Update on pulmonary arterial hypertension research: proceedings from a meeting of experts

Current Medical Research and Opinion

Volumn 34, Issue 2, 2018, Pages 263-273

  McLaughlin, Vallerie ^a   Bacchetta, Matthew ^b   Badesch, David ^c   Benza, Raymond ^d   Burger, Charles ^e   Chin, Kelly ^f   Frantz, Robert ^g   Frost, Adaani ^h   Hemnes, Anna ⁱ   Kim, Nick H ^j   Rosenzweig, Erika B ^k   Rubin, Lewis ^l  

^a UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^b NewYork Presbyterian Columbia University Irving Medical Center   (United States)

^c UNIVERSITY OF COLORADO SCHOOL OF MEDICINE   (United States)

^d ALLEGHENY GENERAL HOSPITAL   (United States)

^e MAYO CLINIC   (United States)

^f UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^g MAYO CLINIC   (United States)

^h HOUSTON METHODIST HOSPITAL   (United States)

ⁱ VANDERBILT UNIVERSITY MEDICAL CENTER   (United States)

^j UNIVERSITY OF CALIFORNIA   (United States)

^k Department of Neurology and the Taub Institute for Research on Alzheimer's Disease and the Aging Brain   (United States)

^l UNIVERSITY OF CALIFORNIA   (United States)

more..

Author keywords

Clinical; health economics; preclinical; proceedings; pulmonary arterial hypertension; registry; technology

Indexed keywords

AMBULATORY CARE; CLINICAL RESEARCH; CONGENITAL HEART DISEASE; EXTRACORPOREAL OXYGENATION; HEALTH ECONOMICS; HUMAN; PEDIATRICS; PULMONARY HYPERTENSION; REGISTER; REVIEW; ECONOMICS; MEDICAL RESEARCH; MEDICAL TECHNOLOGY; PATHOPHYSIOLOGY; PROCEDURES; STATISTICS AND NUMERICAL DATA; TRENDS;

BIOMEDICAL RESEARCH; BIOMEDICAL TECHNOLOGY; HUMANS; HYPERTENSION, PULMONARY; REGISTRIES;

EID: 85041052135     PISSN: 03007995     EISSN: 14734877     Source Type: Journal    
DOI: 10.1080/03007995.2017.1404974     Document Type: Review

References (86)

