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Combination Therapy with Oral Treprostinil for Pulmonary Arterial Hypertension:A Double-Blind Placebo-controlled Clinical Trial

Authors :
R. James White
Carlos Jerjes-Sanchez
Gisela Martina Bohns Meyer
Tomas Pulido
Pablo Sepulveda
Kuo Yang Wang
Ekkehard Grünig
Shirish Hiremath
Zaixin Yu
Zhang Gangcheng
Wei Luen James Yip
Shuyang Zhang
Akram Khan
C. Q. Deng
Rob Grover
Victor F. Tapson
Graciela Noemi Svetliza
Adrian Jose Lescano
Guillermo Roberto Bortman
Fabian Antonio Diez
Christian Edgardo Botta
John Fitzgerald
Eelke Feenstra
Fiona Dawn Kermeen
Anne Margaret Keogh
Trevor John Williams
Peter Paul Yousseff
Benjamin Joh-Han Ng
David McNaughton Smallwood
Nathan Brent Dwyer
Martin Russell Brown
Irene M Lang
Regina Steringer-Mascherbauer
Jaquelina Sonoe Ota Arakaki
Frederico Thadeu Assis Figueiredo Campos
Ricardo de Amorim Correa
Rogerio de Souza
Gisela M. Bohns Meyer
Maria Auxiliadora Carmo Moreira
Hugo Hyung Bok Yoo
Monica Silveira Lapa
John Swiston
Naushad Hirani
Sanjay Mehta
Evangelos Michelakis
Pablo Andres Sepulveda
Monica Maria Zagolin Blancaire
Jimming Liu
Zhang Shuyang
Lei Pan
Bao Chunde
Yi Qun
Cheng Xiaoshu
Yu Zaixin
Xinli Li
Yao Hua
Xianyang Zhu
Yundai Chen
Cheng Zhaozhong
Yuanhua Yang
Zhou Daxin
Shen Jieyan
Jens Erik Nielsen-Kudsk
Jorn Carlsen
Arnaud Bourdin
Eric Hachulla
Claire Dromer
Ari Chaouat
Martine Reynaud-Gauber
Marie-France Seronde
Hans Klose
Michael Halank
Gert Hoffken
Ralf Ewert
Stephan Rosenkranz
Ekkehard Grunig
Ulrich Kruger
Juliane Kronsbein
Barbara Monika Hauptmeier
Andrea Koch
Matthias Held
Tobias Johannes Lange
Claus Neurohr
Heinrike Wilkens
Hubert Rolf Wilhelm Wirtz
Stavros Konstantinides
Paraskevi Argyropoulou-Pataka
Stylianos Orfanos
Prafulla Gopinath Kerkar
Pujar Venkateshacharya Suresh
Hemang Ashwinkumar Baxi
Abraham Oomman
Rajpal Kanaklal Abhaichand
Padma Kumar Edla Arjun
Vijay Chopra
Rahul Mehrotra
Rajeev Kumar Rajput
Jitendra Pal Singh Sawhney
Subir Bimalendu
Kamal Harishchandra Sharma
Bhagavathula Kutumba Srinivasa Sastry
Mordechai Reuben Kramer
Michael Jonathan Segel
Issahar Ben-Dov
Neville Berkman
Mordechai Yigla
Yochai Adir
Michael D’Alto
Carmine Dario Vizza
Laura Scelsi
Patrizio Vitulo
Tomas Rene Pulido
Anko Boonstra
Madelon Clementina Vonk
Bozena Sobkowicz
Tatiana Mularek-Kubzdela
Adam Torbicki
Piotr Podolec
Lim Soo Teik
Hyuk-Jae Chang
Hyung-Kwan Kim
Jun-Bean Park
Sung-A Chang
Duk-Kyung Kim
Wook-Jin Chung
Jong-Min Song
Magnus Nissell
Clara Hjalmarsson
Bengt Rundqvist
Wei-Chun Huang
Chin-Chang Cheng
Chih-Hsin Hsu
Hsao-Hsun Hsu
Kuo-Yang Wang
John Gerard Coghlan
David Gerard Kiely
Joanna Wanda Pepke-Zaba
James Lawrence Lordan
Paul Anthony Corris
Linda Cadaret
Sif Hansdottir
Ronald Jack Oudiz
David B. Badesch
Michael Mathier
Robert Schilz
Nicholas Hill
Aaron Waxman
Catherine J. Markin
Diane Lynn Zwicke
Micah Fisher
Veronica Franco
Namita Sood
Myung H. Park
