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Change to Pegasys access in Australia from 1 September

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For anyone in Australia who is currently being prescribed Pegasys® (peginterferon alfa-2a) as a treatment for MPNs, the MPN AA wants to update you on some important changes to how you will be able to access this medicine moving forward.

While many of you will have been getting Pegasys® dispensed at a local pharmacy, this will start to change from 1 September 2024, with a new Pegasys® Home Delivery Program coming into effect.

This program is delivered by Echo Therapeutics (the sponsor of Pegasys® in Australia) through its EchoCare™ service, in partnership with JustMeds – a local specialist medicines delivery provider.

This means you will now have Pegasys® delivered directly to your home, workplace or another location, rather than having go into a pharmacy to pick up your medicine.  Importantly, 1 September is not a hard deadline to switch over to the new home delivery program. We know it may take some time for people to adjust to this new process, so there is flexibility built in for you to be able to transition across to the Pegasys® Home Delivery Program at the right time for you.

Echo Therapeutics has engaged with doctors and nurse practitioners who are involved in the management and care of people with MPNs, along with the Therapeutic Goods Administration (TGA). Pharmacists have been informed about these changes and will be aware of the new process.

This Pegasys® Home Delivery Program is a short- to medium-term solution.  It will be in place until Echo Therapeutics is satisfied the local supply chain for Pegasys® is more intact and there is a lower risk of unauthorised export.  Once confirmed, it is expected that Pegasys® will become available in retail pharmacies again.

You can find more information about this in the letter attached or visit https://www.echotherapeutics.com.au/echocarepegasyspatient for more information, and step by step guides on how to access Pegasys through the JustMeds partner provider. https://www.justmeds.com.au/pegasys

Alternatively, you can email rx@justmeds.com.au for more information.

As you know, MPN AA does not promote or recommend specific treatments for people living with MPNs. Decisions about any treatments should be made in careful consultation with your haematologist, after taking into account the potential benefits and side effects for each person’s individual circumstances.

Kind regards,
MPN AA Team

Patient Communication Letter from Echo pharmaceuticals re Pegasys

Patient instruction guides

 

 

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Momelotinib for anaemic myelofibrosis? PBS consultation open

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Image: patient receiving a blood transfusion, something that Momelotinib use hopes to minimise

NOTE: CLOSING DATE FOR PATIENT INPUT – 25 SEPTEMBER

What is momelotinib and how does it work?

Momelotinib is a JAK inhibitor, similar to Ruxolitinib. Ruxolitinib is the standard of care for eligible myelofibrosis patients.
Momelotinib, however, has been shown to tackle the issue of anaemia. It blocks an additional receptor on cancer cells, which can mean an increase in the number of red blood cells available.

Australia’s Pharmaceutical Benefits Advisory Committee (PBAC) is currently considering the inclusion of Momelotinib (known as Omjjara) onto the Pharmaceutical Benefits Scheme (PBS), so it is affordable for all eligible patients.

Momelotinib is already approved and available to patients in the US and Europe, but has not been formally registered in Australia by our regulatory agency, the Therapeutic Goods Administration (TGA). We understand this is being progressed at the moment.

The Federal Government is being asked to include Momelotinib on the PBS if used ‘for the treatment of intermediate or high-risk primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis in patients with moderate to severe anaemia who are JAK inhibitor naïve or have been treated with ruxolitinib.’

Patient input to PBS sought by 25 September

To help the Government in its considerations, the Pharmaceutical Benefits Advisory Committee (PBAC) is now taking consumer comments until 25 September.

The MPN AA is supportive of this drug being available on the PBS – as it provides MF patients who have anaemic myelofibrosis with another treatment option.

