Perspectives on the UK CF Registry

Yesterday we released the latestdata report from the UK CF Registry, giving an insight into cystic fibrosis and life for those with the condition in the UK. The report also shows how far we have come in beating cystic fibrosis for good since 1964, when 90% of people with the condition would die before they were 10.

To put perspective on what this data actually means, we’ve asked Sally Smith, a parent of a child with cystic fibrosis, and Dan Longhurst, a 21-year-old student with the condition, to share their stories and hopes for the future

Sally

As a parent of a pre-teenager, I feel the latest Registry Report is very positive and it’s great that CF adults and parents of children with CF agree to be a part of the UK CF Registry. It gives the Cystic Fibrosis Trust valuable insight into patients’ lives enabling their research into the disease and raising the standard of care across all CF centres.

I was pleased to see that there has been a decrease in pseudomonas across the age groups – it is a word that parents dread hearing at their clinic appointments. My daughter took part in a research trial in 2012/13 which looked at taking preventive measures to halt the growth of pseudomonas in the early stages of a chronic cold. As isolating as CF is, for both children and parents, the new protocols instigated across clinics with regards to cross-infection and early treatment have obviously helped. 

As a mum, your natural instinct is to ‘wrap them up in cotton wool’ when you first receive the diagnosis but it’s not really an option. And as I enter into those teenage ‘non-compliant years’, I find the news that 70.9% of people over 16 are in employment or further education encouraging – I just need to find more interesting and fun ways for her to enjoy physical activity.

Dan

I'm Dan, a 21-year-old university student from Essex studying Animal Conservation & Biodiversity, living my life to the fullest with my old pal cystic fibrosis.

I was diagnosed with CF when I was just a month old, and had to battle the highs and lows of the condition all my life (obviously). I’ve never let it stop me accomplishing anything I’ve wanted to do in life.

The first of the two distinct parts of my life that I found the hardest was  when I was at secondary school, when I realised I was not like everyone else. Because I didn't know any different than having CF when I was younger, I was oblivious to the difference between me and my friends, but growing up and releasing I was "different" was hard.

The second was when I went to college: becoming more independent with my treatments and balancing my social life was a struggle.

The hardest part of having CF I feel is not the treatments or the inconveniences of being ill. I found, when I was younger, it was being made to feel different from everyone else, constantly explaining to new people what CF was and why I do certain things (such as taking Creon when I eat), and not having/knowing anyone that was going through what I was (due to cross-infection).

My Mum (Tina), Dad (Martin) and family have always have been there to support me when I have been unwell. The biggest turn around in my health and lifestyle when coming to terms with having CF was meeting my girlfriend, Rosie, who has convinced me that I am no different to any other person and without the condition “you would not be Dan”. Now my outlook is completely different on life and I just simply “get on with it”.

These two perspectives show how the hopes of parents like Sally become the reality for people like Dan. Share your story too and help us show the progress made in beating cystic fibrosis for good.

Understanding Median Predicted Survival

This year’s UK CF Registry Report shows that the median predicted survival for people with cystic fibrosis is a bit lower this year. To explain this change, Dr Janet Allen, Director of Research and Care, explains what we mean by median predicted survival and how “wobbles” happen.

You will have noticed in the annual report for the Registry that the median predicted survival is 36.6 years and that this is lower than the 43.5 years reported last year.

First of all, it is important to understand that median predicted survival is not the same as life expectancy. Median predicted survival is calculated from the registry data and represents the age beyond which half of the people in the UK CF Registry today would be likely to live. The annual values for median predicted survival tends to wobble around from year to year as the calculation is sensitive to the number of deaths recorded in any one year and there is a tendency to over-interpret the yearly wobble. This year the value appears to have dropped because the number of deaths recorded rose to 146 cases (of over 10,330 people on the Registry, representing around 1% of the UK CF population). A similar downward blip was observed in 2009 when the median predicted survival was 34.4 and the deaths recorded at 141. Apart from these dips, the trend in median predicted survival continues to rise and it may be better to report the overall trend rather than the year to year variation.

By way of an analogy, it may help to think of a country’s economy.

Over the past 50 years the UK economy has greatly, and largely consistently, increased, and yet there will be patches where, for a number of reasons, it contracts.

However when it does this, it never goes back to the start. It’s a case of always taking several steps forward, but every now and then one or two back.

The median predicted survival for people with cystic fibrosis has done the same and increased over the past 50 years, and yet, as happened in 2009, last year there was a noticeable but not statistically significant decline, even though the trend remains upwards.

Remember that when the Trust was founded in 1964, 90% of people with cystic fibrosis died before the age of 10.

