bs_bs_banner
Congenital heart disease-associated pulmonary arterial hypertension: preliminary results from a novel registry
imj_2708
874..879
M. L. Rose,1,2 G. Strange,3,6 I. King,1,2 S. Arnup,2,4 S. Vidmar,2,4 C. O’Donnell,9 F. Kermeen,8 L. Grigg,5 R. G. Weintraub1,2 and D. S. Celermajer6,7 1 Department of Cardiology, Royal Children’s Hospital, 2Murdoch Children’s Research Institute, 3Department of Epidemiology and Preventative Medicine, Monash University, 4Department of Paediatrics, University of Melbourne, 5Department of Cardiology, Royal Melbourne Hospital, Melbourne, 6 Department of Cardiology, Royal Prince Alfred Hospital, 7Department of Medicine, The University of Sydney, Sydney, 8Pulmonary Hypertension and Transplant Unit, The Prince Charles Hospital, Brisbane, Australia, and 9Department of Cardiology, Starship Children’s Hospital, Auckland, New Zealand
Key words congenital heart disease, pulmonary arterial hypertension, Eisenmenger syndrome, registry. Correspondence David Celermajer, Cardiology Department, Royal Prince Alfred Hospital, Missenden Road, Camperdown, NSW 2050, Australia. Email:
[email protected] Received 20 September 2011; accepted 6 December 2011. doi:10.1111/j.1445-5994.2011.02708.x
Abstract Background/Aims: Pulmonary arterial hypertension (PAH) frequently accompanies childhood congenital heart disease (CHD) and may persist into adult life. The advent of specific PAH therapies for PAH prompted formation of a national Australian and New Zealand registry in 2010 to document the incidence, demographics, presentation and outcomes for these patients. Methods: This multicentre, prospective, web-based registry enrols patients with CHDassociated PAH being followed in a tertiary centre. The inclusion criteria stipulated patient age ⱖ16 years, a measured mean pulmonary arterial pressure >25 mmHg at rest or echocardiographical evidence of PAH or a diagnosis of Eisenmenger syndrome, and followed since 1 January 2000. A single observer collected standardised data during a series of site visits. Results: Of the first 50 patients enrolled, 30 (60%) were female. The mean age (standard deviation (SD)) at the time of PAH diagnosis or confirmation in an adult centre was 27.23 (10.07) years, and 32 (64%) patients are currently aged >30 years. Fourteen (28%) patients were in World Health Organization Functional Class II and 36 (72%) in Class III at the time of diagnosis. Forty-seven of 50 (94%) had congenital systemic-pulmonary shunts, and 36 (72%) never underwent intervention. Thirteen (26%) had Down syndrome. Confirmation of PAH by recent cardiac catheterisation was available in 30 (60%) subjects. During follow up, a total of 32 (64%) patients received a PAH-specific therapy. Conclusions: CHD associated with PAH in adult life has resulted in a new population with unique needs. This registry will allow documentation of clinical course and longterm outcomes for these patients.
Introduction In Australia, the approximate number of adults living with congenital heart disease (CHD) has been estimated at 40 000,1 and now exceeds the number of children and adolescents with CHD. This number is consistent with epidemiological observations from a national European CHD registry.2 This number continues to expand, as does the complexity of CHD patients surviving to
On behalf of the Australian and New Zealand Adult Congenital Heart Disease Pulmonary Arterial Hypertension Registry. Funding: Actelion Australia Pty Ltd provided financial sponsorshipfor this registry. They had no role in the study design,data collection and analysis, manuscript preparation, ormanuscript review. Conflict of interest: None.
874
adult life.3 As medical management and surgical techniques continue to advance, children previously deemed unsuitable for cardiac surgery are undergoing corrective and palliative procedures. As a result, an increasing population of children with CHD is surviving into adulthood, many of whom will require further medical or surgical management for residual or progressive complications. Early epidemiological studies of CHD4–6 suggested low incidences of about four to five cases per 1000 live births; however, this figure included only the most severe cases referred to specialist referral centres in the USA in an era where paediatricians’ CHD expertise was relatively basic, rigorous diagnostic testing was underutilised and surgical intervention was less sophisticated. Since then, estimates of the incidence of CHD has progressively increased with currently reported incidences of 12–14 cases per 1000 © 2011 The Authors Internal Medicine Journal © 2011 Royal Australasian College of Physicians
PAH-CHD registry
live births, or higher, reported.7 In approximately 6% of adults with congenital heart defects, pulmonary arterial hypertension (PAH) develops,8 the most extreme example being Eisenmenger syndrome (ES).9 CHD has emerged to be one of the commonest associated causes of PAH. A diagnosis of PAH in adults with CHD is associated with more than twofold higher risk for all cause mortality and a threefold higher rate of health service utilisation,8 compared with CHD without PAH. The advent of targeted therapies that influence the key pathogenic pathways involved in PAH has given rise to new therapeutic considerations for patients previously not deemed suitable for such management, such as adults with CHD-associated PAH.10 Arteriolar vasoconstriction and vascular remodelling represent the therapeutic targets of currently available medications that include endothelin receptor antagonists, prostanoids and phosphodiesterase inhibitors. In 2010, a dedicated Australian and New Zealand registry for adults with CHD and PAH was established. The aims of the registry include the following: 1 To describe the prevalence of CHD associated with PAH, as seen in specialist adult CHD (ACHD) centres, 2 To describe the demographics of adults with CHD and coexisting PAH, 3 To understand management patterns and treatment responses to conventional and PAH-targeted therapies, and 4 To describe survival and to develop prognostic variables that may permit risk stratification and early identification of those who are at greatest risk of a poor outcome. We report on the features of the registry and on the first 50 patients entered into the registry.
Materials and methods The registry seeks to enrol cases of CHD associated with PAH in subjects aged at least 16 years at time of visit in a specialist centre and who have been seen in a specialist centre at least once since 1 January 2000. This includes subjects who were initially diagnosed prior to that date, as well as subjects who were referred or diagnosed since 1 January 2000. Potential study subjects were identified through ACHD and PAH tertiary referral centres through the lead clinician at each site using existing local databases. Additional cases were recruited by sending a flier to all cardiologists on the mailing list of the Cardiac Society of Australia and New Zealand. Human Ethics approval was obtained from all participating centres. For the purpose of the registry, PAH has been defined as a mean pulmonary artery pressure of >25 mmHg and a pulmonary artery wedge or left atrial pressure
