Evidence map›Paper›PMID 38123339›Full record

Trial reportAnnals of the rheumatic diseases2024

Randomised trial of genetic testing and targeted intervention to prevent the development and progression of Paget's disease of bone.

Jonathan Phillips, Deepak Subedi, Steff C Lewis, Catriona Keerie, Owen Cronin, Mary Porteous, David Moore, Roseanne Cetnarskyj, Lakshminarayan Ranganath, Peter L Selby and 30 more

Open access · hybridAbstract readRandomized Controlled Trial
In one paragraph

Trial report in Annals of the rheumatic diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
2.5field-weighted citation impact, top 9% 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed, 12 citations in OpenAlex.

  1. Review
  2. Review
  3. Latest developments in Paget's disease of bone.European journal of endocrinology · 2025
    Review
  4. Article
  5. Article
  6. Review
  7. Review
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

40 authors at 20 institutions in 7 countries.

Jonathan PhillipsCentre for Genomic and Experimental Medicine, Institute of Genetics and Cancer, Western General Hospital, University of Edinburgh, Edinburgh, UK.
Deepak SubediDepartment of Radiology and Nuclear Medicine, Western General Hospital, Edinburgh, UK.
Steff C LewisEdinburgh Clinical Trials Unit, The Usher Institute, The University of Edinburgh, Edinburgh, UK.ORCID http://orcid.org/0000-0003-1210-2314
Catriona KeerieEdinburgh Clinical Trials Unit, The Usher Institute, The University of Edinburgh, Edinburgh, UK.ORCID http://orcid.org/0000-0001-5459-6178
Owen CroninRheumatic Diseases Unit, Western General Hospital, Edinburgh, UK.ORCID http://orcid.org/0000-0002-1365-9942
Mary PorteousCentre for Genomic and Experimental Medicine, Institute of Genetics and Cancer, Western General Hospital, University of Edinburgh, Edinburgh, UK.
David MooreSouth East Scotland Molecular Genetics Service, NHS Lothian, Edinburgh, UK.
Roseanne CetnarskyjSchool of Health Sciences, University of Dundee, Dundee, UK.
Lakshminarayan RanganathClinical Biochemistry and Metabolic Medicine, University of Liverpool, Liverpool, UK.ORCID http://orcid.org/0000-0002-4205-2269
Peter L SelbyDepartment of Diabetes, Endocrinology and Metabolism, Manchester Royal Infirmary, Manchester, UK.
Tolga TurgutClinical Genetics, Manchester Centre for Genomic Medicine, Manchester University Hospitals Foundation NHS Trust, Manchester, UK.
Geeta HampsonDepartment of Chemical Pathology, St Thomas' Hospital, London, UK.ORCID http://orcid.org/0000-0001-8465-5247
Rama ChandraKing's College Hospital, London, UK.
Shu HoRheumatology, Robert Jones and Agnes Hunt Orthopaedic and District Hospital NHS Trust, Oswestry, UK.
Jon TobiasMusculoskeletal Research Unit, Translational Health Sciences, Bristol Medical School, University of Bristol, Bristol, UK.ORCID http://orcid.org/0000-0002-7475-3932
Steven Young-MinQueen Alexandra Hospital, Portsmouth, UK.ORCID http://orcid.org/0000-0001-8270-6207
Malachi J McKennaDepartment of Endocrinology and Diabetes Mellitus, St Vincent's University Hospital, Dublin, Ireland.ORCID http://orcid.org/0000-0002-6564-7011
Rachel K CrowleyDepartment of Endocrinology and Diabetes Mellitus, St Vincent's University Hospital, Dublin, Ireland.ORCID http://orcid.org/0000-0003-1472-4117
William D FraserNorwich Medical School, University of East Anglia, Norwich, UK.ORCID http://orcid.org/0000-0003-0556-3358
Jonathan C Y TangDepartments of Endocrinology and Clinical Biochemistry, University of East Anglia, Norwich, UK.ORCID http://orcid.org/0000-0001-6305-6333
Luigi GennariDepartment of Medicine, Surgery and Neurosciences, University of Siena, Siena, Italy.
Rannuccio NutiDepartment of Medicine, Surgery and Neurosciences, University of Siena, Siena, Italy.
Maria Luisa BrandiFIRMO Foundation, Florence, Italy.ORCID http://orcid.org/0000-0002-8741-0592
Javier Del Pino-MontesRheumatology, University of Salamanca, Salamanca, Spain.ORCID http://orcid.org/0000-0002-3145-6137
Jean-Pierre DevogelaerDepartment of Rheumatology, Saint-Luc University Hospital, Université catholique de Louvain, Brussels, Belgium.ORCID http://orcid.org/0000-0002-5836-2771
Anne DurnezDepartment of Rheumatology, Saint-Luc University Hospital, Université catholique de Louvain, Brussels, Belgium.
Giovanni Carlo IsaiaUniversity of Turin, Turin, Italy.
Marco Di StefanoUniversity of Turin, Turin, Italy.
Nuria GuanabensDepartment of Rheumatology, Hospital Clinic, IDIBAPS, University of Barcelona, Barcelona, Spain.ORCID http://orcid.org/0000-0002-7103-9530
Josep Blanch RubioRheumatology Department, Hospital del Mar, Barcelona, Spain.
Markus J SeibelConcord Repatriation General Hospital, Concord, New South Wales, Australia.ORCID http://orcid.org/0000-0002-2701-378X
John P WalshDepartment of Endocrinology and Diabetes, Sir Charles Gairdner Hospital, Nedlands, Western Australia, Australia.ORCID http://orcid.org/0000-0002-1766-2612
Sarah L ReaDepartment of Endocrinology and Diabetes, Sir Charles Gairdner Hospital, Nedlands, Western Australia, Australia.ORCID http://orcid.org/0000-0002-2181-9491
Mark A KotowiczInstitute for Mental and Physical Health and Clinical Translation, Deakin University, Geelong, Victoria, Australia.ORCID http://orcid.org/0000-0002-8094-1411
Geoffrey C NicholsonRural Clinical School, The University of Queensland, Toowoomba, Queensland, Australia.ORCID http://orcid.org/0000-0002-0152-1153
Emma L DuncanEndocrinology Department, Royal Brisbane and Women's Hospital, Herston, Queensland, Australia.ORCID http://orcid.org/0000-0002-8143-4403
Gabor MajorRheumatology, John Hunter Hospital, New Lambton Heights, New South Wales, Australia.ORCID http://orcid.org/0000-0003-3464-7438
Anne HorneDepartment of Medicine, The University of Auckland, Auckland, New Zealand.ORCID http://orcid.org/0000-0003-2783-7452
Nigel GilchristPrincess Margaret Hospital, Christchurch, New Zealand.
Stuart H RalstonCentre for Genomic and Experimental Medicine, Institute of Genetics and Cancer, Western General Hospital, University of Edinburgh, Edinburgh, UK stuart.ralston@ed.ac.uk.ORCID http://orcid.org/0000-0002-2804-7586
Western General Hospital · GBUniversity of East Anglia · GBUniversity of Edinburgh · GBUniversity of Siena · ITUniversity of Turin · ITAZ Sint-Jan · BEBarwon Health · AUConcord Repatriation General Hospital · AUConsorci Institut D'Investigacions Biomediques August Pi I Sunyer · ESEdinburgh Cancer Research · GBHospital Del Mar · ESJohn Hunter Hospital · AUKing's College Hospital · GBKing's College London · GBManchester Royal Infirmary · GBMurdoch University · AUNational University of Ireland · IENHS Lothian · GBPrincess Margaret Hospital · NZQueen Alexandra Hospital · GB

