Article

Real-world versus Clinical Trial Data with NOACs

Register or Login to View PDF Permissions
Permissions× For commercial reprint enquiries please contact Springer Healthcare: ReprintsWarehouse@springernature.com.

For permissions and non-commercial reprint enquiries, please visit Copyright.com to start a request.

For author reprints, please email rob.barclay@radcliffe-group.com.
Average (ratings)
No ratings
Your rating

Abstract

On 12 November 2014, Radcliffe Cardiology, in association with Arrhythmia & Electrophysiology Review (AER) journal, held a roundtable discussion in London, UK. The discussion held between an expert group of physicians was moderated by Professor A John Camm, a renowned authority in anticoagulation and atrial fibrillation. The meeting comprised a series of seven presentations and subsequent panel discussions on a range of topical issues related to the use of non-vitamin K antagonist (novel) oral anticoagulants (NOACs): real-world versus clinical trial data; once or twice daily dosing regimens; spot checks or monitoring for anticoagulation status; antidotes for NOACs; NOACs and dual antiplatelet therapy; NOAC treatment in chronic renal impairment; and choosing between NOACs. This paper summarises the presentations and presents key highlights from the subsequent discussions.

Disclosure:Professor Camm is a consultant/advisor/speaker for Actelion, Astellas, AstraZeneca, Bayer, Boehringer Ingelheim, Boston Scientific, Biotronik, BMS, ChanRX, Daiichi Sankyo, Gilead, GSK, InfoBionic, Incarda, Johnson and Johnson, Medtronic, Menarini, Merck, Mitsubishi, Novartis, Otsuka, Pfizer, Sanofi, Servier, St. Jude Medical, Takeda and Xention; Doctor Alings is a consultant/advisor for Bayer, Boehringer Ingelheim, BMS/Pfizer, Daiichi Sankyo; Professor De Caterina has received a research grant from Boehringer Ingelheim, and is a consultant/advisor/speaker for Bayer, Boehringer Ingelheim, BMS/Pfizer, Daiichi Sankyo, Novartis; Professor Kirchhof has received research grants from BMS/Pfizer, Cardiovascular Therapeutics, Daiichi Sankyo, Sanofi, St. Jude Medical, and is consultant/advisor/speaker for Bayer, Boehringer Ingelheim, BMS, Daiichi Sankyo, Johnson & Johnson, Medtronic, MSD, Pfizer and Servier; Professor Le Heuzey is a consultant/advisor for Bayer, Boehringer Ingelheim, BMS/Pfizer and Daiichi Sanyo; Professor Verheugt is consultant/advisor for Bayer, Boehringer Ingelheim, BMS/Pfizer, Daiichi Sankyo

Received:

Accepted:

Published online:

Support:The roundtable was supported by an unrestricted educational grant from Daiichi-Sankyo.

Copyright Statement:

The copyright in this work belongs to Radcliffe Medical Media. Only articles clearly marked with the CC BY-NC logo are published with the Creative Commons by Attribution Licence. The CC BY-NC option was not available for Radcliffe journals before 1 January 2019. Articles marked ‘Open Access’ but not marked ‘CC BY-NC’ are made freely accessible at the time of publication but are subject to standard copyright law regarding reproduction and distribution. Permission is required for reuse of this content.

There are two types of data available to inform the use of NOACs: that from large clinical trials and that from registries, and these data are of different quality. Professor Kirchhof discussed the current treatment paradigms in valvular AF and presented a decision tree for antithrombotic therapy in patients with non-valvular AF, which combines the recommendations of current European Society of Cardiology (ESC) guidelines and those of the American College of Cardiology Foundation (ACCF)/American Heart Association (AHA)/Heart Rhythm Society (HRS) (see Figure 1).3 These recommendations show that in valvular AF, VKAs are the preferred treatment option. In non-valvular AF, the US recommendations give no preference between VKAs and NOACs, while the European and Canadian guidelines state that NOACs are the preferred option.