Life expectancy answers a different question; for a child born today how long could they be expected to live?

These are complex calculations and are not calculated every year. The last report for the UK was over 20 years ago when it was calculated that life expectancy for a newborn child with cystic fibrosis in 1991 was in the order of 40 years. It is now estimated to be over 50 years for a baby born today, and the Trust is exploring having these calculations done again to show how far we have come since 1964.

You can find the UK CF Registry Report over on the Trust’s website both in Summary Form and as a Full Data Report.

Keeping Cool With Cystic Fibrosis

As temperatures soar, Public Affairs Officer Lynsey Beswick presents her own guide to keeping cool with cystic fibrosis.

So the British summer has finally arrived. But with it the government has issued some important health warnings for those of us who may suffer with long-term health conditions.

So whilst I can confirm that I have been frantically routing to the back of my wardrobe to find my shortest shorts I have also been contemplating some of the advice and what it will mean for me and my cystic fibrosis.

Whilst the government have put together a heat wave plan I have been carefully considering my own CF-proof plan.   

Not only am I armed with inhalers (in every handbag I own) I also plan to ensure that I am extra careful about remembering to take my doses along with any other nebulisers or breathing medication.

We all know that the sun causes sweat – and people with cystic fibrosis love to sweat – so I have also been frantically digging out the salt tablets which are usually only reserved for holidays abroad; to avoid any heat-induced cramps which can be common for people with cystic fibrosis.

 

I have also started to take an antihistamine to act as an anti-inflammatory and to help ensure that any allergies are kept at bay as this can also play havoc with my chest and make me feel worse.

As well as ensuring I have the obvious – lots of fresh water and a good dollop of sun cream – there are some other things that might not be quite so obvious. For example certain antibiotics that I take for my cystic fibrosis can cause photosensitivity – this could mean the difference between a glorious tan and a slightly less glamorous shade of beetroot.

I will certainly be taking things at a slower pace and avoid over exerting myself – a great excuse to get out of going for my evening run – that will have to wait until the weather is cooler I’m afraid!

Instead I will be remaining inside during the hottest hours of the day with my feet up (hopefully), windows open and a fan flowing to keep me as cool and as comfortable as possible. I will also keep all medications in the shade too – particularly digestive enzymes as the heat can apparently damage them and make them ineffective. 

I also need to monitor my food intake – hot weather often means I lose my appetite so making sure I have snacks to hand or supplement drinks will help to ensure I’m still getting essential calories and that I keep my energy levels up.

Of course at the first sign of feeling unwell or breathing difficulty I do have my specialist CF team on speed dial – but I am hoping that by taking sufficient precautions I can avoid any drama and enjoy this great weather while it lasts!

As Lynsey suggests, if you do feel unwell it is important to contact your specialist CF team immediately.

Setting the Parliamentary Agenda on Transplants

James Barrow, Head of Public Affairs, talks about the success of yesterday's debate on organ donation and transplantation in Westminster Hall.

Yesterday’s parliamentary debate on transplantation was an important moment for our community.  Since the launch of our Hope for More report we have been campaigning to improve access to lung transplantation for people with cystic fibrosis. Some 4,500 supporters emailed politicians from across the UK, asking them to urge the Government to take action and our Public Affairs team has met with over 60 MPs, AMs, MLAs and MSPs to discuss the report. This activity triggered the debate in national transplant week.

During the debate MPs raised the use of extended criteria lungs, the ability of surgeons to downsize lungs and the importance of new technology in preserving and reconditioning lungs. Cystic fibrosis was mentioned no less than 20 times during the 90 minute debate.

NHS Blood and Transplant (NHSBT) is now reviewing its existing lung allocation policy and is modelling a national allocation system.  And the Minister for Public Health, Jane Ellison, has expressed concerned at the number of deaths on the waiting list. NHSBT is set to make an announcement on the outcome of its review in the autumn and we will continue to campaign hard on this issue.

Thank you to everyone who continues to support our campaign – your help has made a real difference.

And thank you to those MPs who took part in the debate: Jim Shannon MP, Margaret Ritchie MP, Caroline Nokes MP, Kerry McCarthy MP, Jason McCartney MP, Glyn Davies MP, Madeleine Moon MP, Kate Green MP , Luciana Berger MP, Jane Ellison MP.

If you missed the debate a full transcript is available here.

Seven Things You Learn on the Transplant Waiting List

Robyn has been on the waiting list for a lung transplant for nearly five months. She takes IV antibiotics to keep her as well as she can be, and sometimes has to be on oxygen for long periods of time. A single mum, Robyn is coping with the support of her wonderful daughters Sophie, 11, and Phoebe, 7. Here she shares seven things she has learned from life on the transplant list.

