Patient-reported outcomes from a randomized, active-controlled, open-label, phase 3 trial of burosumab versus conventional therapy in children with X-linked hypophosphatemia

Research output: Contribution to journalArticlepeer-review

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Patient-reported outcomes from a randomized, active-controlled, open-label, phase 3 trial of burosumab versus conventional therapy in children with X-linked hypophosphatemia. / Padidela, Raja; Whyte, Michael P; Glorieux, Francis H; Munns, Craig F; Ward, Leanne M; Nilsson, Ola; Portale, Anthony ; Simmons, Jill ; Namba, Noriyuki; Cheong, Hae Il; Pitukcheewanont, Pisit; Sochett, Etienne; Högler, Wolfgang; Muroya, Koji; Tanaka, Hiroyuki; Gottesman, Gary; Biggin, Andrew ; Perwad, Farzana; Williams, Angela; Nixon, Annabel; Sun, Wei; Chen, Angel; Skrinar, Alison; Imel, Erik.

In: Calcified Tissue International, 23.01.2021.

Research output: Contribution to journalArticlepeer-review

Harvard

Padidela, R, Whyte, MP, Glorieux, FH, Munns, CF, Ward, LM, Nilsson, O, Portale, A, Simmons, J, Namba, N, Cheong, HI, Pitukcheewanont, P, Sochett, E, Högler, W, Muroya, K, Tanaka, H, Gottesman, G, Biggin, A, Perwad, F, Williams, A, Nixon, A, Sun, W, Chen, A, Skrinar, A & Imel, E 2021, 'Patient-reported outcomes from a randomized, active-controlled, open-label, phase 3 trial of burosumab versus conventional therapy in children with X-linked hypophosphatemia', Calcified Tissue International. https://doi.org/10.1007/s00223-020-00797-x

APA

Padidela, R., Whyte, M. P., Glorieux, F. H., Munns, C. F., Ward, L. M., Nilsson, O., Portale, A., Simmons, J., Namba, N., Cheong, H. I., Pitukcheewanont, P., Sochett, E., Högler, W., Muroya, K., Tanaka, H., Gottesman, G., Biggin, A., Perwad, F., Williams, A., ... Imel, E. (2021). Patient-reported outcomes from a randomized, active-controlled, open-label, phase 3 trial of burosumab versus conventional therapy in children with X-linked hypophosphatemia. Calcified Tissue International. https://doi.org/10.1007/s00223-020-00797-x

Vancouver

Author

Padidela, Raja ; Whyte, Michael P ; Glorieux, Francis H ; Munns, Craig F ; Ward, Leanne M ; Nilsson, Ola ; Portale, Anthony ; Simmons, Jill ; Namba, Noriyuki ; Cheong, Hae Il ; Pitukcheewanont, Pisit ; Sochett, Etienne ; Högler, Wolfgang ; Muroya, Koji ; Tanaka, Hiroyuki ; Gottesman, Gary ; Biggin, Andrew ; Perwad, Farzana ; Williams, Angela ; Nixon, Annabel ; Sun, Wei ; Chen, Angel ; Skrinar, Alison ; Imel, Erik. / Patient-reported outcomes from a randomized, active-controlled, open-label, phase 3 trial of burosumab versus conventional therapy in children with X-linked hypophosphatemia. In: Calcified Tissue International. 2021.

Bibtex

@article{21e2035d5744434cb55dedfa7d63eeb3,
title = "Patient-reported outcomes from a randomized, active-controlled, open-label, phase 3 trial of burosumab versus conventional therapy in children with X-linked hypophosphatemia",
abstract = "Changing to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved phosphorus homeostasis, rickets, lower-extremity deformities, mobility, and growth versus continuing oral phosphate and active vitamin D (conventional therapy) in a randomized, open-label, phase 3 trial involving children aged 1–12 years with X-linked hypophosphatemia. Patients were randomized (1:1) to subcutaneous burosumab or to continue conventional therapy. We present patient-reported outcomes (PROs) from this trial for children aged ≥ 5 years at screening (n = 35), using a Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire and SF-10 Health Survey for Children. PROMIS pain interference, physical function mobility, and fatigue scores improved from baseline with burosumab at weeks 40 and 64, but changed little with continued conventional therapy. Pain interference scores differed significantly between groups at week 40 (− 5.02, 95% CI − 9.29 to − 0.75; p = 0.0212) but not at week 64. Between-group differences were not significant at either week for physical function mobility or fatigue. Reductions in PROMIS pain interference and fatigue scores from baseline were clinically meaningful with burosumab at weeks 40 and 64 but not with conventional therapy. SF-10 physical health scores (PHS-10) improved significantly with burosumab at week 40 (least-squares mean [standard error] + 5.98 [1.79]; p = 0.0008) and week 64 (+ 5.93 [1.88]; p = 0.0016) but not with conventional therapy (between-treatment differences were nonsignificant). In conclusion, changing to burosumab improved PRO measures, with statistically significant differences in PROMIS pain interference at week 40 versus continuing with conventional therapy and in PHS-10 at weeks 40 and 64 versus baseline.Trial registration: ClinicalTrials.gov NCT02915705",
keywords = "Burosumab, X-linked hypophosphatemia, Patient-reported outcomes, Patient-reported outcomes measurement information system",
author = "Raja Padidela and Whyte, {Michael P} and Glorieux, {Francis H} and Munns, {Craig F} and Ward, {Leanne M} and Ola Nilsson and Anthony Portale and Jill Simmons and Noriyuki Namba and Cheong, {Hae Il} and Pisit Pitukcheewanont and Etienne Sochett and Wolfgang H{\"o}gler and Koji Muroya and Hiroyuki Tanaka and Gary Gottesman and Andrew Biggin and Farzana Perwad and Angela Williams and Annabel Nixon and Wei Sun and Angel Chen and Alison Skrinar and Erik Imel",
year = "2021",
month = jan,
day = "23",
doi = "10.1007/s00223-020-00797-x",
language = "English",
journal = "Calcified Tissue International",
issn = "0171-967X",
publisher = "Springer",

