Evidence map›Paper›PMID 38229908›Full record

ArticleBone reports2024

Burosumab for the treatment of cutaneous-skeletal hypophosphatemia syndrome.

Lillian Abebe, Kim Phung, Marie-Eve Robinson, Richelle Waldner, Sasha Carsen, Kevin Smit, Andrew Tice, Joanna Lazier, Christine Armour, Marika Page and 4 more

Open access · goldAbstract readCase Reports
PubMed Full text at PMC Publisher
In one paragraph

Article in Bone reports, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
1.6field-weighted citation impact, top 16% 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

6 citing papers in PubMed, 8 citations in OpenAlex.

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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

14 authors at 4 institutions in 1 country.

Lillian AbebeThe Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
Kim PhungThe Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
Marie-Eve RobinsonDepartment of Pediatrics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
Richelle WaldnerDepartment of Pediatrics, University of Alberta, 116 St & 85 Av, Edmonton, AB T6G 2R3, Canada.
Sasha CarsenDepartment of Pediatrics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
Kevin SmitDepartment of Pediatrics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
Andrew TiceDepartment of Pediatrics, Faculty of Medicine, University of Ottawa, 550 Cumberland St, Ottawa, ON K1N 6N5, Canada.
Joanna LazierDepartment of Genetics, Children's Hospital of Eastern Ontario, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
Christine ArmourDepartment of Genetics, Children's Hospital of Eastern Ontario, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
Marika PageThe Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
Saunya DoverThe Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
Frank RauchShriners Hospital for Children, 1003 Decarie Blvd, Montréal, QC H4A 0A9, Canada.
Khaldoun KoujokThe Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
Leanne M WardThe Ottawa Pediatric Bone Health Research Group, Children's Hospital of Eastern Ontario Research Institute, 401 Smyth Road, Ottawa, ON K1H 8L1, Canada.
Children's Hospital of Eastern Ontario · CAUniversity of Ottawa · CAShriners Hospitals for Children - Canada · CAUniversity of Alberta · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Cutaneous-skeletal hypophosphatemia syndrome (CSHS) is a rare bone disorder featuring fibroblast growth factor-23 (FGF23)-mediated hypophosphatemic rickets. We report a 2-year, 10-month-old girl with CSHS treated with burosumab, a novel human monoclonal antibody targeting FGF23. This approach was associated with rickets healing, improvement in growth and lower limb deformity, and clinically significant benefit to her functional mobility and motor development. This case report provides evidence for the effective use of FGF23-neutralizing antibody therapy beyond the classic FGF23-mediated disorders of X-linked hypophosphatemia and tumor-induced osteomalacia.

Indexed as

BurosumabChildConventional treatmentCutaneous-skeletal hypophosphatemia syndromeFibroblast growth factor-23Hypophosphatemic rickets

Identifiers

PMID38229908
PMCPMC10790024
OpenAlexW4388591171

What OpenQuestion 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 OpenQuestion graph.