We are delighted to announce that NICE (the National Institute for Health and Care Excellence) has recently released Final Guidance recommending Ruxolitinib for treating Polycythaemia Vera (PV) in adults in England who cannot tolerate Hydroxycarbamide (also called Hydroxyurea) or when the condition is resistant to it.
Ruxolitinib is already approved, in similar cases, for the treatment of PV patients in Scotland and Northern Ireland and we hope that the NICE recommendation will also be adopted in Wales in due course.
This positive news follows a lengthy appraisal process by NICE, during which MPN Voice, in conjunction with Leukaemia Care, made written submissions on behalf of all PV patients and their families & carers, including results from the UK-specific MAJIC-PV clinical trial, which demonstrated how effective Ruxolitinib is in treating PV patients for whom Hydroxycarbamide hasn’t worked.
Members of the MPN Voice advocacy team, a PV patient who was part of the MAJIC-PV trial and representatives from Leukaemia Care also attended NICE committee meetings at which the suitability of Ruxolitinib as an additional treatment option for PV patients in England was fully evaluated.
We would like to thank the clinicians who worked with us throughout the appraisal process and in particular a number of PV patients, including some who had taken Ruxolitinib as part of a clinical trial, whose first-hand experience of what it is like to live with PV and of the existing treatments, added invaluable real-life testimony to the submissions that we made to NICE.
Polycythaemia Vera can be an extremely debilitating illness that has a significant impact on patients’ lives in terms of day-to-day symptoms and affects not only patients but also their families and carers. Existing therapies for PV can have limitations and side effects and may not adequately reduce many patients’ main symptoms, including fatigue, bone pain and pruritis. Even in cases where patients initially respond well to the existing therapies, those can lose their effectiveness over time. We therefore welcome the approval of Ruxolitinib as an additional treatment option for those PV patients in England who are either intolerant of or unresponsive to treatment with Hydroxycarbamide.
This guidance from NICE underlines how important it is that we continue to support research into the development of new treatments for MPNs. The work that the volunteers on our advocacy team do is a key part of this but that also relies on very valuable input from the whole MPN community, through responses to surveys and questionnaires and above all through fundraising to help us continue our work. If you’d like to know more about what we do and want to help us achieve our mission through volunteering for MPN Voice or helping to raise funds we’d love to hear from you at info@mpnvoice.org.uk.
Written by
Mazcd
Partner
To view profiles and participate in discussions please or .
I have this morning taken my first dose of Rux after about 20 years of hydroxea. The side effects were getting intolerable so extremely grateful I now have this opportunity thanks to the hard work and skill of all those involved.
Fatigue, headache, bone pain, itching were the worst. Not everyday but got worse over the last 12 months. But for many years it controlled my bloods with occasional severe side effects that didn’t last too long. All the best
I've been on Hydroxy now for twelve and a half years with no side effects whatsoever - the only physical indication that I have PV Jak2+ has been fatigue.
However, these last six months have seen a marked change in HCT and platelet levels so after exhaustive test's and increase in Hydroxy dosage (which have brought the HCT levels back to normal) platelet levels remain stubbornly high, so my haemo has decided to move me on to Rux after months of head-scratching trying to work out what's going on....
With progression confirmed as not occurring concensus seems to be that the Hydroxy simply isn't working for me as well as it has in the past.
So, tomorrow morning, after duly signing the relevant forms, I'm being moved over to give Rux a try. I hope to goodness that I'll be as tolerant of this drug and that it has the desired effect and I'd be happy to appraise the Forum of how things progress if there's any interest. I'm lead to believe that it can take around sixteen weeks for things to stabilise but as we're all different, guess it'll be a case of how long's a piece of string ?
This is excellent news! I was a Majic trial patient, currently on Ruxi. Do we know when we’ll be able to get it through standard NHS prescription channels as opposed to named patient supply?
I’m very happy about this outcome as living in Wales and have arranged an appointment with my haematologist straight away to discuss the change to Ruxolitinib. I didn’t tolerate Hydroxycarbamite and changed to Interferon some time ago but the symptom burden has been debilitating, particularly fatigue and pains to the point that I’m on long term sick and at risk of losing my job. I really hope I qualify for Ruxolitinib as there might be a chance that it will improve my symptoms and my quality of life. Fingers crossed 😊
Content on HealthUnlocked does not replace the relationship between you and doctors or other healthcare professionals nor the advice you receive from them.
Never delay seeking advice or dialling emergency services because of something that you have read on HealthUnlocked.
treatment refusal ruxolitinib
Pegasys is applying for formal approval in MPN treatment
Finally start treatment 
