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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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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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February 2025 MPN Education Foundation patient conference videos

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Every two years the MPN Education Foundation hosts a wonderful patient conference in Scottsdale Arizona, with some of the most distinguished MPN expert presenters. The conference is titled the Joyce Niblack Memorial Conference, and is hosted by the Mayo Clinic.

We are very fortunate to be able to view the conference video presentations and slides.

Here is a list of presenters and topics:

Jyoti Nangalia – MPN Molecular Biology

Rupali Bhave -Managing ET in 2025

Raajit Rampal – Managing PV in 2025

Naveen Pemmaraju – Managing ET in 2025

Ruben Mesa – Guidelines & Gaps in MPN

Jeanne Palmer – Transplants in MPN for 2025

Marina Kremyanskaya – New Drugs for PV

John Mascarenhas – New Drugs for MF

Angela Fleischman – Nutrition & Complementary Approaches for MPNs

Jean-Jacques Kiladjian – Long Term Outcomes of Interferon in MPNS

Steffen Koschmieder – Inflammation in MPNs

Susan Leclair – Learning about my Labs: An MPN Patient Guide

 

 

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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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Pegasys: new way for Australians to order via online link

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The company dispensing and arranging delivery for Pegasys® has set up an online ordering link to speed up the ordering process for Australian patients.

This should save patients time and effort and enable patients in Australia who need their Pegasys urgently to get through to the dispensing team.

The link to order can be accessed HERE.

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Pegasys supply update October 2024

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Echo Therapeutics Pty Ltd. has asked us to share this update about Pegasys® with Australian patients.
It is about “an anticipated shortage of Pegasys® in Australia.”

See attached letter from Echo Therapeutics for more details.
Pegasys anticipated shortage letter

This shortage notification appears on the TGA website as an anticipated shortage with supply impact dates shown as March 2025 to June 2025.

The TGA alert notes that “The sponsor is closely controlling the supply. This medicine can be accessed through the Pegasys Home Delivery Program.”

Myelofibrosis with cytopenias – recommendations for routine clinical practice

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A letter in Leukemia has just been published with recommendations from a global consensus group about management of  myelofibrosis (MF) patients with cytopenias. The authors explain that:

‘National and international guidelines exist for the management of MF; however, a need remains for practical guidance applicable in everyday clinical practice, especially for patients experiencing cytopenias or potential failure of current therapy. The landscape is further complicated by the availability of multiple prognostic tools for MF; as such, clinicians may find disease prognostication challenging and confusing. Additionally, to maximize clinical applicability of trial data, inclusivity of eligibility criteria in the context of the real-world MF patient population should be considered.’

‘Recognizing these significant challenges, an international expert consensus group was established to provide best practice recommendations for healthcare professionals, intending to supplement, but not replace, existing guidelines.

25 key clinical questions across five consensus themes were identified:

  1. Defining the thresholds for anemia, and when to initiate/modify treatment
  2. Defining the threshold for thrombocytopenia and when to initiate/modify treatment
  3. Defining JAKi failure and what would warrant switching treatment
  4. How and when to determine prognosis in patients with MF
  5. Unmet needs in MF clinical trials

Consensus recommendations.

Consensus guidelines – see Table 1
Current and emerging treatments for anemia in myelofibrosis – see Table 2

‘In this collaboration between an international panel of physicians with expertise in MF and a diverse Extended Faculty of hematologists and patients, a high level of consensus was achieved on recommendations addressing a wide range of critical gaps in MF management. These recommendations provide a valuable framework to support clinicians in optimizing care for patients with MF’

A link to the full letter is available HERE.

 

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Updated British guidelines on myelofibrosis

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The British Society for Haematology has just released (November and December 2023) new guidelines for myelofibrosis.

As these are British guidelines, not all treatment options are available in Australia and Australian practices may vary.
However the guidelines provide useful insight into diagnosis and prognosis considerations as well as the growing array of treatment options becoming available for myelofibrosis patients.

There are two parts to the guidelines:

The management guideline includes detailed advice on specific situations such as:

  • Management of myelofibrosis-associated anaemia
  • Management of thrombocytopenic patients
  • Role of splenectomy in myelofibrosis
  • Myelofibrosis in paediatric, teenage and young adult population
  • Myelofibrosis and pregnancy
  • Allogeneic stem cell transplantation for myelofibrosis
 

 

 

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Myelofibrosis drug Momelotinib approved by the US FDA

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Here is an Australian news item about the US FDA approval of Momelotinib, a new drug for Myelofibrosis. This is significant as there are few treatment options for those with myelofibrosis, and this is another option for those for whom other therapies are unsuitable. We would love to see it made available to Australian patients in the future.

 

 

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Interferons in MPNs: a conversation with an MPN specialist

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Anne Brazeau, the founder and CEO of MPN Education and Advocacy International has kindly made available an excellent wide ranging video about interferon use in MPNs.

  • The video showcases a presentation about interferons by Gabby Hobbs, MPN specialist and Clinical Director of Leukemia Service at Massachusetts General Hospital as well as being an Assistant Professor at Harvard Medical School.
  • Dr Hobbs discusses issues that haematologists take into account when considering interferon use in pregnancy. (35 minutes into the video).
  • Dr Hobbs also responds to a range of questions from MPN patients about interferon.

Note that while the video refers often to Besremi which is a longer acting pegylated interferon available in the USA, Europe, and some other countries, Pegasys is also a long acting interferon and Australians are extremely fortunate that it is available on Australia’s Pharmaceutical Benefits Scheme (PBS).

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