Roblee Allen
Jeremy P. Feldman
Vijay Balasubramanian
Vandana Kavita Seeram
Abubakr Bajwa
Austin B. Thompson
Christina Migliore
Jean Elwing
John W. McConnell
Jinesh P. Mehta
Franck Farzad Rahaghi
J. Eduardo Rame
Bela Patel
Ron M. Oren
James R. Klinger
Hassan Alnuaimat
Samuel Allen
William Harvey
Michael S. Eggert
Antoine Hage
Chad E. Miller
Rana Lee Adawi Awdish
Hector Cajigas
Daniel Grinnan
Benjamin Howard Trichon
Clark McDonough
Franz Rischard
ACS - Pulmonary hypertension & thrombosis
Pulmonary medicine
University of Rochester Medical Center (URMC)
Unidad de Investigación Clínica En Medicina
Complexo Hospitalar da Santa Casa de Misericórdia de Porto Alegre
Instituto Nacional de Cardiologia Ignacio Chavez
Pontificia Universidad Católica de Chile (UC)
Taichung Veterans General Hospital
Heidelberg University Hospital [Heidelberg]
Ruby Hall Clinic
The Second Xiangya Hospital of Central South University [Hunan, China]
Wuhan Asia Heart Hospital
National Heart Centre Singapore & Duke-National University of Singapore
Peking Union Medical College Hospital [Beijing] (PUMCH)
Oregon Health and Science University [Portland] (OHSU)
United Therapeutics Corporation
Cedars-Sinai Medical Center
Source :
White, R J, Jerjes-Sanchez, C, Bohns Meyer, G M, Pulido, T, Sepulveda, P, Wang, K Y, Grünig, E, Hiremath, S, Yu, Z, Gangcheng, Z, Yip, W L J, Zhang, S, Khan, A, Deng, C Q, Grover, R, Tapson, V F & FREEDOM-EV 2020, ' Combination Therapy with Oral Treprostinil for Pulmonary Arterial Hypertension : A Double-Blind Placebo-controlled Clinical Trial ', American Journal of Respiratory and Critical Care Medicine, vol. 201, no. 6, pp. 707-717 . https://doi.org/10.1164/rccm.201908-1640OC, American Journal of Respiratory and Critical Care Medicine, 201(6), 707-717. American Thoracic Society, American Journal of Respiratory and Critical Care Medicine, White, R J, Jerjes-Sanchez, C, Meyer, G M B, Pulido, T, Sepulveda, P, Wang, K Y, Grünig, E, Hiremath, S, Yu, Z, Zhang, G, Yip, W L J, Zhang, S, Khan, A, Deng, C Q, Grover, R, Tapson, V F, Svetliza, G N, Lescano, A J, Bortman, G R, Diez, F A, Botta, C E, Fitzgerald, J, Feenstra, E, Kermeen, F D, Keogh, A M, Williams, T J, Yousseff, P P, Ng, B J H, Smallwood, D M N, Dwyer, N B, Brown, M R, Lang, I M, Steringer-Mascherbauer, R, Arakaki, J O, Campos, F, De Amorim Correa, R, De Souza, R, Meyer, G M B, Moreira, M C, Yoo, H, Lapa, M S, Swiston, J, Hirani, N, Mehta, S, Michelakis, E, Sepulveda, P, Zagolin, M, Liu, J, Zhang, S, Pan, L, Chunde, B, Qun, Y, Cheng, X, Yu, Z, Li, X, Hua, Y, Zhang, G, Zhu, X, Chen, Y, Cheng, Z, Yang, Y, Zhou, D, Shen, J, Nielsen-Kudsk, J E, Carlsen, J, Bourdin, A, Hachulla, E, Dromer, C, Chaouat, A, Reynaud-Gauber, M, Seronde, M F, Klose, H, Halank, M, Hoffken, G, Ewert, R, Rosenkranz, S, Grunig, E, Kruger, U, Kronsbein, J, Hauptmeier, B M, Koch, A, Held, M, Lange, T