Weight
Almost one year of Kalydeco
Decided it was time for a summary post about my new health on Kalydeco.
I have been on Kalydeco for 11 months through the Named Patient Program, a program where Vertex supply Kalydeco to those who are very unwell. Kalydeco is the first medication that treats the underlying cause of Cystic Fibrosis in patients with the G551D mutation.
Before Kalydeco my lung function was 41% and very unstable. I was in hospital every 3-4 months. I was on medications that treated the CF symptoms; these were becoming less effective as I grew older.
For the last 11 months my FEV1 has been stable at approx 70%. This is amazingly stable compared to before; for 20 years my lung function has varied up to 20% from month to
month, now I am stable for the first time in my life. This is my highest in 3 years.
I have stayed out of hospital for 11 months, which is a 20 year record (I am currently on IVs for mostly prophylactic reasons, I will post about this soon). Before Kalydeco I had over 60 hospital admissions, equalling 3 years of my life.
Every aspect of my health has improved with Kalydeco, I have gained 6kg, I can run up stairs, I can sleep without coughing and I’m attending uni more often. My sweat test results have improved from 102 to 40. This is much lower than the point (60) associated with the CF diagnosis.
Previously I did not plan further ahead than 5 years. At the age of 28, with a life expectancy of 37, I knew transplant was edging closer. Thanks to Kalydeco I now don’t have to plan around hospital and CF, instead I can plan to have a family, a career and to grow old with my lungs.
I hope that Kalydeco can become available to everyone around the world who may benefit as soon as possible. Kalydeco is not approved in Australia yet; we are trying to speed up the reimbursement process and would appreciate your support on facebook and twitter.
Images from Dec 2011 and May 2013. Inflammation and scarring appear white, this has improved significantly on my second xray.
Birthday Wish
This time last year all I wanted for my birthday was Kalydeco.
Each year my birthday wish has been for a CF cure. Last year was the first time that it felt close, frustratingly close as I knew it would be a while before it was approved in Australia. Luckily the named patient program meant I was able to start Kalydeco in September. The change has been astonishing, my lung function has improved 30%, now my highest in 3 years, I am at my highest weight ever, and my sweat test is now below the diagnostic point for Cystic Fibrosis.
So what could I possibly want this birthday?
1. Fast approval/listing in Australia so the 200 people with G551D can access Kalydeco.
2. Fast research for everyone with F508del and other rare mutations, so we can all have a medication like Kalydeco.
3. Someone that can do my uni assignments for me so I can relax on my birthday 😉
6 months of Kalydeco: stable at 70%
The constant productive cough, the overnight cough that disrupted sleep and the breathlessness from walking quickly and up stairs are now distant memories. Instead I have noticed some new changes; my hair is in better condition, my skin has more colour, the dark circles under my eyes have mostly disappeared, even my nails have less small ridges. I can’t remember the last time someone told me you look sick or tired, I was starting to get this daily before Kalydeco. Instead most people now comment about how well I look. I looked back at some photos, and the last time I looked well (not pale & tired) was in May 2010 at my 25th birthday.
For 6 months my lung function has been stable and I have not needed antibiotics. My weight is still increasing. For the first time in my life I need to adjust my CF diet and start to cut down portion sizes. I already eat reasonably healthily (never really liked/agreed with the high fat diet that was promoted when I was younger), but I am hoping some small changes will help. I have started running more regularly, now that it is finally below 35 degrees, which should also help. I am planning to write an in depth post about my exercise in the next few weeks.
Now that I have been busier with uni every day, I have noticed something that is really pretty obvious, that sleep is very important for my lung function. I have been pushing it a little with 6-7 hours of sleep during the week, which starts to catch up with me. My FEV1 can drop 2% or so when I am really tired (a minor drop I know compared to my previous 20% drops, but given how stable I now am 2% starts to concern me!). I am trying to gradually change my body clock and routine as the early starts are going to continue all year.
