The United States Chronic Thromboembolic Pulmonary Hypertension Registry: Protocol for a Prospective, Longitudinal Study

Kim M Kerr, C Greg Elliott, Raymond L Benza, Richard N Channick, Kelly M Chin, R Duane Davis, Sonia Jain, Andrea Z LaCroix, Michael M Madani, Vallerie V McLaughlin, Myung H Park, Victor F Tapson, William R Auger, Kim M Kerr, C Greg Elliott, Raymond L Benza, Richard N Channick, Kelly M Chin, R Duane Davis, Sonia Jain, Andrea Z LaCroix, Michael M Madani, Vallerie V McLaughlin, Myung H Park, Victor F Tapson, William R Auger

Abstract

Background: Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare sequela of acute pulmonary embolism that is treatable when recognized. Awareness of this disease has increased with recent advancements in therapeutic options, but delays in diagnosis remain common, and diagnostic and treatment guidelines are often not followed. Data gathered from international registries have improved our understanding of CTEPH, but these data may not be applicable to the US population owing to differences in demographics and medical practice patterns.

Objective: The US CTEPH Registry (US-CTEPH-R) was developed to provide essential information to better understand the demographics, risk factors, evaluation, and treatment of CTEPH in the United States, as well as the short- and long-term outcomes of surgical and nonsurgical therapies in the modern treatment era.

Methods: Thirty sites throughout the United States enrolled 750 subjects in this prospective, longitudinal, observational registry of patients newly diagnosed with CTEPH. Enrollment criteria included a mean pulmonary artery pressure ≥25 mmHg by right heart catheterization and radiologic confirmation of CTEPH by a multidisciplinary adjudication committee. Following enrollment, subjects were followed biannually until the conclusion of the study. Quality of life surveys were administered at enrollment and biannually, and all other testing was at the discretion of the treating clinician. Details regarding surgical therapy, balloon pulmonary angioplasty, and medical therapy were collected at enrollment and at follow-up, as well as information related to health care utilization and survival.

Results: Data from this registry will improve understanding of the demographics, risk factors, and treatment patterns of patients with CTEPH, and the longitudinal impact of therapies on quality of life, health care utilization, and survival.

Conclusions: This manuscript details the methodology and design of the first large, prospective, longitudinal registry of patients with CTEPH in the United States.

Trial registration: ClinicalTrials.gov NCT02429284; https://www.clinicaltrials.gov/ct2/show/NCT02429284.

International registered report identifier (irrid): DERR1-10.2196/25397.

Keywords: CTEPH; nonsurgical; pulmonary embolism; pulmonary hypertension; registry; surgical; therapy; treatment.

Conflict of interest statement

Conflicts of Interest: KK received a research grant paid to the institution from Bayer HealthCare and is a consultant to Actelion. CGE sits on the steering committee for UCSD (via grant from Bayer). KC is a consultant and sits on the steering/adjudication committees of Actelion, UCSD (via a grant from Bayer), United Therapeutics, Altavant, and Gossamer Bio. KC receives research support (paid to the institution) from Actelion, Ironwood, National Institutes of Health, and SoniVie, and receives financial/material support from the American Heart Association. RB is on the advisory board of Bayer. RC receives research support (paid to institution) from Bayer and Actelion; is a consultant for Actelion, Bayer, United Therapeutics, and Gossamer; and is part of the Speakers Bureau for Actelion and Bayer. MM is a consultant for Bayer, Actelion, and Wexler Surgical. VM receives research support (paid to institution) from Acceleron, Actelion, Gilead, Sonovie, Reata, and United Therapeutics, and performs scientific consulting for Acceleron, Actelion, Altavant, Caremark, LLC, CiVi Biopharma Inc, Gossamer Bio, Liquidia, and United Therapeutics. MP is a consultant for Bayer, Actelion, Abbott, and AstraZeneca; is part of the Speakers Bureau for Bayer; and received travel support from Bayer and Actelion. VFT receives research support (paid to institution) by National Institutes of Health, Actelion, Bayer, BMS/Pfizer, United Therapeutics, EKOS/BTG, Inari, Penumbra, and Johnson & Johnson; is on the advisory board/offers consulting to Actelion, BMS/Pfizer, United Therapeutics, EKOS/BTG, National Institutes of Health, Bayer, Johnson & Johnson, and Thrombolex; and has received speaking honoraria from Janssen and EKOS/BTG. WA is a consultant for Bayer, Cereno Scientific, and Actelion. The other authors have no conflicts of interest to declare.

