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European Myasthenia Gravis Network
Description
Action
Improving information and knowledge for the development of public health.
Area of activity
Developing strategies and mechanisms for preventing, exchanging information on and responding to non-communicable disease threats,including gender specific health threats and rare diseases.
Summary
Myasthenia Gravis (MG) is a rare and heterogeneous neuromuscular disease leading to abnormal fatigability of various muscles, with a prevalence of about 100 per million and an incidence which appears to be increasing. The cause of MG could be genetic or aquired. The genetic forms incriminate several types of mutations in the acetylcholine receptor or associated proteins. Acquired MG has an autoimmune etiology; it is due to auto-antibodies to acetylcholine receptor found in 85% of patients, or other muscle endplate proteins such as MuSK. MG could appear in young adults, essentially females, while the late onset form is distributed equally in males and females. Treatment of autoimmune MG disease is symptomatic, with the use of cholinesterase inhibitors, and empiric, with the use of immunosuppressors, broad anti-immune therapy such as plasma exchange and intravenous immunoglobulins, and thymectomy for selected patients. Clinical classification of MG differentiates between purely ocular and generalised forms, but also between late and early onset forms. To this date, it is not possible to know if these forms are different entities or whether they are a part of a continuum. There are no differential diagnostic tests, the clinical features and evolution are very similar, and currently the same empiric therapies are used for all the groups.
A network concerning acquired
MG has been created several years ago
and was supported by the AFM (French
association against myopathies). This
project gave the basis of a
structured coordinated network on MG
including several scientific
investigators and clinicians. Because
this network has minimal budget, its
activities were limited to exchanges
between the experts in this field and
an annual meeting. However, the
development of this network has
established the necessary background
for developing a sustainable
coordination in the area of health
information, collection of
epidemio-logical data, exchange of
data and information within and
between Member States. EuroMyasthenia
project will cover essentially
acquired MG, although hereditary MG
patients could be integrated in a
further step.
Several activities will be
developed according to the main
objectives:
- Health Information.
Referral laboratories will be
established and experts will provide
professional assistance and
information throughout the European
Community. A leaflet will be prepared
for patients about their disease. An
officially endorsed Website and a
forum will be provided. It will be
open to all citizens but access to
database will be restricted for
professionals. An electronic
newsletter will be sent to all
members every two months.
- European database.
EuroMyasthenia will result in the
creation of a European database that
will collect data from a large part
of Europe and thus permit
epidemiological studies, taking into
account genetic, immunological,
histological, clinical and hormonal
aspects. Health indicators
recommended by the ECHI-2 network and
relevant to MG will be also included.
At least 5000 patients from the
European countries participating to
this network will be included during
the implementation of this project.
- Epidemiological data
collection. Epidemiological studies
will be performed to monitor
occurrence of the different forms of
MG for individual countries and to
identify new markers associated with
disease pathogenic mechanisms. In
turn, a list of recommended
indicators will be established for
the surveillance of Myasthenia Gravis
in the member states and accession
countries.
- Standardization and Quality
Assessment. European Guidelines for
diagnosis and clinical management of
MG will be prepared. A quality
control system for laboratory
diagnosis of the different forms of
MG will be established. By
educational and training visits in
the expert centers, scientific and
medical personnel will improve
healthcare quality. In addition, the
promotion of information between the
different research groups will
contribute to improve the knowledge
of the immunologic and genetic
pathological mechanisms of MG.
The long-term objectives of EuroMyasthenia are to
- define accurately subgroups of MG patients,
- optimise the therapeutic strategies,
- propose new therapeutic protocols according to the clinical subgroup and
- improve the diagnosis of MG patients.
To reach these objectives, Euromyasthenia brings together 25 groups who have made substantial contributions to the understanding and the management of Myasthenia Gravis and 6 organisations of patients. It includes 10 groups of researchers and 15 groups of clinicians with complementary expertise in neurology, immunology, genetics, pathology, biochemical, hormonal and clinical fields. The originality of this project is to combine
- scientific experts who will improve the knowledge,
- clinicians who will promote the databases and the epidemiological studies and
- organisations of patients who will directly be informed on the progress of the project.
Finally such coordinated action on a European level will provide a solid evidence base for healthcare management, and will enhance the efficacy of healthcare systems all over Europe by exchanges with members from regulatory authorities from the represented countries.
More info...
