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CARE Pilot Trial Announcement

The Cavernomas A Randomised Effectiveness (CARE) pilot trial

CAUK is the Patient Group in a consortium also including senior clinicians with a special interest in cavernoma, led by our Medical Advisor Professor Rustam Al-Shahi Salman. The collaboration has been awarded £973,835 from the National Institute of Health Research (NIHR) to run the CARE pilot trial to find out whether treating brain cavernoma with neurosurgery or stereotactic radiosurgery (often known as Gamma-knife radiosurgery) is more effective than not using these treatments, in addition to treating symptoms. This pilot study will investigate whether it will be possible to do a much larger CARE main trial. We aim to recruit patients into the CARE pilot trial from the spring of 2021 onwards. This has been a long journey involving CAUK from the outset as described below.

Background

The main aim is to find out whether treating a symptomatic brain cavernoma directly with neurosurgery or stereotactic radiosurgery (known as ‘active treatment’) is more effective than not using these treatments (known as ‘conservative management’) in addition to treating symptoms.

Guidelines 2012

A study sponsored by CAUK jointly with Genetic Alliance UK in 2012, and the work principally of Ian Stuart working with Rustam Al-Shahi Salman, titled “Guidelines for the management of cerebral cavernous malformations in adults” concluded that there was no robust evidence to help clinicians and patients decide whether active treatment or conservative management is more effective for cavernoma.

What was required were randomized controlled trials (RCTs) comparing different approaches to treatment. At that time, and still today, there were three options (i) neurosurgery to remove the cavernoma via a craniotomy or spinal surgery, (ii) gamma-knife radiosurgery in which the cavernoma is zapped with radiation and (iii) conservative management which avoids (i) and (ii). In all groups, symptoms are treated with e.g. antiepileptic drugs for epilepsy/seizures if required.

Priorities for Research into Cavernoma (2016)

RCTs are expensive and, in order to justify the expense, potential funders, principally the National Institute for Health Research (NIHR), require evidence that the research being proposed is seen as a priority by both patients and clinicians. For this reason, CAUK undertook a “Priority Setting Partnership (PSP)” within the rigid guidelines of the James Lind Alliance, an organisation set up for this reason. The £25,000 funding for this was largely funded in a special appeal to CAUK members. This resulted in a report (Priorities for Research into Cavernoma) which gave a list of the top ten such priorities (and a subsidiary list of the next seventeen) of which the top was “Does treatment (with neurosurgery or stereotactic radiosurgery) or no treatment improve outcome for people diagnosed with brain or spine cavernoma?” and this is the formal question addressed in the CARE trial.

A randomised controlled trial (RCT) to answer the top priority

Since 2016 CAUK has worked to support Rustam in his preparation of getting funding for a RCT to answer the above question. This has involved two surveys of our members to ascertain if they would, in principle, take part in such trials, the NIHR commissioning research to address this uncertainty, Rustam leading our consortium to apply for this funding, and the NIHR awarding funding to our consortium.

Pilot RCT

The main issue concerns whether a sufficient number of patients with symptomatic brain cavernoma will take part in the trial. This uncertainty means that the programme being funded is a pilot RCT to see whether the full-scale RCT is feasible. The QuinteT research group is working with us to understand what influences doctors’ and patients’ decisions to take part when they are approached about the CARE pilot trial. This pilot RCT aims to recruit at least 60 patients, and the results will tell us how many patients might be needed to answer the question and whether it will be possible to recruit these patients in a reasonable timeframe.

The “Plain English Summary” section of the CARE trials application

Background

A cavernoma is a cluster of blood vessels that form blood-filled ‘caverns’ in the brain that look like a raspberry. Cavernomas can bleed into the brain and cause a stroke. Cavernomas can also cause a seizure or epilepsy. These symptoms may lead to disability, handicap and occasionally death. About 160 people in the UK each year are diagnosed with a cavernoma that has caused symptoms. In standard practice in the UK, about one fifth of people with cavernomas have ‘active treatment’ with either brain surgery to remove a cavernoma or stereotactic radiosurgery to treat it with radiation. These treatments themselves can cause death, disability, and handicap.

