Availability of Jakavi (Ruxolitnib) for gvhd in UK
Hi. I currently have chronic sclerotic gvhd, and can hardly manage to mobilise for it. My consultants inform me that the only real medication that will help alongside my current Ecp treatment is Jakavi. Currently as you are aware it is not approved for gvhd in uk but I hear Anthony Nolan is campaigning to have it approved. My life is currently on hold and I am on benefits and have a very low morale. Are you able to let me know if we are any closer to getting it approved for chronic gvhd as I am desperate for it.?
Best Answer
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Hi Dave,
Please accept my apologies for the delay in getting this update to you. I’ve just emailed it over as requested.
Here’s a summary, for anyone else on the Forum who may also be interested:
Sadly ruxolitinib (Jakafi) is still not available in England. The reason: it is now a licensed medication for GvHD which means that, according to NHS England policy, it must be considered by NICE (the National Institute for Health & Care Excellence) before it can be funded by the NHS.
When ruxolitinib was previously available from 2020-22, it was because it was unlicensed for GvHD at the time, so NHS England were able to allow its use temporarily to support with keeping patients out of hospital during the COVID-19 pandemic.
When that temporary policy came to an end last year, NHS England said the system would only start funding it again if the manufacturer submitted ruxolitinib to NICE. The manufacturer (Novartis) has not yet done this.
Anthony Nolan, in collaboration with the BSBMTCT (British Society of Blood and Marrow Transplantation and Cellular Therapy), have tried to get Novartis and NHS England to resolve this situation.
We are waiting to hear from Novartis if/when they think they might submit to NICE, but they have told us this could be a while.
On a more positive note, there is a new drug for chronic GvHD that is currently being considered by NICE for use in England. It is called Rezurock (belumosidil). Anthony Nolan is contributing to the appraisal to illustrate why more treatment options for GvHD are needed. We hope that NICE will make a decision on Rezurock in Sept 2023, although it could be sooner.
I hope you find this update helpful. If anyone has any questions or concerns about your GvHD, and how it is being treated, please continue to raise them with your healthcare team at the earliest opportunity. They will have the fullest understanding of your treatment options going forward.
We can also give you general information on GvHD on our Helpline (0303 303 0303) or you can email us at: patientinfo@anthonynolan.org
All the best,
Tom
Answers
If not available soon, can it be purchased privately?
Hi Dave, I hope someone from Anthony Nolan can give you some facts on this.
You could contact your mp or set up a crowd funding thing. Or try make contact with someone else who's managed to get it to find out how they did it.
I'm really sorry for you. This is awful.
If I get any ideas, however wierd or wonderful, I'll let you know. There's nothing to lose.
Helen
Has anyone else recently been able to get Ruxolitnib subscribed for chronic gvhd in UK?
Hi Dave,
Just info from my side, but I'm not in the UK. my immunsuppressants are not only jakavi.
I also have Myfortic and maybe Cotrim is one too, not sure, and also aciclovir. Maybe you can get them prescribed.
I'm thinking of you a lot. Sorry I didn't take your situation seriously. I didn't get it. It's not fair. It must be awful. If I get any more ideas, I'll let you know.
Love
Helen
I guess nobody from Antony Nolan look at these posts anymore to know how far Anthony Nolan are with thier campaining?
Hi Dave
Call their Hotline. They're doing their best. It's difficult for you to be active when you're suffering so much but stay on it.
Maybe there's a study going on that you could join. Anthony nolan will tell you.
Best... and love,
Helen
Hi. Spoke to Tom on patient services hotline at Anthony Nolan. He will try and get update for me on Jakavi and let me know tomorrow.
Hi Tom,
Many thanks for your reply. It is really appreciated.
That's a relief Dave. Thanks for letting me know. But of course, you need some good news. I'm thinking of you, hoping with you.
Justcame across this on gvhd facebook website. It looks good. Seriously reduced med prices.
I only searched jakavi and it came up.... not available, notify when available.