I have still been noticing a gradual decrease in sputum now that I have been on both Pulmozyme and Bronchitol for a few months. The combination seems to be working really well. I have now reached my goal of being stable and IV free for 6 months, so my new goal is 1 year 🙂
Start: FEV1: 41%, Weight 55.6kg
1 month: FEV1: 70%, Weight 58.5kg
2 months: FEV1: 71%, Weight 60.0kg
4 months: FEV1: 70%, Weight 61.5kg
6 months: FEV1: 70%, Weight 61.8kg
Four months of Kalydeco
I went to clinic a few weeks ago and my FEV1 was 70% 😀
It’s the first time I’ve been out of hospital for five months in the last few years and the first time I’ve been stable without antibiotics in about 15 years!
Start: FEV1: 41%, Weight 55.6kg
4 weeks: FEV1: 70%, Weight 58.5kg
8 weeks: FEV1: 71%, Weight 60.0kg
4 months: FEV1: 70%, Weight 61.5kg
Kalydeco helped me bounce back from my cold, helped stop my PFTs from dropping and helped stop me from getting sicker. With my last three overseas trips I’ve returned home and my FEV1 has been at 49, 50 and 41%. The only way I improved back then was with IVs. Now my body’s ability to cope has improved dramatically. Being stable whilst pushing the limits (travelling, plus the cold temperatures, pollution, smoke and no nebuliser) and still staying at 70% is amazing!
I am planning to stay on Bronchitol and see how this goes for the next few months. I love how convenient it is and it is still helping me clear my lungs well. My cough is back to my new ‘normal’- meaning I am not coughing very much and if I do it is yellow in colour and easy to clear. My energy levels are improving, but I discovered recently that I still struggle with full time university (9-5 every day). Last year I struggled with part time uni, so this has improved a lot, and I am hoping it will continue to improve.
Over the last 10 years I have kept my lung function results in an excel document, so I decided to make some graphs showing my FEV1 and weight. The graphs are pretty amazing, they show my new stability and the very large initial improvement.
I am currently at my highest FEV1 in 2.5 years and only 5% off my highest in 8 years.
This is my highest weight ever. My BMI has changed from 20.0 to 22.1
For anyone who is interested in stats, just for fun (I have been studying stats & evidence based practice for 3 weeks at uni) I had a look to see if my weight and FEV1 were correlated and found there is a strong (r=0.64) relationship between my FEV1 & Weight. There is also a very strong relationship (r=0.92) between my FEV1 & FVC.
7 Weeks on Kalydeco
Thought it was time for another update
My PFTs have been stable at about 70% according to my machine, which is very exciting as I normally would be dropping by now (2 months post IVs). I have also just broken the 60kg mark on the scales so that means I am about 5kg heavier!
My cough has been becoming less productive over the last 3 weeks. I cant remember a time when I have coughed less!
I went 4wdriving and spent lots of time in the sun (cars got stuck on the sand dunes), normally I would have lost heaps of salt and become sick, but I felt fine the next day! I am looking forward to not being restricted in summer- previously I have found it hard to socialise and I have struggled just driving/shopping/running errands when it is above 30 degrees. It has actually become quite restrictive as the summers in Adelaide are very hot, and the salt loss made me feel unwell for several days and often led to bad chest infections.
I generally dont sleep that well and over the last few years I have felt very tired. I have felt tired even with 10 hours of sleep, and I have forced myself to get up. Over the last 3 weeks I have started waking up after 8 hours of sleep and found that I cannot sleep anymore. This seems really weird to me, I am not used to waking up and not feeling tired! I still do get tired throughout the day, but I am hoping this will continue to improve as the inflammation settles and my body becomes more healthy.
I am going to China in December for 4 weeks. The pollution is quite bad in China, so hopefully my lungs will cope better now that I am on Kalydeco. I was not allowed to go on the school trip to China as it was thought that my lungs may not cope with the pollution, so it will be nice to finally make it there!
Before I travel I always have 2 weeks of IVs to maximise my health, particularly given that I can’t get travel insurance that covers CF (can get travel insurance for everything else though). I realised the other day that I had not thought about my normal IVs before travelling to China. It looks like Kalydeco will be the only thing I need before this trip!
Being stable, not declining and not needing IVs like usual really is amazing 😀
70%!!
70% after one month of Kalydeco!!
