Evidence map›Paper›PMID 31362788›Full record

SynthesisActa neuropathologica communications2019

Phase I and phase II sonidegib and vismodegib clinical trials for the treatment of paediatric and adult MB patients: a systemic review and meta-analysis.

Yuchen Li, Qingkun Song, Bryan W Day

Open access · goldAbstract readMeta-AnalysisSystematic Review
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in Acta neuropathologica communications, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 75 papers.

0numbers the graph read from it
0cells of the map it votes in
75citing papers in PubMed
6.2field-weighted citation impact, top 3% 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

75 citing papers in PubMed, 112 citations in OpenAlex.

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  10. A transcription regulator atlas identifies TOX3 as an Atoh1 coactivator in cerebellar development and tumorigenesis.Proceedings of the National Academy of Sciences of the United States of America · 2026
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15 more citing papers are in PubMed but not listed here.

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

3 authors at 3 institutions in 2 countries.

Yuchen LiDepartment of Cell and Molecular Biology, QIMR Berghofer Medical Research Institute, Brisbane, 4006, Australia.
Qingkun SongDepartment of Science and Technology, Beijing Shijitan Hospital, Capital Medical University, Beijing, 10038, China.
Bryan W DayDepartment of Cell and Molecular Biology, QIMR Berghofer Medical Research Institute, Brisbane, 4006, Australia. Bryan.Day@qimrberghofer.edu.au.ORCID 0000-0002-8948-9089
Capital Medical University · CNQueensland University of Technology · AUThe University of Queensland · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundMedulloblastoma (MB) is the most common malignant brain tumour in children but also rarely occur in adults. Sonic Hedgehog (SHH) driven MB is associated with aberrant activation of the SHH signalling pathway. SMO inhibitors, sonidegib and vismodegib, have been used as selective antagonist of the hedgehog pathway that acts by binding to SMO, and inhibits activation of the downstream hedgehog target genes. Several clinical trials investigating SMO inhibitors for the treatment of relapsed MB patients have been published.

methodsWe conducted a systemic review and meta-analysis among these Phase I and II clinical trials. The pooled effect of SMO inhibitors in relapsed MB were analysed using Reviewer Manager 5.3 software. The clinical efficacy of SMO inhibitors on SHH subtype of MB were measured by the objective response rate. The risk difference was obtained by comparing the ORR between SHH and non-SHH subtypes of MB.

resultsThe five studies all had clear criteria for patient recruitment, adequate follow-up time for endpoint assessment and clear definition of tumour responses. MB patients had good compliance in the trials. The pooled objective response rate (ORR) of SMO inhibitor was 37% and 0 against SHH-driven and other MBs. The pooled ORR of sonidegib was 55% among MB

conclusionSonidegib and vismodegib were well tolerated and demonstrated anti-tumour activity in SHH-driven paediatric and adult MB by effectively inhibiting Hh signalling. These results support the ongoing clinical trials using SMO inhibitors in combination with conventional chemotherapies for the treatment of relapsed MB

Indexed as

AnilidesAntineoplastic AgentsBiphenyl CompoundsCerebellar NeoplasmsClinical Trials, Phase I as TopicClinical Trials, Phase II as TopicHedgehog ProteinsHumansMedulloblastomaPyridinesSignal TransductionSmoothened ReceptorTreatment OutcomeAnilidesAntineoplastic AgentsBiphenyl CompoundsHedgehog ProteinsHhAntag691PyridinesSHH protein, humanSmoothened ReceptorsonidegibAnd vismodegibMedulloblastomaSMO inhibitorSonic hedgehog pathwaySonidegib

Identifiers

PMID31362788
PMCPMC6668073
OpenAlexW2964608150

What OpenQuestion holds

Textmetadata
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.