Evidence map›Paper›PMID 39787354›Full record

SynthesisThe Journal of clinical endocrinology and metabolism2025

Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients With X-Linked Hypophosphatemia.

Dalal S Ali, Reza D Mirza, Salma Hussein, Farah Alsarraf, R Todd Alexander, Hajar Abu Alrob, Natasha M Appelman-Dijkstra, Martin Biosse-Duplan, Maria Luisa Brandi, Thomas O Carpenter and 12 more

Abstract readSystematic Review
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in The Journal of clinical endocrinology and metabolism, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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

3 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Guideline
  2. Article
  3. 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

22 authors.

Dalal S AliDivision of Endocrinology and Metabolism, McMaster University, Hamilton, ON, Canada L8S 4L8.ORCID 0000-0002-5378-5548
Reza D MirzaDepartment of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada L8S 4L8.ORCID 0000-0002-0576-4208
Salma HusseinDivision of Endocrinology and Metabolism, McMaster University, Hamilton, ON, Canada L8S 4L8.ORCID 0000-0002-7284-4822
Farah AlsarrafDivision of Endocrinology and Metabolism, McMaster University, Hamilton, ON, Canada L8S 4L8.ORCID 0000-0001-9426-9702
R Todd AlexanderDepartment of Pediatrics, Faculty of Medicine & Dentistry, The University of Alberta, Edmonton, AB, Canada T6G 2R3.ORCID 0000-0001-7396-7894
Hajar Abu AlrobDepartment of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada L8S 4L8.ORCID 0000-0001-7830-3237
Natasha M Appelman-DijkstraDepartment of Internal Medicine, Division of Endocrinology, Center for Bone Quality, Leiden University Medical Center, 2300 ZA Leiden, the Netherlands.ORCID 0000-0001-5035-127X
Martin Biosse-DuplanDepartment of Oral Medicine, Faculty of Dentistry, UMR 1333, Université Paris Cité, 75006 Paris, France.ORCID 0000-0001-9417-3970
Maria Luisa BrandiInstitute of Endocrine and Metabolic Sciences, Vita-Salute San Raffaele University and IRCCS, 20132 Milan, Italy.ORCID 0000-0002-8741-0592
Thomas O CarpenterDepartments of Pediatrics (Endocrinology), and Orthopedics and Rehabilitation, Yale University School of Medicine, New Haven, CT 06520, USA.ORCID 0000-0003-1328-6768
Catherine ChaussainDepartment of Oral Medicine, Faculty of Dentistry, UMR 1333, Université Paris Cité, 75006 Paris, France.ORCID 0000-0002-3463-3936
Karel DandurandDivision of Internal Medicine, Université de Sherbrooke, Sherbrooke, QC, Canada J1H 5N4.ORCID 0000-0003-1759-8419
Guido FillerDepartment of Paediatrics, Endocrinology Division, Western University, London, ON, Canada N6A 3K7.ORCID 0000-0003-1891-6765
Pablo FlorenzanoDepartment of Endocrinology, School of Medicine Pontificia Universidad Católica de Chile, 8320165 Santiago, Región Metropolitana, Chile.ORCID 0000-0001-5936-3146
Seiji FukumotoDepartment of Medicine, Tamaki-Aozora Hospital, 779-3125 Tokushima, Japan.ORCID 0000-0003-3610-3469
Corinna GrasemannDepartment of Pediatrics, Division of Rare Diseases, Katholisches Klinikum Bochum and Ruhr-University Bochum, 44791 Bochum, Germany.ORCID 0000-0003-1793-4603
Erik A ImelDepartment of Medicine and Pediatrics, Endocrinology, Indiana University School of Medicine, Indianapolis, IN 46202, USA.ORCID 0000-0002-7284-3467
Suzanne M Jan de BeurDepartment of Medicine, University of Virginia School of Medicine, Charlottesville, VA 22903, USA.ORCID 0000-0002-9386-7732
Emmett MorganteDepartment of Kinesiology, University of Waterloo, Waterloo, ON, Canada N2L 3G1.ORCID 0009-0009-9271-0379
Leanne M WardChildren's Hospital of Eastern Ontario, Department of Pediatrics, University of Ottawa, Ottawa, ON, Canada K1H 8L1.ORCID 0000-0003-1557-9185
Aliya A KhanDivision of Endocrinology and Metabolism, McMaster University, Hamilton, ON, Canada L8S 4L8.ORCID 0000-0003-3733-8956
Gordon GuyattDepartment of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada L8S 4L8.ORCID 0000-0003-2352-5718

Funding

McMaster University
6 · The paper itself

Abstract

objectiveTo examine the evidence addressing the management of X-linked hypophosphatemia (XLH) in children to inform treatment recommendations.

methodsWe searched Embase, MEDLINE, Web of Science, and Cochrane Central up to May 2023. Eligible studies included randomized controlled trials (RCTs) and observational studies of individuals younger than 18 years with clinically or genetically confirmed XLH. Manuscripts comparing burosumab to either no treatment or conventional therapy (phosphate and active vitamin D) or evaluating conventional therapy to no treatment were included. Two reviewers independently determined eligibility, extracted data, and assessed risk of bias (RoB). GRADE methodology was used to assess evidence certainty.

resultsWe screened 4114 records and assessed 254 full texts. One RCT and one post hoc study proved eligible when comparing burosumab to conventional therapy or no treatment. The open-label RCT was at high RoB, with certainty of evidence ranging from moderate to very low. Burosumab, compared to conventional therapy, probably prevents lower limb deformity and improves physical health quality of life (QoL) (moderate certainty). Burosumab may increase height and enhance the burden of symptoms related to chronic hypophosphatemia (low certainty). Burosumab probably increases treatment-emergent adverse events (moderate certainty) and may increase dental abscesses (low certainty). One observational study assessing conventional therapy vs no treatment was at high RoB, providing very low certainty evidence regarding the impact of conventional therapy on final height.

conclusionOur review indicates that burosumab likely provides benefits to children by preventing lower limb deformity and improving physical health QoL while potentially increasing height. However, burosumab may also increase adverse events. Our review found limited evidence regarding the impact of conventional therapy compared to no treatment on final height. Further research is required to understand the long-term effect of medical therapy in children.

Indexed as

Antibodies, Monoclonal, HumanizedFamilial Hypophosphatemic RicketsAdolescentChildHumansQuality of LifeRandomized Controlled Trials as TopicTreatment OutcomeVitamin DAntibodies, Monoclonal, HumanizedburosumabVitamin Dburosumabchildren XLHconventional therapyefficacypatient-important outcomespediatric XLH

Identifiers

PMID39787354
PMCPMC12012687

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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 Socratic graph.