J, Neurohr, C, Wilkens, H, Wirtz, H, Konstantinides, S, Argyropoulou-Pataka, P, Orfanos, S, Hiremath, S, Kerkar, P G, Suresh, P V, Baxi, H A, Oomman, A, Abhaichand, R K, Edla Kumar, P K, Chopra, V, Mehrotra, R, Rajput, R K, Sawhney, J S, Bimalendu, S, Sharma, K H, Srinivasa Sastry, B K, Kramer, M R, Segel, M J, Ben-Dov, I, Berkman, N, Yigla, M, Adir, Y, D'Alto, M, Vizza, C D, Scelsi, L, Vitulo, P, Pulido, T R, Jerjes-Sanchez, C, Boonstra, A, Vonk, M C, Sobkowicz, B, Mularek-Kubzdela, T, Torbicki, A, Podolec, P, Teik, L S, Yip, W L J, Chang, H J, Kim, H K, Park, J B, Chang, S A, Kim, D K, Chung, W J, Song, J M, Nissell, M, Hjalmarsson, C, Rundqvist, B, Huang, W C, Cheng, C C, Hsu, C H, Hsu, H H, Wang, K Y, Coghlan, J G, Kiely, D G, Pepke-Zaba, J W, Lordan, J L, Corris, P A, Cadaret, L, Hansdottir, S, Oudiz, R J, Badesch, D B, Mathier, M, Schilz, R, Hill, N, Waxman, A, Markin, C J, Zwicke, D L, Fisher, M, Franco, V, Sood, N, Park, M H, Allen, R, Feldman, J P, Balasubramanian, V, Seeram, V K, Bajwa, A, Thompson, A B, Migliore, C, Elwing, J, McConnell, J W, Mehta, J P, Rahaghi, F F, Rame, J E, Khan, A, Patel, B, Oren, R M, Klinger, J R, Alnuaimat, H, Allen, S, Harvey, W, Eggert, M S, Hage, A, Miller, C E, Awdish, R, Cajigas, H, Grinnan, D, Trichon, B H, McDonough, C, White, R J & Rischard, F 2020, ' Combination therapy with oral treprostinil for pulmonary arterial hypertension : A double-blind placebo-controlled clinical trial ', American Journal of Respiratory and Critical Care Medicine, vol. 201, no. 6, pp. 707-717 . https://doi.org/10.1164/rccm.201908-1640OC, American Journal of Respiratory and Critical Care Medicine, American Thoracic Society, 2020, 201 (6), pp.707-717. ⟨10.1164/rccm.201908-1640OC⟩, White, R J, Jerjes-Sanchez, C, Bohns Meyer, G M, Pulido, T, Sepulveda, P, Wang, K Y, Grünig, E, Hiremath, S, Yu, Z, Gangcheng, Z, Yip, W L J, Zhang, S, Khan, A, Deng, C Q, Grover, R, Tapson, V F, FREEDOM-EV Investigators, Svetliza, G N, Lescano, A J, Bortman, G R, Carlsen, J, McDonough, C, White, J R & Rischard, F 2020, ' Combination Therapy with Oral Treprostinil for Pulmonary Arterial Hypertension : A Double-Blind Placebo-controlled Clinical Trial ', American Journal of Respiratory and Critical Care Medicine, vol. 201, no. 6, pp. 707-717 . https://doi.org/10.1164/rccm.201908-1640OC
Publication Year :
2020

Abstract

Rationale: Oral treprostinil improves exercise capacity in patients with pulmonary arterial hypertension (PAH), but the effect on clinical outcomes was unknown.\ud \ud 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.\ud \ud 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.\ud \ud 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.\ud \ud Conclusions: In participants with PAH, addition of oral treprostinil to approved oral monotherapy reduced the risk of clinical worsening.\ud \ud Clinical trial registered with www.clinicaltrials.gov (NCT01560624).

