Irish Haemophilia Society E-zine: May 22, 2020

Irish Haemophilia Society eZine: May 22, 2020
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Update from I.H.S. CEO
May 22, 2020
Gene Therapy

As Gene Therapy clinical trials in haemophilia continue, there is every possibility that we will see the first licenced Gene Therapy for Haemophilia A this year and for Haemophilia B in 2021. There has been ongoing discussion in the community internationally about which centres should be delivering Gene Therapy. EHC and EAHAD have just issued a joint statement stating their view that Gene Therapy should be delivered by expert haemophilia comprehensive care centres delivering Gene Therapy as National hubs with additional support and monitoring by other haemophilia treatment centres. (The main part of their statement is reproduced below).

This strategy makes perfect sense. Licenced Gene Therapy will be an irreversible therapy which will require full informed consent and a lot of detailed engagement between the person with haemophilia getting the therapy and the team delivering the therapy. The centre will require specific facilities to deliver the therapy and there will be an agreed and detailed follow up process between the patient and the centre. If the chosen centres have experience with Gene Therapy clinical trials, that should be a distinct advantage. In addition, this will be an expensive therapy and I foresee health authorities wanting to ensure that this is delivered by expert centres. In Ireland, we now have experience with Gene Therapy clinical trials, with three people with Haemophilia B already having undergone Gene Therapy at the Clinical Trial Facility (CRF) in St. James hospital, working with the team at the National Coagulation Centre (NCC).

Additional Gene Therapy trials for haemophilia will take place in the future and the CRF are also delivering Gene Therapy in clinical trials for other conditions such as spinal muscular atrophy. Clearly, for a geographically small country such as Ireland, it makes sense to have all the haemophilia Gene Therapy (and perhaps all Gene Therapy) delivered by 1 expert facility such as the CRF. It is not difficult to envision Gene Therapy being delivered at the CRF with major visits taking place there and supporting visits (including regular blood tests required in the first month’s post vector infusion) taking place either at the CRF or at other haemophilia comprehensive care or treatment centres.

The national collaborative model for delivery of care in Ireland, where the 3 comprehensive care centres in Dublin and Cork and the 1 treatment centre in Galway work closely together should make this easy to agree and implement. This may prove more difficult in many larger countries where rivalry and perceived prestige linked to Gene Therapy programmes together with different payment models may make agreement harder to reach. The ultimate objective in each country must be to deliver Gene Therapy in the safest possible manner to the person with haemophilia, to monitor optimally and to achieve the best possible outcomes. This must be the guiding principle going forward.

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EAHAD-EHC Joint Statement on: Promoting hub-and-spoke model for the treatment of haemophilia and rare bleeding disorders using gene therapies

To ensure the safe introduction, use, monitoring and optimal learning regarding the delivery of gene therapies over time, EAHAD and EHC jointly call for all first-generation gene therapies to be managed using a hub-and-spoke model, as follows:
* Prescribed and managed exclusively by expert haemophilia comprehensive care centres (as the national hubs), and
* Monitored, by haemophilia treatment centres in close communication with the primary expert hub (as spokes linking into that hub).

These expert hubs should have previous experience with gene therapy trials and/or specialists who can ensure expertise in a timely manner in gene therapy research, education and monitoring, including laboratory monitoring. This will also maximise long-term benefits for gene therapy patients.

EAHAD and the EHC have previously recommended patients being treated with any novel therapies carry an identity card indicating information about their condition, treatment regimen, the haemophilia comprehensive care centre where their treatment is being overseen, and the name and telephone number of the contact person there, who can be reached 24 hours a day, 7 days a week. This should continue with gene therapies.

Any adverse events should be managed by both the expert ‘hubs’ as well as the treatment centre ‘spokes’ to provide the timeliest state-of-the-art treatment options to patients and maximise long-term benefits. All adverse events should be reported to a centralised reporting scheme and should include bleeding episodes.

It is the responsibility of all healthcare stakeholders to ensure the prescription, management and monitoring of gene therapies by expert hubs, at least initially, to ensure the optimal introduction, use and monitoring of these very different and complex new treatments.

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Telemedicine

Telemedicine is defined as the remote diagnosis and treatment of patients by means of telecommunications technology. Telemedicine as a concept is not new but the Covid- 19 pandemic has greatly accelerated and enhanced the practice of telemedicine. Haemophilia care in Ireland has been ahead of the curve for a long time in use of technology. We have had a haemophilia electronic patient record system since 2006 and a specific app for recording home use of coagulation factor concentrates since 2011. More recently, we have been implementing the Lighthouse project which aims to give the person with haemophilia control over their condition enabling them to make or change appointments electronically, interact with the comprehensive care team and receive or request specific information. Through a planned patient portal, each person will be able to access their medical records on their smartphone, tablet or computer. Haemophilia is one of 3 pilot projects funded by the HSE which aimed to optimise the
use of technology in different conditions. We were fortunate to have this project underway as the wider use of technology such as this will become more mainstream in healthcare in the future.

