Evidence map›Paper›PMID 37353295›Full record

ReviewBest practice & research. Clinical haematology2023

Designing and conducting a clinical trial in blood and marrow transplantation.

Michael J Martens, Yan Gao, Aniko Szabo

Erratum issuedOpen access · greenAbstract readReview
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. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.3field-weighted citation impact, top 38% 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.

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

1 citing paper in PubMed, 1 citations in OpenAlex.

  1. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

3 authors at 1 institution in 1 country.

Michael J MartensDivision of Biostatistics, Institute for Health & Equity, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI, 53226, USA; Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, USA. Electronic address: mmartens@mcw.edu.
Yan GaoDivision of Biostatistics, Institute for Health & Equity, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI, 53226, USA. Electronic address: yagao@mcw.edu.
Aniko SzaboDivision of Biostatistics, Institute for Health & Equity, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI, 53226, USA. Electronic address: aszabo@mcw.edu.
Medical College of Wisconsin · US

Funding

Blood and Marrow Transplant Clinical Trials Network DCC- The Medical College of Wisconsin, Inc.U24HL138660 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI Steven M. DeVine, Mehdi Hussain Hamadani · 2017 to 2026
$71.4M
Easy-to-read Informed Consent for HCT Clinical TrialsU10HL069294 · NHLBI · MEDICAL COLLEGE OF WISCONSIN · PI HOROWITZ, MARY MARESCA · 2011 to 2016
$43.8M
NHLBI NIH HHS U10 HL069294NHLBI NIH HHS U24 HL138660
6 · The paper itself

Abstract

Clinical trials form the cornerstone of the science-based approach to improving patient outcomes. A trial needs to be designed and performed carefully to provide valid evidence to inform medical science and to protect the safety and well-being of its participants. The development of a clinical trial involving blood and marrow transplant (BMT) requires special considerations, including the rare disease populations involved and transplant-specific outcomes of interest that necessitate appropriate analysis techniques to evaluate. This article reviews key considerations and best practices for the design and conduct of a clinical trial in BMT, including the selection of patient population, treatment groups, objectives and endpoints, targeted sample size, statistical analysis strategy, provisions for monitoring patient safety and trial progress, and dissemination of trial results. The practical application of these principles is demonstrated using BMT CTN 1301, a recently completed clinical trial evaluating regimens for chronic graft-versus-host disease prevention in transplant patients.

Indexed as

Bronchiolitis Obliterans SyndromeHematopoietic Stem Cell TransplantationBone MarrowBone Marrow TransplantationClinical Trials as TopicHumansBlood and marrow transplantationClinical trial designSafety monitoring committeeSample sizeStudy endpoints

Identifiers

PMID37353295
PMCPMC10845212
OpenAlexW4376126890

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

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.