Thursday, 13 April 2017

UK Guidelines for the Treatment and Management of Scleroderma, Raynaud's, Autoimmune Rare Disease

UK Guidelines for the Treatment and Management
Scleroderma, Raynaud's, Autoimmune Rare Disease

In June 2016, Scleroderma Awareness Month, the first national UK guidelines for the treatment of scleroderma (systemic sclerosis) were published in the international journal ‘Rheumatology’, as well as, on the British Society for Rheumatology (BSR) website.
See more, here   

These guidelines were put together by the BSR and BHPR (British Health Professionals in Rheumatology): 

to develop an expert driven evidence based series of 
recommendations for the management of scleroderma.’ 
Dr Peter Lanyon, Consultant Rheumatologist, has now completed his first year of the 3 year Presidency role of the BSR. 

Dr Lanyon set out his plans and vision for an improved musculoskeletal / rheumatological healthcare / patient experience, with an ‘all hands on deck’ approach. (Although, sclerodactyl hands may not be much use, if taking this in a literal sense).  
It is very encouraging to see that the rare disease, non genetic, rheumatological conditions (this includes scleroderma, vasculitis, myositis etc) have been outlined specifically, for areas of improvement, as by the UK Government’s commitment to such by 2020. 

To read more about this, Click here  

In my view, Dr Lanyon is another unsung hero and blessing to the global scleroderma community, the rare disease community and the musculoskeletal community. 

I have the pleasure of being a member of the NHS Clinical Reference Group (CRG) for Specialised Rheumatology, which Dr Lanyon chaired prior to taking on his BSR Presidency role.  

I witnessed at first hand, the selfless dedication and extra miles which Dr Lanyon demonstrated in order to improve the day to day reality and care for patients, as well as improving the standards of the NHS as a world class leading provider of healthcare.  

The next annual BSR conference is scheduled to take place in Liverpool, 1st - 3rd May 2018.

To watch my presentation ‘Systemic Sclerosis – A Patient’s Perspective’ which I gave at the 2015 BSR annual conference, Click here

Professor Chris Denton, Royal Free Hospital and UCL Division of Medicine, Chair of the BSR/BHPR Scleroderma Guideline development working group, another unsung hero and blessing to the global scleroderma community, provides the following helpful executive summary to the UK national scleroderma guidelines, and, an insight into the important background to their formulation:
           These guidelines are important because they summarise the current best practice for treating the major complications of systemic sclerosis and also address the overall approach to disease management in the UK. 

They have been developed under the auspices of the SAGWG (Standards, Audit and Guidelines Working Group) of BSR that has developed a process that is accredited by NHS evidence.  

This is important since it means that the guideline is NICE accredited and should therefore be taken very seriously within the NHS as defining the standard of care for patients and access to therapies. 

Embedded within the guideline are important NHS England policies for the management of digital ulcers and the pathway developed for assessment and delivery of autologous stem cell transplantation for appropriate cases of diffuse systemic sclerosis.
The guideline process involved establishing a development group that included rheumatologist, scleroderma experts, pharmacists, allied healthcare professionals, specialist nurses, primary care representatives and patients. 

In this way all aspects of the disease and management could be included. A comprehensive literature review of all the evidence supporting treatments for scleroderma was an important starting point and a group of dedicated clinical fellows undertook this work. 

There was a series of telephone and face to face meetings over 2 years that led to the development of the draft guideline. This was then reviewed by BSR SAGWG and by external referees. 

Comments were incorporated and the revised guideline was then finalised and submitted for open consultation so that anyone could comment and have input. After this process the final guideline was written, this was submitted for approval of BSR and then for publication in Rheumatology [3].
This is not the end of the process since the guideline are reviewed and updated every 5 years according to NHS Evidence protocols. 

This is a landmark for UK scleroderma patients and an important one at a time of major NHS change and also challenged and competition for resources for rare diseases. 

It complements the other recommendations being updated such as this of EULAR (European League Against Rheumatism) and the UKSSG (UK Scleroderma Study Group) best practice consensus documents [4].