1. McGoon MD, Kane GC., Pulmonary hypertension: diagnosis and management. Mayo Clin Proc 2009;84:191-207
2. McLaughlin VV, Badesch DB, Delcroix M, et al. End points and clinical trial design in pulmonary arterial hypertension. J Am Coll Cardiol 2009;54:S97-S107
3. Divers C, Platt D, Wang E, et al. A review of clinical trial endpoints of patients with pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension and how they relate to patient outcomes in the United States. J Manag Care Spec Pharm 2017;23:92-104
4. Atkinson C, Stewart S, Upton PD, et al. Primary pulmonary hypertension is associated with reduced pulmonary vascular expression of type II bone morphogenetic protein receptor. Circulation 2002;105:1672-8
5. Loyd JE, Butler MG, Foroud TM, et al. Genetic anticipation and abnormal gender ratio at birth in familial primary pulmonary hypertension. Am J Respir Crit Care Med 1995;152:93-7
6. Rothman AM, Arnold ND, Pickworth JA, et al. MicroRNA-140-5p and SMURF1 regulate pulmonary arterial hypertension. J Clin Invest 2016;126:2495-508
7. Chen X, Talati M, Fessel JP, et al. Estrogen metabolite 16alpha-hydroxyestrone exacerbates bone morphogenetic protein receptor type II-associated pulmonary arterial hypertension through microRNA-29-mediated modulation of cellular metabolism. Circulation 2016;133:82-97
8. Antigny F, Hautefort A, Meloche J, et al. Potassium channel subfamily K member 3 (KCNK3) contributes to the development of pulmonary arterial hypertension. Circulation 2016;133:1371-85
9. Guignabert C, Phan C, Seferian A, et al. Dasatinib induces lung vascular toxicity and predisposes to pulmonary hypertension. J Clin Invest 2016;126:3207-18
10. Malenfant S, Potus F, Mainguy V, et al. Impaired skeletal muscle oxygenation and exercise tolerance in pulmonary hypertension. Med Sci Sports Exerc 2015;47:2273-82
11. Malenfant S, Potus F, Fournier F, et al. Skeletal muscle proteomic signature and metabolic impairment in pulmonary hypertension. J Mol Med (Berl) 2015;93:573-84
12. Brittain EL, Talati M, Fessel JP, et al. Fatty acid metabolic defects and right ventricular lipotoxicity in human pulmonary arterial hypertension. Circulation 2016;133:1936-44
13. Talati MH, Brittain EL, Fessel JP, et al. Mechanisms of lipid accumulation in the bone morphogenetic protein receptor type 2 mutant right ventricle. Am J Respir Crit Care Med 2016;194:719-28
14. Sa S, Gu M, Chappell J, et al. iPSC model of pulmonary arterial hypertension reveals novel gene expression and patient specificity. Am J Respir Crit Care Med 2017;195:930-41
15. Bonnet S, Provencher S, Guignabert C, et al. Translating research into improved patient care in pulmonary arterial hypertension. Am J Respir Crit Care Med 2017;195:583-95
16. Benza RL, Gomberg-Maitland M, Demarco T, et al. Endothelin-1 pathway polymorphisms and outcomes in pulmonary arterial hypertension. Am J Respir Crit Care Med 2015;192:1345-54
17. Hemnes AR, Zhao M, West J, et al. Critical genomic networks and vasoreactive variants in idiopathic pulmonary arterial hypertension. Am J Respir Crit Care Med 2016;194:464-75
18. Hansmann G, Apitz C, Abdul-Khaliq H, et al. Executive summary. Expert consensus statement on the diagnosis and treatment of paediatric pulmonary hypertension. The European Paediatric Pulmonary Vascular Disease Network, endorsed by ISHLT and DGPK. Heart 2016;102(Suppl2):ii86-100
19. Douwes JM, Humpl T, Bonnet D, et al. Acute vasodilator response in pediatric pulmonary arterial hypertension: current clinical practice from the TOPP registry. J Am Coll Cardiol 2016;67:1312-23
20. van Riel AC, Blok IM, Zwinderman AH, et al. Lifetime risk of pulmonary hypertension for all patients after shunt closure. J Am Coll Cardiol 2015;66:1084-6
21. Diller GP, Korten MA, Bauer UM, et al. Current therapy and outcome of Eisenmenger syndrome: data of the German National Register for congenital heart defects. Eur Heart J 2016;37:1449-55
22. Mehta S, Sastry BK, Souza R, et al. Macitentan improves health-related quality of life for patients with pulmonary arterial hypertension: results from the randomized controlled SERAPHIN trial. Chest 2017;151:106-18
23. Hoeper MM, McLaughlin VV, Barbera JA, et al. Initial combination therapy with ambrisentan and tadalafil and mortality in patients with pulmonary arterial hypertension: a secondary analysis of the results from the randomised, controlled AMBITION study. Lancet Respir Med 2016;4:894-901
24. Bourge RC, Waxman AB, Gomberg-Maitland M, et al. Treprostinil administered to treat pulmonary arterial hypertension using a fully implantable programmable intravascular delivery system: results of the DelIVery for PAH trial. Chest 2016;150:27-34