Financing
Leader
organisation
Université de Paris – Sud XI
(UPS)
Public body
15 rue Georges Clémenceau
91405 ORSAY CEDEX
FRANCE
Tel: +33 1 69 15 74 06
Fax: +33 69 15 61 03
Web site: www.u-psud.fr
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Contact Person
Dr Sonia BERRIH-AKNIN
Université Paris-Sud
CNRS UMR 8162
Centre Chirurgical Marie
Lannelongue
133 avenue de la Résistance
92350 Le Plessis-Robinson
FRANCE
Tel: +33 1 45 37 15 51
Fax: +33 1 40 94 67 54
E-mail: sonia.berrih@ccml.u-psud.fr
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Associated
beneficiaries
- Bayerische
Julius-Maximilians-Universität
Würzburg (UW) - established in
Würzburg, Germany
- Hellenic Pasteur Institute
(HPI) - established in Athens,
Greece
- Universitet I Bergen Sedd
(UB) - established in Bergen,
Norway
- Karolinska
Universitetssjukhuset (KU) –
established in Stockholm, Sweden
- Universiteit Maastricht
(UM) – established in Maastricht,
The Netherlands
- Ulleval
Universitetssykehus (UU) –
established in Oslo, Norway
- Istituto Nazionale
Neurologico “Carlo Besta” (NNI) –
established in Milan, Italy
- MDA-Hellas (MDA) –
established in Athens,
Greece
- The Chancellor, Masters and Scholars of the
University of Oxford (The University of Oxford) - established in Oxford, United Kingdom
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Starting
date and duration of
project
- 01/01/2006
- 36 months
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Total
cost
1.302.708,36 €
Subsidy from
the Commission
770.400,00 €
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Outcomes
Results to be achieved
Work package 1: Coordination
of the project
This workpackage is linked
to all objectives:
- To promote the
development of the Internet site
and the database (objectives 3
and 5)
- To ensure that
deliverables and milestones are
obtained in time and that the
studies are performed in
conformity with the National and
European Regulations (all
objectives)
- To check the
justification of the budget in
relation to the technical tasks
(all objectives)
- To organize the network
meetings (all objectives)
- To set up an internal
evaluation committee and an
external evaluation and advisory
board to evaluate the progress of
the network (all objectives)
Work package 2:
Dissemination of the results
This workpackage is linked
to the objectives 1, 2, 3, 4 and
5:
- To standardize
biological, clinical and
histological criteria all over
the EU countries
- To promote a survey
among the associations of MG
patients to determine the
influence of psychological and
socio-economical determinants in
the onset or aggravation of the
disease
- To develop a database
at a European scale including
biological and clinical
parameters
- To promote the
establishment of a specific
European Card for MG patients
- To disseminate data via
a web-site connected to the EU
health portal
Work package 3: Evaluation of
the project
External Evaluation and
Advisory Board will consist of
eminent scientists, clinicians
and association representatives.
In addition, representatives from
relevant clinical and scientific
societies (e.g. International
Society for Neuroimmunology, MGA,
AIM) will be asked to
participate. They will contribute
by evaluating the progress of the
network and will be consulted
periodically for major directions
of the project. Individuals we
are considering to invite
include: J. Newsom-Davis, H.
Wekerle, D. Drachman, A.
Lanzavecchia, J.F. Bach.
Questionnaires will be
sent to 500 myasthenic patients
from all participant countries.
They will be asked about their
current status and their
expectations from the present
Myasthenia network. Another
questionnaire will be sent to 100
neurologists with special
interest on MG. They will be
asked for the difficulties and
problems while working with
myasthenic patients. After the
first 18 months of the network
and at the end of the project,
new questionnaires will be
prepared, asking the same people
(patients or clinicians) to
evaluate the results of our
network. Their responses will be
analyzed and evaluated by the
external and internal Committees.
Work package 4: Database
The aim of WP4 is the
realization of a European
Database on MG
(EuroMyasthenia-DB) that collects
clinical and biological
information to be shared among
scientists and clinicians. The
EuroMyasthenia-DB will allow the
interaction of the European
national registries and hospital
databases containing
country-specific epidemiological,
clinical and biological data.
EuroMyasthenia-DB will be a
crucial tool for the
establishment of a European
cohort of MG patients to be used
for a comprehensive understanding
of the disease natural history,
Public Health evaluation,
cost-care effectiveness and
treatment innovation.
This workpackage is linked
to objective 3
Work package 5: Laboratory
and clinical standardisation
This workpackage will be
the driving force for the
objective 1, that is to
standardise biological, clinical
and histological criteria all
over the EU countries and will
contribute data to objective 3
that is to develop a database on
a European scale. The work done
in this workpackage will be
crucial for the improved
diagnosis and clinical assessment
of patients in those countries in
which MG does not have strong
research or laboratory links.
Work package 6:
Psycho-socioeconomical
determinants
To identify psychological
and socio-economical determinants
that could influence the
development of MG disease and the
clinical severity, a
questionnaire will be established
and will be sent to 3000 patients
through the associations of
patients.
This workpackage is linked
to objectives 2 and 3.
Work package 7:
Epidemiological analysis
This workpackage is linked
to objective 3: develop a
database at a European scale to
serve as a basis of
epidemiological studies
Interim report
 (4 MB)
More info
Statement of project aim(s) and objectives
General objectives
Myasthenia gravis (MG) is
a disabling chronic neuromuscular
disease affecting mostly females.