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

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Step-by-step guide on how to provide input to the PBS 

COMMENTS DUE BY 25 September

  1. The public consultation survey for the November 2024 PBAC meeting can be accessed via this link: https://ohta-consultations.health.gov.au/ohta/pbac-nov-2024/.
  2. On Page 2, enter your name and contact details and select the category that best describes your reason for input. There are several categories, including for health professionals, but those we believe are more relevant for patients are:
    – Individual who would like to access the medicine to treat own health condition
    – Parent, partner or another person directly caring for an individual from the above group
    – Consumer group/organisation submission
    – Other interested individual (including family members, friends, or members of the public interested in the medicine but not directly caring for an individual currently using or wanting access to the medicine)
  3. At Question 7, select ‘Momelotinib – Omjjara®: Myelofibrosis with moderate to severe anaemia’ for the medicine you are providing input on.
  4. On Page 3, provide your input by answering the questions below in the free text boxes OR by attaching a PDF or Word file at the bottom of the page.
    a.  Please outline your experience with the medical/health condition
    b.  How is the medical/health condition currently treated?
    c.  What do you see as the advantages of this proposed medicine, in particular for those with the medical condition and/or
    family and carers?
    d.  What do you see as the main disadvantages of this proposed medicine?
  5. On Page 4, please declare any conflicts of interest relevant to the responses provided in Pages 2 or 3.
  6. 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 you provided on Page 2.

 

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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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Ruxolitinib and risk of non melanoma skin cancer

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A study published in the January 2024 issue of Blood authored by 28 haematologists and researchers throughout the UK titled ‘Outcomes and characteristics of nonmelanoma skin cancers in patients with myeloproliferative neoplasms on ruxolitinib‘ raises serious concerns that patients on ruxolitinib should be aware of the risk of non melanoma skin cancers. The study followed 90 patients (median age 73) who had developed NMSCs whilst on ruxolitinib therapy.

There is an article on the MPN Research Foundation website in the US  which contains a link to the full study HERE.

“Non-melanoma skin cancers in ruxolitinib-treated patients with myeloproliferative neoplasms (MPNs) behave aggressively, with adverse features and high recurrence. In our cohort, mortality from metastatic NMSC exceeded that from myelofibrosis,” the UK study authors report.

Ruxolitinib can be effective in reducing spleen volume and symptom burden as well as potentially prolonging survival in responding patients. “However, benefits need to be balanced against potential toxicities…” the authors suggest.

“Our study highlights the aggressive nature of NMSCs in ruxolitinib-treated patients with MPN, the importance of counseling patients about the risk of skin cancer before starting ruxolitinib, and a requirement for close dermatological monitoring on treatment.”

Optimal MF management following diagnosis of NMSC remains uncertain, according to the authors. “Stopping ruxolitinib may result in MF (myelofibrosis) symptom flare and potentially increase the risk of disease progression, and it is not yet clear whether ruxolitinib cessation (or switching to an alternative JAK inhibitor) impacts NMSC outcomes. Consequently, if a patient develops an NMSC while taking ruxolitinib, the risks and benefits of each treatment option need to be carefully weighed and discussed with the patient, acknowledging the uncertainties alluded to above, before deciding whether to change therapy.”

“Larger, prospective collaborative studies are needed to better understand NMSC risk and outcomes in ruxolitinib-treated patients with MPN, the report concludes, as are similar evaluations of NMSC risk in patients with MPN treated with other JAK inhibitors.”

 

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Important new study – high WCC as risk factor for blood clots in PV

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REVEAL study

Findings from an important new study for the management of polycythemia (PV) patients have just been released.

Called the REVEAL study*, it shows that leukocytosis (a high white cell count) is a risk factor for thrombosis (blood clots) in patients with PV. The study showed thrombosis to occur in high and low-risk patients, and even in patients in whom hematocrit was adequately controlled.

To date, known risk factors for thrombosis in PV patients included advanced age, previous history of blood clots, and elevated hematocrit >.45. There was insufficient data to include elevated white blood cell (WBC) counts as a known risk factor.

