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Efficacy of Burosumab in Adults with X-linked Hypophosphatemia (XLH): A Post Hoc Subgroup Analysis of a Randomized Double-Blind Placebo-Controlled Phase 3 Study.

Publication ,  Journal Article
Brandi, ML; Jan de Beur, S; Briot, K; Carpenter, T; Cheong, HI; Cohen-Solal, M; Crowley, RK; Eastell, R; Imanishi, Y; Imel, EA; Ing, SW ...
Published in: Calcif Tissue Int
October 2022

The anti-fibroblast growth factor 23 monoclonal antibody burosumab corrects hypophosphatemia in adults with X-linked hypophosphatemia (XLH) and improves pain, stiffness, physical function, and fatigue. This post hoc subgroup analysis used data from the 24-week placebo-controlled period of a phase 3 study in 134 adults with XLH (ClinicalTrials.gov NCT02526160), to assess whether the benefits of burosumab are evident in 14 clinically relevant subgroups defined by baseline demographic and functional criteria, including sex, Brief Pain Inventory-short form (BPI-SF) Average And Worst Pain, region, race, Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC®) Stiffness, Physical Function and Pain domains and total score, use of opioid/other pain medication, active fractures/pseudo-fractures, and 6-min walk test distance. There were no statistically significant interactions between any of the subgroups and treatment arm for any endpoint. Higher proportions of subjects achieved mean serum phosphate concentration above the lower limit of normal (the primary endpoint) with burosumab than with placebo in all subgroups. For the key secondary endpoints (WOMAC Stiffness and Physical Function; BPI-SF Worst Pain) individual subgroup categories showed improvements with burosumab relative to placebo. For additional efficacy endpoints, burosumab was favored in some subgroups but differences were not significant and confidence intervals were wide. For some endpoints the treatment effect is small at 24 weeks in all subjects. This subgroup analysis shows that burosumab was largely superior to placebo across endpoints in the 14 clinically relevant subgroup variables at 24 weeks and is likely to benefit all symptomatic adults with active XLH.

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Published In

Calcif Tissue Int

DOI

EISSN

1432-0827

Publication Date

October 2022

Volume

111

Issue

4

Start / End Page

409 / 418

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Pain
  • Humans
  • Familial Hypophosphatemic Rickets
  • Endocrinology & Metabolism
  • Double-Blind Method
  • Antibodies, Monoclonal, Humanized
  • Adult
  • 4003 Biomedical engineering
  • 3202 Clinical sciences
 

Citation

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Brandi, M. L., Jan de Beur, S., Briot, K., Carpenter, T., Cheong, H. I., Cohen-Solal, M., … Takeuchi, Y. (2022). Efficacy of Burosumab in Adults with X-linked Hypophosphatemia (XLH): A Post Hoc Subgroup Analysis of a Randomized Double-Blind Placebo-Controlled Phase 3 Study. Calcif Tissue Int, 111(4), 409–418. https://doi.org/10.1007/s00223-022-01006-7
Brandi, Maria Luisa, Suzanne Jan de Beur, Karine Briot, Thomas Carpenter, Hae Il Cheong, Martine Cohen-Solal, Rachel K. Crowley, et al. “Efficacy of Burosumab in Adults with X-linked Hypophosphatemia (XLH): A Post Hoc Subgroup Analysis of a Randomized Double-Blind Placebo-Controlled Phase 3 Study.Calcif Tissue Int 111, no. 4 (October 2022): 409–18. https://doi.org/10.1007/s00223-022-01006-7.
Brandi ML, Jan de Beur S, Briot K, Carpenter T, Cheong HI, Cohen-Solal M, et al. Efficacy of Burosumab in Adults with X-linked Hypophosphatemia (XLH): A Post Hoc Subgroup Analysis of a Randomized Double-Blind Placebo-Controlled Phase 3 Study. Calcif Tissue Int. 2022 Oct;111(4):409–18.
Brandi, Maria Luisa, et al. “Efficacy of Burosumab in Adults with X-linked Hypophosphatemia (XLH): A Post Hoc Subgroup Analysis of a Randomized Double-Blind Placebo-Controlled Phase 3 Study.Calcif Tissue Int, vol. 111, no. 4, Oct. 2022, pp. 409–18. Pubmed, doi:10.1007/s00223-022-01006-7.
Brandi ML, Jan de Beur S, Briot K, Carpenter T, Cheong HI, Cohen-Solal M, Crowley RK, Eastell R, Imanishi Y, Imel EA, Ing SW, Insogna K, Ito N, Javaid K, Kamenicky P, Keen R, Kubota T, Lachmann RH, Perwad F, Pitukcheewanont P, Portale A, Ralston SH, Tanaka H, Weber TJ, Yoo H-W, Sun W, Williams A, Nixon A, Takeuchi Y. Efficacy of Burosumab in Adults with X-linked Hypophosphatemia (XLH): A Post Hoc Subgroup Analysis of a Randomized Double-Blind Placebo-Controlled Phase 3 Study. Calcif Tissue Int. 2022 Oct;111(4):409–418.
Journal cover image

Published In

Calcif Tissue Int

DOI

EISSN

1432-0827

Publication Date

October 2022

Volume

111

Issue

4

Start / End Page

409 / 418

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Pain
  • Humans
  • Familial Hypophosphatemic Rickets
  • Endocrinology & Metabolism
  • Double-Blind Method
  • Antibodies, Monoclonal, Humanized
  • Adult
  • 4003 Biomedical engineering
  • 3202 Clinical sciences