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 details of poster presented at ASH by Dr Indu Raman

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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More benefits of exercise

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Happy new year to MPN patients and their families.

Over a year ago, we publicised an article from Australian oncologist and author Dr Ranjana Srivastava, about the benefits of exercise.  Dr Srivastava stated that  ‘in addition to standard [cancer] treatments, exercise really is one of the best daily “pills” cancer patients can take to improve their life expectancy. Indeed, if exercise could be marketed as a pill, it would be an outright blockbuster.‘

In the last few days, the MPN AA has seen some further information and evidence about the benefits of exercise…. and because it’s a new year, we thought you might appreciate being inspired by them.

  1. Some practical easy to implement advice  from The Conversation
    ‘Five ways to make your daily walks even more beneficial’. 
  2. Recent findings from researchers at Stanford university, posted by MPN haematologist and researcher Dr Ann Mullally. It is a video interview (with transcript) from PBS News in the US.
    How exercise may be the ‘most potent medical intervention ever known’.

Caution: Haematologists and GPs still caution however to be sure to check with your GP and haematologist before launching on any new exercise program and start slowly and gently if you haven’t exercised before or not for some length of time.

Best wishes from the MPN AA for a wonderful and healthy 2025.

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Patients with CalR mutation – promising ASH update

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The American Society of Hematology’s December 2024 conference included developments in treatments for MPN patients with the CalR mutation. A range of Australian and international immunotherapy research is underway and some treatments are already in clinical trials. It is too early to present results.

A fascinating video about CalR treatment research is linked below.
In the video, Drs Alex Rampotas and Zoë Wong explain that there are already specific immunotherapies being trialled against the CalR mutation, all of which may well be effective.  They specifically mention a ‘B specific T cell engager’ and ‘a blocking antibody against it’.

However their collaboration is about a third type of immunotherapy option, a novel second generation CAR-T cell therapy. They advise that CAR-T is the ‘strongest immunotherapy so potentially  ……. able to overcome some of the immune suppression of myelofibrosis and directly eliminate the malignant stell cells.’

The full video is 8 minutes long, unfortunately with background noise.  However if you have the CalR mutation, watching the video will give you a Christmas present of great promise!

https://www.vjhemonc.com/video/qsoln_mbhbk-development-and-evaluation-of-a-first-in-class-car-t-therapy-against-calreticulin-mutant-neoplasms/

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Ruxolitinib available for some PV patients? PBAC call for submissions by 29 January 2025

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In March 2025, Australia’s Pharmaceutical Benefits Advisory Committee (PBAC) will consider an extension to the existing listing of Ruxolitinib (known as Jakavi®) onto the Pharmaceutical Benefits Scheme (PBS). The extension sought will be for the treatment of adult patients with PV who are resistant to or intolerant of hydroxycarbamide (hydroxyurea).

NOTE: CLOSING DATE FOR PATIENT INPUT – 29 JANUARY 2025

To help the Government in its considerations, PBAC is taking consumer comments until 29 January 2025.
* See below for advice on making a submission.

The MPN AA is supportive of ruxolitinib being available on the PBS for PV patients. It would provide another treatment option when first line therapy fails. Thanks to the MAJIC-PV trial conducted in the UK, Ruxolitinib has now been established as a viable second line treatment option for PV.

For more information please see our News post about the Majic-PV trial.

*Next steps if you’d like to make a submission

Making a submission to the PBAC is a way for PV patients to have a voice by explaining how having access to this treatment could impact on their own quality of life.

If you follow the instructions below you will be able to make your comments via a simple process.

  1. To comment for the  2025 PBAC meeting, start by clicking on this LINK.
  2. On Page 2, enter your name and contact details and select the category that best describes your reason for input, ie:
    – Individual who would like to access the medicine to treat own health condition
    The drug you are commenting on is ‘Ruxolitinib Jakavi®.
    You will see that this is a ‘Resubmission to request a General Schedule Authority Required (STREAMLINED) listing for the treatment of adult patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxycarbamide (hydroxyurea).’ This is because the drug company unsuccessfully sought approval in 2019. However as more results are now available from the MAJIC study, the pharmaceutical company is resubmitting its application.
  3. On Page 3, provide your input by answering the questions in the free text boxes OR by attaching a PDF or Word file at the bottom of the page.
  4. On Page 4, please declare any conflicts of interest relevant to the responses provided in Pages 2 or 3.
  5. When you have completed the form, select ‘Submit Response’ on Page 5. You will be sent a confirmation receipt and link to a PDF copy of your response to the email address you provided on Page 2.

If you’d like any support in making your submission, you can contact the Department of Health at HTAconsumerengagement@health.gov.au or watch one of their facebook Q&A recordings HERE.

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