1. Paediatric Cardiac Research Across the Region Professor Massimo Caputo Cardiac Surgeon Karen Sheehan RGN, RSCN, MSc Cardiac Research Sister

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4. Need for research in CHD in UK and Ireland • 10 published RCTs in paediatric cardiac surgery in UK and Ireland since 2000 • All single-centre phase II trials • 431 patients randomised in total or < 1% of approx. 65,000 children (most of these pts were recruited in Bristol)

5. Strength of Bristol Network • The only BHF chair in congenital heart surgery in the UK • Congenital heart surgery is one of the leading theme in the cardiovascular section of the BRC • Bristol congenital heart surgery academic team has conducted the highest number of RCTs over the last 10 years in the UK • Bristol Heart Institute is one of 7 Specialised Research Institutes that were created by UoB in recognition of their world-class, leading-edge research

6. Cardiac Research Nurses

7. An overview of Research in the Network Region • The paediatric research nurse team has been in existence for 6 years and has drawn together cardiac research studies under one umbrella to ensure a single approach to our families. • We are leading some of the most cutting edge research in Congenital Heart Disease from Stem Cell work to alternative drug therapies for patients requiring lifelong anticoagulation • Studies are offered to ALL eligible patients who access care through Bristol Royal Hospital for Children

8. Research in the new CHD standards G1 (L1) Each Specialist Children’s Surgical Centre is expected to participate in research • We recruit to local BRC, NIHR, Charity and Commercially funded studies. The studies include local, national and international collaborations. • We currently recruit to: 3 surgical and 2 cardiology studies; are in follow up/results for 5 surgical and 2 cardiology studies; in set up for 2 surgical studies and 1 cardiology studies; and have potential interest in 2 surgical and 1 cardiology studies. • They are a combination of observational (drug and non-drug) and observational studies

9. G2 (L1) Each Congenital Heart Network must have, and regularly update, a research strategy and programme that documents current and planned research activity in the field of paediatric cardiac disease and the resource needed to support the activity and objectives for development. This must include commitment to working in partnership with other Specialist Children’s Surgical Centres and Specialist Children’s Cardiology Centre, and Local Children’s Cardiology Centres as appropriate, in research activity which aims to address issues that are important for the further development and improvement of clinical practice, for the benefit of children and young people with CHD and their families.

10. Our mission statement is ‘To provide high quality, relevant research which advances the care of patients with CHD and their families’ by ensuring our research is • Patient and family focused • A positive experience for patients and families irrespective of whether they take part in a study or not • Is inclusive • Is at the forefront of new treatments and technologies • Is at the centre of clinical care • Is fully collaborative and multidisciplinary • Represents local, national and international collaborations • Utilises all funding streams i.e. BRC,NIHR, Commercial collaboration and charitable organisations

11. As the research Sister I work with the cardiac surgeons and Cardiologists to resource any studies they wish to undertake. I also work with the commercial manager in R&D and NIHR study managers to screen potential cardiac studies we might be able to support before directing to an appropriate PI. • We work as part of the Clinical Trials and Evaluation Unit of Bristol University and run studies funded by the Biomedical Research Centre’s cardiovascular theme with a strong PPI framework. • We have a trust wide research and innovation strategy • http://www.uhbristol.nhs.uk/media/2216772/research_and_innovation_strategy_2014-2019_final.pdf • as well as an NIHR clinical research nurse strategy • https://www.nihr.ac.uk/our-faculty/clinical-research-staff/clinical-research-nurses/

12. We work collaboratively with other cardiac centres in both national (MORBIDITY) and international (APIXABAN) studies. • We tie in with the PICU research group to keep abreast of what research is running in PICU which may include cardiac patients in their participants and vice versus.

13. G3 (L1) Each Congenital Heart network must demonstrate close links with one or more academic department(s) in Higher Education Institutions.

14. As well as working closely with the CTEU, part of the University of Bristol, we also work with other institutions depending on the study, e.g. UCL for MORBIDITY, London School of Hygiene & Tropical Medicine for AIMS. • We have strong links with University of the West of England (UWE) with a joint Public and Patient Involvement (PPI) lead appointment working between UWE, UoB and UHBristol. We work closely to develop strong PPI to help drive research forward in CHD and to ensure it is designed with the patient and family in mind. We currently have a parent PPI group and in set up for a young person’s PPI group. • http://cteu.bristol.ac.uk/ppi/patient-and-public-advisory-group-pag/ • I am cardiovascular lead for PPI for the NIHR Bristol BRC and help move forward PPI within the speciality. • I also provide BRP (Building Research Partnerships) workshops in PPI for members of the public and clinicians who are interested in getting involved in PPI as part of UWE’s PPI programme.

16. G4 (L1) Where they wish to do so, patients should be supported to be involved in trials of new technologies, medicines etc. • Patients come from all over the network and we endeavour to ensure equal opportunity to all our patients and families to take part in any research study they are eligible for. Some special considerations have to be addressed during set up of studies due to the large geographical area covered by the cardiac service. • This can range from finding a more local unit which are also running a study (e.g. AIMS) or ensuring appropriate funding comes with the study to ensure travel expenses etc. are realistic. • The charts below demonstrate the breakdown of regional recruitment to 3 different studies.

17. The AIMS study • This is a national, multi centre, double blinded, randomised control study looking at the use of Irbesartan (an ARB) to reduce the aortic root dilatation in patients with Marfan syndrome • Marfan syndrome is a genetic disorder of the connective tissue • This patient population is relatively small as it’s condition specific • Highest recruiter in the UK

18. AIMS Study Percentage recruitment by region

19. Morbidity Study • This is a national multicentre observational study driven by NICOR and recruits from five centre around the UK (Birmingham, Evelina, Bristol, Glasgow and GOSH) • The study aims to identify complications following cardiac surgery and their impact on the patient and family • The complications have been identified by a team of clinicians and public/parents because they are clinically important, have a detrimental impact of the child and family and can be easily identified • The families are followed up for 6 months from surgery to look at the IMPACT of complications from a physical, developmental, psychological and practical perspective for patient and the family • The study population was ALL children having cardiac surgery over an 18 month period

20. MORBIDITY Study Percentage recruitment by region

21. DECISION Study • This is a single centre observational study looking at predictors of post operative bleeding in children following cardiac surgery • The study population was children having cardiac surgery requiring cardiopulmonary bypass

22. DECISION Study Percentage recruitment by region

23. How to get involved in Research