Although widely used, aspirin is not recommended: clinical trial data suggest that warfarin reduces the risk of major stroke, arterial embolism and other intracranial haemorrhages compared with aspirin in elderly patients.4 In addition, in a comparison of apixaban and aspirin in 5,599 patients with AF who were at increased risk of stroke and for whom VKA therapy was unsuitable, the same amount of major bleeding seen in both, but the study was terminated early because of a clear benefit in favour of apixaban.5 Lifelong oral anticoagulation is indicated for all AF patients with two or more CHA2DS2VASc (chronic heart failure, hypertension, age >65, diabetes, prior stroke or transient ischaemic attack [TIA], vascular disease, sex category) factors. Patients with only one of these risk factors may also benefit from oral anticoagulation. Almost everyone with AF is eligible for oral anticoagulation.6 In valvular AF, VKAs are the preferred treatment option. In non-valvular AF, the US guidelines give no preference between VKAs and NOACs, while the European and Canadian guidelines state that NOACs are the preferred option.7

Four landmark trials of NOACs have formed the basis for guidelines: RE-LY (Randomized Evaluation of Long-Term Anticoagulation Therapy; dabigatran),8 ROCKET AF (Rivaroxaban Once Daily, Oral, Direct Factor Xa Inhibition Compared with Vitamin K Antagonism for Prevention of Stroke and Embolism Trial in Atrial Fibrillation),9 ARISTOTLE (Apixaban for Reduction in Stroke and Other Thromboembolic Events in Atrial Fibrillation),10 and ENGAGE AF-TIMI (Effective Anticoagulation with Factor Xa Next Generation in Atrial Fibrillation – Thrombolysis in Myocardial Infarction 48; edoxaban).11 A recent meta-analysis of all 71,683 participants included in these trials showed that NOACs had a favourable risk–benefit profile, with significant reductions in haemorrhagic stroke and intracranial haemorrhage, but increased gastrointestinal bleeding.12 In addition, a 10 % mortality benefit was seen with NOACs.

In terms of real-world data, the GARFIELD (Global Anticoagulant Registry in the FIELD) study, an observational study of patients newly diagnosed with non-valvular AF, found that NOACs are not being used according to stroke risk scores and guidelines, with overuse in patients at low risk and underuse in those at high risk of stroke.13 Recent 1-year data from the EORP-AF Pilot (EURObservational Research Programme-Atrial Fibrillation General Registry Pilot Phase) suggest the majority of patients (84 %) who were taking a VKA at baseline, remain on a VKA afterwards.

Decision Tree for Antithrombotic Therapy in Patients with Non-valvular Atrial Fibrillation

Article image

Likewise, patients initiated on a NOAC were likely to remain on a NOAC. Encouragingly, many patients who had received antiplatelet therapies have switched to OACs after a year.14 Another registry study, PREFER in AF (Prevention of Thromboembolic Events – European Registry in Atrial Fibrillation) found that NOACs constitute 5–10 % of use.15

The panel discussed the value of registries. It was considered that they were necessary as they generate useful data next to the phase III clinical trial data. Many patients do not participate in clinical trials because they do not meet the exclusion criteria or do not want to participate in a trial. The data generated by clinical trials can be to some extent artificial because, for example, drug intake is monitored for each patient. Furthermore, not all side-effects are picked up in phase III trials as several 100,000 people need to be exposed to the drug to reveal certain rare effects. However, registries are rarely controlled properly in terms of randomisation, therefore it can be difficult to put the results into perspective. Professor Le Heuzey considered that registries generate valuable data e.g. GARFIELD shows a lower rate of anticoagulation than in clinical trials. Registries give us an idea of the different settings in which NOACs are being used, the uptake of NOAC therapy and improved safety information, but efficacy is hard to assess. We need to know in which settings registries are run (e.g. by cardiologists or GPs) and what the inclusion criteria are for registries in order to interpret them correctly, according to Professor De Caterina. They can also help inform why data arising from clinical trials are not put into practice. For example, based on clinical trial data, the use of aspirin is discouraged in AF patients and yet registry data tell us that aspirin is still in widespread use. The PREFER registry showed that doctors treat vascular disease and AF as two independent entities so therefore when these conditions coexist, two different drugs are used. In order to maximise their usefulness, registries need more outcome data, as can be generated by pragmatic trials. NOACs perform as well in real world registries as in clinical trials but VKAs perform worse in real world data. So more trials in daily use setting are needed. In summary, the panel concluded that registry data are extremely helpful and complementary to clinical trial data and in many instances confirmatory, particularly in terms of safety.