Subjects

Subjects :
Male
Administration, Oral
Oral treprostinil
Critical Care and Intensive Care Medicine
Pulmonary arterial hypertension
[SDV.MHEP.PSR]Life Sciences [q-bio]/Human health and pathology/Pulmonology and respiratory tract
combination therapy
oral
epoprostenol
0302 clinical medicine
pulmonary arterial hypertension
middle aged
Clinical endpoint
double-blind method
MESH: Double-Blind Method
Familial Primary Pulmonary Hypertension
030212 general & internal medicine
humans
MESH: Aged
MESH: Middle Aged
Epoprostenol/analogs & derivatives
adult
Hazard ratio
Middle Aged
[SDV.SP]Life Sciences [q-bio]/Pharmaceutical sciences
antihypertensive agents
3. Good health
aged
female
MESH: Young Adult
oral treprostinil
MESH: Administration, Oral
young adult
Female
Pulmonary Arterial Hypertension/drug therapy
medicine.drug
Adult
Pulmonary and Respiratory Medicine
MESH: Pulmonary Arterial Hypertension
medicine.medical_specialty
Randomization
Adolescent
clinical study
sequential therapy
administration, oral
adolescent
male
placebos
Sequential therapy
MESH: Placebos
MESH: Epoprostenol
Lower risk
Placebo
administration
Clinical study
Young Adult
03 medical and health sciences
Double-Blind Method
[SDV.MHEP.CSC]Life Sciences [q-bio]/Human health and pathology/Cardiology and cardiovascular system
Internal medicine
medicine
Humans
Combination therapy
Adverse effect
Placebos/therapeutic use
Aged
MESH: Adolescent
Pulmonary Vascular Disease
MESH: Antihypertensive Agents
MESH: Humans
business.industry
MESH: Adult
Original Articles
MESH: Male
Clinical trial
030228 respiratory system
Antihypertensive Agents/administration & dosage
business
MESH: Female
Treprostinil

Details

Language :
English
ISSN :
1073449X and 15354970
Database :
OpenAIRE
Journal :
White, R J, Jerjes-Sanchez, C, Bohns Meyer, G M, Pulido, T, Sepulveda, P, Wang, K Y, Grünig, E, Hiremath, S, Yu, Z, Gangcheng, Z, Yip, W L J, Zhang, S, Khan, A, Deng, C Q, Grover, R, Tapson, V F & FREEDOM-EV 2020, ' Combination Therapy with Oral Treprostinil for Pulmonary Arterial Hypertension : A Double-Blind Placebo-controlled Clinical Trial ', American Journal of Respiratory and Critical Care Medicine, vol. 201, no. 6, pp. 707-717 . https://doi.org/10.1164/rccm.201908-1640OC, American Journal of Respiratory and Critical Care Medicine, 201(6), 707-717. American Thoracic Society, American Journal of Respiratory and Critical Care Medicine, White, R J, Jerjes-Sanchez, C, Meyer, G M B, Pulido, T, Sepulveda, P, Wang, K Y, Grünig, E, Hiremath, S, Yu, Z, Zhang, G, Yip, W L J, Zhang, S, Khan, A, Deng, C Q, Grover, R, Tapson, V F, Svetliza, G N, Lescano, A J, Bortman, G R, Diez, F A, Botta, C E, Fitzgerald, J, Feenstra, E, Kermeen, F D, Keogh, A M, Williams, T J, Yousseff, P P, Ng, B J H, Smallwood, D M N, Dwyer, N B, Brown, M R, Lang, I M, Steringer-Mascherbauer, R, Arakaki, J O, Campos, F, De Amorim Correa, R, De Souza, R, Meyer, G