Evidence map›Paper›PMID 32188704›Full record

Trial reportJournal for immunotherapy of cancer2020

Phase 2 study of pembrolizumab in patients with advanced rare cancers.

Aung Naing, Funda Meric-Bernstam, Bettzy Stephen, Daniel D Karp, Joud Hajjar, Jordi Rodon Ahnert, Sarina A Piha-Paul, Rivka R Colen, Camilo Jimenez, Kanwal P Raghav and 30 more

Erratum issued 2 registry-linked trialsOpen access · goldAbstract readClinical Trial, Phase IIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal for immunotherapy of cancer, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to 2 registered trials, which are not on this map. Cited by 95 papers, 5 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
95citing papers in PubMed, 5 pooled it
8.2field-weighted citation impact, top 2% 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.

NCT02721732 phase2active not recruitingnot on this map

Phase II Study for the Evaluation of Efficacy of Pembrolizumab (MK-3475) in Patients With Rare Tumors

TypeinterventionalSponsorM.D. Anderson Cancer CenterRan2016 to 2028Enrolled157ConditionsAdvanced Malignant Solid Neoplasm, Carcinoma of Unknown Primary, Metastatic Adrenal Gland Pheochromocytoma, Metastatic Kidney Medullary CarcinomaArmsLaboratory Biomarker Analysis, Pembrolizumab, Questionnaire Administration
NCT07085572 phase2recruitingnot on this mapstarted 2025, after this paper: background citation

Cemiplimab as Maintenance Treatment for Advanced Adrenocortical Cancer

TypeinterventionalSponsorAzienda Socio Sanitaria Territoriale degli Spedali Civili di BresciaRan2025 to 2028Enrolled31ConditionsAdrenal Cortical CarcinomaArmsCemiplimab
3 · Its place in the literature

Who cites it

95 citing papers in PubMed, 5 syntheses or guidelines pooled it, 127 citations in OpenAlex.

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  7. SWOG/NCI Phase II Dual Anti-CTLA-4/PD-1 Blockade in Rare Tumors: Nonepithelial Ovarian Cancer.Clinical cancer research : an official journal of the American Association for Cancer Research · 2024
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35 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

40 authors at 2 institutions in 1 country.

Aung NaingInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA anaing@mdanderson.org.ORCID 0000-0002-4803-8513
Funda Meric-BernstamInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Bettzy StephenInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Daniel D KarpInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Joud HajjarSection of Immunology, Allergy and Rheumatology, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.
Jordi Rodon AhnertInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Sarina A Piha-PaulInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Rivka R ColenCancer Systems Imaging, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Camilo JimenezEndocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Kanwal P RaghavGastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Renata FerrarottoThoracic/Head & Neck Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Shi-Ming TuGenitourinary Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Matthew CampbellGenitourinary Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Linghua WangGenomic Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Sarjeel H SabirInterventional Radiology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Coya TapiaTranslational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Chantale BernatchezMelanoma Medical Oncology-Research, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Michael FrumovitzGynecologic Oncology and Reproductive Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Nizar TannirGenitourinary Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Vinod RaviSarcoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Saria KhanInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Jeane M PainterInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Abulrahman AbonofalInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Jing GongInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Anas AlshawaInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Lacey M McQuinnInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Mingxuan XuInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Sara AhmedCancer Systems Imaging, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Vivek SubbiahInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
David S HongInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Shubham PantInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Timothy A YapInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Apostolia M TsimberidouInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Ecaterina E Ileana DumbravaInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Filip JankuInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Siqing FuInvestigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Richard M SimonR Simon Consulting, Potomac, Maryland, USA.
Kenneth R HessBiostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Gauri R VaradhacharyGastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Mouhammed Amir HabraEndocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.ORCID 0000-0002-4148-9637
The University of Texas MD Anderson Cancer Center · USBaylor College of Medicine · US

Funding

Tumor Evolution and Metastasis ProgramP30CA016672 · NCI · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI Fabien Delerue · 1985 to 2026
$290.8M
Convalescent Plasma to Limit Coronavirus Associated ComplicationsUL1TR003167 · NCATS · UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON · PI KARP, DANIEL D, MCPHERSON, DAVID D · 2019 to 2023
$45.3M
Paul Calabresi Clinical Oncology AwardK12CA088084 · NCI · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI David S. Hong, Scott Kopetz · 2000 to 2026
$14.0M
NCATS NIH HHS UL1 TR003167NCI NIH HHS K12 CA088084NCI NIH HHS P30 CA016672
6 · The paper itself

Abstract

backgroundPatients with advanced rare cancers have poor prognosis and few treatment options. As immunotherapy is effective across multiple cancer types, we aimed to assess pembrolizumab (programmed cell death 1 (PD-1) inhibitor) in patients with advanced rare cancers.

methodsIn this open-label, phase 2 trial, patients with advanced rare cancers whose tumors had progressed on standard therapies, if available, within the previous 6 months were enrolled in nine tumor-specific cohorts and a 10th cohort for other rare histologies. Pembrolizumab 200 mg was administered intravenously every 21 days. The primary endpoint was non-progression rate (NPR) at 27 weeks; secondary endpoints were safety and tolerability, objective response rate (ORR), and clinical benefit rate (CBR).

resultsA total of 127 patients treated between August 15, 2016 and July 27, 2018 were included in this analysis. At the time of data cut-off, the NPR at 27 weeks was 28% (95% CI, 19% to 37%). A confirmed objective response (OR) was seen in 15 of 110 (14%) evaluable patients (complete response in one and partial response in 14). CBR, defined as the percentage of patients with an OR or stable disease ≥4 months, was 38% (n=42). Treatment was ongoing in 11 of 15 patients with OR at last follow-up. In the cohort with squamous cell carcinoma (SCC) of the skin, the NPR at 27 weeks was 36%, ORR 31%, and CBR 38%. In patients with adrenocortical carcinoma (ACC), NPR at 27 weeks was 31%, ORR 15%, and CBR 54%. In the patients with carcinoma of unknown primary (CUP), NPR at 27 weeks was 33%, ORR 23%, and CBR 54%. In the paraganglioma-pheochromocytoma cohort, NPR at 27 weeks was 43%, ORR 0%, and CBR 75%. Treatment-related adverse events (TRAEs) occurred in 66 of 127 (52%) patients, and 12 (9%) had grade ≥3 TRAEs. The most common TRAEs were fatigue (n=25) and rash (n=17). There were six deaths, all of which were unrelated to the study drug.

conclusionsThe favorable toxicity profile and antitumor activity seen in patients with SCC of skin, ACC, CUP, and paraganglioma-pheochromocytoma supports further evaluation of pembrolizumab in this patient population. TRIAL REGISTRATION NUMBER: NCT02721732.

Indexed as

AdultAgedAged, 80 and overAntibodies, Monoclonal, HumanizedAntineoplastic Agents, ImmunologicalCarcinoma, Squamous CellCohort StudiesFemaleFollow-Up StudiesHumansMaleMiddle AgedNeoplasmsPrognosisRare DiseasesSurvival RateAntibodies, Monoclonal, HumanizedAntineoplastic Agents, Immunologicalpembrolizumaboncologytumours

Identifiers

PMID32188704
PMCPMC7078933
OpenAlexW3010680101

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

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.