References

  1. Camm AJ, Lip GY, De Caterina R, et al. 2012 focused update of the ESC Guidelines for the management of atrial fibrillation: an update of the 2010 ESC Guidelines for the management of atrial fibrillation. Developed with the special contribution of the European Heart Rhythm Association. Eur Heart J 2012;33:2719–47.
    Crossref | Pubmed
  2. Heidbuchel H, Verhamme P, Alings M, et al. EHRA practical guide on the use of new oral anticoagulants in patients with non-valvular atrial fibrillation: executive summary. Eur Heart J 2013;34:2094–106.
    Crossref | Pubmed
  3. Mohl W, Komamura K, Kasahara H, et al. Myocardial protection via the coronary sinus. Circ J 2008;72:526–33.
    Crossref | Pubmed
  4. Mant J, Hobbs FD, Fletcher K, et al. Warfarin versus aspirin for stroke prevention in an elderly community population with atrial fibrillation (the Birmingham Atrial Fibrillation Treatment of the Aged Study, BAFTA): a randomised controlled trial. Lancet 2007;370:493–503.
    Crossref | Pubmed
  5. Connolly SJ, Eikelboom J, Joyner C, et al. Apixaban in patients with atrial fibrillation. N Engl J Med 2011;364:806–17.
    Crossref | Pubmed
  6. Singer DE, Hylek EM. Optimal oral anticoagulation for patients with nonrheumatic atrial fibrillation and recent cerebral ischemia. N Engl J Med 1995;333:1504.
    Crossref | Pubmed
  7. Kirchhof P, Curtis AB, Skanes AC, et al. Atrial fibrillation guidelines across the Atlantic: a comparison of the current recommendations of the European Society of Cardiology/European Heart Rhythm Association/European Association of Cardiothoracic Surgeons, the American College of Cardiology Foundation/American Heart Association/Heart Rhythm Society, and the Canadian Cardiovascular Society. Eur Heart J 2013;34:1471–4.
    Crossref | Pubmed
  8. Connolly SJ, Ezekowitz MD, Yusuf S, et al. Dabigatran versus warfarin in patients with atrial fibrillation. N Engl J Med 2009;361:1139–51.
    Crossref | Pubmed
  9. Patel MR, Mahaffey KW, Garg J, et al. Rivaroxaban versus warfarin in nonvalvular atrial fibrillation. N Engl J Med 2011;365:883–91.
    Crossref | Pubmed
  10. Granger CB, Alexander JH, McMurray JJ, et al. Apixaban versus warfarin in patients with atrial fibrillation. N Engl J Med 2011;365:981–92.
    Crossref | Pubmed
  11. Giugliano RP, Ruff CT, Braunwald E, et al. Edoxaban versus warfarin in patients with atrial fibrillation. N Engl J Med 2013;369:2093–104.
    Crossref | Pubmed
  12. Ruff CT, Giugliano RP, Braunwald E, et al. Comparison of the efficacy and safety of new oral anticoagulants with warfarin in patients with atrial fibrillation: a meta-analysis of randomised trials. Lancet 2014;383:955–62.
    Crossref | Pubmed
  13. Kakkar AK, Mueller I, Bassand JP, et al. Risk profiles and antithrombotic treatment of patients newly diagnosed with atrial fibrillation at risk of stroke: perspectives from the international, observational, prospective GARFIELD registry.
    PLoS One 2013;8:e63479.
    Crossref | Pubmed
  14. Lip GY, Laroche C, Ioachim PM, et al. Prognosis and treatment of atrial fibrillation patients by European cardiologists: one year follow-up of the Eurobservational Research Programme-Atrial Fibrillation General Registry pilot phase (EORP-AF Pilot registry), Eur Heart J 2014;35:3365–76.
    Crossref | Pubmed
  15. Kirchhof P, Ammentorp B, Darius H, et al. Management of atrial fibrillation in seven European countries after the publication of the 2010 ESC Guidelines on atrial fibrillation: primary results of the Prevention of Thromboembolic Events – European Registry in Atrial Fibrillation (PREFER in AF). Europace 2014;16:6–14.