Before I started I was
FEV1: 1.41L (41%), FVC: 2.48L (62%) & MMEF: 0.52L (14%). Weight 55.6kg.
This is the highest I have been in 2.5 years, and only 5% off my highest in the last 8 years! I was hoping to get to the 60s and 70 is absolutely amazing! My first blow was about 66, I realised 70 might be possible and I managed to get there! It didn’t seem real when I was blowing these numbers on my small machine, and even now that it has been confirmed at clinic it still doesn’t seem real!
I think the large amount of physio, nebs and exercise helped me get to that 70%. I am hoping that I will be able to maintain my pfts and avoid antibiotics for a while.
After one month of Kalydeco my energy levels have improved, I have put on 3kg, I have less reflux, no cough overnight, no salt on my face after exercise, no crackles and no chest pain.
I still have a productive cough in the morning and with physio, but it has decreased significantly. Past late afternoon I rarely cough. The sputum is light green in the morning, but by night it is now often light yellow/almost white! I remember being told by my physios at the children’s hospital that white sputum was normal, but I found this very hard to believe! Looks like it might be true!
I am so grateful to the scientists at Vertex that developed this medication. It is hard to express in words the difference this medication can make to our lives, helping us to work, have a family and have lives that are much more normal/less affected by CF. I have been reading about the trials with VX809 & Kalydeco for F508del (90% of the CF population), the second generation medications (in the lab these medications are more effective than VX809, VX661 and Kalydeco) and how multiple pharmaceutical companies (Pfizer, Genzyme, and N30) are now researching these CF medications. I hope that soon all CFs will benefit from a medication like this as well. I feel very lucky and grateful that I have had this amazing opportunity.
The two week point
Now that I have been on Kalydeco for 2 weeks I thought it was time for another update. After the initial coughing period for the first few days, I was coughing much less for the next week. My cough increased slightly a few days ago, but it is still much less than my ‘normal’ cough. There are still no crackles, and my lungs feel less restricted or tight. My pfts increased significantly in the first few days (15%), and this was stationary for the next week. I thought I had hit my max as my pfts were not changing, it was about day 12 and I knew the clinical trial results had hit close to the maximum pft results at day 14. I was very excited to find that this week my pfts have been gradually going up slightly, now at about 20% higher. I don’t know exactly how accurate my machine is, but I am seeing the highest numbers I have seen on it (I bought it this year), so that has to be good! Normally the only way my pfts ever get higher is with IVs, so it is very exciting to see the number go up and stay there! I am hoping that now that there is less mucus, and therefore less infection, the inflammation will start to settle. I think this is what is helping my pfts at the moment.
I have put on 2kgs, I think this is due to less coughing and less infection stealing my energy, and also a slight increase in appetite. I played tennis and although I was still puffed out, I had more stamina, and did not have salt on my face afterwards. Previously when I have exercised (eg 1-2 hours at the gym with body pump/balance/jam or playing tennis) it has taken me a few days to recover as I have lost so much salt, after tennis I felt fine and not at all tired. So my recovery seems to be much faster.
I have been keeping up with ventolin, hypertonic & pep and pulmozyme. For the first time in my life I am excited to do physio as I can see the results on my pft machine (It’s possible that I could have improved equally without all my effort but it seems to be helping). I have read posts by other cfs about the impact of pft results on our emotions, and I particularly agreed with a post by Alex (http://kalydecoforaustralians.blogspot.com.au/).
The FEV1 score is the main objective measure we have to tell us how our lungs are going. When I was younger I did not worry about this much as I was pretty stationary in the 90s. They would go up and down a bit, but in 2003 at 85% I felt like I had a large ‘buffer zone’ between my pfts and the transplant point. I followed the research and I was pretty optimistic as I felt that medications would be developed before I got to the transplant point. Since then I have been going up and down, with the down parts dominating. Besides when I have IVs, I can only think of a handful of times that my pfts have gone up in between clinics. I am used to seeing numbers that get lower, and I have been very frustrated when they haven’t gone back up with IVs. I used to graph my pfts, but I stopped a few years ago when my pfts started dropping faster. Most people my age know that 20-30% generally means lung transplant assessment, so I was worried when I first got into the 60s, then the 50s, and this year the 40s. I was expecting that within 5-10 years I would need a lung transplant. It is hard to explain exactly what it feels like to see the numbers going up, it doesn’t seem real as going up does not normally happen often. Going up 20% is not something that I ever thought was possible, even with Kalydeco. It feels like I’ve won the lottery twice, first with G551D, and second with it helping so well! I don’t want to look too far into the future as it is only week 2, but I am hoping Kalydeco can help delay or even stop the need for a transplant. Might have to actually care and look into my superannuation, something that has never been a priority for me!
