Evidence map›Paper›PMID 38811045›Full record

Trial reportThe European respiratory journal2024

Inhaled treprostinil in pulmonary hypertension associated with COPD: PERFECT study results.

Steven D Nathan, Rahul Argula, Maria G Trivieri, Sameh Aziz, Elizabeth Gay, Boris Medarov, Joseph Parambil, Amresh Raina, Michael G Risbano, Thenappan Thenappan and 11 more

Registry-linked trialAbstract readRandomized Controlled TrialMulticenter Study
In one paragraph

Trial report in The European respiratory journal, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03496623 (A Phase 3, Randomized, Placebo-controlled, Double-blind, Adaptive Study to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Patients With Pulmonary Hypertension Due to Chronic Obstructive Pulmonary Disease), which is not on this map. Cited by 41 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
41citing papers in PubMed, 3 pooled it
–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.

NCT03496623 phase3terminatednot on this map

A Phase 3, Randomized, Placebo-controlled, Double-blind, Adaptive Study to Evaluate the Safety and Efficacy of Inhaled Treprostinil in Patients With Pulmonary Hypertension Due to Chronic Obstructive Pulmonary Disease (PH-COPD)

TypeinterventionalSponsorUnited TherapeuticsRan2018 to 2022Enrolled188ConditionsPulmonary Hypertension, Chronic Obstructive Pulmonary DiseaseArmsInhaled treprostinil solution, Placebo solution
3 · Its place in the literature

Who cites it

41 citing papers in PubMed, 3 syntheses or guidelines pooled it.

  1. Pooled it
  2. Guideline
  3. Pooled it
  4. Trial
  5. Review
  6. Review
  7. Article
  8. Review
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  10. Review
  11. Review
  12. Article
  13. Review
  14. Article
  15. Update on pulmonary hypertension.Respiratory research · 2026
    Review
  16. Article
  17. Article
  18. Article
  19. Article
  20. Review
4 · The record

Corrections and comments

  • Commented on by
    2024
5 · Who and what money

Authors and funding

21 authors.

Steven D NathanInova Fairfax Hospital, Falls Church, VA, USA steven.nathan@inova.org.ORCID https://orcid.org/0000-0002-6270-1617
Rahul ArgulaMedical University of South Carolina, Charleston, SC, USA.
Maria G TrivieriDivision of Cardiology, Icahn School of Medicine, Mount Sinai Hospital, New York, NY, USA.
Sameh AzizCarilion Clinic, VTC School of Medicine, Roanoke, VA, USA.
Elizabeth GayBrigham and Women's Hospital, Boston, MA, USA.
Boris MedarovAlbany Medical Center, Albany, NY, USA.ORCID https://orcid.org/0000-0002-8132-7122
Joseph ParambilCleveland Clinic, Cleveland, OH, USA.
Amresh RainaAllegheny General Hospital, Pittsburgh, PA, USA.
Michael G RisbanoUniversity of Pittsburgh Medical Center, Pittsburgh, PA, USA.ORCID https://orcid.org/0000-0003-3334-7046
Thenappan ThenappanUniversity of Minnesota Medical Center, Minneapolis, MN, USA.
Jose Soto SotoAscension St Vincent's Southside Hospital, Jacksonville, FL, USA.
Heidi BellUnited Therapeutics, Research Triangle Park, NC, USA.
Victoria LacasseUnited Therapeutics, Silver Spring, MD, USA.
Prakash SistaUnited Therapeutics, Silver Spring, MD, USA.
Michael Di MarinoUnited Therapeutics, Silver Spring, MD, USA.
Aimee SmartUnited Therapeutics, Silver Spring, MD, USA.
Brittanie HawkesUnited Therapeutics, Silver Spring, MD, USA.
Elizabeth NelsonUnited Therapeutics, Silver Spring, MD, USA.
Todd BullUniversity of Colorado, Denver, CO, USA.
Victor TapsonCedars Sinai Medical Center, Los Angeles, CA, USA.
Aaron WaxmanBrigham and Women's Hospital, Boston, MA, USA.ORCID https://orcid.org/0000-0001-7797-0361

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundPulmonary hypertension (PH) accompanying COPD (PH-COPD) is associated with worse outcomes than COPD alone. There are currently no approved therapies to treat PH-COPD. The PERFECT study (ClinicalTrials.gov: NCT03496623) evaluated the safety and efficacy of inhaled treprostinil (iTRE) in this patient population.

methodsPatients with PH-COPD (mean pulmonary arterial pressure ≥30 mmHg and pulmonary vascular resistance ≥4 WU) were enrolled in a multicentre, randomised (1:1), double-blind, placebo-controlled, 12-week, crossover study. A contingent parallel design was also prespecified and implemented, based on a blinded interim analysis of missing data. Patients received treatment with iTRE up to 12 breaths (72 µg) 4 times daily or placebo. The primary efficacy end-point was change in peak 6-min walk distance (6MWD) at week 12.

resultsIn total, 76 patients were randomised, 64 in the original crossover design and 12 in the contingent parallel design; 66 patients received iTRE and 58 received placebo. The study was terminated early at the recommendation of the data and safety monitoring committee based on the totality of evidence that iTRE increased the risk of serious adverse events and suggestive evidence of an increased risk of mortality. The change in 6MWD was numerically worse with iTRE exposure than with placebo exposure.

conclusionsThe risk-benefit observations associated with iTRE in patients with PH-COPD did not support continuation of the PERFECT study. The results of this study do not support iTRE as a viable treatment option in patients with PH-COPD.

Indexed as

Antihypertensive AgentsCross-Over StudiesEpoprostenolHypertension, PulmonaryPulmonary Disease, Chronic ObstructiveWalk TestAdministration, InhalationAgedDouble-Blind MethodFemaleHumansMaleMiddle AgedTreatment OutcomeAntihypertensive AgentsEpoprostenoltreprostinil

Identifiers

PMID38811045
PMCPMC11154754

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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.