The pros and cons of active treatment or avoiding it by just treating the symptoms (also known as ‘conservative management’) are finely balanced. The most reliable way of finding out which management is best is to do a randomised trial, in which suitable patients are allocated to active treatment or conservative management at random. This has never been done with cavernomas, and this was the top priority identified by a Priority Setting Partnership for cavernoma.

Aim

The NIHR wants research to be done to find out whether enough patients can be found for a randomised trial comparing ‘active treatment’ with ‘conservative management’ of symptomatic cavernomas. We need to know this because cavernomas are rare and we do not know whether patients and doctors will take part.

Design and methods used

In three years we will:

  1. Create a network of specialists to do this study. We will include the UK and Ireland patient support organisations for people with cavernoma (Cavernoma Alliance UK) and doctors representing the relevant specialties at all the major hospitals specialising in decisions about cavernoma treatment in the UK and Ireland.
  2. Invite newly diagnosed patients to join a pilot phase of a randomised controlled trial. Of 190 people diagnosed with brain cavernoma in 18 months, we estimate that 60 of them will enrol in the randomised trial. We will study why some patients take part in the randomised trials and others don’t. We will use this information to change the methods of the trial if recruitment to the randomised trial goes slowly.
  3. Estimate whether enough patients can be found for a full-scale randomised trial to be done to find out whether active treatment or conservative management of symptomatic brain cavernomas is best.

Patient and public involvement

We involved people with cavernoma, carers, and representatives of Cavernoma Alliance UK in the design of this application. We held a focus group meeting with patients and carers on 6 July 2019: all approved the design of the project and the extent of patient and public involvement. The focus group wanted the trials to be as inclusive of patients as possible. The focus group recognised how the project would benefit from them contributing their ‘lived experience’ of brain cavernoma. People with cavernoma, carers, and representatives of Cavernoma Alliance UK will also keep an eye on the research by forming an advisory group and meeting regularly to discuss the research. Two representatives of this group will join and advise the steering committee.

Dissemination

We will publish our findings in medical journals. We will work with Cavernoma Alliance UK to produce a plain English summary and circulate it to patients via newsletters, email, the web, and social media.

Our new CEO, Helen Evans on her first day

We are delighted to announce that today is the first day for Helen Evans as our new Chief Executive. Helen brings a wealth of relevant skills and experience that we are confident will take CAUK through to its third phase of development, building on the success of our Big Lottery funded Big Step project. Until recently Helen was Chief Executive of Getting Heard, an Oxfordshire based advocacy charity. Under her leadership the charity was awarded the prestigious Quality Performance Mark, secured multi-year Big Lottery funding and was one of the first providers of Care Act advocacy in the UK.

Before working in the UK charity sector Helen was Head of Safeguarding for a global aid agency, having transitioned from an earlier 10 year career in international Human Resources. Helen is also an alum of the Jo Cox Women in Leadership Programme and former select committee expert witness. Helen will start on the 17th September 2018 and will be home-based, travelling as needed, working from 9.30am to 2.30pm every day except Monday.

Helen has created a special video for her first day, which you can see below…

CaverChat Closing

The CaverChat facility of our website will be closing from today, 17th September 2018, due mainly to lack of use. The majority of our members have chosen to use the facebook groups to communicate and so we highly recommend people visit those groups instead.

ANNOUNCEMENT – Appointment of Helen Evans as our new Chief Executive

Helen Evans Profile picture

Helen Evans Profile picture

We are delighted to announce the appointment of Helen Evans as our new Chief Executive. Helen brings a wealth of relevant skills and experience that we are confident will take CAUK through to its third phase of development, building on the success of our Big Lottery funded Big Step project. Until recently Helen was Chief Executive of Getting Heard, an Oxfordshire based advocacy charity. Under her leadership the charity was awarded the prestigious Quality Performance Mark, secured multi-year Big Lottery funding and was one of the first providers of Care Act advocacy in the UK.

