Evidence map›Paper›PMID 33484279›Full record

Trial reportCalcified tissue international2021

Patient-Reported Outcomes from a Randomized, Active-Controlled, Open-Label, Phase 3 Trial of Burosumab Versus Conventional Therapy in Children with X-Linked Hypophosphatemia.

Raja Padidela, Michael P Whyte, Francis H Glorieux, Craig F Munns, Leanne M Ward, Ola Nilsson, Anthony A Portale, Jill H Simmons, Noriyuki Namba, Hae Il Cheong and 14 more

Registry-linked trialOpen access · hybridAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Calcified tissue international, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02915705 (A Randomized, Open-Label, Phase 3 Study to Assess the Efficacy and Safety of KRN23 Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia), which is not on this map. Cited by 33 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
33citing papers in PubMed, 2 pooled it
4.3field-weighted citation impact, top 5% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02915705 phase3completednot on this map

A Randomized, Open-Label, Phase 3 Study to Assess the Efficacy and Safety of KRN23 Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia (XLH)

TypeinterventionalSponsorKyowa Kirin, Inc.Ran2016 to 2019Enrolled61ConditionsX-Linked HypophosphatemiaArmsburosumab, Oral Phosphate Supplement, active vitamin D
3 · Its place in the literature

Who cites it

33 citing papers in PubMed, 2 syntheses or guidelines pooled it, 47 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Trial
  4. Article
  5. Article
  6. Review
  7. Article
  8. Article
  9. Article
  10. Article
  11. Article
  12. Review
  13. Article
  14. Article
  15. Observational
  16. Article
  17. Article
  18. Review
  19. Article
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

24 authors at 19 institutions in 8 countries.

Raja PadidelaDepartment of Paediatric Endocrinology, Royal Manchester Children's Hospital, Manchester, UK. Raja.Padidela@mft.nhs.uk.ORCID https://orcid.org/0000-0002-3350-9896
Michael P WhyteShriners Hospitals for Children -Washington University School of Medicine in St Louis, St Louis, MO, USA.ORCID https://orcid.org/0000-0002-8755-8490
Francis H GlorieuxShriners Hospital for Children - Canada, McGill University, Montreal, QC, Canada.ORCID https://orcid.org/0000-0001-6616-3484
Craig F MunnsThe University of Sydney Children's Hospital Westmead Clinical School, The Children's Hospital at Westmead, Westmead, NSW, Australia.ORCID https://orcid.org/0000-0001-5898-5808
Leanne M WardDepartment of Pediatrics, University of Ottawa, Ottawa, ON, Canada.ORCID https://orcid.org/0000-0003-1557-9185
Ola NilssonDivision of Pediatric Endocrinology & Center for Molecular Medicine, Karolinska Institute, Stockholm, Sweden.ORCID https://orcid.org/0000-0002-9986-8138
Anthony A PortaleDepartment of Pediatrics, University of California, San Francisco, San Francisco, CA, USA.ORCID https://orcid.org/0000-0003-2054-6898
Jill H SimmonsDepartments of Pediatrics, Division of Endocrinology and Diabetes, Vanderbilt University School of Medicine, Vanderbilt University, Nashville, TN, USA.ORCID https://orcid.org/0000-0002-4189-6718
Noriyuki NambaDepartment of Pediatrics, Osaka Hospital, Japan Community Healthcare Organization, Osaka, Japan.ORCID https://orcid.org/0000-0002-3803-4500
Hae Il CheongSeoul National University Children's Hospital, Seoul, Republic of Korea.ORCID https://orcid.org/0000-0001-7556-1265
Pisit PitukcheewanontCenter of Endocrinology, Diabetes and Metabolism, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Etienne SochettDepartment of Paediatrics, Hospital for Sick Children, Toronto, ON, Canada.ORCID https://orcid.org/0000-0002-7027-7123
Wolfgang HöglerDepartment of Paediatrics and Adolescent Medicine, Johannes Kepler University Linz, Linz, Austria.ORCID https://orcid.org/0000-0003-4328-6304
Koji MuroyaDepartment of Endocrinology and Metabolism, Kanagawa Children's Medical Center, Yokohama, Japan.ORCID https://orcid.org/0000-0002-1810-9894
Hiroyuki TanakaOkayama Saiseikai General Hospital Outpatient Center, Okayama, Japan.ORCID https://orcid.org/0000-0002-9117-3534
Gary S GottesmanShriners Hospitals for Children, St Louis, MO, USA.ORCID https://orcid.org/0000-0002-0302-3852
Andrew BigginThe University of Sydney Children's Hospital Westmead Clinical School, The Children's Hospital at Westmead, Westmead, NSW, Australia.ORCID https://orcid.org/0000-0002-3443-9359
Farzana PerwadDepartment of Pediatrics, University of California, San Francisco, San Francisco, CA, USA.ORCID https://orcid.org/0000-0001-7983-0127
Angela WilliamsKyowa Kirin International, Marlow, UK.
Annabel NixonChilli Consultancy, Salisbury, UK.ORCID https://orcid.org/0000-0003-3454-9491
Wei SunKyowa Kirin Pharmaceutical Development, Princeton, NJ, USA.
Angel ChenUltragenyx Pharmaceutical, Novato, CA, USA.
Alison SkrinarUltragenyx Pharmaceutical, Novato, CA, USA.
Erik A ImelDepartment of Medicine and Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.ORCID https://orcid.org/0000-0002-7284-3467
Shriners Hospitals for Children - St. Louis · USThe University of Sydney · AUUltragenyx Pharmaceutical (United States) · USUniversity of California, San Francisco · USChildren's Hospital of Los Angeles · USHospital for Sick Children · CAIndiana University School of MedicineJohannes Kepler University of Linz · ATKanagawa Children's Medical Center · JPKirin (United States) · USKyowa Kirin International (United Kingdom) · GBManchester University NHS Foundation Trust · GBMontreal Children's Hospital · CAOkayama Saiseikai General Hospital · JPÖrebro University · SEOsaka Hospital · JPSeoul National University Children's Hospital · KRUniversity of Ottawa · CAVanderbilt University · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

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.

Indexed as

Familial Hypophosphatemic RicketsAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedChildHumansPatient Reported Outcome MeasuresAntibodies, MonoclonalAntibodies, Monoclonal, HumanizedburosumabBurosumabPatient-reported outcomesPatient-reported outcomes measurement information systemX-linked hypophosphatemia

Identifiers

PMID33484279
PMCPMC8064984
OpenAlexW3125103770

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.