Ruxolitinib on PBS for polycythemia vera

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The MPN AA is delighted to advise that, as of today, 1 September 2025, ruxolitinib is available on the pharmaceutical benefits schedule (PBS) for the treatment of adult patients with polycythemia vera who are resistant to, or intolerant of hydroxycarbamide (hydroxyurea). We have copied and pasted below what we believe are the most relevant details from the Pharmaceutical Benefits Advisory Committee (PBAC)’s recommendations.

‘‘…..the PBAC is satisfied that ruxolitinib provides, for some patients, a significant improvement in efficacy over best available therapy. The PBAC noted that while no statistically significant differences in progression-free survival or overall survival were reported in the clinical trial evidence, differences in these outcomes were assumed in the economic model and maintained throughout the 20 year time horizon……’

If you wanted to read any more detail, here is the link to the relevant PBAC page.

 

 

 

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Polycythemia vera – new way of monitoring disease activity?

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A European study has just been released suggesting a new way to monitor polycythemia vera patients’ disease activity.

Titled ‘Neutrophil-to-Lymphocyte ratio as surrogate for JAK2V617F suppression and event-free survival in polycythemia vera’ it explores the value of using neutrophil-to-lymphocyte ratio (NLR) as a means of monitoring PV disease activity.

The study used data from three of the largest PV clinical trials, and evaluated the impact of PV therapies on the neutrophil-to-lymphocyte ratio – and then its relationship with molecular response (reduction in allele burden) and clinical outcomes.

The full article is free to access HERE.

Conclusion

‘This study highlights the distinct effects of Hydroxyurea and Ropeg on leukocyte-derived biomarkers in PV.  While both reduce leukocytosis and neutrophilia -key thrombosis drivers- Ropeg also normalizes NLR, lowers JAK2V617F VAF, and better preserves lymphopoiesis, suggesting potential to reduce thrombosis and modify disease progression.  These findings support NLR as a predictive biomarker, reinforcing the importance of inflammation-targeted strategies in PV management.’

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MPN Education Symposium in Perth and online

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Publicity flyer for MPN Education Symposium 25 October 2025

‘What does optimal management of MPNs look like in 2025?’

MPN AA, in partnership with the University of Western Australia School of Biomedical Sciences and Royal Perth Hospital, is proudly presenting an MPN Education Symposium.

The symposium will be all day on Saturday 25 October, WA time.
It is free to attend in person or online.

Who should attend?
MPN patients, family members, carers, friends, clinicians, researchers and people interested in learning more about MPNs are welcome to attend.
There will be opportunities to ask questions and meet and chat with other participants throughout the day.

FAQs are available on the Symposium registration page.

And for more details, including the Event program, see this page  or use the QR code above. 

Please register to attend either in person, or online. 

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Pegasys update 4 August 2025

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Pegasys® is now available – the Irish stock has arrived, however it will not be on the PBS until 1 September 2025

The MPN AA is happy to share the advice that Pegasys® is once again available for Australian patients. BUT it will only be available as a private script during August.

For a full explanation of the situation, please see the advice from the supplier of Pegasys® in Australia, Echo Therapeutics on the JustMeds website.

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Atrial fibrillation and MPN patients with Jak2 mutation

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A recently published research article may be of interest to MPN patients with the Jak2 mutation. It is titled ‘Incidence, Outcomes and Risk Factors for Atrial Fibrillation in Patients With JAK2V617F-Positive Myeloproliferative Neoplasms‘. The article was published in Cancer Medicine in July 2025.

The researchers retrospectively analysed clinical profiles of 439 Chinese MPN patients with the Jak2 mutation from several hospitals in China between 2017 and March 2025. The researchers’ intention was to consider specific risk factors for atrial fibrillation (AF) in the MPN patient population which they felt “have not been fully elucidated”.

The researchers concluded that:

  • “atrial fibrillation was significantly associated with higher risks of stroke and mortality in patients with JAK2V617F-positive MPN.
  • TET2 mutation and increased IL-1ß were independent risk factors of AF in patients with JAK2V617F-positive MPN.”
  • They also noted that “interferon-alfa and ruxolitinib were associated with improved AF-free survival in patients in the high-risk group.”

The paper is freely available to read in full HERE.

 

 

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Video interview re Pegasys supply

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Haematologist Dr Cecily Forsyth and MPN AA advocate Nathalie Cook OAM have prepared an explanatory video for patients about the current Australian Pegasys supply.

The video addresses the current and future supply issues and outlines strategies for patients and their haematologists to minimise any problems or manage any short breaks in supply.

In summary, the expected timeline for Pegasys Australian supplies is:

May – July
2025		 Just under one month of 135 ug syringes remain.
Australia is currently out of 180 ug supply.
Patients with 180 ug scripts who may not have enough Pegasys supply to last til July may need a 135 ug script for the interim period.
July
2025		 New Irish stock expected to arrive in Australia.  This stock is from the new certified factory in Austria that we will receive stock from later in the year.
Patients with 135 ug scripts may need to obtain 180 ug scripts as the Irish stock will have far more 180 ug supplies than 135 ug supplies
July – November
2025		 Patients will be using the Irish supplied stock and this will continue to be delivered by JustMeds until permanent supplies are in place.
Patients will still need to multidose to preserve supplies.
November 2025 New Australian stock direct from the factory anticipated to arrive

A transcript of this interview is available HERE

Background

On 20 May we posted a  Letter from Echo Therapeutics also explaining more detail around the Pegasys supply situation.