25. ClinicalTrials.gov. A study to test the effects of riociguat in patients with pulmonary hypertension associated with left ventricular systolic dysfunction (LEPHT). National Institutes of Health, Bethesda, MD. October 2016. Available online at: https://clinicaltrials.gov/ct2/show/NCT01065454. [Last accessed 2 January 2017]
26. Hoendermis ES, Liu LC, Hummel YM, et al. Effects of sildenafil on invasive haemodynamics and exercise capacity in heart failure patients with preserved ejection fraction and pulmonary hypertension: a randomized controlled trial. Eur Heart J 2015;36:2565-73
27. ClinicalTrials.gov. ARTEMIS-PH - Study of ambrisentan in subjects with pulmonary hypertension associated with idiopathic pulmonary fibrosis (ARTEMIS-PH). National Institutes of Health, Bethesda, MD. May 2014. Available online at: https://clinicaltrials.gov/ct2/show/results/NCT00879229?term=NCT008792-29&rank = 1. [Last accessed 3 January 2017]
28. ClinicalTrials.gov. (ARTEMIS-IPF) Randomized, placebo-controlled study to evaluate safety and effectiveness of ambrisentan in IPF (ARTEMIS-IPF). National Institutes of Health, Bethesda, MD. February 2014. Available online at: https://clinicaltrials.gov/ct2/show/results/NCT00768300?term=NCT00768300&rank = 1. [Last accessed 3 January 2017]
29. ClinicalTrials.gov. Efficacy and safety of riociguat in patients with symptomatic pulmonary hypertension (PH) associated with idiopathic interstitial pneumonias (IIP) (RISE-IIP). National Institutes of Health, Bethesda, MD. September 2016. Available online at: https://clinicaltrials.gov/ct2/show/NCT021388-25?term=NCT02138825&rank = 1. [Last accessed 3 January 2017]
30. ClinicalTrials.gov. Sildenafil trial of exercise performance in idiopathic pulmonary fibrosis (STEP-IPF). National Institutes of Health, Bethesda, MD. June 2015. Available online at: https://clinicaltrials.gov/ct2/show/results/NCT00517933?term=NCT00517933&rank = 1§=X430156#othr. [Last accessed 3 January 2017]
31. ClinicalTrials.gov. Sildenafil therapy for pulmonary hypertension and sickle cell disease. National Institutes of Health, Bethesda, MD. December 2015. Available online at: https://clinicaltrials.gov/ct2/show/results/NCT00492531?term=NCT-00492531&rank = 1§=X30156#evnt. [Last accessed 3 January 2017]
32. ClinicalTrials.gov. Clinical study to evaluate the safety and tolerability of macitentan in subjects with combined pre- and post-capillary pulmonary hypertension due to left ventricular dysfunction (MELODY-1). National Institutes of Health, Bethesda, MD. December 2015. Available online at: https://clinicaltrials.gov/ct2/show/NCT02070991?term=MELODY-1+Macitentan&rank = 1. [Last accessed 2 January 2017]
33. ClinicalTrials.gov. Clinical study to assess the efficacy, safety and tolerability of macitentan in subjects with inoperable chronic thromboembolic pulmonary hypertension (MERIT-1). National Institutes of Health, Bethesda, MD. October 2016. Available online at: https://clinicaltrials.gov/ct2/show/NCT0202129-2?term=NCT02021292&rank = 1. [Last accessed 2 January 2017]
34. Actelion announces positive results of the MERIT study with macitentan in patients with chronic thromboembolic pulmonary hypertension. Actelion Pharmaceuticals Ltd, Allschwil, Switzerland. November 7, 2016. Available online at: https://www1.actelion.com/en/journalists/news-archive.page?newsId=2054623. [Last accessed 3 January 2017]
35. ClinicalTrials.gov. Riociguat versus balloon pulmonary angioplasty in non-operable chronic thromboembolic pulmonary hypertension (RACE). National Institutes of Health, Bethesda, MD. January 2016. Available online at: https://clinicaltrials.gov/ct2/show/NCT02634203?term=NCT02634203&rank = 1. [Last accessed 2 January 2017]
36. Centers for Medicare & Medicaid Services. Research, statistics, data and systems - National health expenditure date: Projected. US Centers for Medicare & Medicaid Services, Baltimore, MD. Available online at: https://www.cms.gov/Research-Statistics-Data-and-Systems/Statistics-Trends-and-Reports/NationalHealthExpend-Data/NationalHealthAccountsProjected.html. [Last accessed 18 July 2016]
37. Stettin G, Henderson R., Express scripts 2015 drug trend report. Express Scripts Holding Company, St. Louis, MO. March 2016. Available online at: https://lab.express-scripts.com/lab/drug-trend-report. [Last accessed December 2016]
38. Burger CD, Long PK, Shah MR, et al. Characterization of first-time hospitalizations in patients with newly diagnosed pulmonary arterial hypertension in the REVEAL registry. Chest 2014;146:1263-73
39. Burke JP, Hunsche E, Regulier E, et al. Characterizing pulmonary hypertension-related hospitalization costs among Medicare Advantage or commercially insured patients with pulmonary arterial hypertension: a retrospective database study. Am J Manag Care 2015;21:S47-S58
