Impact of Stopping Burosumab Treatment at the End of Skeletal Growth in Adolescents with X-linked Hypophosphatemia (XLH)

Overview

Journal Bone Rep

Date 2024 Dec 16

PMID 39679164

Authors

Charlotte Jarvis

Renuka Ramakrishnan

Poonam Dharmaraj

Talat Mushtaq

Sanjay Gupta

Angela Williams

Angela J Rylands

Helen Barham

Annabel Nixon

Suma Uday

Affiliations

Soon will be listed here.

Abstract

Many adolescents with X-linked hypophosphatemia (XLH) currently have to stop treatment with burosumab at the end of skeletal growth. We describe the experience of a cohort of adolescents with XLH before, during, and after stopping burosumab (median treatment duration 37.5 months). Improvements in serum phosphate, pain, mobility, function, and quality of life noted during burosumab treatment were reversed after treatment cessation. Further real-world data are needed to explore the value of uninterrupted burosumab treatment in adolescents.

References

Skrinar A, Dvorak-Ewell M, Evins A, Macica C, Linglart A, Imel E . The Lifelong Impact of X-Linked Hypophosphatemia: Results From a Burden of Disease Survey. J Endocr Soc. 2019; 3(7):1321-1334. PMC: 6595532. DOI: 10.1210/js.2018-00365. View

Ferizovic N, Marshall J, Williams A, Mughal M, Shaw N, Mak C . Exploring the Burden of X-Linked Hypophosphataemia: An Opportunistic Qualitative Study of Patient Statements Generated During a Technology Appraisal. Adv Ther. 2019; 37(2):770-784. PMC: 7004427. DOI: 10.1007/s12325-019-01193-0. View

Beck-Nielsen S, Mughal Z, Haffner D, Nilsson O, Levtchenko E, Ariceta G . FGF23 and its role in X-linked hypophosphatemia-related morbidity. Orphanet J Rare Dis. 2019; 14(1):58. PMC: 6390548. DOI: 10.1186/s13023-019-1014-8. View

Cheung M, Rylands A, Williams A, Bailey K, Bubbear J . Patient-Reported Complications, Symptoms, and Experiences of Living With X-Linked Hypophosphatemia Across the Life-Course. J Endocr Soc. 2021; 5(8):bvab070. PMC: 8272533. DOI: 10.1210/jendso/bvab070. View

Padidela R, Whyte M, Glorieux F, Munns C, Ward L, Nilsson O . Patient-Reported Outcomes from a Randomized, Active-Controlled, Open-Label, Phase 3 Trial of Burosumab Versus Conventional Therapy in Children with X-Linked Hypophosphatemia. Calcif Tissue Int. 2021; 108(5):622-633. PMC: 8064984. DOI: 10.1007/s00223-020-00797-x. View

Baroncelli G, Grandone A, Aversa A, Sessa M, Pelosini C, Michelucci A . Safety and efficacy of burosumab in improving phosphate metabolism, bone health, and quality of life in adolescents with X-linked hypophosphatemic rickets. Eur J Med Genet. 2024; 70:104958. DOI: 10.1016/j.ejmg.2024.104958. View

Trombetti A, Al-Daghri N, Brandi M, Cannata-Andia J, Cavalier E, Chandran M . Interdisciplinary management of FGF23-related phosphate wasting syndromes: a Consensus Statement on the evaluation, diagnosis and care of patients with X-linked hypophosphataemia. Nat Rev Endocrinol. 2022; 18(6):366-384. DOI: 10.1038/s41574-022-00662-x. View

Romagnoli C, Iantomasi T, Brandi M . Impact of X-Linked Hypophosphatemia on Muscle Symptoms. Genes (Basel). 2022; 13(12). PMC: 9778127. DOI: 10.3390/genes13122415. View

Briot K, Portale A, Brandi M, Carpenter T, Cheong H, Cohen-Solal M . Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension. RMD Open. 2021; 7(3). PMC: 8458321. DOI: 10.1136/rmdopen-2021-001714. View

10.

Imel E, Glorieux F, Whyte M, Munns C, Ward L, Nilsson O . Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial. Lancet. 2019; 393(10189):2416-2427. PMC: 7179969. DOI: 10.1016/S0140-6736(19)30654-3. View

11.

Insogna K, Briot K, Imel E, Kamenicky P, Ruppe M, Portale A . A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial Evaluating the Efficacy of Burosumab, an Anti-FGF23 Antibody, in Adults With X-Linked Hypophosphatemia: Week 24 Primary Analysis. J Bone Miner Res. 2018; 33(8):1383-1393. DOI: 10.1002/jbmr.3475. View

12.

Chen Y, Kao T, Chou C, Wu C, Yang H, Lai C . Exploring the Link between Serum Phosphate Levels and Low Muscle Strength, Dynapenia, and Sarcopenia. Sci Rep. 2018; 8(1):3573. PMC: 5824959. DOI: 10.1038/s41598-018-21784-1. View

13.

Linglart A, Biosse-Duplan M, Briot K, Chaussain C, Esterle L, Guillaume-Czitrom S . Therapeutic management of hypophosphatemic rickets from infancy to adulthood. Endocr Connect. 2014; 3(1):R13-30. PMC: 3959730. DOI: 10.1530/EC-13-0103. View

14.

Cremonesi I, Nucci C, DAlessandro G, Alkhamis N, Marchionni S, Piana G . X-linked hypophosphatemic rickets: enamel abnormalities and oral clinical findings. Scanning. 2014; 36(4):456-61. DOI: 10.1002/sca.21141. View

15.

Kamenicky P, Briot K, Brandi M, Cohen-Solal M, Crowley R, Keen R . Benefit of burosumab in adults with X-linked hypophosphataemia (XLH) is maintained with long-term treatment. RMD Open. 2023; 9(1). PMC: 9980374. DOI: 10.1136/rmdopen-2022-002676. View

16.

Mughal M, Baroncelli G, de Lucas-Collantes C, Linglart A, Magnolato A, Raimann A . Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries. Front Endocrinol (Lausanne). 2023; 13:1034580. PMC: 9928183. DOI: 10.3389/fendo.2022.1034580. View

17.

Ariceta G, Beck-Nielsen S, Boot A, Brandi M, Briot K, Collantes C . The International X-Linked Hypophosphatemia (XLH) Registry: first interim analysis of baseline demographic, genetic and clinical data. Orphanet J Rare Dis. 2023; 18(1):304. PMC: 10523658. DOI: 10.1186/s13023-023-02882-4. View

18.

Padidela R, Nilsson O, Makitie O, Beck-Nielsen S, Ariceta G, Schnabel D . The international X-linked hypophosphataemia (XLH) registry (NCT03193476): rationale for and description of an international, observational study. Orphanet J Rare Dis. 2020; 15(1):172. PMC: 7329472. DOI: 10.1186/s13023-020-01434-4. View

19.

Orlando G, Bubbear J, Clarke S, Keen R, Roy M, Anilkumar A . Physical function and physical activity in adults with X-linked hypophosphatemia. Osteoporos Int. 2022; 33(7):1485-1491. DOI: 10.1007/s00198-022-06318-w. View

20.

Portale A, Carpenter T, Brandi M, Briot K, Cheong H, Cohen-Solal M . Continued Beneficial Effects of Burosumab in Adults with X-Linked Hypophosphatemia: Results from a 24-Week Treatment Continuation Period After a 24-Week Double-Blind Placebo-Controlled Period. Calcif Tissue Int. 2019; 105(3):271-284. DOI: 10.1007/s00223-019-00568-3. View