Impact of stopping burosumab treatment at the end of skeletal growth in adolescents with X-linked hypophosphatemia (XLH)

被引:1
作者
Jarvis, Charlotte [1 ]
Ramakrishnan, Renuka [1 ]
Dharmaraj, Poonam [1 ]
Mushtaq, Talat [2 ]
Gupta, Sanjay [3 ]
Williams, Angela [4 ]
Rylands, Angela J. [4 ]
Barham, Helen [5 ]
Nixon, Annabel [5 ]
Uday, Suma [6 ]
机构
[1] Alder Hey Childrens NHS Fdn Trust, Liverpool L12 2AP, England
[2] Leeds Teaching Hosp NHS Trust, Leeds LS9 7TP, England
[3] Hull Univ Teaching Hosp NHS Trust, Kingston Upon Hull HU3 2JZ, England
[4] Kyowa Kirin Int Plc, Marlow SL7 1HZ, England
[5] Chilli Consultancy, Salisbury SP1 1JS, England
[6] Birmingham Womens & Childrens NHS Fdn Trust, Birmingham B15 2TG, England
来源
BONE REPORTS | 2025年 / 24卷
关键词
X -linked hypophosphatemia; Burosumab; Case series; RICKETS;
D O I
10.1016/j.bonr.2024.101819
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
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.
引用
收藏
页数:5
相关论文
共 20 条
[1]   The International X-Linked Hypophosphatemia (XLH) Registry: first interim analysis of baseline demographic, genetic and clinical data [J].
Ariceta, Gema ;
Beck-Nielsen, Signe Sparre ;
Boot, Annemieke M. ;
Brandi, Maria Luisa ;
Briot, Karine ;
Collantes, Carmen de Lucas ;
Emma, Francesco ;
Giannini, Sandro ;
Haffner, Dieter ;
Keen, Richard ;
Levtchenko, Elena ;
Makitie, Outi ;
Mughal, M. Zulf ;
Nilsson, Ola ;
Schnabel, Dirk ;
Tripto-Shkolnik, Liana ;
Liu, Jonathan ;
Williams, Angela ;
Wood, Sue ;
Zillikens, M. Carola .
ORPHANET JOURNAL OF RARE DISEASES, 2023, 18 (01)
[2]   Safety and efficacy of burosumab in improving phosphate metabolism, bone health, and quality of life in adolescents with X-linked hypophosphatemic rickets [J].
Baroncelli, Giampiero I. ;
Grandone, Anna ;
Aversa, Antonio ;
Sessa, Maria Rita ;
Pelosini, Caterina ;
Michelucci, Angela ;
Toschi, Benedetta ;
Manca, Mario ;
Isola, Alessandro ;
Comberiati, Pasquale .
EUROPEAN JOURNAL OF MEDICAL GENETICS, 2024, 70
[3]   FGF23 and its role in X-linked hypophosphatemia-related morbidity [J].
Beck-Nielsen, Signe Sparre ;
Mughal, Zulf ;
Haffner, Dieter ;
Nilsson, Ola ;
Levtchenko, Elena ;
Ariceta, Gema ;
Collantes, Carmen de Lucas ;
Schnabel, Dirk ;
Jandhyala, Ravi ;
Makitie, Outi .
ORPHANET JOURNAL OF RARE DISEASES, 2019, 14 (1)
[4]   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 [J].
Briot, Karine ;
Portale, Anthony A. ;
Brandi, Maria Luisa ;
Carpenter, Thomas O. ;
Cheong, Hae Ii ;
Cohen-Solal, Martine ;
Crowley, Rachel K. ;
Eastell, Richard ;
Imanishi, Yasuo ;
Ing, Steven ;
Insogna, Karl ;
Ito, Nobuaki ;
Jan de Beur, Suzanne ;
Javaid, Muhammad K. ;
Kamenicky, Peter ;
Keen, Richard ;
Kubota, Takuo ;
Lachmann, Robin H. ;
Perwad, Farzana ;
Pitukcheewanont, Pisit ;
Ralston, Stuart H. ;
Takeuchi, Yasuhiro ;
Tanaka, Hiroyuki ;
Weber, Thomas J. ;
Yoo, Han-Wook ;
Nixon, Annabel ;
Nixon, Mark ;
Sun, Wei ;
Williams, Angela ;
Imel, Erik A. .
