Sunday, 2 April 2017

Taking Part in Raynaud's, Scleroderma Research Trials, Autoimmune Rare Disease, Nicola Whitehill








The theme for this year’s Rare Disease Day 2016 was ‘Research, where all possibilities are Limitless’. 

This is particularly relevant to the rare disease patient whose daily reality is governed by a poorly understood medical condition, where treatments are targeted at symptom suppression, with little known understanding to cause and cure. 

And, of course, to have actually attained a diagnosis, is an accomplishment in itself, since on average this takes 4 years. 

I wrote about the importance of expert specialist centres, here

I wrote about the importance of an early diagnosis, here
 
In 1997, aged 24, having developed swollen fingers and joints, a specialised blood test confirmed that I had the rare, autoimmune rheumatic disease known as Scleroderma (Systemic Sclerosis) along with the circulatory disease, Raynaud’s. 

I was given a 15month prognosis by my diagnosing doctor, as, there is no cure, and still, there remains no cure. 

I was prescribed a cocktail of chemotherapy and immunosuppressive drugs, which eventually slowed the Scleroderma progression. 

I am now 13 years free from this regime, with just taking a daily tablet to open my blood vessels and remain indoors at much as possible, to prevent Raynauds attacks.  


TAKING PART IN RESEARCH

Throughout my 20 year Scleroderma patient experience, I have taken part in several clinical trials, all based at the expert Scleroderma Unit at The Royal Free Hospital in London, with Prof Chris Denton being the clinical lead.

It is 2 years since my last clinical trial experience, where, over the course of 6months I had to take ‘mystery tablets’ at set times, communicated to me via an electronic bleeper, which I was unable to turn off! 

WHAT CAN I EXPECT? 

At the start and end of any clinical trial, all baseline measurements must be taken, so that any difference in results can be seen. 

This required me to have to travel to the Scleroderma Unit in London. 

I was able to remain at home in Southport for the trial duration, with the lovely Yvonne, the clinical trial nurse, staying in regular phone contact with me, for that time. 

On this particular trial, I am sure that my ‘mystery tablets’ were the live active ingredient, since I experienced some quite unpleasant side effects, which I noted for the study purposes. I was quite relieved when that trial finished.

My clinical trial experience prior to the ‘mystery tablets’ involved me having to inject myself with a ‘mystery liquid’. 

Having a rare disease certainly takes you down life paths which you would never have dreamed of going, as well as, makes you find inner strength, which you never knew you had. 

I have always hated needles, and still do, I can not even watch the phlebotomist put the needle into my arm at my current bi-monthly blood tests. 

However, for the best part of 18months ‘Trainspotting’ came to my house here, in Southport. 

I was required to inject myself with a liquid which was stored in a small vial, which I had just removed from the freezer. 

I had several minutes to warm the vial, so that the frozen ingredient became a liquid, draw the liquid into the needle and then inject myself, twice a week, including Christmas Day 2008. 

I used my stomach as my injection site, giving myself the reward of cake, to keep my injection site maintained properly. 

I would also play my favourite music at that time, to get me in to ‘the zone’ for doing what I needed to get on and do.

I have the utmost respect for all patients who have to inject themselves on a daily basis. The ‘mystery liquid’, although my worst nightmare to administer, made me feel a lot better, with less pain and muscle rigidity. 

Sadly, I understand that the results were not promising enough for the pharmaceutical company to consider further investment in this area, which is disappointing to me as a patient, as I did feel a lot better when having the ‘mystery liquid’.

I would urge every rare disease patient to take part in a research trial if the opportunity presents, by a respected research centre.

I wrote about the role of medical research, here

I discussed the Journal of Scleroderma and Related Disorders, here


As a rare disease patient, this (taking part in trials), is a way in which we can help to pioneer the future for fellow rare disease patients. 

As well as, by taking part in a trial there is the opportunity to try newer innovative treatments which may provide most welcome symptom relief.

An edited version of this article features on the Rare Disease UK website, here 

Raremark website here 





October 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      



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  
  




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



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 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
#Raynauds
#RareDisease  

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.
#HOPE 


 

Last Update: April 2018




 


2 comments:

  1. Hi,
    Well done Nichola, you are an amazing lady. Immune diseases seem to be on the increase. I is quite strong in my family. I have raynauds & am currently being monitored for potential Lupus as the bloods were variable & inconclusive. One of my cousins have raynauds too & is being investigated following serious weight loss and stiffness. My son also has an autoimmune disorder, Vasculitis, he has been very ill at times & has had ops due to the disease but like yourself he fights every step of the way. I live with worry whilst he lives with what life has thrown him. He is brave & admirable like yourself.He is 27 yrs old now & injects him self weekly with a Chemo drug in order to fight relapses.
    One of the things he struggles with is from the outside he looks so well but when hes off work sick people cant understand the invisible symptoms, pain exhaustion & worse during flare ups.
    I am interested in genetic links to immune disease, have you come across any articles, as we have a few family members with various immune issues? Anything you can send to read I would much appreciate it.
    All the best, Kate x

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  2. Hi Nichola - thank you for your articles. You are lucky on so many levels. Mainly? I envy you in that you are lucky enough to be able to communicate your experiences eloquently in a professional manner publishable. Also, through your experiences you gain wisdom (as I) and able to pass that on. I would love to be able to articulate my experience with Scleroderma and from the perspective as a black woman am in more heightened fear of things escalating to death because Scleroderma affects any people of colour more severely and rapidly. This also tails into "trials". The fear of going on a trial to help future prognosis is fraught with fear. The anonymous medicine perhaps making one worse and the doctors find it extremely difficult to help black people from the outset - fumbling in the dark - let alone trials medicine having the potential to make one feel better/worse appears a bit of a self sacrifice for black people fearing moving to an early grave - catch 22. It just worries me that where it is extremely difficult for doctors to better understand our white cousins compatriots, it just seems no man's land and a fait accompli doctors trying to bring someone of colour back from the brink of death that this disease can take them from relatively coping to it's all gone tits up with the trial medicine. What are we to do - can be a lonely scary place and no assurances in any shape or form. When I see my doctor, I sometimes feel they are just prescribing protocol with the though it's touch and go whether it works for you (as a black person) as I hear the words all the time "I don't know!!!". Sometimes, this is when I feel being black the medical system has not given near enough equal research our way. This is why predisease I hated going to the doctors because so many times they would say "it's hard to see on your type of skin or I can't see anything" - when for me I could clearly see things! It can mean "game over" if you are black and diagnosed with an autoimmune disease. Thankfully, I feel someone is looking out for me, because as a black woman, I am doing extremely well and coming up to my third year. This I know is partly due to always being active once and researching the disease when I was coming to terms with the diagnosis and realised from the outset that diet and the right type of supplements help in lessening the severity - it worked, within months my rheumatic pains disappeared still to-date and my other symptoms became less by the second (figure of speech). Although I think I've had the disease much longer before diagnosis but didn't go to the doctors because I was certain they wouldn't see the thickening of skin located to just both my wrists at the time. However, in all that said, you are doing a great job highlighting and bringing Scleroderma to the public forefront which benefits everyone. I'm happy for any parts of what I've written wants to be used.

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