The PReS SCLERODERMA Working Group
UP DATE FROM THE CHAIRMAN
At our meeting in June we had an outstanding programme on present and future scleroderma research projects. We started the programme with an informative overview of achievements reached by PReS juvenile scleroderma working group (JSWG) in the past year. The friendship, commitment and support provided by the members of JSWG reassure me that our future research projects will deliver innovative thoughts and actions in this challenging field of paediatric rheumatology. I want to thank all the participants of the programme for making it a great success for our JSWG.
In this section of the newsletter we will provide a brief summary of a selection of our current research studies.
Looking for minimum standards in care for children with localized scleroderma - Result of the consensus meeting regarding diagnosis, assessment and treatment of the disease.
Hamburg, Germany on the 11th of December 2011
The meeting was proposed to the JSWG by I Foeldvari at the annual PReS conference in Bruges in 2011 and it was advertised by PReS. Members of the PRES scleroderma working group were invited to participate. Two paediatric dermatologists were invited to reflect the multidisciplinary care for these children. P. Clements was invited to moderate the meeting. A nominal group technique was used. 75% consensus was defined as agreement.
Although there are no solid guidelines currently present as the ‘standard of care’ for jLs, these suggestions are part of routine care for most physicians specializing in the care of paediatric localized scleroderma and are non-invasive measures. Using them as general ‘guidelines’ prospectively in a large cumulative jLs cohort will help determine which are more relevant than others to the management of these patients.
Topical treatment of small superficial circumscribed morphea lesions and systemic treatment with MTX +/- corticosteroids for all other subtypes of jLS was unanimously agreed upon. The duration of treatment and alternative therapies require more investigation.
T Avcin welcomed the project and proposed further continuation and extension of the project. As the second step, all diagnostic and treatment topics and Hamburg agreements will be circulated to all members of our WG.
I Foeldvari drew attention to legal and more importantly to financial requirements of such an evaluation. All members can seek funding to cover the expenses arising out of developing jLS standards. If anyone raising funds on our behalf needs advice, please contact us and we will do our best to help and coordinate the effort.
Immunosuppressive treatment for juvenile Systemic Scleroderma and juvenile Systemic Scleroderma Overlaps for Stem Cell reinfusion: a retrospective study from the EBMT ADWP and PDWP in collaboration with the PRES
Aim of the project: To describe the long term clinical outcome following autologous haematopoietic stem cell transplantation in children and adolescents transplanted before the age of 21 years, for juvenile systemic scleroderma, and juvenile systemic scleroderma overlaps.
- Patient survival
- Transplant Related Mortality
- Progression-free survival for systemic sclerosis, defined as survival in the absence of relapse or progression.
Question to the JSWG regarding this project: Could we create a control group of severe jSSc patients, who were treated at least with a two consecutive DMARD or a combination, and who did not went into remission?
If you are interested in the study please feel free to contact I Foeldvari.
To decipher the optimal management of systemic sclerosis
Type of funding scheme: Collaborative project (small-scale focussed research project)
Work programme topic addressed: HEALTH.2012.2.4.4.2 - Observational trials in rare diseases.
Coordinator: Ulf Müller-Ladner, MD, Professor for Internal Medicine and Rheumatology, Justus-Liebig University Giessen
Duration of the project: 36 months
DeSScipher aims at improving clinical practice in the management of the rare autoimmune rheumatic disease systemic sclerosis (SSc) for which no orphan drug but only off-label treatments are available.
Over a three-year period, building upon the expertise of a multidisciplinary, experienced consortium combining clinicians, physicians, biostatisticians, biologists and chemists, DeSScipher will provide a systemic, integrated approach to SSc.
This approach will be based on observational trials, which will address different but crucial dimensions of this systemic disease:
- early detection/prevention and treatment,
- functional impairment and severe complications,
- affection of different organ systems (skin, joints, lungs and heart)
- juvenile and adult patients.
The Juvenile Scleroderma International (JUSI) Database
Since 2002 the Juvenile Scleroderma Working Group (JSWG) of the Paediatric Rheumatology European Society (PReS) has created an international database including more than 1000 children with different varieties of Scleroderma. At present, the database has been transformed into a web-based instrument for the prospective collection of clinical data and biological samples. Aim of this Database is to provide a well-characterized cohort of scleroderma patients, according to the current classification criteria, and collect adequate information enabling to uniform clinical assessment and diagnostic tests, to stimulate clinical and basic research projects, to gather sufficient numbers of patients for clinical trials. The executive committee of the JSWG of PReS established the minimal set of clinical and laboratory data of scleroderma patients to be included in the database, the regulatory issues related to the ethical aspects and to the access modality to the data by investigators.
The JUSI database has an Administrative structure including a Database Executive Committee, responsible for data collection, bio statistical support and evaluation of the projects progress and an International Advisory Committee in charge to evaluate proposed projects that require access to the database data. This is an associated project of a working group member.
F Zulian offered open positions in the International Advisory Committee to JSWG members.
F Zulian invited O Kasapçopur, I Foeldvari expressed his interest. F Zulian will decide who will finally participate on the advisory board.
Members of JSWG agreed to launch the following projects:
- Prospective study of patients with presentation of Raynaud and long term follow up- Characterization of this patients ( Paediatric Vedos project)
- Uveitis in loc Sc patients – cross sectional study of currently followed patients
- TMJ involvement of loc Sc patients- cross sectional study of currently followed patients
- Developing consensus on follow up of children with Raynaud phenomenon ((planned for the 09.12.12 in Hamburg on Sunday after the course)
- Developing a consensus for treatment of systemic sclerosis (planned for the 09.12.12 in Hamburg on Sunday after the course)
Study documentation for uveitis and TMJ involvement in paediatric localized scleroderma will be available soon.
Save the date! Our forthcoming traditional annual meeting will be held in Hamburg on 09th of December 2012.
If you are interested to participate on the consensus conference , please send an email to Tamas Constantin or Ivan Foeldvari
I Foeldvari is willing to extend his term as chairman. T Avcin passed secretary responsibilities to T Constantin. Executive Committee (M Zak, J Anton, F Zulian) was expanded by opening 2 new positions (T Avcin and Severine Guillaume Czitrom). All positions were affirmed by members of JSWG.
I Foeldvari announced that Kim Fligelstone-Kimflig, as the representative of FESCA, will be a member of our JSWG.
Capillaroscopy for Paediatric Rheumatologists,
Fifth Hamburg Symposium on juvenile Scleroderma - update on new developments
- Date: December 08, 2012, 10.00-18.30
- Location: Schön Klinik Eilbek, Haus 7, Dehnhaide 120, 22081 Hamburg, Germany
- Attendance at the meeting is free of charge.
For further information please visit PReS website.
The EULAR/EUSTAR 2013 Scleroderma Course
The course is dedicated to a limited number of students, young rheumatologists interested in scleroderma, mainly from EUSTAR centres.
Please follow information on EUSTAR webpage (www.eustar.org), where registration will be open.
DATES FOR YOUR DIARY
Developing a consensus for treatment of systemic sclerosis (planned for the 09 December 2012 in Hamburg on Sunday after the capillaroscopy course)
PReS annual meeting in 2013 will be held at Ljubljana, Slovenia (26-30 September 2013) T Avcin announced that scleroderma will be one of the main topics of the meeting with at least 2 oral presentations on localized and systemic scleroderma.
If you have an idea, issue, or question related to the working group, let us hear from you. Please send your articles, messages or announcements for the next issue to Tamas Constantin (e-mail: firstname.lastname@example.org)
|Members of executive committee:||
Severine Guillaume Czitrom