Raynauds Scleroderma Global Patients: Clinical pearls from the World Congress on Systemic Sclerosis, scleroderma, February, 2016

Clinical Pearls From The World Congress On Systemic Sclerosis, Scleroderma.

February, 2016, JSRD Vol.2 Issue 1 Jan – Apr 2017,

Dr. Daniel E. Furst.

Journal of Scleroderma and Related Disorders (JSRD), Vol.2 Issue 1 Jan – Apr 2017 pp.1-68, excerpts from the article written by Dr. Daniel E. Furst.

Introduction

Every second year, the World Scleroderma Foundation holds a World Congress on Systemic Sclerosis where more than 1000 rheumatologists and those interested in scleroderma meet to review clinical and translational aspects of systemic sclerosis.

The World Congress was held during February 18-20, 2016 in Lisbon, Portugal.

The complete abstract book is available as a supplement to the Journal of Scleroderma and Related Diseases but a summary of some of the interesting aspects of this Congress is appropriate.

While most aspects of systemic sclerosis (SSC) were included during the World Scleroderma Foundation Congress, this manuscript will concentrate on selected aspects of the disease that could be of particular interest.

Specifically, some of the therapeutic trials relating to SSc, ultrasound, capillaroscopy and selected clinical aspects including fatigue, disease classification plus gastrointestinal disease.

Perforce, only summaries can be included, sometimes with commentary regarding their usefulness or interest.

All the abstracts below come from the Journal of Scleroderma and Related Disorders 2016;1(1):43-169. Doi: 10.531/jsrd.5000203.

Main points:

PAH:

Survival in SSC PAH has improved. Although survival in SSc-PAH is improved, survival in scleroderma PAH leaves room for further improvement. It is still only approximately 60% four plus year survival.

Gastro-intestinal:

These are encouraging results showing that pro-kinetics help symptoms although it is worth trying more inexpensive therapies before going on to the more expensive medications such as domperidone or equivalent.

Measurement of Disease:

Ultrasound is being used more and more frequently in rheumatology.

This is also true in SSc. The first steps towards defining SSC skin ulcers were demonstrated, showing only 40% concordance between clinical observation and ultrasound measurement.

This inaccuracy of the clinical observation could, speculatively, be the reason studies of healing skin ulcers were often unsuccessful, since one would not expect healing of an ulcer if it really was not one.

When comparing skin ultrasound for skin thickening in SSc patients and normal healthy subjects, the ultrasound seemed able to separate the SSC patients from healthy subjects in the forearm, hands and fingers but was less successful for the lower extremity.

The skin of the abdomen in SSC patients appeared clearly different than that in healthy subjects and this is important because the abdomen is hard to score accurately.

Capillaroscopy is another inexpensive tool that is being used in SSc.

This interesting abstract seemed to imply that capillaroscopic patterns, like the skin in general, may become stable over time and that the meaning of capillary patterns may need to be reconsidered in long-standing disease.

Clinical aspects:

Juvenile SSc has been a somewhat neglected area within the scleroderma community.

A response index has been developed for adult diffuse SSc, called the Combined Response Index for Systemic Sclerosis. A similar response index is being developed for juvenile SSC.

Using a Delphi approach, paediatric rheumatologists interested in SSc found, initially, 21 domains and 131 measurements that might be used.

This was winnowed down to 11 domains and 41 items. Thus far, the domains were: global activity, biomarkers, Raynaud’s phenomenon, digital ulcers, skin, pulmonary, cardiac, GI, renal, health-related quality of life, function and childhood development.

Fatigue is a greater problem than sleep in SSC, diffuse in particular. These provocative abstracts need to make us think about the multiple causes of fatigue, beyond difficulty sleeping.

These abstracts point to the importance of the GIT in multiple spheres of SSc patients’ lives and also begin parsing out how better to measure and, eventually, treat SSc gastrointestinal disease more effectively.

Conclusion:

The World Scleroderma Foundation Congress abstracts describe many areas of research and interest, including new data on therapeutics, measurement of disease and clinical issues.

To view the full article, Click here

May 2017.

5^th Systemic Sclerosis World Congress, Click here

To read my articles:

Rare Disease Day 2018, Research - Taking Part in Clinical Trials, Click here

March Autoimmune Disease Awareness Month 2018, Click here

Global patient video, Click here

Becoming a Patient Research Ambassador for the NIHR, Click here

If we only had more RESEARCH investment for Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here

The Importance of Medical Research and Awareness to the Scleroderma, Raynaud's, Autoimmune Rare Disease patient, Click here

Scleroderma Awareness Month 2017 Patient Profiles Campaign, Click here

Scleroderma Awareness Month 2017 Patient Profiles Campaign Patient Index, Click here

The Pandora’s Box of the rare autoimmune disease Scleroderma, Raynaud's and Cancer, Click here

Key Event Dates 2018, Click here

Importance of an Early Diagnosis, Click here

Taking Part in Clinical Research Trials, Click here

The Importance of a Multi Disciplinary Medical ‘Dream Team’, Click here

Expert Specialist Centres, Click here

My Skin is Cured from Scleroderma, Click here

UK Guidelines for Managing and Treating Scleroderma, Click here

Fatigue, Click here

Mobility, Click here

Diet and nutrition, Click here

Raynaud's, Click here

World Scleroderma Day 29^th June 2016, Click here

World Scleroderma Day 29^th June 2017, Click here

Why Global Collaboration is important to the Rare Disease Patient, Click here

RARE DISEASE DAY:

The theme to this years Rare Disease Day is Research

Rare Disease Day 2018 – Research, Taking Part in Clinical Trials. Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here

2016 Rare Disease Day Patient Voice

2016 Rare Disease UK Parliamentary Reception

2017 Rare Disease Day Flashback

2017 Rare Disease Day unmet clinical needs to the scleroderma patient

Rare Disease Day is a fantastic opportunity for the entire rare disease community to shine a spotlight on their reality, combining as one unified voice. Where, at least one commonality presides –

Medical Research provides the brightest light

for the illumination of the rare disease patients’ plight.

I highlighted other areas of medical research interest within Week 3 of my Patient Profiles Campaign for Scleroderma Awareness Month 2017.

VIDEO

INDEX

Although rare disease patients are few in number, eg. 2.5 million scleroderma patients worldwide, (the World Scleroderma Foundation), the commonalities and golden hallmark for each rare disease patient are the same:

Early Diagnosis

Expert Specialist Centres

Access to Innovative Medicines

Where MEDICAL RESEARCH investment is VITAL.

Research is the key. Abstracts from 2016 World Congress, Click here

Scleroderma Awareness Month Campaign 2016, Click here

Links

View video, here

Preamble - here

The Family Day at the Scleroderma Unit, The Royal Free Hospital is taking place on the 19^th May 2018. Browse the program, here

This year, I am celebrating 20 years of being a patient at this world leading expert specialist research centre.

Sept 2017

I am eternally grateful to the global scleroderma trail blazers Dame Prof Black and Prof Chris Denton, whose commitment and dedication to unlocking the scleroderma enigma, is nothing other than, superhuman.

I am truly humbled and inspired by their work ethic. I am wholly appreciative for Prof Denton’s continued medical expertise and support, especially during my barrister qualifying years, 1997 - 2004.

1^st March 2004, I qualified as a self employed practising barrister. Further to having been told in 1997, by my diagnosing doctor, that I was looking at a 15month prognosis.

I very much hope to utilise my professional skills and qualifications along with my patient experience, to help achieve the #SclerodermaFreeWorld dream, hoping to improve understanding and best practice, in the meantime.

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