Cardiomyopathy PSP protocol

Contents

Published: 23 May 2023

Version: 1.0

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Purpose of the PSP and background

The purpose of this Protocol is to clearly set out the aims, objectives and commitments of the Cardiomyopathy PSP in line with James Lind Alliance (JLA) principles.  The Protocol is a JLA requirement and will be published on the PSP’s page of the JLA website.  The Steering Group will review the Protocol regularly and any updated version will be sent to the JLA.

The JLA is a non-profit making initiative, established in 2004.  It brings patients, carers and clinicians together in PSPs.  These PSPs identify and prioritise the evidence uncertainties, or ‘unanswered questions’, that they agree are the most important for research in their topic area.  Traditionally PSPs have focused on uncertainties about the effects of treatments, but some PSPs have chosen to broaden their scope beyond that.  The aim of a PSP is to help ensure that those who fund health research are aware of what really matters to patients, carers and clinicians.  The National Institute for Health and Care Research (NIHR) coordinates the infrastructure of the JLA to oversee the processes for PSPs, based at the NIHR Evaluation, Trials and Studies Coordinating Centre (NETSCC), University of Southampton. 

Cardiomyopathy is a disease of the heart muscle. (‘Cardio’ means heart, ‘myo’ means muscle and ‘pathy’ means disease.) It isn’t a single condition, but a group of conditions that affect the structure of the heart and reduce its ability to pump blood around the body. Cardiomyopathy can affect anyone, at any age and is thought to occur in around 1 in 300 people in the UK.

The main symptoms of cardiomyopathy include tiredness, breathlessness, swelling in the abdomen, and ankles, palpitations, pain in the chest, and dizziness or fainting. It can be difficult to diagnose cardiomyopathy at first as these symptoms can also occur in other conditions and some people may have no symptoms at all in the early stages of cardiomyopathy. 

There are many possible causes of cardiomyopathy. Some types are genetic, caused by a mutation in the person’s DNA which affects how their heart develops. Genetic conditions may be inherited so cardiomyopathy sometimes runs in families. Often if one person is diagnosed with cardiomyopathy it is recommended that their close family members are tested for the condition too. Other causes of cardiomyopathy include viral infections, autoimmune diseases and some medications used to treat cancer.

Although a diagnosis of cardiomyopathy can be life-changing, with the right treatment and support, people with cardiomyopathy can live full and active lives.

Cardiomyopathy UK is keen to identify the research priorities of people living with cardiomyopathy and people caring for those who live with the condition, to help inform the research priorities of the charity, help researchers in cardiomyopathy identify questions of importance to users and to inform the priorities of other research funders.

Aims, objectives and scope of the PSP

The aim of the Cardiomyopathy PSP is to identify the unanswered questions about cardiomyopathy from patient, carer and clinical perspectives and then prioritise those that patients, carers, and clinicians agree are the most important for research to address.

The objectives of the PSP are to:

  1. Work with people affected by cardiomyopathy (patients (0+ years) and carers) and clinicians to identify uncertainties about all aspects of the management of cardiomyopathy.
  2. Agree by consensus a prioritised list of those uncertainties, for research.
  3. Publicise the results of the PSP and the process.
  4. Take the results to research commissioning bodies to be considered for funding.

For the purposes of this PSP cardiomyopathy is defined as an ‘inheritable intrinsic disease/primary problem with the heart muscle’.

The scope of the Cardiomyopathy PSP will include:

  1. Diagnosis (to include assessment of symptoms and investigations);
  2. Treatment (including ongoing care and management);
  3. Genetic testing, and/or family screening;
  4. Lifestyle, physical activity & exercise;
  5. Causes;
  6. Comorbidities;
  7. Outcomes of the conditions for individuals and their family;
  8. Impact of the conditions on individuals and their family (including i.e., physical, psychological, social, educational, travel);

associated with all types of cardiomyopathies, including rarer types. It will exclude from its scope questions about:

  • Ischemic cardiomyopathy (cardiomyopathy due to coronary artery disease);
  • Syndromic cardiomyopathies (cardiomyopathy as part of wider genetic syndromes associated with cardiomyopathy); and
  • Hypertension in those without cardiomyopathy and with hypertensive heart disease only due to high blood pressure.

It is recognised that international participants may respond to the surveys in which case their priorities will be separated and shared with their relevant overseas groups.

