Evidence map›Paper›PMID 24440717›Full record

Trial reportCell reports2014

Assessing PIK3CA and PTEN in early-phase trials with PI3K/AKT/mTOR inhibitors.

Filip Janku, David S Hong, Siqing Fu, Sarina A Piha-Paul, Aung Naing, Gerald S Falchook, Apostolia M Tsimberidou, Vanda M Stepanek, Stacy L Moulder, J Jack Lee and 5 more

Registry-linked trialOpen access · goldAbstract readClinical Trial
In one paragraph

Trial report in Cell reports, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05983159 (A Modular Open Label, Signal Seeking, Phase II Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations), which is not on this map. Cited by 146 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
146citing papers in PubMed, 1 pooled it
14.5field-weighted citation impact, top 1% 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.

NCT05983159 phase2recruitingnot on this mapstarted 2024, after this paper: background citation

A Modular Open Label, Signal Seeking, Phase II Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations (TARGET-VM)

TypeinterventionalSponsorMurdoch Childrens Research InstituteRan2024 to 2027Enrolled50ConditionsSlow-Flow Vascular Malformation, Fast-Flow Vascular Malformation, Vascular Malformations, Venous MalformationArmsAlpelisib, Mirdametinib
3 · Its place in the literature

Who cites it

146 citing papers in PubMed, 1 synthesis or guideline pooled it, 237 citations in OpenAlex.

  1. Pooled it
  2. PIK3CB Inhibitor GSK2636771 in Cancers WithJCO precision oncology · 2025
    Trial
  3. Trial
  4. Phase II study of BKM120 in patients with advanced esophageal squamous cell carcinoma (EPOC1303).Esophagus : official journal of the Japan Esophageal Society · 2022
    Trial
  5. Trial
  6. Phase II Study of Copanlisib in Patients With Tumors WithJournal of clinical oncology : official journal of the American Society of Clinical Oncology · 2022
    Trial
  7. Trial
  8. Trial
  9. Trial
  10. Trial
  11. Article
  12. Review
  13. Review
  14. Article
  15. Article
  16. HPV-Independent Cervical Cancer-A New Challenge of Modern Oncology.International journal of molecular sciences · 2025
    Review
  17. Article
  18. Review
  19. Review
  20. The treatment landscape of triple-negative breast cancer.Medical oncology (Northwood, London, England) · 2024
    Review

86 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

15 authors at 2 institutions in 1 country.

Filip JankuDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA. Electronic address: fjanku@mdanderson.org.
David S HongDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Siqing FuDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Sarina A Piha-PaulDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Aung NaingDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Gerald S FalchookDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Apostolia M TsimberidouDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Vanda M StepanekDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Stacy L MoulderDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
J Jack LeeDepartment of Biostatistics, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Rajyalakshmi LuthraMolecular Diagnostics Laboratory, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Ralph G ZinnerDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Russell R BroaddusDepartment of Pathology, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Jennifer J WhelerDepartment of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA.
Razelle KurzrockMoores Cancer Center, University of California, San Diego, 3855 Health Sciences Drive, La Jolla, CA 92093, USA.
The University of Texas MD Anderson Cancer Center · USUniversity of California, San Diego · US

Funding

Tumor Evolution and Metastasis ProgramP30CA016672 · NCI · UNIVERSITY OF TX MD ANDERSON CAN CTR · PI Scott Kopetz · 1985 to 2026
$290.8M
U OF TEXAS HLTH SCI CTR HOUSTON CENTER FOR CLINICAL AND TRANSLATIONAL SCIENCESUL1RR024148 · NCRR · UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON · PI MCPHERSON, DAVID D · 2006 to 2010
$27.9M
NCI NIH HHS P30 CA016672NCRR NIH HHS RR024148NCRR NIH HHS UL1 RR024148
6 · The paper itself

Abstract

Despite a wealth of preclinical studies, it is unclear whether PIK3CA or phosphatase and tensin homolog (PTEN) gene aberrations are actionable in the clinical setting. Of 1,656 patients with advanced, refractory cancers tested for PIK3CA or PTEN abnormalities, PIK3CA mutations were found in 9% (146/1,589), and PTEN loss and/or mutation was found in 13% (149/1,157). In multicovariable analysis, treatment with a phosphatidylinositol 3-kinase (PI3K)/AKT/mammalian target of rapamycin (mTOR) inhibitor was the only independent factor predicting response to therapy in individuals harboring a PIK3CA or PTEN aberration. The rate of stable disease ≥6 months/partial response reached 45% in a subgroup of individuals with H1047R PIK3CA mutations. Aberrations in the PI3K/AKT/mTOR pathway are common and potentially actionable in patients with diverse advanced cancers. This work provides further important clinical validation for continued and accelerated use of biomarker-driven trials incorporating rational drug combinations.

Indexed as

AdolescentAdultAgedAged, 80 and overClass I Phosphatidylinositol 3-KinasesColorectal NeoplasmsFemaleHumansMaleMelanomaMiddle AgedMutationOvarian NeoplasmsPhosphatidylinositol 3-KinasesPhosphoinositide-3 Kinase InhibitorsProtein Kinase InhibitorsClass I Phosphatidylinositol 3-KinasesPhosphatidylinositol 3-KinasesPhosphoinositide-3 Kinase InhibitorsPIK3CA protein, humanProtein Kinase InhibitorsProto-Oncogene Proteins c-aktPTEN PhosphohydrolasePTEN protein, humanTOR Serine-Threonine Kinases

Identifiers

PMID24440717
PMCPMC4409143
OpenAlexW2086924064

What OpenQuestion holds

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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.