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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Momelotinib (OMJJARA) now on PBS

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The MPN AA is delighted to advise that the Australian government has now approved the inclusion of momelotinib, now known as OMJJARA, onto the Pharmaceutical Benefits Scheme (PBS).

Momelotinib was originally developed in Melbourne.  A short news item about its approval is HERE.

What is momelotinib (OMJJARA) and how does it work?

Momelotinib is a JAK inhibitor, similar to Ruxolitinib. By inhibiting JAKs, ‘momelotinib reduces the inflammation resulting from abnormal production of blood cells which relieves splenomegaly and symptoms caused by myelofibrosis.’

Momelotinib also tackles the issue of anaemia. It ‘blocks an additional receptor on cancer cells involved in regulating iron levels in the body, which can mean an increase in the number of red blood cells available. This may result in improvement of anaemia, including the need for transfusion of red blood cells.’

The formal Australian government approval is as follows:  ‘OMJJARA is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib.’

The MPN AA is pleased to advise that we provided a submission to government supporting the availability of momelotinib on the PBS. MF patients who have anaemic myelofibrosis now have another much needed treatment option available to them.

A patient information brochure for momelotinib (Omjjara) has been prepared by the sponsor, and is available from the TGA (Therapeutic Goods Administration) website.
It is titled Consumer Medicine Information and is the last link on that page – HERE.

The MPN AA has also provided a link to momelotinib (OMJJARA) patient information on our MPN Treatments pages.
We hope to provide further patient information on that treatment page in the next couple of weeks.

 

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International review series on MPNs published

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The April 2025 edition of Haematologica features an excellent review series on MPNs.

The authors of the introduction paper to the series, Australia’s Drs Steven Lane and Yin Yuan, explain that ‘the review series is not designed to be a definitive review of all aspects of MPN biology and treatment. Rather, we have selected a few key topics of particular interest to the field that have been informed by recent advances in basic or clinical research’.

In addition to the paper from Drs Steven Lane and Yin Yuan, paper no 4, ‘Pathogenesis and management of high molecular risk myeloproliferative neoplasms’ also features two Australian coauthors, haematologist and researcher Dr Victoria Ling and researcher Dr Megan Bywater.

The papers are all free to access and linked below:

  1. Prevention, diagnosis and management of myeloproliferative neoplasms: an introduction to a review series,
  2. Evolution of myeloproliferative neoplasms from normal blood stem cells,
  3. ‘Clinical and laboratory approaches to target and eradicate early disease-initiating stem cells’:
    Paper is titled New approaches to standard of care in early-phase myeloproliferative neoplasms: can interferon-a alter the natural history of the disease?,
  4. Pathogenesis and management of high molecular risk myeloproliferative neoplasms,
  5. ‘Strategies to prevent or treat the devastating clinical consequence of AML arising from antecedent MPN, also known as blast-phase MPN’. Paper is titled Prevention and treatment of transformation of myeloproliferative neoplasms to acute myeloid leukemia.

 

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PV treatment ‘rusfertide’ trial results

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Early results from the Phase 3 clinical trial of the hepciden mimetic ‘rusfertide’ have recently been published.

Rusfertide aims to overcome the need for phlebotomy in patients with polycythemia vera (PV), by keeping haematocrit below .45.

Early phase 3 trial results show that rusfertide was well tolerated and effective in keeping PV patients’ haematocrit below .45.

These results are extremely promising for PV patients.

The linked article provides more detail, including commentary from Australia’s Dr Cavan Bennett from the Walter and Eliza Hall Institute  HERE.

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Pegasys shortage update: where to from here

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The Therapeutic Goods Administration has updated its advice on a possible shortage of Pegasys in Australia. Its website now states that there is an anticipated shortage from June to September 2025.

To date, prompt action by the supplier of Pegasys in Australia, Echo Therapeutics in partnership with JustMeds, has enabled Australian patients to continue to access Pegasys despite the worldwide shortage.

Echo Therapeutics has written a letter to MPN patients providing more context about the shortage and its likely duration, what is being done to minimise the shortage, etc.
See: Letter-to-Patients-Update-on-Pegasys®-supply-Australia-March-2025.

As to where to from here for MPN patients being treated with Pegasys…..
Valuable information and strategies for patients to discuss with their haematologist are outlined in the Q and A video interview below with haematologist Dr Cecily Forsyth and MPN AA team member and advocate Nathalie Cook OAM.

Multidosing information

During the period of the anticipated Pegasys shortage, some patients may wish to know how some of us multidose from a pegasys vial.  A video has kindly been prepared by haematology nurse practitioner, Jacqui Jagger to provide patients with information on how this can be done safely and effectively (see below).


An information sheet on multidosing has also been prepared – see How some patients multidose.

For future updates, please continue to check the News section of this website.

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Diet, exercise and myelofibrosis: a holistic approach

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The Medscape organisation in the US produces podcasts about Myelofibrosis.

If you’d like to listen to the podcasts or even just read the transcripts, it’s simple to log in via Google, Apple or to set up a log in and password.

The most recent podcast (March 2025) discussing diet and exercise is informative and helpful.