1) Make lasting memories

Last year my parents took us all to Disneyworld in Florida which was absolutely amazing. We wanted to ensure we had the holiday of a lifetime and a chance to build some everlasting memories in case we didn’t get that chance again.

2) You may want to keep the whole truth from the ones you love

The girls have always known that their mummy is not well but I keep the frightening statistic that one in three people with cystic fibrosis die on the lung transplant list hidden from them. It wouldn’t be fair and could damage what we have if they stopped acting normally around me.

3) Life will change

The one thing I miss the most is running around with the girls. We live in the country and up until I became this ill, I enjoyed a very outdoorsy lifestyle with our horses, dogs and long country walks.

4) It’s okay to ask for help

I really miss doing these activities with my girls and have had to hand over the reins to my long suffering parents who take them out all the time. They have been a brilliant support to me, especially since my husband and I are no longer together.

5) Improvise!

I do consider myself very luck, because my daughters are content when we curl up on the sofa and have movie nights instead. We do lots of indoor activities too, like baking, and they like to have their friends over for sleepovers.

6) Be prepared

I can’t travel further than two hours from the hospital, in case I get that life-changing phone call. My parents are on standby and when the time comes no matter what time of day it is my mum is ready to look after the kids and my dad is ready to drive me to the hospital.

7) Anything could happen

I have written the girls a letter each for when I go in to have my transplant, in case the operation goes wrong. I’ve also started to write letters for them to open further down the line. Things like special messages for all of their birthdays, their wedding day and even for when a boy might break their heart for the first time. I want to give them these letters so that when they read them they can feel close to me and know that I have planned ahead for them, but most importantly to let them feel my love when they will need it most.

Extracts from a Short and Merry Life - Part Three: Adherence and Commitment

Ahead of tomorrow's book signing at Waterstones in Wimbledon the final extract from 'How Have I Cheated Death? A Short and Merry Life with Cystic Fibrosis' by Tim Wotton is on drug adherence and commitment:

Why would I want to cut corners and miss my medication? Even when I take all my medication, it just about maintains my health – it never cures. I would feel a lot worse if I missed any of my treatment regime, so what’s in it for me to risk that? After all, I’m only as healthy as my last treatment. It is important to control the parts of my illness that can be controlled. Manage the manageable. In rowing terms, I control what’s going on inside my own boat rather than fixate on external factors.

I have calculated that for every 24 hours in a day, at least two hours are spent doing something medical. A positive way to view this is that leaves me with 22 hours to do what I want to do each day so I take pleasure in the hours I have and not the hours I lose.

22 hours for Tim Wotton and 2 hours for CF.

My treatments are something I do to get out the door and get on with my busy life – they are my enablers – they have become a second nature activity like brushing my teeth. This is engrained in my subconscious by constant repetition so that eventually my body begins to remind me what to take and when. This can only happen by taking ownership of the condition rather than relying on others to be my conscience. For me, it’s been a case of building habits and over the years I have become pretty competitive with myself to adhere to the regimen. Conversely I am prone to getting annoyed with myself when I forget any part of my treatment.

If my routine is altered which is likely when I’m travelling, on holiday or in a work conference, it does affect my medical management and I have to focus even harder not to miss any facets of my regime.

In dealing with CF I have tended to match fire with fire. I’m bullish about getting my own way from hospital visits and I’m probably a pain to medical staff at times. I read my own body signals and thus don’t always wait for the doctor to tell me about changing my antibiotics when I already know what drug to take and when to take them. I try to keep on top of the condition and look for new ways to ‘boss’ my illness with changes of drug, alternative therapy or new ways of positive thinking.

I won’t back down.

I one hundred per cent respect my illness, know my many limitations and look to avoid the ‘CF banana skin’ of trying to dismiss it and not take it seriously enough.

A lot of willpower is required to win this ongoing battle. When you get to a certain age you tend to balk at doing things that you just don’t want to do and believe me doing my treatment is the last thing in the world I want to do. I have to knuckle down every day of my life and continually do these unpleasant acts of medication. I only get out of my body what I am prepared to put into it.

I do at times have to bite my tongue when people I encounter complain about their short-term health issue but do nothing about it. Or those that make a big fuss at the dinner table about taking just one tablet. I’ve also witnessed guys who milk their condition or injury for all it’s worth and feed off of the novelty factor. These are often the same people who don’t take any medication to get it fixed but prolong their own agony and that of those around them. I prefer to say nothing and let my actions do the talking. But I’ve had nearly 40 years to get used to my afflictions – I’ve never known anything different.

