Evidence map›Paper›PMID 42167248›Full record

Trial reportCell reports. Medicine2026

Biomarker study of pembrolizumab in patients with advanced rare cancers.

Aung Naing, Xiqi Li, Qi Wang, Xiangjun Jun Tian, Sharjeel Sabir, Priya R Bhosale, Mohamed H Derbala, Bettzy Stephen, Mingxuan Xu, Joud Hajjar and 20 more

Abstract readClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Cell reports. Medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the 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.

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

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

30 authors.

Aung NaingInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA. Electronic address: anaing@mdanderson.org.
Xiqi LiGenomic Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Qi WangBioinformatics & Comp Biology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Xiangjun Jun TianBioinformatics & Comp Biology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Sharjeel SabirInterventional Radiology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Priya R BhosaleAbdominal Imaging, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Mohamed H DerbalaInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Bettzy StephenInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Mingxuan XuInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Joud HajjarSection of Immunology, Allergy and Rheumatology, Baylor College of Medicine and Texas Children's Hospital, Houston, TX 77030, USA.
Serdar A GursesInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Yali YangInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Hassan Ahmed MominInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Ignacio Ivan WistubaTranslational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Anas AlshawaInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Kenna R ShawInstitute for Personalized Cancer Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Abdulrazzak ZarifaInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Gopal SinghInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Renganayaki Krishna PandurenganTranslational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Gabriela Maria RasoTranslational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Edwin Roger ParraTranslational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Juhee SongBiostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Mohammad Moustaf MohammadInstitute for Personalized Cancer Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Jordi Rodon AhnertInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Siqing FuInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Vivek SubbiahInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Sarina Anne Piha-PaulInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Jing WangBioinformatics & Comp Biology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Scott Eric WoodmanGenomic Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Funda Meric-BernstamInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Rare cancers lack robust, evidence-based treatment options. We conduct a prospective, multi-histology phase 2 clinical trial of pembrolizumab in 154 patients (142 evaluable), observing an objective response rate of 14.8% and clinical benefit (CB) in 26.8%. Multi-modal profiling is performed on baseline, on-treatment, and progression samples. CB associates with high microsatellite instability (MSI-H)/high tumor mutation burden (TMB-H) (odds ratio [OR]: 13.9, p = 0.0013) and programmed cell death ligand 1 (PD-L1) combined positive score (CPS) ≥10 (p = 0.0285), although responses also occur in biomarker-negative tumors and vary by histology. CB tumors exhibit higher pre-treatment immune infiltration and T cell activation, whereas no CB (NCB) tumors show proliferative programs. In moderately infiltrated tumors, CB associates with increased immune content during treatment. Multiplex immunofluorescence confirms higher baseline T cell densities in CB and limited remodeling in NCB tumors. These findings suggest that tumor immune microenvironment features may serve as predictive markers beyond genomic assays and highlight immune cell recruitment during therapy in moderately infiltrated tumors.

Indexed as

Antibodies, Monoclonal, HumanizedAntineoplastic Agents, ImmunologicalBiomarkers, TumorNeoplasmsAdultAgedFemaleHumansMaleMicrosatellite InstabilityMiddle AgedMutationProspective StudiesT-LymphocytesTumor MicroenvironmentAntibodies, Monoclonal, HumanizedAntineoplastic Agents, ImmunologicalBiomarkers, Tumorpembrolizumabbiomarkerimmune infiltrationpembrolizumabrare cancerspatial immune contexttumor microenvironment

Identifiers

PMID42167248
PMCPMC13293929

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Registered trials

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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.