There is a need for combining the
efforts to improve the knowledge
on this disease for the following
reasons:
- MG is a rare disease
(prevalence about 10/100 000);
- it is heterogeneous;
- it could be
life-threatening, when the
respiratory muscles are affected;
- the diagnosis is not
always easy to make;
- the clinical criterion
and therapeutic approaches are
different among the European
countries;
- there are aspects in
the pathogenesis of the disease
that are not yet clear;
- there is no cure for
this disease;
- many of the drugs used
for the treatment of MG have
severe side-effects
- some pharmaceutical
molecules, such as anaesthesia
agents are life-threatening for
the patients.
Our main objectives are:
- to improve knowledge
and information on the different
forms of MG
- to improve
standardisation of data
collection
- to promote education
and training, namely for the
integration of new member states
- to identify important
health indicators for MG
- to collect data and
perform epidemiological studies
at European level in order to
improve the classification of MG
disease' subtypes.
The strategy of
EuroMyasthenia is to bring
together 31 groups from 14
different countries, including 3
major categories:
- scientists
who have made substantial
contributions to the
understanding of MG;
- clinicians experienced with the
management of MG patients;
- associations of MG patients.
The members of the network will join
forces to construct a database
including some indicators of
health recommended by the ECHI-2
network as well as clinical,
biological and
psycho-socioeconomical factors
collected by the members of the
network. Further analyses of this
database will enable to achieve
main objectives of the EU call
and promote a better
classification and treatment of
MG patients.
A better knowledge of the
disease and more detailed
information for the general
practioners and neurologists
should avoid erroneous diagnosis,
and improve treatment and health.
In the long term, such
coordinated action on a European
level will provide a solid
evidence base for healthcare
management, and will enhance the
efficacy of healthcare systems
all over Europe.
Specific objectives
Improving exchange of
information and promoting a
better classification is a
priority for Europe (2.1.4 in
Work Plan 2005). EuroMyasthenia
aims to set-up a Europe-wide
Myasthenia Gravis (MG) network to
improve information and knowledge
for MG, promote a better
classification, optimize
therapeutic strategies and
support actions to reduce
inequalities in MG care.
The specific objectives of
EuroMyasthenia are to:
- standardize biological,
clinical and histological
criteria all over the EU
countries
- promote surveys among
the organisations of MG patients
to determine the influence of
psychological and
socio-economical determinants in
the onset and/or aggravation of
the disease
- develop a database at a
European scale to serve as a
basis for epidemiological studies
- promote the
establishment of a specific
European Card for MG patients
- disseminate data via a
web-site connected to the EU
health portal
These objectives will be
achieved by
- Building on
complementary experience of 10
scientists expert in the field of
Myasthenia Gravis (Objectives 1,
3, 5), of 15 teams of clinicians
(objectives 1, 2, 3, 4, 5) and of
6 organisations of patients
(objectives 2, 4, 5).
- Reinforcing the exchange
of information via an Internet
Website, by exchanges of
laboratory, medical and
paramedical personnel, by the
production of a regular
newsletter and organisation of
regular network meetings and an
international conference.
- Supporting close
collaboration with other networks
supported by the EU on rare
diseases (Orphanet and Eurordis).
- Establishing
questionnaires to be sent to MG
patients and neurologists among
the associated partners and
collaborators of the project, but
also among new European countries
that could be integrated to the
project during the
implementation.
- Producing a European set
of Guidelines for Myasthenia
Gravis diseases. This will be
possible by the participation of
members from regulatory
authorities from the represented
countries.
Methods
The creation of an
internet site available to the
European health portal will be
based on previous European
experience in Public Heath
networks (WP1 and WP2). A part of
this site will be open to all
citizens. It will include all
information reporting the
progress of the project and a
forum.
The
psycho-socio-economical
determinants will be analysed
through a questionnaire prepared
in several languages and sent to
MG patients through the
organisations of patients (WP6).
Finally the epidemiological
analysis will be implemented with
the help of technical competences
of several collaborators (WP7).
The European database will
be based on national databases,
and will include Standardized
clinical, biological and genetic
data from MG patients (WP4).
Indicators recommended by the
ECHI-2 network will be
introduced. Collaborating with
other European projects, in
particular Orphanet and Eurordis
will promote synergy and avoid
duplication.
To standardise the
clinical and biological
parameters, reference reagents
will be distributed to the
centers performing the tests and
personnel will be educated in
expert centers (WP5).
Methodological problems will be
discussed during the meetings.
Evaluating the progress of
the project will be done through
the external Evaluation and
Advisory Board (WP3). The
preparation of the various
Guidelines and leaflets will be
discussed during the meetings.
All reports and communication
documents will be under the
supervision of the internal
evaluation committee.
This project brings
together multiple competences and
will enrol several personnel to
fit the tasks described in the
workpackages. However for
specific technical tasks such as
the creation of a professional
website or database, the internal
evaluation committee could decide
whether subcontracting with SMEs
is required.
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