©Kim M Kerr, C Greg Elliott, Raymond L Benza, Richard N Channick, Kelly M Chin, R Duane Davis, Sonia Jain, Andrea Z LaCroix, Michael M Madani, Vallerie V McLaughlin, Myung H Park, Victor F Tapson, William R Auger. Originally published in JMIR Research Protocols (https://www.researchprotocols.org), 25.05.2021.

References

    1. Kim NH, Delcroix M, Jenkins DP, Channick R, Dartevelle P, Jansa P, Lang I, Madani MM, Ogino H, Pengo V, Mayer E. Chronic thromboembolic pulmonary hypertension. J Am Coll Cardiol. 2013 Dec 24;62(25 Suppl):D92–D99. doi: 10.1016/j.jacc.2013.10.024.
    1. Rich S, Dantzker DR, Ayres SM, Bergofsky EH, Brundage BH, Detre KM, Fishman AP, Goldring RM, Groves BM, Koerner SK. Primary pulmonary hypertension. A national prospective study. Ann Intern Med. 1987 Aug;107(2):216–223. doi: 10.7326/0003-4819-107-2-216.
    1. Badesch DB, Raskob GE, Elliott CG, Krichman AM, Farber HW, Frost AE, Barst RJ, Benza RL, Liou TG, Turner M, Giles S, Feldkircher K, Miller DP, McGoon MD. Pulmonary arterial hypertension: baseline characteristics from the REVEAL Registry. Chest. 2010 Feb;137(2):376–387. doi: 10.1378/chest.09-1140.
    1. Humbert M, Sitbon O, Chaouat A, Bertocchi M, Habib G, Gressin V, Yaici A, Weitzenblum E, Cordier J, Chabot F, Dromer C, Pison C, Reynaud-Gaubert M, Haloun A, Laurent M, Hachulla E, Simonneau G. Pulmonary arterial hypertension in France: results from a national registry. Am J Respir Crit Care Med. 2006 May 01;173(9):1023–1030. doi: 10.1164/rccm.200510-1668OC.
    1. Ling Y, Johnson MK, Kiely DG, Condliffe R, Elliot CA, Gibbs JSR, Howard LS, Pepke-Zaba J, Sheares KKK, Corris PA, Fisher AJ, Lordan JL, Gaine S, Coghlan JG, Wort SJ, Gatzoulis MA, Peacock AJ. Changing demographics, epidemiology, and survival of incident pulmonary arterial hypertension: results from the pulmonary hypertension registry of the United Kingdom and Ireland. Am J Respir Crit Care Med. 2012 Oct 15;186(8):790–796. doi: 10.1164/rccm.201203-0383OC.
    1. McGoon MD, Benza RL, Escribano-Subias P, Jiang X, Miller DP, Peacock AJ, Pepke-Zaba J, Pulido T, Rich S, Rosenkranz S, Suissa S, Humbert M. Pulmonary arterial hypertension: epidemiology and registries. J Am Coll Cardiol. 2013 Dec 24;62(25 Suppl):D51–D59. doi: 10.1016/j.jacc.2013.10.023.
    1. Dournes G, Verdier D, Montaudon M, Bullier E, Rivière A, Dromer C, Picard F, Billes M, Corneloup O, Laurent F, Lederlin M. Dual-energy CT perfusion and angiography in chronic thromboembolic pulmonary hypertension: diagnostic accuracy and concordance with radionuclide scintigraphy. Eur Radiol. 2014 Jan;24(1):42–51. doi: 10.1007/s00330-013-2975-y.
    1. Gopalan D, Delcroix M, Held M. Diagnosis of chronic thromboembolic pulmonary hypertension. Eur Respir Rev. 2017 Jan;26(143):160108. doi: 10.1183/16000617.0108-2016.