Best of luck all,
Helen
Hi Helen,
Thanks for looking, I really do appreciate it. These days I seem so fatigued I never seem up to doing much research. I was never like this before. Not sure if is the low dose steroids. I have had a look at your website and tried to input rezurock as well which may help me but it can’t find that either. If I enter an immune suppressant cellcept which will not help me it says prescription only. Thanks for trying. I may try Facebook group to see if they know of any other jak2/jak2 inhibitors that target fibrotic skin.
It's understandable that you're not in the mood, have the energy to research. We're allowed to feel **** Dave, I was told by ally in the Facebook group that it's ok to be in bed all day and have Pyjama days. Go with it. Don't criticize yourself. This helped me a lot. I'm so critical of myself too. Let's ease up on ourselves. I'm not like it either but it's ok.
Sleep well Dave
Hi Helen ,
Many thanks. Because I am so fatigued and immobile all my days are pyjama days. I was never like this before. It worries me. I hope if I can get off these low dose steroids I will improve a bit. I joined the prednisone uk group and lots of people suffer really badly when on low dose prednisone with fatigue, not being able to walk upstairs, muscle weakness, joint pain, big mood swings, not being able to get out of bed. So hopefully can come off them soon as not doing anything positive for me, as not helping my gvhd.
Hi Dave
Glad to hear you've found the prednisone uk group.
Here's something from the facebook group from today. I did straight copy and paste.
"Jakafi helped me get off steroids for good. I really didn’t notice any side effects. I also had skin and gut gvhd. Also eye and mouth. My gut was the worst. Kept me on steroids forever until Jakafi. I get a grant through something called the PAN Foundation. I have no co-pay. Call Jakafi directly and they can help you figure out help with the cost. Good luck!"
Enjoy your day Dave, pj's or not. I'm thinking my flare up might have gone to my lungs, I'd got through that one, ticked it off. I'll wait and see.
Kindness and compassion to us all,
Helen
Hi Helen,
many thanks. I hope you do not get flare up to the lungs as I think probably worst place to have it. We are all suffering in our own ways. I hope we all start making progress soon as it mentally helps. I think without noticing physical progress it does affect us in a negative fashion.
My son has severe chronic pulmonary gvhd. He started on rezurock approx 3 weeks ago. The Royal marsden are his primary carers.
The cost is £7.5k per month and we did a go fund page to help raise funds. We have raised enough for 5 months and then hope it will either be nhs approved or if not, they will agree to fund the remaining treatment if my son is responding. He is 20 and was diagnosed with PMAL at 17.
Hi Su0906,
I am really happy you have managed to get Rezurock for your Son and I hope he gets improvement really soon, as I know how hard it is to suffer with gvhd as I am still suffering 2 years out from transplant. I would really like to have rezurock to help loosen up my tight skin and give me some mobility back. Since getting gvhd it has really affected me psycologically and I could never concentrate on putting a go fund me page together for me, and I would never raise enough for one month. Hopefully NICE will approve it for NHS in or before September and will become available on prescription. Hopefully also soon Axatilimab may also become available but believe it is currently under trials. Good luck it has improvements shortly.
Hi Dave,
I met my dermatologist this morning before my ecp.
I asked her a few questions and she's been quite helpful... we'll have to see what really works.
Her aim is to soften up the thick , hard layers of skin (maybe a bit like yours) I don't have the prescription yet, but I'll let you know.
I can shower but should just disinfect afterwards and moisture but not put lots on the open wounds.
She said I could swim with waterproof plasters and disinfect/moisturise afterwards.
Instead of physio massage, because they can't massage my skin anymore cos it's so broken and weepy, she said movement and gymnastics. Of course not the normal, very very gentle.
I was just reading your posts and it feels like the steroids are really holding you back. I had them for ages too. I got really depressed on them.
Don't forget, you're not that person, like you say yourself. I have had soo many pyjama days... I was doing what i needed. It's more than ok, it's right.
Do please be kind to yourself Dave. You're doing really well. I can't imagine how you're coping. You can be proud of yourself.
The ecp was mixed, one awful day, really awful, and today went really easy. The nurses yesterday got stressed because my veins weren't working (very high thrombozytes (sticky cloggy blood) and dehydrated) and so we all got stressed, the machine too... peeping. Quite horrible. One particular nurse gets really uptight and takes it out on others.