RMD OPEN, 2021, 7 (03)
[5]   Exploring the Link between Serum Phosphate Levels and Low Muscle Strength, Dynapenia, and Sarcopenia [J].
Chen, Yuan-Yuei ;
Kao, Tung-Wei ;
Chou, Cheng-Wai ;
Wu, Chen-Jung ;
Yang, Hui-Fang ;
Lai, Ching-Huang ;
Wu, Li-Wei ;
Chen, Wei-Liang .
SCIENTIFIC REPORTS, 2018, 8
[6]   Patient-Reported Complications, Symptoms, and Experiences of Living With X-Linked Hypophosphatemia Across the Life-Course [J].
Cheung, Moira ;
Rylands, Angela J. ;
Williams, Angela ;
Bailey, Karen ;
Bubbear, Judith .
JOURNAL OF THE ENDOCRINE SOCIETY, 2021, 5 (08)
[7]   X-linked Hypophosphatemic Rickets: Enamel Abnormalities and Oral Clinical Findings [J].
Cremonesi, Ilaria ;
Nucci, Cesare ;
D'Alessandro, Giovanni ;
Alkhamis, Nadia ;
Marchionni, Silvia ;
Piana, Gabriela .
SCANNING, 2014, 36 (04) :456-461
[8]   Exploring the Burden of X-Linked Hypophosphataemia: An Opportunistic Qualitative Study of Patient Statements Generated During a Technology Appraisal [J].
Ferizovic, Nermina ;
Marshall, Jade ;
Williams, Angela E. ;
Mughal, M. Zulf ;
Shaw, Nicholas ;
Mak, Catherine ;
Gardiner, Oliver ;
Hossain, Pushpa ;
Upadhyaya, Sheela .
ADVANCES IN THERAPY, 2020, 37 (02) :770-784
[9]   Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial [J].
Imel, Erik A. ;
Glorieux, Francis H. ;
Whyte, Michael P. ;
Munns, Craig F. ;
Ward, Leanne M. ;
Nilsson, Ola ;
Simmons, Jill H. ;
Padidela, Raja ;
Namba, Noriyuki ;
Cheong, Hae Il ;
Pitukcheewanont, Pisit ;
Sochett, Etienne ;
Hoegler, Wolfgang ;
Muroya, Koji ;
Tanaka, Hiroyuki ;
Gottesman, Gary S. ;
Biggin, Andrew ;
Perwad, Farzana ;
Mao, Meng ;
Chen, Chao-Yin ;
Skrinar, Alison ;
San Martin, Javier ;
Portale, Anthony A. .
LANCET, 2019, 393 (10189) :2416-2427
[10]   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].
Insogna, Karl L. ;
Briot, Karine ;
Imel, Erik A. ;
Kamenicky, Peter ;
Ruppe, Mary D. ;
Portale, Anthony A. ;
Weber, Thomas ;
Pitukcheewanont, Pisit ;
Cheong, Hae Il ;
de Beur, Suzanne Jan ;
Imanishi, Yasuo ;
Ito, Nobuaki ;
Lachmann, Robin H. ;
Tanaka, Hiroyuki ;
Perwad, Farzana ;
Zhang, Lin ;
Chen, Chao-Yin ;
Theodore-Oklota, Christina ;
Mealiffe, Matt ;
Martin, Javier San ;
Carpenter, Thomas O. .
JOURNAL OF BONE AND MINERAL RESEARCH, 2018, 33 (08) :1383-1393