Not sure how to multi-dose?
The MPN AA previously prepared an information sheet on  How some patients multidose.
It includes information on where patients can buy the correct size needles online if their pharmacy or hospital does not have any available.
And below is a short video prepared previously by haematology nurse practitioner Jacqui Jagger showing how to safely multidose.

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Pegasys supply update as of May 2025

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The MPN AA has been advised of some very encouraging news about the Pegasys supply in Australia into the future.

For details, please see the attached letter from the Australian supplier, Echo Therapeutics.

Letter-to-Clinicians Patients and HCPs -Update-on-Pegasys®-supply-Australia-May-2025.

 

 

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“Sentry” myelofibrosis phase 3 clinical trial recruiting in Australia

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An international clinical trial for myelofibrosis patients is currently in phase 3 and is actively enrolling in almost 150 sites across the world.
The MPN AA has been asked to raise awareness because there are trial sites in Australia (see below).  If you have myelofibrosis and think it might be relevant for you, you would need to ask your haematologist.

We understand that this phase 3 trial may be suitable for MPN adults patients with myelofibrosis who have not been treated with JAK inhibitor, have a platelet count >100 × 109/L, and with active symptoms.

The trial is studying selinexor in combination with ruxolitinib versus ruxolitinib plus placebo in patients with myelofibrosis.

This trial’s international identification is NCT04562389.  The clinical trial is titled XPORT-MF-034 (SENTRY), and is in phase 3.

The following Australian trial sites are either active or planned for activation:

LOCATION HAEMATOLOGIST CONTACT
QLD: Icon Cancer Centre – Wesley  Dr Ian Irving
NSW: Liverpool Hospital  Dr Bartlomiej Getta
NSW: Campbelltown Hospital  Dr Bartlomiej Getta
NSW: Bankstown-Lidcombe Hospital Dr Bartlomiej Getta
SA: Royal Adelaide Hospital Dr David Ross

For further information about the SENTRY trial and whether this is something that might be suitable for you, you would need to speak to your haematologist.

Please note that the MPN AA is a volunteer patient group. We are not experts and therefore not in a position to endorse any clinical trial. You need to make your own inquiries together with your medical team.

For further information about clinical trials see the Australian and New Zealand Clinical Trials Registry information for Participants or the Leukaemia Foundation Clinical Trials page.

 

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Interferons in the treatment of myeloproliferative neoplasms

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The MPNAA has just become aware of this extensive article about interferon, written by US haematologists and published in 2024 in Therapeutic Advances in Hematology.

As so many more MPN patients are now being treated with interferon, we have copied the abstract as well as a link to the full article which is free to access.

The abstract explains that ‘Interferons are cytokines with immunomodulatory properties and disease-modifying effects that have been used to treat myeloproliferative neoplasms (MPNs) for more than 35?years. The initial use of interferons was limited due to difficulties with administration and a significant toxicity profile. Many of these shortcomings were addressed by covalently binding polyethylene glycol to the interferon structure, which increases the stability, prolongs activity, and reduces immunogenicity of the molecule.

In the current therapeutic landscape, pegylated interferons are recommended for use in the treatment of polycythemia vera, essential thrombocythemia, and primary myelofibrosis. We review recent efficacy, molecular response, and safety data for the two available pegylated interferons, peginterferon alfa-2a (Pegasys) and ropeginterferon alfa-2b-njft (BESREMi). The practical management of interferon-based therapies is discussed, along with our opinions on whether to and how to switch from hydroxyurea to one of these therapies. Key topics and questions related to use of interferons, such as their safety and tolerability, the significance of variant allele frequency, advantages of early treatment, and what the future of interferon therapy may look like, will be examined. Pegylated interferons represent an important therapeutic option for patients with MPNs; however, more research is still required to further refine interferon therapy.’

Here is the link Interferons in the treatment of myeloproliferative neoplasms

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PBAC has recommended ruxolitinib be listed on the PBS for PV

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We are thrilled to advise that the Pharmaceutical Benefits Committee (PBAC) at its March meeting has recommended that ruxolitinib (jakavi) be listed on the Pharmaceutical Benefits Scheme (PBS) for adult polycythemia vera patients who are resistant to or intolerant of hydroxycarbamide (hydroxyurea).

This does not mean it’s on the PBS and available for us to be prescribed yet.
The approvals process will now continue. For example, cost still needs to be considered by government and so automatic approval and listing is not a given.  Also this may still take some time (eg, as in the case of momelotinib – Omjjara approval for anaemic myelofibrosis).

However it’s a really welcome step in the PBAC approval process and the MPNAA would like to thank those people who made a submission to the PBAC in support of its inclusion on the PBS. The MPN AA also made a submission.

Here is the excerpt from the PBAC report.

Patients may be wondering why the language ‘resistant to or intolerant of hydroxycarbamide (hydroxyurea)’ has been used when so many patients are in fact being treated with Pegasys. However this recommendation is couched in the words of the submission from the pharmaceutical company. That submission in turn was based on results of clinical trials that reported on hydroxyurea versus ruxolitinib.

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