Take care Dave, I could do with a coffee if you want one too? Milk? Maybe you could bring the biscuits:)
Love Helen
Hi Helen,
you state you are waiting for prescription? Is that for Rituximab? I believe I have Myositis and my main issue is I believe because of this bought on by gvhd. Myositis being muscle inflammation and this I believe why muscles are so week and I fatigue so easily. I do believe I also have some skin gvhd but how bad I am not sure as I believe the Myositis is masking it. Rituximab looks like a quite good 2nd line cure in UK for muscoskeleter issues such as myositis. This effects mainly my thighs that feel they are continually swelled and sort of numb because of it and also burning and tingling in thighs which are all also typical of Myositis. When I go to clinic next week I will see if I can mri to confirm or not if I have Myositis. Today my I went to day therapy clinic where I had my third Rituximab infusion over hours. So far it has been a month since my first infusion and to date I have not really seen improvements yet, but hoping I may get some slow improvements in next month. Again I have been too impatient hoping to see some results by now.
sounds like good news for you.! You are able to shower and swim. Be careful though as you don’t want infection and up as inpatient. Softening the skin sounds a good idea as it is likely to heal better if soft, but how do they plan to do that? Rezurock sounds like a good drug for that but it again takes time.
I am currently on really low prendisone steroids 5mg every 2 days. I don’t know how much the steroids contribute to my muscle weakness, mood swings, I just hope the doctor takes them down soon so we can stop.
as my progress does seem so non existence currently and has been like this for some time now, I guess it worries me as I need to sort my my mobility issue otherwise nothing else will improve, so I guess I am sometimes hard on myself and I am not really a person who sits around to wait for things to happen, I like to go and do, and I can’t do that I just have to wait and is frustrating and this gets me on low ebb. It is worse as Ias I have been like this for so long.
on your note I hope doctor has something she has something to soften your skin that does not take to long. I know Ecp also does this but it takes so long.
hoping for us both that we start having more gradual improvements now we are into our third year after sct where things should hopefully get better.
would love a coffee.
lots of love Dave
Hi Dave,
Just a quick update, I've got to eat something.
So far today I'm very sceptical about everything the dermatologist said yesterday. She, and a nurse, bandaged my feet ankles and last night I had lots of fluff stuck in the open wet wounds and caught pulling and tearing, pulling the flakey skin off. I doubt her permission to swim is sensible too. She said in the same breath not to take a bath. All a bit dodgy. She said she'd give me this that and the other and nothings materialised yet. Patience ;) But I was really low this morning. My senior gymnastics helped me get out of it. Being helped by 80 year olds and being the only one sitting... but still..
Let's have the coffee later, just got to force myself to eat for now LOL.
Hi Helen,
Try not to get too low. I know it is easier said than done in our condition. I understand what you say about the wounds and being sceptical. For a wound to heal you want it to scab and the more it is interfered with the more chance for the scam to come off. You also don’t want bits and pieces in there if it is weeping. I think a lot of what doctors say is to try and help pick up up. Mine does not really say anything. Don’t know which is worse. I am struggling like you to eat, never really get feel for hunger so forcing things down myself to eat, I keep hoping this will one day go. Everything seems to be at a standstill for both of us. I just hope our bodies don’t leave us here as I want to improve as I am sure you want to. I hate hanging around waiting for something to happen.
keep the coffee pot hot
Hi Dave,
I won't get rituximab. I trust my team with that.
The dermatologist doesn't belong to my sct team thank goodness. I just can't get an appointment with another dermatologist practice so she comes to me when it's aaabsolutely necessary. I won't bother again. She's based in the university dermatology department. I've got an appointment in a different clinic in July up in the Black forest. I'm sceptical to say the least. Maybe it's good to be cautious. I can so easily get my hopes up. It's not a good idea.
I found a good thing that seems to have helped me emotionally Dave. It's an ap from the BBC called reels. There's a short video on it about changing the way we feel about very dark challenging experiences. If you want I can try and post it here, or on Facebook. I was sceptical and a bit scared, but it's light and helpful.
By for now, thanks for being there for me.
Helen