I have to respect this illness 365 days a year and especially at times when I would rather kick-back and have some time off: Christmas day, birthdays, my wedding day and honeymoon, holidays, travel, before and after work – I don’t get a day off with this condition. The symptoms of CF and diabetes don’t take a day off and neither can I. I can take a holiday but CF doesn’t take a holiday from me. I’ve calculated that if I had just two weeks off from taking all my various medications, I would be seriously ill in hospital.

If I fail to prepare for daily eventualities, I am preparing to be caught out and potentially be affected. Following a ‘just in case’ mantra, I always plan for the next day, work, social commitments and the weather. My work bag is always stocked with my daily pills, insulin and dextrose energy sweets. I bet I’m one of the few men who would go out on a stag do armed with an umbrella so that I don’t get wet and damp! As soon as I’ve played sport or if I get caught in the rain, I’ll take a shower to avoid catching a chill. It’s being spectacularly practical rather than spontaneous. Spontaneity is something that happens to other people who live in a parallel universe to me.

I get angry on a daily basis about my drug regime, but I have learned to channel that anger and frustration. I focus my thoughts on what activity – tube to work, playing sport, game with my son, meal with my wife, night out with friends – I will do once that piece of medication has been completed, rather than on the actual treatment itself. I very rarely vent my anger and frustration with those around me and I’m learning to be more open as I write this diary.

I re-join my body back on the bench. I’m now calm and at peace as I sit in the delightfully calm twilight moment that only really pervades on a still summer’s evening. I think of friends who can just exist without having to put themselves through this form of exercise torture and I envy them. My God, I envy them. Sometimes I feel that ignorance would be bliss.

However tough it is for me to bear, exercise is my version of treading water in the ocean. Once I stop, I’ll drown and that will be it. I’ve literally got no other choice but to keep on going with it. By forcing myself to breathe hard while exercising, I in turn breathe life into my lungs. While there’s still breath left in me, I will continue to run for my very life.

Everyday I’m shaken by my disease but I try not to be too stirred and respond by shaking it back. After all, I’m still here. I’m still running. I won’t back down.

Tim will be signing copies of his book at Waterstones in Wimbledon from 6:30-8:00pm this Saturday. You can purchase 'How Have I Cheated Death? A Short and Merry Life with Cystic Fibrosis' from Austin Macauley and Amazon, as well as WH Smith, Waterstones and Foyles.

The Next Steps for Combination Therapy and Cystic Fibrosis

Ed Owen, Chief Executive of the Cystic Fibrosis Trust, discuss how this week's announcement on the ivacaftor and lumacaftor combination therapy affects the wider landscape of beating cystic fibrosis for good.

The results of the Phase III trial of Vertex’s combination therapy for people with the most common mutation of cystic fibrosis is a further sign of the extraordinary new period we have entered.

Over the last five decades, we have made great progress from the time when cystic fibrosis was a condition that killed most people living with it before they reached the age of 10.

Yet it is only now that therapies targeting the basic genetic defect – the holy grail of so much work since the discovery of the cystic fibrosis gene in 1989 - are becoming a distinct reality. Indeed, Kalydeco (Ivacaftor) is doing just that for those with the G551D mutation already!

Of course, it is important to sound an important note of caution here, not just because the results of the combination therapy for those with two copies of the F508Del gene are not as dramatic as that seen with Kalydeco – and neither is a cure.

There is also still some way to go before this treatment can be available to people with cystic fibrosis, Vertex need to receive a license to market it from the relevant regulatory authorities.  Here in Europe that’s the European Medicines Agency (EMA) which can take many months to assess the evidence that is presented to it.

And even if regulatory approval is granted, there will then be the often protracted process by which each of the four NHS systems across the UK decide whether to fund this therapy or not.

In all, whatever the final decisions of the NHS, this whole process will probably take at least another year, and probably more – and that’s a desperately long time for many.

Those with other mutations will have to wait longer still before transformational therapies come along.  But there is hope in these areas too.

We can afford to be very positive – and our voice as a cystic fibrosis community will play a vital part in persuading decision-makers and manufacturers to enable new treatments of this sort to be funded in the UK.

This week is a further step forward on the road to beating cystic fibrosis for good - but there is still a long way to travel.  In the words of the late Casey Kasem we need to “keep our feet on the ground and keep reaching for the stars”.

For more information on these trial results do read Dr Janet Allen’s blog from yesterday and a great piece by our Special Adviser on Research & Patient Involvment, Oli Rayner 

You can also get more information about the combination threapy from the Trust