Title :

Combination therapy with oral treprostinil for pulmonary arterial hypertension. A double-blind placebo-controlled clinical trial

Author(s) :

White, R. James [Auteur]
University of Rochester Medical Center [URMC]
Jerjes-Sanchez, Carlos [Auteur]
Bohns Meyer, Gisela Martina [Auteur]
Hospital de Clínicas de Porto Alegre [HCPA]
Pulido, Tomas [Auteur]
Instituto Nacional de Cardiologia Ignacio Chavez
Sepulveda, Pablo [Auteur]
Pontificia Universidad Católica de Chile [UC]
Wang, Kuo Yang [Auteur]
Grunig, Ekkehard [Auteur]
Heidelberg University Hospital [Heidelberg]
Hiremath, Shirish [Auteur]
Yu, Zaixin [Auteur]
Gangcheng, Zhang [Auteur]
Yip, Wei Luen James [Auteur]
National University of Singapore [NUS]
Zhang, Shuyang [Auteur]
Peking Union Medical College Hospital [Beijing] [PUMCH]
Khan, Akram [Auteur]
Oregon Health and Science University [Portland] [OHSU]
Deng, C. Q. [Auteur]
Grover, Rob [Auteur]
Tapson, Victor F. [Auteur]
Cedars-Sinai Medical Center

Journal title :

American Journal of Respiratory and Critical Care Medicine

Abbreviated title :

Am. J. Respir. Crit. Care Med.

Volume number :

201

Pages :

707-717

Publication date :

2020-03-15

ISSN :

1535-4970

Keyword(s) :

sequential therapy
pulmonary arterial hypertension
oral treprostinil
clinical study
combination therapy

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

Rationale: Oral treprostinil improves exercise capacity in patients with pulmonary arterial hypertension (PAH), but the effect on clinical outcomes was unknown. Objectives: To evaluate the effect of oral treprostinil ...
Show more >Rationale: Oral treprostinil improves exercise capacity in patients with pulmonary arterial hypertension (PAH), but the effect on clinical outcomes was unknown. Objectives: To evaluate the effect of oral treprostinil compared with placebo on time to first adjudicated clinical worsening event in participants with PAH who recently began approved oral monotherapy. Methods: In this event-driven, double-blind study, we randomly allocated 690 participants (1:1 ratio) with PAH to receive placebo or oral treprostinil extended-release tablets three times daily. Eligible participants were using approved oral monotherapy for over 30 days before randomization and had a 6-minute-walk distance 150 m or greater. The primary endpoint was the time to first adjudicated clinical worsening event: death; hospitalization due to worsening PAH; initiation of inhaled or parenteral prostacyclin therapy; disease progression; or unsatisfactory long-term clinical response. Measurements and Main Results: Clinical worsening occurred in 26% of the oral treprostinil group compared with 36% of placebo participants (hazard ratio, 0.74; 95% confidence interval, 0.56–0.97; P = 0.028). Key measures of disease status, including functional class, Borg dyspnea score, and N-terminal pro–brain natriuretic peptide, all favored oral treprostinil treatment at Week 24 and beyond. A noninvasive risk stratification analysis demonstrated that oral treprostinil–assigned participants had a substantially higher mortality risk at baseline but achieved a lower risk profile from Study Weeks 12–60. The most common adverse events in the oral treprostinil group were headache, diarrhea, flushing, nausea, and vomiting. Conclusions: In participants with PAH, addition of oral treprostinil to approved oral monotherapy reduced the risk of clinical worsening.Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

CHU Lille
Inserm
Université de Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Submission date :

2021-07-06T12:46:49Z
2024-02-02T13:53:42Z