The Steering Group is responsible for discussing what implications the scope of the PSP will have for the evidence-checking stage of the process. Resources and expertise will be put in place to do this evidence checking.

The Steering Group

The Steering Group includes membership of patients and carers and clinicians, as individuals or representatives from a relevant group.

The Cardiomyopathy PSP will be led and managed by a steering group involving the following:

PSP Chair

  • Louise Dunford (JLA Adviser)

PSP Lead

  • Vacant, Cardiomyopathy UK

PSP Coordinator

  • Rebecca Gray, Cardiomyopathy UK

Patient representatives

  • Jenny Moon, Cardiomyopathy UK
  • Marcia Malcolm, Cardiomyopathy UK
  • Sandra Miller, Cardiomyopathy UK
  • Robbie Jones, Cardiomyopathy UK
  • LaRisha Porter, Cardiomyopathy UK
  • Andy Smith, Cardiomyopathy UK

Patient Lead:

  • Alison Fielding, Vice President/Patient Focus, Cardiomyopathy UK

Patient family member/Trustee

  • Libby Jarman, Senior Research Physiotherapist, University Hospitals Southampton NHS Trust and University of Southampton/Trustee, Cardiomyopathy UK

Clinical representatives

  • Jayne Partridge, Cardiomyopathy Specialist Nurse, Cardiomyopathy UK
  • Dr Tootie Bueser, RN PhD, Director for Nursing & Midwifery, South East Genomic Medicine Service Alliance, Guy’s & St Thomas’ NHS Foundation Trust and Chief Nurse, North Thames Genomic Medicine Service Alliance UCL Partners /Trustee, Cardiomyopathy UK
  • Ella Field, Research Nurse Inherited Cardiovascular Disease, Great Ormand Street Hospital
  • Helen Alexander, Physiotherapist, St Bartholomew’s Hospital, Barts Health NHS Trust
  • Dr Brian Halliday, Senior Clinical Lecturer at the National Heart and Lung Institute and Consultant Cardiologist at Royal Brompton Hospital
  • Professor Ruth Newbury-Ecob, MB ChB, MD, FRCP, FRCPCH, Consultant Clinical Geneticist, Department of Clinical Genetics, University Hospitals Bristol NHS Foundation Trust
  • Dr Caroline Coats, Consultant Cardiologist, NHS Greater Glasgow and Clyde
  • Pauline Aiston, Specialist Health Psychologist, Adult Congenital Heart Disease (ACHD) Lead for Psychology at the Bristol Heart Institute, University Hospitals Bristol and Western NHS Trust.
  • Dr Jenn Zhang, Clinical Psychologist, Bart’s Heart Centre Psychology Service, St Bartholomew’s Hospital, Barts Health NHS Trust

The Partnership and the priority setting process will be supported and guided by

  • The James Lind Alliance – Louise Dunford (PSP Chair, JLA Adviser)

The Steering Group will agree the resources, including time and expertise that they will be able to contribute to each stage of the process, with input and advice from the JLA.

Partners

Organisations and individuals will be invited to be involved with the Cardiomyopathy PSP as partners. This section will be updated as partners are confirmed. Partners are organisations or groups who will commit to supporting the PSP, promoting the process and encouraging their represented groups or members to participate. Organisations which can reach and advocate for these groups will be invited to become involved in the PSP.

Partners will represent the following groups:

  • people who have cardiomyopathy
  • carers of people who have cardiomyopathy
  • health and social care professionals with experience of cardiomyopathy.

Exclusion criteria:

Some organisations may be judged by the JLA or the Steering Group to have conflicts of interest. These may be perceived to potentially cause unacceptable bias as a member of the Steering Group. As this is likely to affect the ultimate findings of the PSP, those organisations will not be invited to participate. It is possible, however, that interested parties may participate in a purely observational capacity when the Steering Group considers it may be helpful.

The methods the PSP will use

This section describes a schedule of proposed steps through which the Cardiomyopathy PSP aims to meet its objectives.  The process is iterative and dependent on the active participation and contribution of different groups.  The methods used in any step will be agreed through consultation between the Steering Group members, guided by the PSPs aims and objectives.

