Evidence map›Paper›PMID 42156385›Full record

Observational studyNature communications2026

Precision-guided therapy in H3K27-altered diffuse midline glioma.

Campbell J L McKay, Chelsea Mayoh, Phoebe Power, Sophie Jessop, Shampavi Sriharan, Jordan Staunton, Sarah Trinder, Nataliya Zhukova, Cristina Mignone, Dinisha Govender and 22 more

Registry-linked trialAbstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in Nature communications, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03336931 (A Multicenter Prospective Study of the Feasibility and Clinical Value of a Diagnostic Service for Identifying Therapeutic Targets and Recommending Personalised Treatment for Children and Adolescents With High-risk Cancer), which is not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–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.

NCT03336931 active not recruitingnot on this map

A Multicenter Prospective Study of the Feasibility and Clinical Value of a Diagnostic Service for Identifying Therapeutic Targets and Recommending Personalised Treatment for Children and Adolescents With High-risk Cancer

TypeobservationalSponsorSydney Children's Hospitals NetworkRan2017 to 2032Enrolled550ConditionsChildhood Cancer, Childhood Solid Tumor, Childhood Brain Tumor, Childhood LeukemiaArmsMolecular profiling and drug testing
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

32 authors.

Campbell J L McKayThe Royal Children's Hospital, Melbourne, VIC, Australia.ORCID http://orcid.org/0009-0006-3497-3144
Chelsea MayohChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0002-6398-3046
Phoebe PowerKids Cancer Centre, Sydney Children's Hospital, Randwick, NSW, Australia.
Sophie JessopChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.
Shampavi SriharanQueensland Children's Hospital, Brisbane, QLD, Australia.
Jordan StauntonChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.
Sarah TrinderPerth Children's Hospital, Perth, WA, Australia.
Nataliya ZhukovaMonash Children's Hospital, Clayton, VIC, Australia.
Cristina MignoneThe Royal Children's Hospital, Melbourne, VIC, Australia.
Dinisha GovenderCancer Centre for Children, The Children's Hospital at Westmead, Westmead, NSW, Australia.
Frank AlvaroJohn Hunter Hospital, Newcastle, NSW, Australia.
Peter DownieMonash Children's Hospital, Clayton, VIC, Australia.ORCID http://orcid.org/0000-0002-6616-5102
Jordan R HansfordMichael Rice Centre for Haematology and Oncology, Women's and Children's Hospital, Adelaide, SA, Australia.ORCID http://orcid.org/0000-0001-7733-383X
Tim HassallQueensland Children's Hospital, Brisbane, QLD, Australia.
Santosh ValviPerth Children's Hospital, Perth, WA, Australia.
Marion K MateosChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0002-2763-5853
Paulette BarahonaChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0002-3184-8499
Andrew J GiffordChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0002-4092-2347
Gabor TaxChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0003-4530-0798
Maria TsoliChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0003-1155-0510
Emmy M DolmanChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.
Marie WongChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0003-0066-6606
Mark CowleyChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0002-9519-5714
Glenn M MarshallChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0002-2503-0762
Toby N TrahairChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0002-3295-228X
Paul G EkertChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0002-2976-8617
Michelle HaberChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0003-2036-8817
Vanessa TyrellChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0002-5162-517X
Loretta M S LauChildren's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia.ORCID http://orcid.org/0000-0002-3172-0970
Neevika Manoharan *Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia. neevika.manoharan@health.nsw.gov.au.
David S Ziegler *Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, NSW, Australia. d.ziegler@unsw.edu.au.ORCID http://orcid.org/0000-0001-7451-7916
Dong-Anh Khuong-Quang *The Royal Children's Hospital, Melbourne, VIC, Australia. donganh.khuongquang@rch.org.au.ORCID http://orcid.org/0000-0001-6305-7790

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Precision-guided therapy (PGT) approaches have shown improved survival in children with high-risk cancer, but it is unclear whether children with H3K27-altered diffuse midline glioma (DMG) benefit from this approach. Here we report on the cohort of patients with DMG who were consecutively enroled in the Australian Childhood Cancer PRecISion Medicine for Children with Cancer (PRSIM) clinical trial (NCT03336931), a prospective cohort study aimed at providing PGT recommendations based on comprehensive molecular profiling. A total of 68 patients with DMG were enroled and 58 (85%) had WGS and RNAseq performed. PGT was recommended for 50 patients (74%). Eighteen patients (26%), who received 21 PGTs, were eligible for survival analysis. The overall response rate (CR or PR) was 48% (n = 10). Median overall survival for the PGT group was 21.3 months versus 12.1 months for those that did not receive PGT (p = 0.34). Patients who received PGT after tumor progression had significantly improved overall survival (p = 0.007) and subsequent progression-free survival (p = 0.001) compared to those that received unguided therapy or reirradiation alone. These results highlight the feasibility of a precision-guided approach and demonstrate its potential clinical benefit in patients with DMG.

Indexed as

Brain NeoplasmsGliomaHistonesPrecision MedicineAdolescentChildChild, PreschoolFemaleHumansInfantMaleProgression-Free SurvivalProspective StudiesTreatment OutcomeHistones

Identifiers

PMID42156385
PMCPMC13381652

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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 OpenQuestion graph.