Prior to the Covid-19 pandemic, the NCC had initiated some clinic “visits” for people with mild haemophilia by telephone. This has now been widened to include all severities of bleeding disorders. People with severe haemophilia are typically asked to attend their centre twice per year for out- patient clinics. At present, these visits are being conducted by telephone or by video consultation from the NCC. Each person with a bleeding disorder who is being contacted have the choice of a telephone or video consultation with the consultant. We will also see more consultations with an advanced nurse practitioner. Telephone consultations are also taking place with the teams from the other comprehensive care centres at Crumlin and Cork and with the treatment centre in Galway.

Early indications are that people with bleeding disorders are quite satisfied with the options of a telephone or video consultation. The clinical issues can be discussed fully. It does not require the person to travel, find parking or wait to be seen. Of course, it cannot fully take the place of an actual visit to the centre. Blood tests can not be done remotely. For a first visit where a diagnosis of a bleeding disorder is required, this would have to be in person. When the first wave of the pandemic is abated, it would be sensible to survey the outcomes of and attitude to telephone or video consultations as a way forward. I can foresee a situation where, for a person with severe Haemophilia, von Willebrand’s or other severe bleeding disorder, an in- person clinic visit could be scheduled annually where blood tests could be performed as required. A second visit could be virtual using telemedicine. Information flow could be both ways and also include the GP using the information from the
patient portal. Innovation has also extended to other areas of comprehensive care.

The physiotherapy team at the NCC have been carrying out reviews and appointments by video. The dental team has consulted a significant number of people in emergency situations. It is not possible to do most dental work over a video consultation, but they have been able to see photos sent of the problem area in the mouth and talk people through inserting temporary fillings. Psychological and social work interventions should also be possible by phone or video consultation. We may come to see the accelerated expansion of telemedicine in haemophilia care as a silver lining of this pandemic.

Quality of Life Updates

Over the past 2 years, we have been periodically and anonymously the monitoring the quality of life of adults with severe haemophilia using a specific quality of life tool developed by patient experts- the Patient Reported Outcome Burden and Experience (PROBE) tool. We will be sending this out electronically to all members with haemophilia in the next week and we ask you to please fill in the survey and return to us.

Brian O’Mahony
Chief Executive

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Upcoming Webinars

Continuing with our series of Webinars and Q&A Sessions, we have further meetings coming up on;
Wednesday, May 27th @ 11am: Hemlibra Update for Parents with Dr Beatrice Nolan and CHI Crumlin Team

Tuesday, June 3rd @ 11am – Exercise and Physical Activity for Children with Paula Loughnane, Senior Physiotherapist at CHI Crumlin

Friday, June 5th @ 11am – an Update on the iPATH Research project with Professor James O'Donnell

Wednesday, June 10th @ 11am - Update on New Children’s Hospital with Eilish Hardiman, CEO

To join us for any of these meeting, please use the link & meeting ID below:

Join Zoom Meeting
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Meeting ID: 905 221 0070

If you plan to join, we would appreciate it if you could drop Barry an email at [email protected] (mailto:[email protected]) to give us an idea of numbers.

FYI: We shall be recording the audio and slides from some Webinars to make them available to those that cannot attend. Please be assured that only audio from speaker's presentations and not Q&As will be recorded. Attendees will not be identifiable on the recordings.

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EHC Webinars on Women and Bleeding Disorders

In light of current events, the European Haemophilia Consortium (EHC) had to cancel their 2020 Conference on Women and Bleeding Disorders, however, in its place, the EHC are organising two very interesting webinars on Women and Bleeding Disorders.
* The Management of Heavy Menstrual Bleeding; Thursday 28 May (12.00 noon to 13.30)
* The Place of Women in Haemophilia Comprehensive Care; Friday 29 May (9am to 10.30)

Registration for the webinars is free and you can do so via this link ([link removed]) .

There are more details about the sessions and speakers below and we hope that you will be able to join us in attending.

EHC Online Webinar on the Management of Heavy Menstrual Bleeding – Thursday 28 May (12.00 noon to 13.30)

This webinar will look at:
* The basics: ‘How do periods change from adolescence to menopause?’
* What does a normal menstrual cycle look like and how it is impacted by issues in coagulation?
* Case-studies on how to manage your periods in different situations including:
* Getting a teenager ready for her first period.
* Managing heavy menstrual bleeding – what are the options?
* Planning to get pregnant – how do you stop contraception and manage your periods.
* Reaching menopause – what to expect and how to manage it?

