Newsletter n°2
The European Working Group on Gaucher Disease
EWGGD Virtual Symposium 25 May 2020
As I hope you are aware due to the postponement of the EWGGD face to face meeting to October we are inviting to you a full day virtual meeting on 25th May 2020
The meeting will run during the day to cover various time zones. Once we have your registration we will send you a meeting link. We would be pleased to receive further abstracts for presentation and will try to coordinate this with your time zones.
Our theme is ‘Gaucher Disease: Setting the agenda for the next decade’ and abstract categories are pathophysiology, clinical phenotype and therapy.
For those of you who have already sent abstracts and received acknowledgement there is no need to resubmit.
Revised abstract deadline : 15th May 2020
Deadline for slides : 22nd May 2020
Please follow the link for registration and abstract submission
The aim of the EWGGD is to promote clinical and basic research into Gaucher disease for the ultimate purpose of improving the lives of patients with this disease; it brings together clinicians, scientists and patients in an open forum for discussion on all aspects of the condition.
The opportunity for presenting unpublished scientific data as well as free discussion is a central premise of the Group. For the dissemination of information, a two yearly workshop is organized. Although a limited number of representatives from industrial partners are invited to attend the meetings, the Working Group has always been an independent organization with a charitable purpose.
Notes from the EWGGD webinar on 1 April on Gaucher
disease and Covid-19
Self-isolation:
Follow Ministry of Health guidelines for individual countries
Gaucher patients with co-morbidities and chronic illnesses have been advised to self-isolate
Patients with underlying lung disease or splenectomy or other high-risk factors and our GD 3 community have been advised to self-isolate completely (UK:12 weeks).
Pregnant Gaucher patients being told to self-isolate.
Gaucher treatment:
General consensus was that it was ok for patients to miss some infusions rather than visit hospitals for treatment where there was a higher risk of covid-19
In some countries home therapy has been given to those patients who usually went to hospital to protect them. In other regions, implementation of home therapy not immediately recommended because of geographic difficulties. The EWGGD believes home treatment should be strongly considered during this crisis
If a patient is on Cerdelga then they should inform clinicians before starting any new therapies to check for drug interactions. However, the safest approach is to stop Cerdelga for a short while that other treatments are necessary for covid-19 Hydroxychloroquine interferes with Eliglustat so should not be taken together. One suggestion is to use MedScape’s drug interaction checker
No problems are reported with supply chain if enzyme or Cerdelga.
Physicians should report any safety issues for their databases/registries
Covid-19 disease course:
In general, admitted patients requiring ventilation have markers associated with macrophage activation.
So far, no reports of Gaucher patients with severe manifestations of covid-19
Data collection:
The EWGGD board suggest capturing data relating to covid-19 in Gaucher patients in a standard way with the aim to answer two important question- are GD patients more susceptible to infection or complications?
Data will be collected locally according to local regulations but should not be shared unless there is ethical approval
Gaucher related data
Last lab before COVID 19
COVID 19 related data
Lab during COVID 19
Situation of these patients at end of crisis: deceased, effects of loss o treatment, etc.
Please find attached and above the newsletter the very brief survey in the link and the excel spread sheet for more information attached:
IGA: aims is reach out to global community to let them know that we’re here and that we are communicating with Health Care Providers who know and
Scientific Working Groups
To further develop the aims of the EWGGD we propose to develop a number of scientific working groups who will meet on a regular basis between the scientific symposia.
The objectives of the working groups will be to address issues of importance to our Gaucher community and formulate consensus statements, guidelines or implement research studies to investigate the relevant question.
Our first working groups will be
1) Biomarkers and Materials addressing issues relating to laboratory diagnostics (see below)
2) Clinical guidelines working group
Biomarkers and Materials Working Group
The EWGGD will organize working groups, including a “biomarkers and materials” working group.
The heterogeneity of assay techniques and biomarker results for Gaucher disease makes it difficult to form international cohorts and hinders their exploitation. This is one of the reasons why these biomarkers are little or not involved in prognostic scores. It therefore seems logical that a process of homogenization of practices for the determination of these biomarkers should be implemented and the EWGGD network is an appropriate organization for this objective. Moreover, this is indeed a specific mission of an academic group such as the EWGGD.
With Professor Hans Aerts, we propose, to coordinate a working group whose objectives are 1) to carry out a descriptive analysis of practices in 10-15 European countries, 2) to set up a strategy to standardise techniques and results, 3) to carry out comparative analyses after exchanges of biological samples (a link will be established with the biobanks concerned), 4) to consider the constitution of well characterised samples, in conjunction with clinical managers and national registers in order to be able to propose a research project aimed at screening new molecules that could play the role of biomarkers.
A process is underway with a representative per country to form a first group of 10 to 15 people who will develop the first steps of these objectives. A report of the group will be made at each EWGGD plenary meeting, as will be the case for all working groups, a procedure validated by the EWGGD board.
Please join us on 25th May to hear more or contact any member of the EWGGD board for further information.
Chairman
Derralynn Hughes, London, UK
Vice Chairman
Marc Berger, Clermont-Ferrand, France
Executive Committee
Dardis Andrea, Udine, Italy
Maciej Machaczka, Stockholm, Sweden
Shoshana Revel-Vilk, Jerusalem, Israel (Secretary)
President founder
Hans Aerts, Leiden, The Netherlands
Treasurer
Carla Hollak, Amsterdam, The Netherlands
IGA representative
Jeremy Manuel O.B.E., London, UK
