We are happy to share with you the latest newsletter from FESCA.
You can for instance read some inspiring stories from our members and how they spread awareness of scleroderma during the awareness month towards World Scleroderma Day 2019.
The juvenile scleroderma inception cohort project (www.juvenile-scleroderma.com) collects data from juvenile systemic scleroderma (jSSc) patients prospectively with a standardised assessment of the organ systems. We included now over 100 children with jSSc in this cohort. This is the largest prospective cohort of the world. jSSc is an orphan disease, with a prevalence of 3 per 1 000 000 children. Each recruited patient is important for us.
Please tell your treating physician to participate in this cohort.
The juvenile scleroderma inceptions project has the aim to learn more about jSSc; the evolvement of the organ involvement, about the prognosis of the disease and about the factors influencing the prognosis. In this project the responsivness of the outcome measures are assessed, if they are sensible enough to find occurring changes over time. This is a very important issue, as early recognition of an organ involvement helps to guide an intensification of the therapy. As in other autoimmune diseases, there is a feeling, that early intervention, leads to prevention of damage of the organ systems and significantly improves survival. There are currently no controlled studies, which explore the effectivity of certain medications in jSSc, but based on the inception cohort data, clinical trials for licencing medication for jSSc are on the way.
The validation of the paediatric outcome measures for jSSc , which is one an other aim of the project will enable us to conduct prospective therapeutic trials for our paediatric patients more sufficiently.
An other important question is for the paediatric patients and their parents, what is the long term prognosis of patients with jSSc. Currently we have only indirect data from adult patient cohorts, where juvenile onset patients are incorporated. During the longitunidal follow up, we will learn more about the long term prognosis too.
With your help, with each included patient, we will learn more about jSSc and jSSc will be a much more controllable disease in the near future.
We are very happy to show you the video made via Kill the Film, who we have worked with before.
This year we are again focusing on Scleroderma will not take my smile, and this year we are happy to have a whole world of support.
Read more of the campaign for this year at www.worldsclerodermaday.org, and remember to post pictures with you and friends, smiling away, on social medias. Remember to use the #ScleroSmile. We DO love your smile – and your world of support.
You really liked our campaign from last year: Scleroderma will not take my smile. So why not do the campaign another year, but still a bit different than last year.
No matter which platform you use: Again we will invite you to join us for World Scleroderma Day on social medias by using #ScleroSmile.
We will have a stand at the EULAR 2018 congress in Amsterdam. If you are at EULAR, drop by and say hello.
On the 16th February 2018, I found myself on the steps of the entrance of the Bordeaux Congress Centre. Set in the Quartier du Lac, or the Lake District, the modern venue spans an impressive 12,000 square metres, contains a range of exhibition and meeting rooms as well as several large amphitheatres, and over the 15-17 February 2018 was the home of the 5th Systemic Sclerosis World Congress.
The congress was an opportunity for doctors, healthcare professionals and patients to gather, share and present new medical findings, research and results. Alongside the question and answer panels, patient talks and presentations in the large amphitheatres, there were also smaller workshops and focus groups taking place in the seminar rooms upstairs. There was also the chance for patients to participate in physical classes such as posture and breathing exercises and yoga.
Whilst I had previous knowledge of systemic sclerosis, also known as scleroderma, and have attended several fundraising events in aid of those affected by the condition in the past, I was keen to learn more about the disease. I attended the congress to support a member of my family who was invited to speak as a patient representative at the event.
On arrival we were warmly welcomed into the brightly lit and spaciously designed building and received our patient passes in order to gain access to the event. The lobby of the Congress Centre was abuzz with friendly chatter and conversation as there were several stalls and information stands set up to provide a space for introductions and networking. There were also information leaflets and samples of products created to help those with systemic sclerosis on offer.
After browsing the main lobby area, we attended a SPIN focus group with the aim of identifying and facilitating barriers to physical activity in scleroderma. It was a small group session lasting around an hour with patients from all over the world in attendance. The seminar revolved around conducting specialised research in aid of creating new solutions for patient problems. It was great to be able to see that genuine patient concerns were being addressed directly and personally. The focus group allowed patients to share their own experiences and offer support and advice to each other.
During the rest of my time at the 5th Systemic Sclerosis World Congress, I attended several other talks by healthcare professionals discussing the treatment of pain, regarding nutrition for those with scleroderma and also a talk by a professor sharing research on kidney failure. These talks were available for translation into French and Spanish and were followed by the option for audience members to ask questions. The presentations were well planned and ran smoothly, allowing for listeners to come in and choose which talks they wished to listen to, or to exit and attend another seminar or activity.
The congress was a brilliant example of how important sharing information and experiences can benefit others, as well as being a big advancement in improving the priorities of systemic sclerosis research. Over the few days that I attended, there were abundant opportunities for patients and doctors to interact and lots of discussion of medical research carried out to help improve patient care. The smaller group sessions and larger medical talks provided a wealth of support and advice from doctors in order to improve the quality of life for scleroderma patients.
The 5th Systemic Sclerosis World Congress was a huge success with doctors and patients attending from all around the world. Over 1,200 people attended from 56 countries, including France, Belgium, China, UK, USA and Canada. The organisation behind the hugely successful event was FESCA, the Federation of European Scleroderma Associations. FESCA managed everything in association with the patient congress, for example, organising the programme, inviting the speakers and helping to arrange their accommodation, accommodating patients and doctors from other European countries by providing translation services and also supplying the event with refreshments.
