Evidence map›Paper›PMID 40775758›Full record

ArticleBMC medical research methodology2025

Longitudinal graphics of patient-reported physical function in patients treated for hematologic malignancies.

Gita Thanarajasingam, Vishal Bhatnagar, Brie N Noble, Ting-Yu Chen, Mallorie H Fiero, Ron Hoffman, Molly Jeffery, Gina L Mazza, John Mascarenhas, Ruben Mesa and 6 more

2 registry-linked trialsAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in BMC medical research methodology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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.

NCT01259817 phase2completednot on this map

Single Arm Salvage Therapy With Pegylated Interferon Alfa-2a for Patients With High Risk Polycythemia Vera or High Risk Essential Thrombocythemia Who Are Either Hydroxyurea Resistant or Intolerant or Have Had Abdominal Vein Thrombosis

TypeinterventionalSponsorRonald HoffmanRan2011 to 2016Enrolled135ConditionsHigh Risk Polycythemia Vera, High Risk Essential ThrombocythemiaArmsPEGASYS, Aspirin
NCT01259856 phase3completednot on this map

Randomized Trial of Pegylated Interferon Alfa-2a Versus Hydroxyurea Therapy in the Treatment of High Risk Polycythemia Vera (PV) and High Risk Essential Thrombocythemia (ET)

TypeinterventionalSponsorRonald HoffmanRan2011 to 2017Enrolled168ConditionsHigh Risk Polycythemia Vera, High Risk Essential ThrombocythemiaArmsPEGASYS, Hydroxyurea, Aspirin
3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Trial
  2. Review
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

16 authors.

Gita ThanarajasingamDivision of Hematology, Mayo Clinic, 200 First Street SW, Rochester, MN, USA. thanarajasingam.gita@mayo.edu.
Vishal BhatnagarOncology Center for Excellence U.S. Food and Drug Administration (U.S. FDA), Silver Spring, MD, USA.
Brie N NobleDepartment of Quantitative Health Sciences, Mayo Clinic, Scottsdale, AZ, USA.
Ting-Yu ChenOffice of Biostatistics, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
Mallorie H FieroOffice of Biostatistics, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
Ron HoffmanTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Molly JefferyDivision of Health Care Delivery Research, Kern Center for the Science of Health Care Delivery, Mayo Clinic, Rochester, MN, USA.
Gina L MazzaDepartment of Quantitative Health Sciences, Mayo Clinic, Scottsdale, AZ, USA.
John MascarenhasTisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Ruben MesaAtrium Health Wake Forest Baptist Comprehensive Cancer Center, Winston-Salem, NC, USA.
Meena MurugappanOffice of Biostatistics, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
Joseph RossDepartment of Health Policy and Management, Yale School of Public Health, New Haven, CT, USA.
Surbhi SidanaDivision of Blood and Marrow Transplantation and Cellular Therapy, Department of Medicine, Stanford University School of Medicine, Stanford, CA, USA.
Rahma WarsameDivision of Hematology, Mayo Clinic, 200 First Street SW, Rochester, MN, USA.
Paul G KluetzOncology Center for Excellence U.S. Food and Drug Administration (U.S. FDA), Silver Spring, MD, USA.
Amylou C DueckDepartment of Quantitative Health Sciences, Mayo Clinic, Scottsdale, AZ, USA.

Funding

Women's Cancer ProgramP30CA015083 · NCI · MAYO CLINIC ROCHESTER · PI AKHILESH PANDEY · 1985 to 2026
$151.3M
Tissue BankP01CA108671 · NCI · UNIVERSITY OF ILLINOIS AT CHICAGO · PI Ronald Hoffman · 2006 to 2026
$82.0M
NCI NIH HHS P01 CA108671NIH HHS P01CA108671NIH HHS P30CA015083Oncology Center of Excellence, Food and Drug Administration U01FD005938
6 · The paper itself

Abstract

backgroundThe US Food and Drug Administration (FDA) released a draft guidance document detailing core patient-reported outcomes in cancer clinical trials, including physical function (PF). The objectives of this study were to develop analytic methods and visualizations of patient-reported PF in patients with cancer.

methodsWe applied an estimand framework to a patient-reported tolerability endpoint to develop data summaries cross-sectionally and over time, along with visualizations. We accomplished this through iterative feedback with clinicians, statisticians, and FDA stakeholders using three clinical trial datasets in hematologic malignancies. Graphical approaches were applied to three datasets in hematologic malignancies: (1) patients with myeloproliferative neoplasms enrolled in MPN-RC 111/112 trials completed EORTC QLQ-C30 over 12 months; (2) patients with hematologic malignancies undergoing CAR-T cell therapy or autologous transplant who completed FACT questionnaires over 6 months; and (3) patients with multiple myeloma or amyloidosis who completed the PROMIS-29 questionnaire over 6 months. Zoom polls were administered to two stakeholder groups (clinicians/clinical investigators and patient advocates) to elicit feedback.

resultsVisualizations included stacked bar charts, line plots of arithmetic mean changes from baseline, pie charts, waffle plots, and waterfall plots of PF data. Graphics considered scaled scores and individual items and included delineation of PRO completion rate at each time point. Confidence intervals and reference lines were included as applicable, and colorblind accessible colors were implemented to ensure inclusivity of all visualizations. Data summaries over time reporting "worst" change were difficult to interpret. In terms of stakeholders' preference, patients preferred stacked bar charts while clinicians equally favored stacked bar charts and line plots; both patients and clinicians preferred waterfall plots to pie charts. Patient feedback highlighted the need for various graphics to convey group level trends and granular individual-patient level information.

conclusionPatient-reported PF informs the evaluation of treatment tolerability in cancer trials. Data summaries and visualizations of physical function developed through an iterative process were reviewed favorably by patients, clinicians and FDA stakeholders in this study. Future work to systematically assess accuracy of interpretation of the various analytic and visualization methods is a necessary next step across clinical, regulatory, payer and patient stakeholders.

trial registrationNCT01259817, NCT01259856.

Indexed as

Hematologic NeoplasmsPatient Reported Outcome MeasuresClinical Trials, Phase II as TopicClinical Trials, Phase III as TopicCross-Sectional StudiesFemaleHumansLongitudinal StudiesMaleMiddle AgedMulticenter Studies as TopicQuality of LifeRandomized Controlled Trials as TopicSurveys and QuestionnairesUnited StatesUnited States Food and Drug AdministrationCancerGraphicsLongitudinal analysisPhysical functionTreatment tolerability

Identifiers

PMID40775758
PMCPMC12329971

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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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.