    Crossref | Pubmed
  16. Zamorano JL, Greiner W, Sandberg A, et al. Patient preferences for chronic treatment for stroke prevention: results from the European Patient Survey in Atrial Fibrillation (EUPS-AF). Poster P1789. Presented at European Society of Cardiology Congress, 25–29 August 2012; Munich, Germany.
  17. Weitz JI, Connolly SJ, Patel I, et al. Randomised, parallel-group, multicentre, multinational phase 2 study comparing edoxaban, an oral factor Xa inhibitor, with warfarin for stroke prevention in patients with atrial fibrillation. Thromb Haemost 2010;104:633–41.
    Crossref | Pubmed
  18. Bae JP, Dobesh PP, Klepser DG, et al. Adherence and dosing frequency of common medications for cardiovascular patients. Am J Manag Care 2012;18:139–46.
    Pubmed
  19. Laliberte F, Nelson WW, Lefebvre P, et al. Impact of daily dosing frequency on adherence to chronic medications among nonvalvular atrial fibrillation patients. Adv Ther 2012;29:675–90.
    Crossref | Pubmed
  20. Evers T, Diamantopoulos A. Clinical impact of treatment persistence in patients with atrial fibrillation. Value in Health 2013;16:A276.
    Crossref
  21. Douxfils J, Mullier F, Robert S, et al. Impact of dabigatran on a large panel of routine or specific coagulation assays. Laboratory recommendations for monitoring of dabigatran etexilate. Thromb Haemost 2012;107:985–97.
    Crossref | Pubmed
  22. Douxfils J, Mullier F, Loosen C, et al. Assessment of the impact of rivaroxaban on coagulation assays: laboratory recommendations for the monitoring of rivaroxaban and review of the literature. Thromb Res 2012;130:956–66.
    Crossref | Pubmed
  23. Becker RC, Yang H, Barrett Y, et al. Chromogenic laboratory assays to measure the factor Xa-inhibiting properties of apixaban – an oral, direct and selective factor Xa inhibitor.
    J Thromb Thrombolysis 2011;32:183–7.
    Crossref | Pubmed
  24. Cuker A, Siegal DM, Crowther MA, et al. Laboratory measurement of the anticoagulant activity of the non-vitamin K oral anticoagulants. J Am Coll Cardiol 2014;64:1128–39.
    Crossref | Pubmed
  25. Blann AD, Lip GY. Laboratory monitoring of the non-vitamin K oral anticoagulants. J Am Coll Cardiol 2014;64:1140–2.
    Crossref | Pubmed
  26. Makris M, Van Veen JJ, Tait CR, et al. Guideline on the management of bleeding in patients on antithrombotic agents. Br J Haematol 2013;160:35–46.
    Crossref | Pubmed
  27. Phase 2 healthy volunteer study to evaluate the ability of prt064445 to reverse the effects of several blood thinner drugs on laboratory tests. Available at: http://clinicaltrials.gov/ct2/show/NCT01758432?term=NCT01758432&rank=1 (accessed 27 November 2014).
  28. Crowther M, Vandana M, Michael K, et al. A phase 2 randomized, double-blind, placebo-controlled trial demonstrating reversal of rivaroxaban-induced anticoagulation in healthy subjects by andexanet alfa (PRT064445), an antidote for Fxa inhibitors. Abstract no 3636. Presented at the 55th Americal Society of Haematology (ASH) Annual Meeting and Exposition 7–10 December 2013; New Orleans, LA, US.
  29. US National Institutes of Health. A study in older subject to evaluate the safety and ability of andexanet alfa to reverse the anticoagulation effect of rivaroxaban. http://clinicaltrials.gov/ct2/show/NCT02220725 (accessed 27 November 2014).