If you’re interested, you can access the information HERE: Diet, Exercise, and Myelofibrosis: A Holistic Approach

This MPNAA website also has a wealth of information about LIVING WELL WITH AN MPN.

 

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ASH 2024 overview podcast from Claire Harrison from the UK

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Haematologist Claire Harrison from Guys and St Thomas’ Hospital in the UK is one of the world’s leading MPN experts.

In this podcast she provides an overview of some of the MPN presentations from the American Society of Hematology meeting (ASH) in December 2024.

Yet again, the amount of research into MPNs here in Australia and world-wide is extremely reassuring for patients.

The podcast can be accessed HERE.

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New Australian research into causes of thrombosis in MPNs

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The MPN AA is delighted to report on some very recent Australian MPN research into causes of thrombosis in MPNs and, in particular, polycythemia vera (PV).
As MPN patients are aware, MPNs are associated with an increased risk of thrombosis. This Australian research has added a new insight into the mechanism underlying thrombosis which can be such a risk for MPN patients, especially PV patients.

This research was presented at the December 2024 American Society of Hematology (ASH) meeting by haematologist and researcher, Dr Indu Raman. The research was undertaken by Dr Indu Raman, Dr Cavan Bennett, and colleagues at Walter and Eliza Hall Institute of Medical Research, Melbourne, Molecular Oncology and Cancer Immunology, Epworth Healthcare, and Royal Melbourne Hospital.

 

The poster presentation and accompanying paper are titled: Dysregulated Complement Activation in Polycythemia Vera: A Novel Mechanism for Thrombosis in Myeloproliferative Neoplasms Uncovered By Proteomic Analysis

Full poster available for download

While research has identified factors such as increased activation of platelets, neutrophils and elevated expression of molecules directly involved in clot formation, underlying mechanisms of thrombosis are not fully understood. This study aimed to investigate potential mechanisms underlying the increased thrombotic risk in MPNs. This was done through mass spectrometry-based proteomic analysis of bone marrow trephines.

To provide some definitions before reading on:
–  Bone marrow trephines are the solid cores of bone marrow tissue recovered from a patient during a bone marrow biopsy.
–  Proteomic analysis is the analysis of the entire set of proteins that is expressed by a genome, that is the genetic information contained in the cells in that bone marrow sample
–  Complement proteins are a group of immune system proteins – some of many identifiable from the proteomic analysis.

Experiments undertaken by Dr Raman and the research team suggest increased activation of complement proteins, a group of immune system proteins. Overactivation of complement proteins can enhance both inflammation and clot formation.

These findings are novel and have not been well described previously. Dysregulated complement protein activation may contribute to the heightened clotting and inflammation observed in PV.

Dr Raman and team advise that further studies are needed to confirm this finding, which may lead to alternative therapeutic options.

Further detail about the research is available via the ASH Abstract

If you have specific questions for Dr Raman or Dr Cavan Bennett, about this research please first contact the MPNAA at mpnaa@mpnallianceaustralia.org.au

The MPN AA thanks Drs Raman, Bennett and the research team for this important research.

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Fedratinib for myelofibrosis? Call for submissions

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At its meeting in May this year, Australia’s Pharmaceutical Benefits Advisory Committee (PBAC) will consider a listing of fedratinib (Inrebic ®) onto the Pharmaceutical Benefits Scheme (PBS) for the treatment of patients with intermediate-2/high-risk myelofibrosis.

To help consider the merits of this proposal for fedratinib (Inrebic ®), the PBAC would like to learn more about myelofibrosis patients’ experience of their disease: symptom burden, treatment, side effects, how patients manage in their daily lives, impact on work, and so on.

The availability of this drug would be a welcome development for myelofibrosis patients.  Currently there is only one Jak inhibitor, ruxolitinib (Jakavi ®), on the PBS for myelofibrosis.

For myelofibrosis patients with anaemia, momelotinib has recently been recommended to government by the PBAC for inclusion on the PBS (although there are more processes to still be considered by government before it may be approved).

Fedratinib, if also recommended by the PBAC and then approved by government, would be an extremely useful addition to the small number of options haematologists have to treat myelofibrosis. Overseas experience indicates that fedratinib is particularly useful in MF patients with low platelets.

NOTE: CLOSING DATE FOR PATIENT INPUT – 26 MARCH 2025

The MPN AA is very supportive of fedratinib (Inrebic ®) being made available to patients on the PBS. It could provide an important treatment option for myelofibrosis patients, especially those with low platelets.

If you have myelofibrosis, please consider making a submission. Or equally providing input to the MPN AA to include in our submission.
Here is a link to the PBAC website where you can make a submission for the inclusion of fedratinib (Inrebic ®)onto the PBS.

At that link, the PBAC has attached a document that clearly sets out what type of information they are seeking from you.  That document is called ‘Copy of public consultation survey for items to be considered by the PBAC (May 2025)’ and we found it helpful to read through that document before completing the survey.

If you’d like some help or to discuss this process with the MPN AA, please don’t hesitate to contact us at mpnaa@mpnallianceaustralia.org.au.

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