Evidence map›Paper›PMID 39209468›Full record

ReviewThe European respiratory journal2024

Clinical trial design, end-points, and emerging therapies in pulmonary arterial hypertension.

Jason Weatherald, Thomas R Fleming, Martin R Wilkins, Thomas M Cascino, Mitchell A Psotka, Roham Zamanian, Werner Seeger, Nazzareno Galiè, Mardi Gomberg-Maitland

Abstract readReview
In one paragraph

Review in The European respiratory journal, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing 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

17 citing papers in PubMed.

  1. Trial
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  8. Review
  9. Article
  10. Article
  11. Article
  12. Article
  13. Pulmonary hypertension aetiologies in different parts of the world.International journal of cardiology. Congenital heart disease · 2025
    Review
  14. Another Act in the Story of Oral Imatinib for Pulmonary Arterial Hypertension.American journal of respiratory and critical care medicine · 2025
    Article
  15. Article
  16. Review
  17. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

9 authors.

Jason WeatheraldDepartment of Medicine, Division of Pulmonary Medicine, University of Alberta, Edmonton, AB, Canada weathera@ualberta.ca.ORCID https://orcid.org/0000-0002-0615-4575
Thomas R FlemingDepartment of Biostatistics, University of Washington, Seattle, WA, USA.
Martin R WilkinsNational Heart and Lung Institute, Imperial College London, London, UK.ORCID https://orcid.org/0000-0003-3926-1171
Thomas M CascinoDivision of Cardiovascular Medicine, University of Michigan, Ann Arbor, MI, USA.
Mitchell A PsotkaInova Schar Heart and Vascular, Falls Church, VA, USA.
Roham ZamanianVera Moulton Wall Center for Pulmonary Vascular Disease, Stanford University School of Medicine, Palo Alto, CA, USA.
Werner SeegerDepartment of Internal Medicine, Universities of Giessen and Marburg Lung Center (UGMLC), Institute for Lung Health (ILH), Cardio-Pulmonary Institute (CPI), Member of the German Center for Lung Research (DZL), Giessen, Germany.ORCID https://orcid.org/0000-0003-1946-0894
Nazzareno GalièCardiology Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna and Dipartimento DIMEC, Università di Bologna, Bologna, Italy.
Mardi Gomberg-MaitlandDivision of Cardiovascular Medicine, Department of Medicine, George Washington University, School of Medicine, Washington, DC, USA.

Funding

The Cellular Response to Elafin in PAHP01HL108797 · NHLBI · STANFORD UNIVERSITY · PI ZAMANIAN, ROHAM · 2011 to 2021
$19.7M
STATISTICAL ISSUES IN AIDS RESEARCHR37AI029168 · NIAID · UNIVERSITY OF WASHINGTON · PI JAMES P HUGHES · 1999 to 2026
$9.8M
Training to Advance Care Through Implementation Science in Cardiac And Lung Illnesses (TACTICAL)K12HL138039 · NHLBI · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI BARNES, GEOFFREY DOUGLAS, PRESCOTT, HALLIE CHRISTINE · 2017 to 2021
$3.1M
NHLBI NIH HHS K12 HL138039NHLBI NIH HHS P01 HL108797NIAID NIH HHS R37 AI029168
6 · The paper itself

Abstract

Clinical trials in pulmonary arterial hypertension (PAH) have led to the approval of several effective treatments that improve symptoms, exercise capacity and clinical outcomes. In phase 3 clinical trials, primary end-points must reflect how a patient "feels, functions or survives". In a rare disease like PAH, with an ever-growing number of treatment options and numerous candidate therapies being studied, future clinical trials are now faced with challenges related to sample size requirements, efficiency and demonstration of incremental benefit on traditional end-points in patients receiving background therapy with multiple drugs. Novel clinical trial end-points, innovative trial designs and statistical approaches and new technologies may be potential solutions to tackle the challenges facing future PAH trials, but these must be acceptable to patients and regulatory bodies while preserving methodological rigour. In this World Symposium on Pulmonary Hypertension task force article, we address emerging trial end-points and designs, biomarkers and surrogate end-point validation, the concept of disease modification, challenges and opportunities to address diversity and representativeness, and the use of new technologies such as artificial intelligence in PAH clinical trials.

Indexed as

Clinical Trials as TopicEndpoint DeterminationPulmonary Arterial HypertensionResearch DesignAntihypertensive AgentsArtificial IntelligenceBiomarkersHumansTreatment OutcomeAntihypertensive AgentsBiomarkers

Identifiers

PMID39209468
PMCPMC11525337

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.