Before working in the UK charity sector Helen was Head of Safeguarding for a global aid agency, having transitioned from an earlier 10 year career in international Human Resources. Helen is also an alum of the Jo Cox Women in Leadership Programme and former select committee expert witness. Helen will start on the 17th September 2018 and will be home-based, travelling as needed, working from 9.30am to 2.30pm every day except Monday.

You can view Helen’s introduction video by clicking here

Helen Evans, new Chief Executive says:

“I am delighted to be joining CAUK and to have the opportunity to take CAUK forward in its third phase of development, building on the success of the Big Lottery funded Big Step project. The work that CAUK leads is critical to meeting the needs of those impacted by cavernoma, and I very much appreciate the opportunity to be a part of this.

On starting at CAUK I anticipate my priorities being twofold: to meet, listen and respond to the needs of the CaverCommunity; and to also lead fundraising efforts ensuring the continuation of activities funded until September 2018 by the Big Lottery. Whilst I don’t have personal experience of cavernoma, I do know what it’s like living in a family impacted by long-term ill-health. I will be coming to this role with the passion of someone who knows first-hand what a difference a charity like CAUK can make, and will be drawing on my experience as a former Chief Executive to ensure CAUK continues building on its successes to date.

May I take this opportunity to acknowledge and thank your Chair of Trustees, Dr David White, who will shortly be taking a well-deserved step back from his role as Chair. I am also thankful to CAUK’s founder, Dr Ian Stuart, without whom this charity would not have been established and who will continue to be our Co-Ordinator. I am very excited to have the opportunity to work for CAUK and look forward to getting to know you all come mid-September”.

Dr David White, Chair of Trustees says:

I am thrilled with the appointment of Helen as the first Chief Executive of Cavernoma Alliance UK. Helen’s experience in the charity sector, both large and small, provides a wonderful background for us as we move into a new phase in our existence.

Dr Ian Stuart, Founder and Co-Ordinator says:

I am very excited at Helen’s appointment, and I looking forward immensely to working with her. She will enable us to move forward with confidence at an exciting time for supporting our greatly increasing membership and the development of new forms of therapy.

Next Steps

The Board of Trustees will be overseeing Helen’s induction and will ensure she has as many opportunities as possible to meet with cavernoma members, within the 18 hours per week her role permits. As discussed at our Annual General Meeting our Big Lottery funding ends this September, meaning one of Helen’s key priorities will be securing alternative funding as well as getting to meet CAUK members. If anyone has any questions or comments about Helen’s appointment please do not hesitate to get in touch.

11th International Cavernoma Alliance UK Forum (2017)

CAUK Members at lunchtime, Annual CAUK Forum, London, 2016

CAUK Members at lunchtime, Annual CAUK Forum, London, 2016

Our 11th International Cavernoma Alliance UK Forum is to be held for the first time away from London and in the north of England at the University of York. This will be an opportunity to especially benefit members in the north. For those who wish to attend from the south, fast trains from London to York take less than 2 hours.

Everyone is welcome, and as always, this event is free of charge – including lunch – and we would like you all to save the date, Saturday 24 June 2017.

The Forum will be held at the University’s prestigious Ron Cooke Hub (Lakeside Room) and will include Dr Connie Lee, President, Angioma Alliance USA, Dr Jenny Thomson geneticist, Chapel Allerton Hospital, Leeds and Mr Nitin Mukerji, neurosurgeon, James Cook University Hospital, Middlesbrough.

Campus accommodation will be available at Franklin House, on a first-come-first-served basis. This is a bed and breakfast service; both single and double rooms are en-suite and we have arranged a CAUK supper on Friday evening for those who arrive in York on Friday.

Accommodation can also be booked for the Saturday evening, 24 June 2017, so members can attend the CaverMorning which takes place at the Priory Street Centre in the middle of York, Sunday 25 June 9.30-1.30pm. This will be an informal time with a few fun workshops including one on drumming. There will be ample time for participants to attend the Jorvik Centre, the Minster, the Railway Museum etc.

