Expert Consensus and Artificial Intelligence in Medical Decision Making in Patients with Malignant Brain Tumors (EC-AIM Brain)

October 14, 2024 updated by: Tufts Medical Center

A Survey Based Study Assessing the Feasibility of Using Standardized Clinical Vignettes to Aid in Medical Decision in Patients with Malignant Brain Tumors

Nearly 23,000 adults are diagnosed with primary central nervous system (CNS) malignancy yearly. An additional 200,000 adults are diagnosed with brain metastasis. There are significant variations in CNS tumor treatment. However, due to significant heterogeneity in patient baseline factors, identifying unwarranted variation is challenging. Ghogawala et al have previously demonstrated that, among patients undergoing surgical treatment of cervical myelopathy and lumbar degenerative spinal disease, an expert panel consisting of surgeon experts can identify variations in proposed surgical procedure and demonstrated superior patient outcomes when the surgery performed matched the procedure recommended by expert consensus. Expert panel surveys have not previously been used to identify variations in care among patients with CNS malignancy.

The primary aim is to determine whether patient outcomes are superior when treatment aligns with recommendations made by a clinical expert neurosurgical panel. The study also seek to identify patient factors that predispose to variability in care. Our long-term aim is to determine whether predictive artificial learning algorithms can achieve the same outcomes, or better, as clinical expert panels, but with greater efficiency and greater capacity to be available for more patients. The investigators hypothesize that:

  • When a team of 10 medical experts has greater than 80% consensus regarding optimal treatment and when the doctor and patient select that specific treatment, the outcome is superior than when a patient and doctor select an alternative procedure.
  • When a team of 10 medical experts has greater than 80% consensus regarding optimal treatment, the structured data used by the experts can be processed and trained by computing algorithms to predict the pattern recognized by the experts - i.e. - the computer can predict how an expert panel would vote.

Procedures include the following:

  1. Chart review portion of study: Patients will be identified from case logs of the principal investigators from July 2017 through July 2023. Data will be collected retrospectively and will include age, non-identifier demographics, diagnosis details, operative/treatment characteristics, post-treatment characteristics, and follow-up characteristics. Images reviewed will include pre and post-treatment MRIs obtained as part of routine care. Data will be abstracted from the medical record (Epic/Soarian and PACS) and recorded in an excel database.
  2. Survey portion of study: De-identified structured radiographic data and a brief clinical vignette without patient identifiers will be uploaded to Acesis Healthcare Process Optimization Platform (http://www.acesis.com/our-platform). A survey will be generated by Acesis and emailed to the subject experts/participants. This portion is prospective.

  3. Cohort definitions:

    1. Patients will be assigned to either "expert-treatment consensus" or "no expert-treatment consensus" arms based on whether greater than 80% consensus is achieved
    2. Patients will be assigned to either "Expert consensus-aligned" or "Expert consensus - unaligned" arms based on whether expert survey results match actual treatment given.
  4. Data will then be analyzed using appropriate packages with SAS statistical analysis software. Survival analysis will be performed to determine whether consensus predicts improved progression free survival (PFS).
  5. The structured and de-identified radiographic images used by the experts in surveys will be used for training and development of an AI algorithm. The aim of this portion of the study is to determine whether standardized and structured imaging can be used to train an algorithm to predict whether expert consensus is achieved and the recommended treatment.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

1) Statistical analysis plan:

  1. this document provides the details of statistical analyses planned for the EC-AIM Brain study.
  2. Objective of this study:

i. The primary objective is to determine whether patients whose treatment aligns with expert consensus have a superior outcome to those who treatment does not align with expert consensus.

  1. This will be determined with a primary endpoint of progression free survival (alive and without tumor growth at last follow up).
  2. This will be tested with the log-rank test for equality of survival curves (EC/aligned vs EC/unaligned).

2) Abbreviations:

  1. EC - expert consensus
  2. HR - hazard ratio
  3. OS - overall survival
  4. PFS - progression free survival
  5. KPS - Karnofsky Performance Score 3) Target population:

a. Inclusion criteria: i. Consecutive patients treated by neurosurgeons at a single center between April 2018 and July 2023 for malignant brain tumors, including glioma, metastasis, and lymphoma.

b. Exclusion criteria:

  1. Patients without available MRI dicom images
  2. Patients with other CNS malignancies
  3. Patients with multiply recurrent gliomas undergoing treatment for primarily palliative purposes
  4. Patients younger than 16 years old 4) Sampling: Consecutive 5) Sample size assessment: a. It is challenging to determine an estimated effect size that EC would provide. From previous research on expert panels in lumbar spondylolisthesis, a sample size of approximately 200 patients was noted to be sufficient for initial assessment. As such, the study will include 225 subjects as our cohort size in order to account for ~10% patient loss to follow up.

