 2022-08 | - |
dc.identifier.citation | Journal of Clinical Endocrinology and Metabolism, Vol.107 No.8, pp.E3241-E3253 | - |
dc.identifier.issn | 0021-972X | - |
dc.identifier.uri | https://hdl.handle.net/10371/185909 | - |
dc.description.abstract | Context Younger age at treatment onset with conventional therapy (phosphate salts and active vitamin D; Pi/D) is associated with improved growth and skeletal outcomes in children with X-linked hypophosphatemia (XLH). The effect of age on burosumab efficacy and safety in XLH is unknown. Objective This work aimed to explore the efficacy and safety of burosumab vs Pi/D in younger (< 5 years) and older (5-12 years) children with XLH. Methods This post hoc analysis of a 64-week, open-label, randomized controlled study took place at 16 academic centers. Sixty-one children aged 1 to 12 years with XLH (younger, n = 26; older, n = 35) participated. Children received burosumab starting at 0.8 mg/kg every 2 weeks (younger, n = 14; older, n = 15) or continued Pi/D individually titrated per recommended guidelines (younger, n = 12; older, n = 20). The main outcome measure included the least squares means difference (LSMD) in Radiographic Global Impression of Change (RGI-C) rickets total score from baseline to week 64. Results The LSMD in outcomes through 64 weeks on burosumab vs conventional therapy by age group were as follows: RGI-C rickets total score (younger, +0.90; older, +1.07), total Rickets Severity Score (younger, -0.86; older, -1.44), RGI-C lower limb deformity score (younger, +1.02; older, +0.91), recumbent length or standing height Z-score (younger, +0.20; older, +0.09), and serum alkaline phosphatase (ALP) (younger, -31.15% of upper normal limit [ULN]; older, -52.11% of ULN). On burosumab, dental abscesses were not reported in younger children but were in 53% of older children. Conclusion Burosumab appears to improve outcomes both in younger and older children with XLH, including rickets, lower limb deformities, growth, and ALP, compared with Pi/D. | - |
dc.language | 영어 | - |
dc.publisher | The Endocrine Society | - |
dc.title | Effect of Burosumab Compared With Conventional Therapy on Younger vs Older Children With X-linked Hypophosphatemia | - |
dc.type | Article | - |
dc.identifier.doi | 10.1210/clinem/dgac296 | - |
dc.citation.journaltitle | Journal of Clinical Endocrinology and Metabolism | - |
dc.identifier.wosid | 000805283400001 | - |
dc.identifier.scopusid | 2-s2.0-85134428269 | - |
dc.citation.endpage | E3253 | - |
dc.citation.number | 8 | - |
dc.citation.startpage | E3241 | - |
dc.citation.volume | 107 | - |
dc.description.isOpenAccess | N | - |
dc.contributor.affiliatedAuthor | Cheong, Hae Il | - |
dc.type.docType | Article | - |
dc.description.journalClass | 1 | - |
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