Step 1: Identification and invitation of potential partners

Potential partner organisations will be identified through a process of peer knowledge and consultation, through the Steering Group members’ networks.  Potential partners will be contacted and informed of the establishment and aims of the Cardiomyopathy PSP.

Potential partner organisations that have been identified so far are as follows:

  • British Heart Foundation (BHF)
  • Heart Research UK (HRUK)
  • Pumping Marvellous (PM)
  • British Society for Heart Failure (BSHF)
  • Heart Research Institute (HRI)
  • British Association for Nursing in Cardiovascular Care (BANC)
  • British Cardiovascular Society (BCS)
  • Association of Inherited Cardiac Conditions (AICC)
  • Arrhythmogenic Cardiomyopathy Trust (ACT)
  • Blood Pressure UK (BPUK)
  • Community Heart Trust (CHT)
  • British Heart Rhythm Society (BHRS)
  • British Heart Valve Society (BHVS)
  • British Society of Echocardiology (BSE)
  • British Society of Genetic Medicine (BSGM)
  • British Junior Cardiologists Association (BJCA)
  • Centre for Mental Health (CFME)
  • Heart Failure Investigators Research Network (HFIRN)
  • UK Maternal Cardiology Society (UKMCS)
  • British Heart Foundation Clinical Research Collaborative (BHFCRC)
  • Myocarditis UK (MUK)
  • UK ATTR Amyloidosis Patient Association (UKATTR)
  • Genomics England (GENG)
  • Sudden Cardiac Arrest UK (SCAUK)
  • Society for Cardiothoracic Surgeons (SCTS)
  • British Association of Cardiovascular Prevention & Rehabilitation (BACPR)
  • Cardiovascular and the Ethics & Social Sciences Genomics England Clinical Interpretation Partnership (CESGE)

Step 2: Initial stakeholder meeting / awareness raising

The Steering Group should advise on when to do this. Awareness raising has several key objectives – to present the proposed plan for the PSP, to generate support for the process, to encourage participation in the process, to initiate discussion, answer questions and address concerns

The initial stakeholder meeting / awareness raising will have several key objectives:

  • to welcome and introduce potential members of the Cardiomyopathy PSP
  • to present the proposed plan for the PSP
  • to initiate discussion, answer questions and address concerns
  • to identify those potential partner organisations which will commit to the PSP and identify individuals who will be those organisations’ representatives and the PSP’s principal contacts
  • to establish principles upon which an open, inclusive and transparent mechanism can be based for contributing to, reporting and recording the work and progress of the PSP.

Step 3: Identifying evidence uncertainties

The Cardiomyopathy PSP will carry out a consultation to gather uncertainties from patients, carers and clinicians. A period of up to 2 months will be given to complete this exercise.

The Cardiomyopathy PSP recognises that the following groups may require additional consideration:

  • Youths (16 - 25 years)
  • Ethnic, or ethnic minority groups, and people from ethnic minority backgrounds.

The Steering Group will use the following methods to reach the special groups:

  • Online, paper and telephone survey, social media, pilot of the survey, two community engagement workshops (youth; ethnic groups) and further tailored communications to increase responses from groups identified as poor responders to the survey.
  • The PSP has appointed Jayne Partridge Cardiomyopathy UK Specialist Nurse as a special group advocate to – engage and represent the interests of those communities within the PSP, communicate information to help the steering group make informed decisions, ensure that the PSP implements findings and produce a report at end of the project to evaluate this work.

The starting point for identifying sources of uncertainties and research recommendations is NHS Evidence. 

Sources of uncertainties for research recommendations may include:

  • Cochrane database
  • NHS Evidence
  • NICE Guidance and NICE Research recommendations database
  • BMJ clinical evidence
  • Relevant treatment guidelines as published by:
    • British Journal of Cardiac Nursing
    • Journal of the American Society of Cardiology (JASC)
    • European Society of Cardiology
    • American Heart Association (AHA)
    • Circulation
    • Heart
    • Journal of American Medical Association (JAMA)
    • European Journal of Cardiovascular Nursing

Step 4: Refining questions and uncertainties

The consultation process will produce ‘raw’ questions and comments indicating patients’, carers and clinicians’ areas of uncertainty.  These raw questions will be categorised and refined by Kristina Staley into summary questions which are clear, addressable by research, and understandable to all.  Similar or duplicate questions will be combined where appropriate.  Out-of-scope and ‘answered’ submissions will be compiled separately. The Steering Group will have oversight of this process to ensure that the raw data is being interpreted appropriately and that the summary questions are being worded in a way that is understandable to all audiences.  The JLA Adviser will observe to ensure accountability and transparency.