}

RIS

TY - JOUR

T1 - Patient-reported outcomes from a randomized, active-controlled, open-label, phase 3 trial of burosumab versus conventional therapy in children with X-linked hypophosphatemia

AU - Padidela, Raja

AU - Whyte, Michael P

AU - Glorieux, Francis H

AU - Munns, Craig F

AU - Ward, Leanne M

AU - Nilsson, Ola

AU - Portale, Anthony

AU - Simmons, Jill

AU - Namba, Noriyuki

AU - Cheong, Hae Il

AU - Pitukcheewanont, Pisit

AU - Sochett, Etienne

AU - Högler, Wolfgang

AU - Muroya, Koji

AU - Tanaka, Hiroyuki

AU - Gottesman, Gary

AU - Biggin, Andrew

AU - Perwad, Farzana

AU - Williams, Angela

AU - Nixon, Annabel

AU - Sun, Wei

AU - Chen, Angel

AU - Skrinar, Alison

AU - Imel, Erik

PY - 2021/1/23

Y1 - 2021/1/23

N2 - Changing to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved phosphorus homeostasis, rickets, lower-extremity deformities, mobility, and growth versus continuing oral phosphate and active vitamin D (conventional therapy) in a randomized, open-label, phase 3 trial involving children aged 1–12 years with X-linked hypophosphatemia. Patients were randomized (1:1) to subcutaneous burosumab or to continue conventional therapy. We present patient-reported outcomes (PROs) from this trial for children aged ≥ 5 years at screening (n = 35), using a Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire and SF-10 Health Survey for Children. PROMIS pain interference, physical function mobility, and fatigue scores improved from baseline with burosumab at weeks 40 and 64, but changed little with continued conventional therapy. Pain interference scores differed significantly between groups at week 40 (− 5.02, 95% CI − 9.29 to − 0.75; p = 0.0212) but not at week 64. Between-group differences were not significant at either week for physical function mobility or fatigue. Reductions in PROMIS pain interference and fatigue scores from baseline were clinically meaningful with burosumab at weeks 40 and 64 but not with conventional therapy. SF-10 physical health scores (PHS-10) improved significantly with burosumab at week 40 (least-squares mean [standard error] + 5.98 [1.79]; p = 0.0008) and week 64 (+ 5.93 [1.88]; p = 0.0016) but not with conventional therapy (between-treatment differences were nonsignificant). In conclusion, changing to burosumab improved PRO measures, with statistically significant differences in PROMIS pain interference at week 40 versus continuing with conventional therapy and in PHS-10 at weeks 40 and 64 versus baseline.Trial registration: ClinicalTrials.gov NCT02915705

AB - Changing to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved phosphorus homeostasis, rickets, lower-extremity deformities, mobility, and growth versus continuing oral phosphate and active vitamin D (conventional therapy) in a randomized, open-label, phase 3 trial involving children aged 1–12 years with X-linked hypophosphatemia. Patients were randomized (1:1) to subcutaneous burosumab or to continue conventional therapy. We present patient-reported outcomes (PROs) from this trial for children aged ≥ 5 years at screening (n = 35), using a Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaire and SF-10 Health Survey for Children. PROMIS pain interference, physical function mobility, and fatigue scores improved from baseline with burosumab at weeks 40 and 64, but changed little with continued conventional therapy. Pain interference scores differed significantly between groups at week 40 (− 5.02, 95% CI − 9.29 to − 0.75; p = 0.0212) but not at week 64. Between-group differences were not significant at either week for physical function mobility or fatigue. Reductions in PROMIS pain interference and fatigue scores from baseline were clinically meaningful with burosumab at weeks 40 and 64 but not with conventional therapy. SF-10 physical health scores (PHS-10) improved significantly with burosumab at week 40 (least-squares mean [standard error] + 5.98 [1.79]; p = 0.0008) and week 64 (+ 5.93 [1.88]; p = 0.0016) but not with conventional therapy (between-treatment differences were nonsignificant). In conclusion, changing to burosumab improved PRO measures, with statistically significant differences in PROMIS pain interference at week 40 versus continuing with conventional therapy and in PHS-10 at weeks 40 and 64 versus baseline.Trial registration: ClinicalTrials.gov NCT02915705

KW - Burosumab

KW - X-linked hypophosphatemia

KW - Patient-reported outcomes

KW - Patient-reported outcomes measurement information system

U2 - 10.1007/s00223-020-00797-x

DO - 10.1007/s00223-020-00797-x

M3 - Article

JO - Calcified Tissue International

JF - Calcified Tissue International

SN - 0171-967X

ER -