Whilst the days are free, accommodation and an evening meal the night before (Friday 23 June 2017), are chargeable as is the accommodation for Saturday night. Accommodation rates include breakfast and VAT: £52.80 single; £90 double. (£20 per person for the Friday evening meal). The university has limited availability so visit the payment page and reserve with your credit card as soon as possible. Please note there is abundant parking (free after 6pm and all day at the weekends) and a free regular campus bus service to the venue from Franklin House.

When the accommodation becomes full at Franklin House, members can stay at either at the nearby Travelodge, Hull Road, York, or in the city centre a short bus journey away where accommodation can also be found at the Premier Inn, Blossom Street and at the Bar Convent both of which are just five minutes walk from the train station. (Please go to their websites for pricing and availability.) At this point, flexible rates (which allow for amendments/refunds) are extremely reasonable. As the event gets closer, accommodation will command a higher price if it’s available at all.

You can now book a place at the CAUK 2017 Forum here, and the CaverMorning.

Important survey

Please can we ask members and those interested in cavernoma to take this survey as
it may have an effect on how the wider neurological community are treated. The
deadline for your response is 30th September 2016. To access the survey, please
click here. Thank you.

Review of events in May and June

Prearing for their talks; Heather Band (Batten Disease Family Association) and Professor David C S White (Chair, CAUK Trustees) with support from Pat Spallone (CAUK member) and Ian Stuart (Co-ordinator)

Prearing for their talks; Heather Band (Batten Disease Family Association) and Professor David C S White (Cahir, CAUK Trustees) with support from Pat Spallone (CAUK member) and Ian Stuart (Co-ordinator)

David White participated in a workshop for Findacure in the City of London, Friday 24th June 2016. The workshop dealt with how support groups, like Cavernoma Alliance UK, can work with researchers. (Audio of the presentation ‘How Rare Disease Patient Groups Can Work With Researchers’ can be found at the end of this article.

David spoke for 20 minutes on ‘Setting a research agenda with the JLA Priority Setting Partnership’ which discussed how CAUK had developed the project. Tracing its evolution from the CAUK/Genetic Alliance UK sponsored 2012 booklet ‘Guidelines on the management of cerebral cavernous malformations in adults’, David and Ian’s first meeting with the JLA team at the University of Southampton, through to acquiring the £25,000 required (provided by grants from The Hospital Saturday Fund, the Lothian Health Board Endowment Fund, the National Hospital Development Foundation CAUK members, and the CAUK trustees match funding) to the complex process of soliciting, sifting and ranking priorities as an attempt was made to reduce the questions from 2268 to a “long list” of 27 for the Final Workshop. David’s results are available on our website here, or as a free hard copy (£2.00 P & P) from CAUK, Suites 4 & 5, Somerleigh Gate, Somerleigh Road, Dorchester, Dorset, DT1 1TL.
Finally, David took part in a panel discussion with Dr. Sara Ellis (from the Association of Medical Research Charities), Heather Band (Batten Disease Family Association) and Dr. Jutta Roth (Oxford Rare Disease Initiative) covering wide-ranging questions from the audience allowing David the opportunity to explain how CAUK operates CaverCentres, CaverHubs and CaverClinics with patients meeting clinicians that ordinarily they would not have access to.

CAUK Chair David White presents the Priority Settings Partnership Final Report on cavernoma to Hospital Saturday Fund

David White, Chair of CAUK, presents the Priority Setting Partnership Final Report on cavernoma to Zenaid Richards and John Greenwood at the AGM of the Hospital Saturday Fund, at the Guildhall in London. (12th May 2016)

A successful members’ fundraising campaign in 2014 allowed CAUK to administer a Priority Settings Partnership Project through the James Lind Alliance and in collaboration with medical specialists in cavernoma. This is to help determine treatment uncertainties in the field of cavernoma to enable research aims to be established. This will in future provide clearer guidelines in the management and treatment of cavernoma. The Final Report has now been completed and can be downloaded here. For further information on the PSP Project click here.

PSP Report Cover 1200

Click here to download a copy of the report

 

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