Evidence map›Paper›PMID 37611997›Full record

ReviewBest practice & research. Clinical haematology2023

Endpoint selection and evaluation in hematology studies.

Ruta Brazauskas, Mary Eapen, Tao Wang

Erratum issuedAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Best practice & research. Clinical haematology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. 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

5 · Who and what money

Authors and funding

3 authors.

Ruta BrazauskasDivision of Biostatistics, Institute for Health and Equity, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI, 53226, USA. Electronic address: ruta@mcw.edu.
Mary EapenDivision of Hematology and Oncology, Department of Medicine, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI, 53226, USA. Electronic address: meapen@mcw.edu.
Tao WangDivision of Biostatistics, Institute for Health and Equity, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI, 53226, USA. Electronic address: taowang@mcw.edu.

Funding

Data Resource for Analyzing Blood &Marrow TransplantsU24CA076518 · NCI · MEDICAL COLLEGE OF WISCONSIN · PI Amy M Moskop, Bronwen Shaw · 1998 to 2026
$105.2M
NCI NIH HHS U24 CA076518
6 · The paper itself

Abstract

Observational studies and clinical trials in hematology aim to examine treatments for blood disorders. The outcomes being studied must address the goals of the study and provide meaningful information about treatment course, disease progression, describe patients' survival experience and quality of life. Endpoints are the specific measures of these outcomes, and much consideration should be given to their selection. In this review, we describe the outcomes and endpoints frequently used in studying hematologic diseases and provide general guidelines for their statistical analysis. The main focus is on clinical outcomes which are commonly used in establishing treatment safety and efficacy. We also briefly discuss the role surrogate and composite endpoints play in hematology studies. The importance of patient reported outcomes to comprehensive assessment of the treatment effectiveness is highlighted. Provided practical considerations for choosing primary and secondary endpoints may be helpful in designing hematology clinical trials.

Indexed as

HematologyQuality of LifeDisease ProgressionHumansClinical trialsComposite endpointsEndpointsHematologyOutcomesPatient reported outcomesPrimary endpointsSurrogate endpoints

Identifiers

PMID37611997
PMCPMC10979628

What OpenQuestion holds

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Registered trials

None linked

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.