40. OPSUMIT® (macitentan) tablets, for oral use. South San Francisco, CA: Actelion Pharmaceuticals US Inc; 2013
41. Frantz RP, Schilz RJ, Chakinala MM, et al. Hospitalization and survival in patients using epoprostenol for injection in the PROSPECT observational study. Chest 2015;147:484-94
42. Galie N, Barbera JA, Frost AE, et al. Initial use of ambrisentan plus tadalafil in pulmonary arterial hypertension. N Engl J Med 2015;373:834-44
43. Ghofrani HA, Galie N, Grimminger F, et al. Riociguat for the treatment of pulmonary arterial hypertension. N Engl J Med 2013;369:330-40
44. Ghofrani HA, D’Armini AM, Grimminger F, et al. Riociguat for the treatment of chronic thromboembolic pulmonary hypertension. N Engl J Med 2013;369:319-29
45. Rubin LJ, Galie N, Grimminger F, et al. Riociguat for the treatment of pulmonary arterial hypertension: a long-term extension study (PATENT-2). Eur Respir J 2015;45:1303-13
46. Simonneau G, D’Armini AM, Ghofrani HA, et al. Predictors of long-term outcomes in patients treated with riociguat for chronic thromboembolic pulmonary hypertension: data from the CHEST-2 open-label, randomised, long-term extension trial. Lancet Respir Med 2016;4:372-80
47. UPTRAVI® (selexipag) tablets, for oral use. South San Francisco, CA: Actelion Pharmaceuticals US Inc.; 2015
48. Medicare Payment Advisory Commission. Report to the Congress - Medicare and Health Care Delivery System. Washington, DC. June 2013. Available online at: http://www.medpac.gov/docs/default-source/reports/jun13_entirereport.pdf?sfvrsn = 0. [Last accessed December 2016]
49. The Commonwealth Fund. Aiming Higher - Results from a state scorecard on health system performance, 2009. New York, NY. October 2009. Available online at: http://www.commonwealthfund.org/∼/media/files/publications/fund-report/2009/oct/mccarthy_state_scorecard_2009_executive_summary_only.pdf. [Last accessed December 2016]
50. Mazimba S, Mejia-Lopez E, Black G, et al. Diastolic pulmonary gradient predicts outcomes in group 1 pulmonary hypertension (analysis of the NIH primary pulmonary hypertension registry). Respir Med 2016;119:81-6
51. Frost AE, Badesch DB, Barst RJ, et al. The changing picture of patients with pulmonary arterial hypertension in the United States: how REVEAL differs from historic and non-US Contemporary Registries. Chest 2011;139:128-37
52. Montani D, Bergot E, Gunther S, et al. Pulmonary arterial hypertension in patients treated by dasatinib. Circulation 2012;125:2128-37
53. Benza RL, Miller DP, Frost A, et al. Analysis of the lung allocation score estimation of risk of death in patients with pulmonary arterial hypertension using data from the REVEAL Registry. Transplantation 2010;90:298-305
54. Naumburg E, Soderstrom L, Huber D, et al. Risk factors for pulmonary arterial hypertension in children and young adults. Pediatr Pulmonol 2017;52:636-41
55. Richter MJ, Tiede SL, Sommer N, et al. Circulating angiopoietin-1 is not a biomarker of disease severity or prognosis in pulmonary hypertension. PLoS One 2016;11:e0165982
56. Girerd B, Montani D, Coulet F, et al. Clinical outcomes of pulmonary arterial hypertension in patients carrying an ACVRL1 (ALK1) mutation. Am J Respir Crit Care Med 2010;181:851-61
57. McLaughlin VV, Langer A, Tan M, et al. Contemporary trends in the diagnosis and management of pulmonary arterial hypertension: an initiative to close the care gap. Chest 2013;143:324-32
58. Chung L, Farber HW, Benza R, et al. Unique predictors of mortality in patients with pulmonary arterial hypertension associated with systemic sclerosis in the REVEAL registry. Chest 2014;146:1494-504
59. Gall H, Felix JF, Schneck FK, et al. The Giessen Pulmonary Hypertension Registry: survival in pulmonary hypertension subgroups. J Heart Lung Transplant 2017;36:957-67
60. Preston IR, Roberts KE, Miller DP, et al. Effect of warfarin treatment on survival of patients with pulmonary arterial hypertension (PAH) in the registry to evaluate early and long-term PAH disease management (REVEAL). Circulation 2015;132:2403-11
61. Olsson KM, Delcroix M, Ghofrani HA, et al. Anticoagulation and survival in pulmonary arterial hypertension: results from the Comparative, Prospective Registry of Newly Initiated Therapies for Pulmonary Hypertension (COMPERA). Circulation 2014;129:57-65
62. Opitz CF, Hoeper MM, Gibbs JS, et al. Pre-capillary, combined, and post-capillary pulmonary hypertension: a pathophysiological continuum. J Am Coll Cardiol 2016;68:368-78
63. Zafrir B, Salman N, Crespo-Leiro MG, et al. Body surface area as a prognostic marker in chronic heart failure patients: results from the Heart Failure Registry of the Heart Failure Association of the European Society of Cardiology. Eur J Heart Fail 2016;18:859-68