    1. Mahmud E, Madani MM, Kim NH, Poch D, Ang L, Behnamfar O, Patel MP, Auger WR. Chronic thromboembolic pulmonary hypertension: evolving therapeutic approaches for operable and inoperable disease. J Am Coll Cardiol. 2018 May 29;71(21):2468–2486. doi: 10.1016/j.jacc.2018.04.009.
    1. Kim NH, Delcroix M, Jais X, Madani MM, Matsubara H, Mayer E, Ogo T, Tapson VF, Ghofrani H, Jenkins DP. Chronic thromboembolic pulmonary hypertension. Eur Respir J. 2019 Jan;53(1):1801915. doi: 10.1183/13993003.01915-2018.
    1. Pepke-Zaba J, Delcroix M, Lang I, Mayer E, Jansa P, Ambroz D, Treacy C, D'Armini AM, Morsolini M, Snijder R, Bresser P, Torbicki A, Kristensen B, Lewczuk J, Simkova I, Barberà JA, de Perrot M, Hoeper MM, Gaine S, Speich R, Gomez-Sanchez MA, Kovacs G, Hamid AM, Jaïs X, Simonneau G. Chronic thromboembolic pulmonary hypertension (CTEPH): results from an international prospective registry. Circulation. 2011 Nov 01;124(18):1973–1981. doi: 10.1161/CIRCULATIONAHA.110.015008.
    1. Delcroix M, Lang I, Pepke-Zaba J, Jansa P, D'Armini AM, Snijder R, Bresser P, Torbicki A, Mellemkjaer S, Lewczuk J, Simkova I, Barberà JA, de Perrot M, Hoeper MM, Gaine S, Speich R, Gomez-Sanchez MA, Kovacs G, Jaïs X, Ambroz D, Treacy C, Morsolini M, Jenkins D, Lindner J, Dartevelle P, Mayer E, Simonneau G. Long-term outcome of patients with chronic thromboembolic pulmonary hypertension: results from an international prospective registry. Circulation. 2016 Mar 01;133(9):859–871. doi: 10.1161/CIRCULATIONAHA.115.016522.
    1. Remy-Jardin M, Remy J, Artaud D, Fribourg M, Beregi JP. Spiral CT of pulmonary embolism: diagnostic approach, interpretive pitfalls and current indications. Eur Radiol. 1998;8(8):1376–1390. doi: 10.1007/s003300050557.
    1. Kreitner K, Kunz RP, Ley S, Oberholzer K, Neeb D, Gast KK, Heussel C, Eberle B, Mayer E, Kauczor H, Düber C. Chronic thromboembolic pulmonary hypertension - assessment by magnetic resonance imaging. Eur Radiol. 2007 Jan;17(1):11–21. doi: 10.1007/s00330-006-0327-x.
    1. Auger WR, Fedullo PF, Moser KM, Buchbinder M, Peterson KL. Chronic major-vessel thromboembolic pulmonary artery obstruction: appearance at angiography. Radiology. 1992 Feb;182(2):393–398. doi: 10.1148/radiology.182.2.1732955.
    1. Gopalan D, Blanchard D, Auger WR. Diagnostic evaluation of chronic thromboembolic pulmonary hypertension. Ann Am Thorac Soc. 2016 Jul;13(Suppl 3):S222–39. doi: 10.1513/AnnalsATS.201509-623AS.
    1. Yorke J, Corris P, Gaine S, Gibbs JSR, Kiely DG, Harries C, Pollock V, Armstrong I. emPHasis-10: development of a health-related quality of life measure in pulmonary hypertension. Eur Respir J. 2014 Apr;43(4):1106–1113. doi: 10.1183/09031936.00127113.

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