6) Data Acquisition Methods:

  1. Standardized clinical vignettes with de-identified clinical data and standardized radiographic images and video will be uploaded to Acesis HPOP platform.

i. Uploaded files will include representative multiplanar magnetic resonance or computed tomography images b. An email with a link to the survey will be distributed to the brain tumor expert panel c. Expert respondents will have 72 hours to respond to a standardized survey d. Expert respondents may answer questions regarding suggested treatment as well as any adjunctive technologies needed 7) Blinding: The chart review portion will be performed by a separate study investigator without knowledge of cohort assignment; cohort allocation (based on survey results and actual treatment) will be performed by a separate study investigator.

8) Missing data:

a. All subjects will be included in primary outcome analysis. They will be censored at the last follow up assessment. All missing data will be assumed to be missing at random.

9) Quality assurance plan: 10) Outcome Group Allocation:

a. Treatment will be defined into the following four categories: i. Observation/no treatment ii. Biopsy only iii. Resection iv. Radiation treatment b. Expert consensus will be defined as at least 80% agreement among at least 10 survey respondents c. Aligned will be defined as the recommended treatment category above matching the actual treatment provided 11) Outcome Analysis:

a. Primary Outcome Variable Analysis: i. The primary outcome measure is progression free survival (alive and without evidence of tumor growth at follow up) ii. Hypothesis:

  1. H0: hEC_aligned ≤ hEC_unaligned
  2. HA: hEC_aligned > hEC_unaligned iii. This will be tested with the log-rank test for equality of survival curves (EC/aligned vs EC/unaligned).

iv. Test will be one-sided (alpha=0.025) b. Additional analyses of the primary outcome: i. Adjustment for known confounders including diagnosis, age, KPS, extent of resection ii. Exploratory data analysis using descriptive statistics and logistic regression/linear regression will be used to evaluate for interactions and confounding co-variates c. Secondary Outcome Variable Analysis: i. Overall survival ii. Data analysis will be exploratory and supportive, and, thus, the statistical plan will not account for multiplicity iii. Secondary analyses will be performed for OS and PFS for two- and three- cohort groups

  1. EC/aligned vs EC/unaligned
  2. EC vs no EC

  3. EC/aligned vs EC/unaligned vs no EC 12) Planned analysis for predictors of EC

    1. Data analysis will be exploratory and supportive, and, thus, the study will not account for multiplicity
    2. Descriptive statistics will be used to evaluate differences between EC and no EC groups
    3. Descriptive statistics will be used to evaluate differences between EC/aligned and EC/unaligned groups
    4. Fisher exact tests and Chi-squared tests will be used for categorical variables depending on test assumptions
    5. Student's t test and Mann-Whitney U tests will be used for normally distributed and non-normally distributed continuous variables, respectively
    6. Logistic regression models will be used to evaluate for predictors of EC
    7. Concordance between treatment performed and expert consensus will be evaluated using Spearman coefficients
    8. All statistical tests used for descriptive statistics will be two-tailed (alpha=0.05) 13) Planned interim analysis: None 14) Adverse events: None expected as this is a retrospective chart review and survey based study.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
225

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02111
        • Tufts Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The population from which sampling will occur is all patients with CNS gliomas, metastatic disease, and lymphoma treated by neurosurgeons.

Description

Inclusion Criteria:

  • consecutive patients treated at a single center between April 2018 and July 2023 for malignant brain tumors, including glioma, metastasis, and lymphoma.

Exclusion Criteria:

  • Patients without available MRI dicom images
  • Patients with other CNS malignancies
  • Patients with multiply recurrent gliomas undergoing treatment for primarily palliative purposes
  • Patients younger than 18 years old

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
EC/aligned
Cohort with expert consensus aligned with actual treatment
Behavioral: Survey
The intervention is an email based survey as described in the study description.
EC/unaligned
Cohort with expert consensus not aligned with actual treatment
Behavioral: Survey
The intervention is an email based survey as described in the study description.
No EC
Expert panel recommended treatment did not achieve 80% agreement
Behavioral: Survey
The intervention is an email based survey as described in the study description.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Overall Survival
Time Frame: Through study completion, an average of 2 years
Patients alive at last follow up through study completion, an average of 2 years
Through study completion, an average of 2 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Progression free survival
Time Frame: Through study completion, an average of two years
Patients without definite evidence of progression by last follow up through study completion, an average of two years
Through study completion, an average of two years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Tufts Medical Center

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Marie Roguski, MD MPH, Tufts Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2017

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2025

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 9, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 14, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 21, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 21, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 14, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • STUDY00003110

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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