The patient congress was brought to a jovial end by an Australian duo, a systemic sclerosis patient and her husband, providing musical entertainment to bring closure to the proceedings. The unexpected singalong created a cheerful atmosphere and worked to unite the crowd in song. Whilst I do not have systemic sclerosis, I found the congress very interesting and am grateful for the opportunity to be able to learn more about the disease. It was an enlightening experience and I look forward to the next World Congress in 2020, which will take place in Prague.
– a niece to 1 of the patient participants
FESCA is very happy to share the news, that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to nintedanib for the treatment of systemic sclerosis with associated interstitial lung disease. (SSc-ILD).
This designation is based on Boehringer Ingelheim’s Investigational New Drug application (IND) of nintedanib for the treatment of SSc-ILD and the anticipated efficacy and safety data from SENSCIS™ (Safety and Efficacy of Nintedanib in Systemic SClerosIS), a double-blind, randomized, placebo-controlled global Phase III trial. This pivotal study is fully enrolled, including more than 520 patients from 32 countries.
The President of FESCA, Annelise Rønnow, says: “There are only a very few drugs assessed in clinical trials for scleroderma with lung involvement – a devastating reality for people living with the disease. We appreciate that the FDA recognised the importance of the development in this field”.
Read the entire pressrelease here.
The American company, Cumberland Pharmaceuticals, is conducting a phase 2 clinical study evaluating the safety and effectiveness of a new oral drug product in patients with diffuse cutaneous systemic sclerosis (dcSSc) or patients with pulmonary arterial hypertension (PAH) associated with their scleroderma. The study is open at 6 Scleroderma Centers of Excellence and opening at more centers with active Scleroderma Research programs.
General study information can be found at the study’s NCT website (NCT02682511). Eligible subjects will be reimbursed for their time and travel. If you are interested in learning more, please contact us at 615-627-4121 or firstname.lastname@example.org
At the moment the study is targeted Australia, Canada, South Africa, India and U.S.
Details on the trial – please go to: https://clinicaltrials.gov/ct2/show/NCT02682511?cond=systemic+sclerosis+ifetroban&rank=1
We invite you from all over the world to post a picture/video with smiles on social medias like twitter, facebook, instagram etc with the hashtag #ScleroSmile
Scleroderma is a rare autoimmune disease that turns simple daily activities into real challenges. It is life-threatening and currently has no cure. But patient organisations are fighting back. Join us as we spread awareness and support research. We refuse to lose our smiles!
Also known as Systemic Sclerosis, scleroderma is a chronic disease of the connective tissue, immune and vascular systems, and presents a major diagnostic and management problem for patients and their physicians. Despite this, the past two years have witnessed important improvements in therapies and care. Early diagnosis is essential, and the first symptom is nearly always Raynaud’s.
Help us to keep smiling by donating to research!
The Federation of European Scleroderma Associations aisbl. (FESCA) is a focal umbrella support and advocacy organisation composed of the national scleroderma associations of 19 EU countries.
The Federation of European
Scleroderma Associations aisbl.
Throughout 2016, Project Scleroderma has been brainstorming new and innovative ways of helping the scleroderma community, in addition to the awareness component which was their first focus. Now they have officially launched the Project Scleroderma Patient Support App., in conjunction with FESCA, so that you and scleroderma patients everywhere can download this app for free at any time.
The Project Scleroderma Patient Support App, among other features, allows patients to track and chart symptoms on a daily basis and keep a running diary of notes to share with their physicians. It offers a space to interact privately and securely with other scleroderma patients, and will serve as a hub for patient resources and educational videos.
To download the app
STEP 1: Download “Mobile Health Library” available on App Store, Google Play and Kindle Fire.
STEP 2: Enter MHL app code: SCLERO
Project Scleroderma promises to update this app continually, and add useful content on a regular basis. Its main purpose is to simplify the daily experience for scleroderma patients as they tackle everyday challenges, to help ease the emotional stress that is a part of chronic illness, and to help patients who are newly diagnosed to better navigate this new and overwhelming diagnosis.
Also included is a wellness tracker that allows patients to track their overall well-being on a daily basis, and a dosing reminder for patients to set for their medications.
The button for FESCA is in the patient resources tab, and contains the link for our Facebook and website and video. The app is completely secure, and HIPPA compliant. No information will be shared or sold. It is strictly for the benefit of the patient. We will periodically be running surveys through the app to see how people are feeling about it, and to find ways in which they can help improve it.
Both FESCA and Project Scleroderma are committed to supporting the scleroderma community in as many ways as we can, in addition to our ongoing mission to dramatically increase the global level of awareness for this disease. We hope that this app will truly help scleroderma patients from every part of the world to simplify their experience and to connect with others who are battling this disease.
Today, Boehringer Ingelheim sent out a press release, announcing that, “… the European Commission (EC) and U.S. Food and Drug Administration (FDA) have granted Orphan Drug Designation to nintedanib for the treatment of systemic sclerosis (SSc, also known as scleroderma), including the associated interstitial lung disease (SSc-ILD)”.
Joep Welling from Fesca says: “Scleroderma and associated interstitial lung disease have a devastating impact on the patient community, and we welcome this important news that a potential new treatment has received Orphan Drug Designation. It’s a crucial step forward in helping to address an unmet need and represents important progress for patients with this rare disease.”
Read the entire press release here: https://www.boehringer-ingelheim.com/press-release/orphan-drug-designation-granted-nintedanib-treatment-systemic-sclerosis