This will result in a long list of in-scope summary questions.  These are not research questions and to try and word them as such may make them too technical for a non-research audience.  They will be framed as researchable questions that capture the themes and topics that people have suggested.

The summary questions will then be checked against evidence to determine whether they have already been answered by research.  This will be done by Kristina Staley, Information Specialist.  The PSP will complete the JLA Question Verification Form, which clearly describes the process used to verify the uncertainty of the questions, before starting prioritisation.  The Question Verification Form includes details of the types and sources of evidence used to check uncertainty.  The Question Verification Form should be published on the JLA website as soon as it has been agreed to enable researchers and other stakeholders to understand how the PSP has decided that its questions are unanswered, and any limitations of this.

Questions that are not adequately addressed by previous research will be collated and recorded on a standard JLA template by Kristina Staley. This will show the checking undertaken to make sure that the uncertainties have not already been answered.  The data should be submitted to the JLA for publication on its website on completion of the priority setting exercise, taking into account any changes made at the final workshop, in order to ensure that PSP results are publicly available.   

The Steering Group will also consider how it will deal with submitted questions that have been answered, and questions that are out of scope.

Step 5: Prioritisation – interim and final stages

The aim of the final stage of the priority setting process is to prioritise through consensus the identified uncertainties about cardiomyopathy. This will involve input from patients, carers and clinicians. The JLA encourages PSPs to involve as wide a range of people as possible, including those who did and did not contribute to the first consultation. There are usually two stages of prioritisation.

  1. Interim prioritisation is the stage where the long list of questions is reduced to a shorter list that can be taken to the final priority setting workshop. This is aimed at a wide audience, and is done using similar methods to the first consultation. With the JLA’s guidance, the Steering Group will agree the method and consider how best to reach and engage patients, carers and clinicians in the process. The most highly ranked questions (around 25) will be taken to a final priority setting workshop. Where the interim prioritisation does not produce a clear ranking or cut off point, the Steering Group will decide which questions are taken forwards to the final prioritisation.
  2. The final priority setting stage is generally a one-day workshop facilitated by the JLA. With guidance from the JLA and input from the Steering Group, up to 30 patients, carers and clinicians will be recruited to participate in a day of discussion and ranking, to determine the top 10 questions for research. All participants will declare their interests. The Steering Group will advise on any adaptations needed to ensure that the process is inclusive and accessible.

Dissemination of results

The Steering Group will identify audiences with which it wants to engage when disseminating the results of the priority setting process, such as researchers, funders and the patient and clinical communities. They will need to determine how best to communicate the results and who will take responsibility for this. Previous PSPs’ outputs have included academic papers, lay reports, infographics, conference presentations, journey slide deck and videos for social media.

Findings and research

It is anticipated that the findings of the Cardiomyopathy PSP will be reported to funding and research agenda setting organisations such as the NIHR and the major research funding charities. Steering Group members and partners are expected to develop the prioritised uncertainties into research questions, and to work to establish the research needs of those unanswered questions to use when approaching potential funders, or when allocating funding for research themselves, if applicable.

Publicity

As well as alerting funders, partners and Steering Group members are encouraged to publish the findings of the Cardiomyopathy PSP using both internal and external communication mechanisms. The Steering Group may capture and publicise the results through descriptive reports of the process itself in Plain English. This exercise will be distinct from the production of an academic paper, which the partners are also encouraged to do. However, production of an academic paper should not take precedence over publicising of the final results.

It should be noted that the priorities are not worded as research questions. The Steering Group should discuss how they will work with researchers and funders to establish how to address the priorities and to work out what the research questions are that will address the issues that people have prioritised. The dissemination of the results of the PSP will be co-led by PSP lead Charlotte Gallagher and PSP Patient lead Alison Fielding.

The JLA encourages PSPs to report back about any activities that have come about because of the PSP, including funded research. Details can be sent to jla@soton.ac.uk.

Agreement of the Steering Group

The Cardiomyopathy PSP Steering Group agreed the content and direction of this Protocol on 27th April 2023.