64. Farber HW, Miller DP, McGoon MD, et al. Predicting outcomes in pulmonary arterial hypertension based on the 6-minute walk distance. J Heart Lung Transplant 2015;34:362-8
65. Frost AE, Badesch DB, Miller DP, et al. Evaluation of the predictive value of a clinical worsening definition using 2-year outcomes in patients with pulmonary arterial hypertension: a REVEAL Registry analysis. Chest 2013;144:1521-9
66. Benza RL, Miller DP, Barst RJ, et al. An evaluation of long-term survival from time of diagnosis in pulmonary arterial hypertension from the REVEAL Registry. Chest 2012;142:448-56
67. Benza RL, Miller DP, Foreman AJ, et al. Prognostic implications of serial risk score assessments in patients with pulmonary arterial hypertension: a Registry to Evaluate Early and Long-Term Pulmonary Arterial Hypertension Disease Management (REVEAL) analysis. J Heart Lung Transplant 2015;34:356-61
68. Sadoughi A, Roberts KE, Preston IR, et al. Use of selective serotonin reuptake inhibitors and outcomes in pulmonary arterial hypertension. Chest 2013;144:531-41
69. Javidfar J, Brodie D, Sonett J, et al. Venovenous extracorporeal membrane oxygenation using a single cannula in patients with pulmonary hypertension and atrial septal defects. J Thorac Cardiovasc Surg 2012;143:982-4
70. Javidfar J, Brodie D, Takayama H, et al. Safe transport of critically ill adult patients on extracorporeal membrane oxygenation support to a regional extracorporeal membrane oxygenation center. ASAIO J 2011;57:421-5
71. Biscotti M, Vail E, Cook KE, et al. Extracorporeal membrane oxygenation with subclavian artery cannulation in awake patients with pulmonary hypertension. ASAIO J 2014;60:748-50
72. Chicotka S, Rosenzweig EB, Brodie D, et al. The “Central sport model”: extracorporeal membrane oxygenation using the innominate artery for smaller patients as bridge to lung transplantation. ASAIO J 2017;63:e39-e44
73. Humbert M., Update on the European respiratory review. Eur Respir J 2010;36:993-4
74. Thenappan T, Shah SJ, Rich S, et al. A USA-based registry for pulmonary arterial hypertension: 1982–2006. Eur Respir J 2007;30:1103-10
75. Iyngkaran P, Toukhsati SR, Biddagardi N, et al. Technology-assisted congestive heart failure care. Curr Heart Fail Rep 2015;12:173-86
76. Bui AL, Fonarow GC., Home monitoring for heart failure management. J Am Coll Cardiol 2012;59:97-104
77. Jehn M, Schmidt-Trucksaess A, Schuster T, et al. Accelerometer-based quantification of 6-minute walk test performance in patients with chronic heart failure: applicability in telemedicine. J Card Fail 2009;15:334-40
78. Jehn M, Prescher S, Koehler K, et al. Tele-accelerometry as a novel technique for assessing functional status in patients with heart failure: feasibility, reliability and patient safety. Int J Cardiol 2013;168:4723-8
79. Mainguy V, Provencher S, Maltais F, et al. Assessment of daily life physical activities in pulmonary arterial hypertension. PLoS One 2011;6:e27993
80. Pugh ME, Buchowski MS, Robbins IM, et al. Physical activity limitation as measured by accelerometry in pulmonary arterial hypertension. Chest 2012;142:1391-8
81. ClinicalTrials.gov. Electronic activity level monitoring pilot in pulmonary hypertension (e-MOTION PH). National Institutes of Health, Bethesda, MD. October 2016. Available online at: https://clinicaltrials.gov/ct2/show/NCT02536534?term=EMotion+Bayer&rank = 1. [Last accessed December 2016]
82. Chen H, Taichman DB, Doyle RL., Health-related quality of life and patient-reported outcomes in pulmonary arterial hypertension. Proc Am Thorac Soc 2008;5:623-30
83. Galie N, Hoeper MM, Humbert M, et al. Guidelines for the diagnosis and treatment of pulmonary hypertension: the Task Force for the Diagnosis and Treatment of Pulmonary Hypertension of the European Society of Cardiology (ESC) and the European Respiratory Society (ERS), endorsed by the International Society of Heart and Lung Transplantation (ISHLT). Eur Heart J 2009;30:2493-537
84. US Department of Health and Human Services (FDA). Guidance for industry: Patient-reported outcome measures - use in medical product development to support labeling claims. Washington, DC. December 2009. Available online at: http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf. [Last accessed December 2016]
85. McCollister D, Shaffer S, Badesch DB, et al. Development of the pulmonary arterial hypertension-symptoms and impact (PAH-SYMPACT(R)) questionnaire: a new patient-reported outcome instrument for PAH. Respir Res 2016;17:72
86. ClinicalTrials.gov. Clinical study of macitentan in patients with pulmonary arterial hypertension to psychometrically validate the PAH-SYMPACT instrument. National Institutes of Health, Bethesda, MD. June 2016. Available online at: https://clinicaltrials.gov/ct2/show/NCT01841762?term=PAH%2C+SYMPHO-NY&rank = 2. [Last accessed December 2016]