M B, Moreira, M C, Yoo, H, Lapa, M S, Swiston, J, Hirani, N, Mehta, S, Michelakis, E, Sepulveda, P, Zagolin, M, Liu, J, Zhang, S, Pan, L, Chunde, B, Qun, Y, Cheng, X, Yu, Z, Li, X, Hua, Y, Zhang, G, Zhu, X, Chen, Y, Cheng, Z, Yang, Y, Zhou, D, Shen, J, Nielsen-Kudsk, J E, Carlsen, J, Bourdin, A, Hachulla, E, Dromer, C, Chaouat, A, Reynaud-Gauber, M, Seronde, M F, Klose, H, Halank, M, Hoffken, G, Ewert, R, Rosenkranz, S, Grunig, E, Kruger, U, Kronsbein, J, Hauptmeier, B M, Koch, A, Held, M, Lange, T J, Neurohr, C, Wilkens, H, Wirtz, H, Konstantinides, S, Argyropoulou-Pataka, P, Orfanos, S, Hiremath, S, Kerkar, P G, Suresh, P V, Baxi, H A, Oomman, A, Abhaichand, R K, Edla Kumar, P K, Chopra, V, Mehrotra, R, Rajput, R K, Sawhney, J S, Bimalendu, S, Sharma, K H, Srinivasa Sastry, B K, Kramer, M R, Segel, M J, Ben-Dov, I, Berkman, N, Yigla, M, Adir, Y, D'Alto, M, Vizza, C D, Scelsi, L, Vitulo, P, Pulido, T R, Jerjes-Sanchez, C, Boonstra, A, Vonk, M C, Sobkowicz, B, Mularek-Kubzdela, T, Torbicki, A, Podolec, P, Teik, L S, Yip, W L J, Chang, H J, Kim, H K, Park, J B, Chang, S A, Kim, D K, Chung, W J, Song, J M, Nissell, M, Hjalmarsson, C, Rundqvist, B, Huang, W C, Cheng, C C, Hsu, C H, Hsu, H H, Wang, K Y, Coghlan, J G, Kiely, D G, Pepke-Zaba, J W, Lordan, J L, Corris, P A, Cadaret, L, Hansdottir, S, Oudiz, R J, Badesch, D B, Mathier, M, Schilz, R, Hill, N, Waxman, A, Markin, C J, Zwicke, D L, Fisher, M, Franco, V, Sood, N, Park, M H, Allen, R, Feldman, J P, Balasubramanian, V, Seeram, V K, Bajwa, A, Thompson, A B, Migliore, C, Elwing, J, McConnell, J W, Mehta, J P, Rahaghi, F F, Rame, J E, Khan, A, Patel, B, Oren, R M, Klinger, J R, Alnuaimat, H, Allen, S, Harvey, W, Eggert, M S, Hage, A, Miller, C E, Awdish, R, Cajigas, H, Grinnan, D, Trichon, B H, McDonough, C, White, R J & Rischard, F 2020, ' Combination therapy with oral treprostinil for pulmonary arterial hypertension : A double-blind placebo-controlled clinical trial ', American Journal of Respiratory and Critical Care Medicine, vol. 201, no. 6, pp. 707-717 . https://doi.org/10.1164/rccm.201908-1640OC, American Journal of Respiratory and Critical Care Medicine, American Thoracic Society, 2020, 201 (6), pp.707-717. ⟨10.1164/rccm.201908-1640OC⟩, White, R J, Jerjes-Sanchez, C, Bohns Meyer, G M, Pulido, T, Sepulveda, P, Wang, K Y, Grünig, E, Hiremath, S, Yu, Z, Gangcheng, Z, Yip, W L J, Zhang, S, Khan, A, Deng, C Q, Grover, R, Tapson, V F, FREEDOM-EV Investigators, Svetliza, G N, Lescano, A J, Bortman, G R, Carlsen, J, McDonough, C, White, J R & Rischard, F 2020, ' Combination Therapy with Oral Treprostinil for Pulmonary Arterial Hypertension : A Double-Blind Placebo-controlled Clinical Trial ', American Journal of Respiratory and Critical Care Medicine, vol. 201, no. 6, pp. 707-717 . https://doi.org/10.1164/rccm.201908-1640OC
Accession number :
edsair.doi.dedup.....247f9389052b9bd4d58d6383633fc9da