Funding

Versus Arthritis 18163Wellcome Trust
6 · The paper itself

Abstract

introductionPaget's disease of bone (PDB) frequently presents at an advanced stage with irreversible skeletal damage. Clinical outcomes might be improved by earlier diagnosis and prophylactic treatment.

methodsWe randomised 222 individuals at increased risk of PDB because of pathogenic

resultsThe median duration of follow-up was 84 months (range 0-127) and 180 participants (81%) completed the study. At baseline, 9 (8.1%) of the ZA group had PDB lesions vs 12 (10.8%) of the placebo group. Two of the placebo group developed new lesions versus none in the ZA group (OR 0.41, 95% CI 0.00 to 3.43, p=0.25). Eight of the placebo group had a poor outcome (lesions which were new, unchanged or progressing) compared with none of the ZA group (OR 0.08, 95% CI 0.00 to 0.42, p=0.003). At the study end, 1 participant in the ZA group had lesions compared with 11 in the placebo group. Biochemical markers of bone turnover were significantly reduced in the ZA group. One participant allocated to placebo required rescue therapy with ZA because of symptomatic disease. The number and severity of adverse events did not differ between groups.

conclusionsGenetic testing for pathogenic TRIAL REGISTRATION NUMBER: ISRCTN11616770.

Indexed as

DiphosphonatesOsteitis DeformansBiomarkersGenetic TestingHumansSequestosome-1 ProteinZoledronic AcidBiomarkersDiphosphonatesSequestosome-1 ProteinZoledronic AcidPatient Reported Outcome MeasuresPharmacogeneticsTherapeutics

Identifiers

PMID38123339
PMCPMC10958267
OpenAlexW4389990966

What OpenQuestion holds

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LicenceCC BY
Read underepoch 390

Registered trials

None linked

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 OpenQuestion graph.