EHC Online Webinar on the Place of Women in Haemophilia Comprehensive Care – Friday 29 May (09.00 to 10.30 hrs)

This webinar will look at:
* What is comprehensive care and why it is important in rare bleeding disorders?
* What are some of the issues faced by women to access comprehensive care services? – Discussion between a clinician and patient.
* Case studies on initiatives that have worked and have helped to better include women in CCC?
* Strong WBD committee and cooperation with clinicians,
* EAHAD survey – Highlight some of the structural barriers,
* Joint clinics – how does it work?

Confirmed Speakers:

Rezan Abdul-Kadir, Consultant Gynaecologist, Royal Free Hospital, London, United Kingdom

Yannick Collé, Member, EHC Women and Bleeding Disorders Committee, France

Michelle Lavin, Clinical Lead for coagulation haematology research in Irish Centre for Vascular Biology at the Royal College of Surgeons in Ireland

Declan Noone, President, EHC, Ireland

Karin Van Galen, Consultant Haematologist, Van Kreveldkliniek, University Medical Centre Utrecht, The Netherlands

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Send Us Your Photos!

We've all been been finding ways to keep occupied (and keep younger ones entertained) while spending more time at home to help suppress the spread of Covid-19.

With that in mind, we would love to include a photo gallery of I.H.S. members surviving the 'lockdown' in our Summer Magazine

So, if you have snaps of you and yours gardening, tackling DIY, BBQs, online quizzes, home schooling, home workouts or just chilling - please, send them on to barry@haemophilia (mailto:) .

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Physiotherapy Update: NCC, St James's Hospital

The physiotherapy team in the National Coagulation Centre (NCC) in St James's Hospital, is doing a survey of people with severe haemophilia that attend the National Coagluation Centre.

They are looking for feedback on their annual physiotherapy and Haemophilia Joint Health Score (HJHS) assessment service for people with severe haemophilia. They hope to use the information to develop services to better fit the needs and demands of their service users.

The survey will be sent out this week and the team would really value your contribution. We ask all members who received this survey to please complete and return the survey.

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Barretstown Summer Camps

As they are unable to run their traditional summer camps this year, Barretstown have developed a new interactive programme which will bring the magic of camp straight to the camper’s home through our NEW private online streaming service, Barretstown Live.

They have built a Studio in Barretstown from where the team will be live streaming interactive activities and fun.

For more information on Barretstown LIVE, please contact [email protected] (mailto:[email protected])

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Annual Report 2019

Our Annual Report 2019 is available now with overviews on our activities, events, support and finance, and more from last year.

A hard copy will be be printed and mailed to member's in the future but for now, it's digital only.

To read or download, pleaseclick here ([link removed]) .

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Hyde Square Apartments

Just to remind you all, that the Hyde Square apartments will continue to be available for members and bookings will be taken as normal.


▪ People with haemophilia or related bleeding disorders from outside of Dublin, when attending St. James’s Hospital or Our Lady’s Children’s Hospital, Crumlin for treatment, for a hospital appointment or for a review clinic.

▪ An immediate family member, a spouse, a partner and/or child of the person with haemophilia or related bleeding disorder from outside Dublin, when attending St. James’s Hospital or Our Lady’s Children’s Hospital, Crumlin for treatment, for a hospital appointment or for a review clinic, or while a family member is an in-patient.

To make a booking, please contact Julia on 01 657 9900*.

For more info, please click here ([link removed]) .

* A nominal fee of €10.00 per booking, per night will be levied to offset the cost of cleaning and routine maintenance.



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Events Update

Hardly a surprise give the current situation we find ourselves in but so far we've had to make some changes to our events:

May

Women & Bleeding Disorders Info Day

Date: Saturday, May 9th
Venue: Hodson Bay Hotel, Athlone, Co. Westmeath

FYI: This event has been postponed and will be held in 2021 instead.

June

Mother & Daughter(s) Overnight

Date: Saturday June 20th & Sunday, June 21st
Venue: Lilliput Adventure Centre

This event has unfortunately been cancelled.

July

Father & Son(s) Overnight

Dates: Saturday July 4th & Sunday, July 5th
Venue: Lilliput Adventure Centre

FYI: This event has unfortunately been cancelled.

Mild Haemophilia & Haemophilia B Information Day

Date: Saturday 18th July

This event will now take place online, via Zoom.

August

Family Day Out

Date: Monday, August 10th
Venue: Dublin Zoo

FYI: This event has unfortunately been cancelled.

Von Willebrand’s Information Day

Date: Saturday, August 29th
Venue: Castleknock Hotel, Dublin

It is currently planned to proceed with this event

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Strategic Plan 2020 - 2024

Following much work and consultation, the board and staff of the I.H.S. completed a new Strategic Plan to run from 2020 to 2024.

This new plan will guide our direction and activities for the next number of years as we continue to provide the optimum information, services and support to people living with haemophilia, von Willebrand’s and other inherited bleeding disorders, and their families.

You can read / download the Strategic Plan here ([link removed]) .

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