  30. US National Institutes of Health. Study to evaluate the safety, pharmacokinetics and pharmacodynamics of BI 655075 administered alone or with dabigatran etexilate. Available at: http://clinicaltrials.gov/ct2/show/NCT01688830 (accessed 27 November 2014).
  31. US National Institutes of Health. Reversal of dabigatran anticoagulant effect with idarucizumab. Available at: http://clinicaltrials.gov/ct2/show/NCT02104947 (accessed 27 November 2014).
  32. US National Institutes of Health. Effects of a double-blind, single dose of per977 administered alone, and following a single dose of edoxaban (PER977-P1). Available at: http://clinicaltrials.gov/ct2/show/NCT01826266 (accessed 27 November 2014).
  33. Boehringer Ingelheim. US FDA grants breakthrough therapy designation to Pradaxa® (dabigatran etexilate) specific investigational antidote. Press release. Available at: www.boehringer-ingelheim.com/news/news_releases/press_releases/2014/30_june_2014_dabigatranetexilate.html (accessed 27 November 2014).
  34. van Ryn J, Sghmoll M, Pillu H, et al. Effect of dabigatran on the ability to generate fibrin at a wound site and its reversal by idarucizumab, the antidote to dabigatran, in healthy volunteers: an exploratory marker of blood loss. Abstract no 18403. Presented at the American Heart Association Scientific Sessions, 18 November 2014; Chicago, USA.
  35. Lip GY, Huber K, Andreotti F, et al. Antithrombotic management of atrial fibrillation patients presenting with acute coronary syndrome and/or undergoing coronary stenting: executive summary – A consensus document of the European Society of Cardiology Working Group on Thrombosis, endorsed by the European Heart Rhythm Association (EHRA) and the European Association of Percutaneous Cardiovascular Interventions (EAPCI), Eur Heart J 2010;31:1311–8.
    Crossref | Pubmed
  36. Hansen ML, Sorensen R, Clausen MT, et al. Risk of bleeding with single, dual, or triple therapy with warfarin, aspirin and clopidogrel in patients with atrial fibrillation. Arch Intern Med 2010;170:1433–41.
    Crossref | Pubmed
  37. European Heart Rhythm Association, European Association for Cardio-Thoracic Society, Camm AJ, et al. Guidelines for the management of atrial fibrillation: the Task Force for the Management of Atrial Fibrillation of the European Society of Cardiology (ESC). Eur Heart J 2010;31:2369–429.
    Crossref | Pubmed
  38. Faxon DP, Eikelboom JW, Berger PB, et al. Antithrombotic therapy in patients with atrial fibrillation undergoing coronary stenting: a North American perspective: executive summary. Circ Cardiovasc Interv 2011;4:522–34.
    Crossref | Pubmed
  39. US National Institutes of Health. A study exploring two strategies of rivaroxaban (JNJ39039039; BAY-59-7939) and one of oral vitamin K antagonist in patients with atrial fibrillation who undergo percutaneous coronary intervention. Available at: https://clinicaltrials.gov/ct2/show/NCT01830543 (accessed 17 March 2015).
  40. US National Institutes of Health. Evaluation of dual therapy with dabigatran vs. triple therapy with warfarin in patients with af that undergo a pci with stenting (REDUAL-PCI). Available at: http://clinicaltrials.gov/ct2/show/NCT02164864 (accessed 1 December 2014).
  41. Ogata K, Mendell-Harary J, Tachibana M, et al. Clinical safety, tolerability, pharmacokinetics, and pharmacodynamics of the novel factor Xa inhibitor edoxaban in healthy volunteers. J Clin Pharmacol 2010;50:743–53.
    Crossref | Pubmed
  42. Peacock WF, Gearhart MM, Mills RM. Emergency management of bleeding associated with old and new oral anticoagulants. Clin Cardiol 2012;35:730–7.
    Crossref | Pubmed