The structure of the UK national guideline is divided into 3 sections:  

Part A: general approach to SSc management
This section highlights the importance of an early diagnosis with referral to a specialist scleroderma centre followed up with management within the framework of a multidisciplinary team.

Part B: key therapies and treatment of organ-based disease
This section highlights all of the major complications and potential organ involvement, as well as possible treatment options.
Topics include: Raynaud’s phenomenon, digital ulcers, lung fibrosis, pulmonary arterial hypertension, gut involvement, renal involvement, cardiac manifestations, skin manifestations, calcinosis, musculoskeletal manifestations, ASCT.  
I am particularly honoured to have been a member of the medical team who compiled the current NHS guidelines for digital ulcer management in scleroderma. 

Part C: service organization and delivery within NHS England
Scleroderma, SSc should be diagnosed promptly, investigated appropriately and managed within an integrated system of primary, secondary and tertiary level care.
The guidelines are scheduled to be reviewed by the NHS in 2019
References for more information:
BSR and BHPR guideline for the treatment of systemic sclerosis
  • Christopher P. Denton1, Michael Hughes2, Nataliya Gak1, Josephine Vila3, Maya H. Buch4, Kuntal Chakravarty1, Kim Fligelstone1, Luke L. Gompels5, Bridget Griffiths3, Ariane L. Herrick2, Jay Pang6, Louise Parker7, Anthony Redmond4, Jacob van Laar8, Louise Warburton9, Voon H. Ong1, on behalf of the BSR and BHPR Standards, Guidelines and Audit Working Group
Author Affiliations
  • 1Centre for Rheumatology, Royal Free Hospital, London
  • 2Rheumatology Department, Salford Royal NHS Foundation Trust, The University of Manchester, Manchester Academic Health Science Centre, Manchester
  • 3Department of Rheumatology, Freeman Hospital, Newcastle upon Tyne
  • 4Leeds Institute of Musculoskeletal and Rheumatic Medicine, Chapel Allerton Hospital, Leeds
  • 5Rheumatology Department, Musgrove Park Hospital, Taunton,
  • 6Pharmacy Department
  • 7Centre for Rheumatology, Royal Free Hospital, London, UK
  • 8Rheumatology and Immunology, UMC Utrecht, Utrecht, The Netherlands
  • 9Primary Care, Telford and Wrekin NHS Trust, Telford, Shropshire, UK

For the scleroderma patient, these guidelines are a quantum leap in progress for scleroderma best practice management and care. 

These guidelines can be used universally around the world, especially for those countries who, due to their size and economic climate, are not able to produce such recommendations for their scleroderma patients.

As with all things, change is a constant. I very much hope that by the time of the NHS scheduled review for the guidelines in 2019, more, and improved treatments have been made available, as well as, identification of the causative factors of scleroderma have been discovered.

It is with huge gratitude and thanks to the super-human Prof Chris Denton for championing the formulation of the best practice management guidelines for the treatment of scleroderma in the UK, along with the numerous expert medics who assisted with this.

To read more:

the importance of an early diagnosis   

taking part in Clinical Research Trials  

Living the dream, scleroderma style, hoping for a cure….

#SclerodermaFreeWorld #RaynaudsFreeWorld  

An edited version of this article was published here, in my Column with Scleroderma News. July 2016. 

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   

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 29th June 2016, Click here

World Scleroderma Day 29th June 2017, Click here   

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

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  

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. 


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


View video, here   

Preamble - here

The Family Day at the Scleroderma Unit, The Royal Free Hospital is taking place on the 19th 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. 

1st 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.
For latest updates follow: 

Facebook Page:

Twitter: @SclerodermaRF  @RaynaudsRf  

#SclerodermaFreeWorld           #RaynaudsFreeWorld
#ADAM #Scleroderma

Living the dream, scleroderma style.  

Please DONATE to help fund medical research at The Scleroderma Unit,The Royal Free Hospital, London.  

100% of your monies will be used for medical research purposes only. No wages or admin costs. Thank You.


Last Update: April 2018


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