More changes…
On tuesday I noticed that I was coughing up less after I took Kalydeco, so I decided to try hypertonic saline with PEP. So far I had not tried any hypertonic since I started Kalydeco as I was curious to see what Kalydeco could do on its own.
About an hour after I took Kalydeco I tried the hypertonic and PEP and it worked really well. I coughed up a reasonable amount of thin light green mucus. For the past few days I have been taking Kalydeco at 10am & 10pm with ventolin via a spacer, and then hypertonic about an hour later. I have been more productive with the morning dose- I have always been much more productive in the first few hours after I get up. I have then done pulmozyme around dinner time. Today even with hypertonic & PEP I was less productive, I think maybe there is just less there now.
Some people have asked me questions about improvements in other areas: I have not noticed any sinus changes as I have not had sinus problems for the last few years (no current blocked nose/headaches) and reflux is about the same (hopefully may improve after a while). I am hoping that my impaired glucose tolerance (half way to CF diabetes) may also improve in the long term.
It has been quite hot recently and I have not noticed salt in my sweat, will be interesting to see how this goes over summer. Over the last few years I have not coped well with heat & loosing salt, so I am looking forward to any improvement! Might have to test the Kalydeco with a tropical holiday to somewhere in Asia! According to the scales I have put on 1kg. My pfts are still at the 15% increase point, but they vary a lot on my small machine (pico1) so I am waiting to see how they go over the next few weeks and at clinic.
I am extremely happy with the 15%, I tried not to have high expectations before I started and I knew the trial had an average improvement of 10%. So if I do manage to get any higher that would be fantastic! It will take some time for the inflammation to settle (once you decrease the sputum & infection) so that might help the pfts to improve further.
2012: Before Kalydeco
I’ve decided to write a blog so that anyone who is interested can follow my progress with Kalydeco. I was approved on to the Named Patient Program in Australia last week, and hopefully will start Kalydeco later this week.
2012 has not been a great year for me in terms of my health. In December 2011 I was travelling in Vietnam when I reacted to pantoprazole and developed acute kidney failure, with a creatinine of 460. Whilst my creatinine and kidneys are now normal, my reflux has been much worse as I cannot take that class of reflux medications (proton pump inhibitors). I already had a nissen fundoplication at the age of 12, so it looks like there are limited current surgical options.
My lung function has been lower this year, so I have been having IV antibiotics more frequently, about every 3 months. I have had much less energy this year, I cant remember the last time I woke up and didnt feel really tired. I have been struggling to keep up with part time uni and work and have had to decrease my commitments recently. I have been coughing overnight and in the morning much more than usual. So I am looking forward to any possible improvement with Kalydeco!
Hopefully it will arrive this week, I dont think it will seem real until it is here and probably not until I hopefully see an improvement!
Current:
Age: 27
Genes: G551D and F508del, diagnosed at age 3.5
Sweat Test: 102 (110 when diagnosed in 1989)
FEV1: 41% (90-100 as a child, 85% in 2003, 75% 2005-2010, 65% 2011)
Chest: Extensive bronchiectasis according to CT, pseudomonas since age 3 (nothing besides pseudomonas cultured over the last 15 years)
Weight: 55.6kg (55-60 over the last 10 years)
Height: 166.7cm (my BMI of 20.0 is reasonable for someone with CF. I had overnight feeds from age 12-17 so that helped me get to a normal height & weight)
Current Meds: Creon Forte, Azithromycin, Abdeck (vitamin), Ranitidine (reflux), Pulmozyme, Hypertonic Saline, inhaled TOBI as needed, intravenous antibiotics every 3-4 months.
magicbluepill 