However, in an analysis of 2271 PV patients, the REVEAL study found 142 blood clots in 106 patients.
Specifically. during the study period of just under 4 years, it identified:

  • Significant associations with initial blood clot occurrence for hematocrit level >45% and WBCs >11 × 109/L .
  • Elevated WBC count was significantly associated with initial thrombosis in both low and high-risk PV patients.
  • Even when hematocrit was controlled at 45% or lower, WBC count >12 × 109/L was significantly associated with thrombosis.
  • There are different risk factors for arterial and venous thrombosis, an observation that has also been reported in essential thrombocythemia. Both leukocytosis and thrombocytosis (high platelet count), poor hematocrit control, and age were associated with a higher risk of arterial thrombosis, whereas female sex, history of previous thrombosis, and leukocytosis turned out to be the main risk factors for venous thrombosis.

The study’s authors feel that, even without a large study proving that lowering leukocytosis reduces blood clots, there is sufficient evidence to ‘support the inclusion of leukocytosis in the definition of high-risk polycythemia vera’. They also suggest that two elements of clinical practice could be changed:
– ‘low-risk patients with persistent leukocytosis could be elevated to the high-risk category and, therefore, be candidates for cytoreduction’ and
– ‘normalization of leukocytes should be included as an objective of treatment in patients receiving cytoreduction.’

There are two abstracts available discussing the REVEAL study’s findings.

REVEAL puts leukocytes into risk stratification 

Association between elevated white blood cell counts and thrombotic events in polycythemia vera: analysis from REVEAL

* REVEAL is a prospective observational study that enrolled 2510 patients with PV, with median follow-up of 44.7 months (range, 2-59 months). REVEAL stands for The Prospective Observational Study of Patients with Polycythemia Vera in US Clinical Practices.

 

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QIMR Berghofer Medical Research Institute – Blood cancer treatment could be transformed by discovery

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QIMR Berghofer Medical Research Institute researchers have discovered a potential new treatment for Acute Myeloid Leukaemia and other cancers.

Watch their exciting news HERE

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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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Polycythemia vera: 2024 update on diagnosis, risk-stratification and management

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MPN specialist haematologists, the USA’s Ayalew Tefferi and Italy’s Tiziano Barbui have just released a very extensive overview of polycythemia vera.

The overview is detailed and includes:

  • an important update that recognises interferon as a first line treatment option along with hydroxyurea, as well as much discussion of the latest research into treatment options,
  • advice on pregnancy management in PV patients,
  • a detailed explanation of the genetic landscape and potential implications,
  • discussion of research on life expectancy,
  • and much more.

The article appears in the American Journal of Hematology and is open sourced and can be accessed HERE.

If you’d like to see more articles on polycythemia vera, our guidelines page can be accessed HERE.

 

 

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MPN experts: Roundtable series on PV

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MPN specialists  Professor Claire Harrison, Guy’s and St Thomas’ NHS Foundation Trust from the UK, Professor Jean-Jacques Kiladjian, Saint Louis Hospital, Paris,  and Alessandro Rambaldi, MD, University of Milan have collaborated to provide an up to the minute roundtable series on management of polycythemia vera (PV). (Courtesy of the Oncology Learning Network).
Each video includes an accompanying transcript.

Part 1: Hematocrit control and cardiovascular assessment – HERE

Part 2: Aims of treatment and choice of appropriate first-line therapy – HERE.

Part 3: Identifying when first-line therapy for PV might be failing – HERE

Part 4: Second-line therapy and reviewing data on Ruxolitinib – HERE

 

 

 

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PV – hematocrit control and cardiovascular assessment

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A recent presentation from MPN specialists Professor Jean-Jacques Kiladjian, Saint Louis Hospital, Paris, and Professor Claire Harrison, Guy’s and St Thomas’ NHS Foundation Trust from the UK discusses the importance of maintaining hematocrit control in patients with polycythemia vera (PV) as well as monitoring cardio-vascular health.

The discussion, part 1 of an expert roundtable series, is wide ranging and covers the risk factors for patients with polycythemia vera.
Of particular interest is the manner in which cardiovascular health is treated by Professor Kiladjian’s team in Paris.

Claire Harrison: “Jean-Jacques, we’ve recently been looking across some PV patients and thinking about how do we do cardiovascular risk stratification and assessment. I found out that we do this in a slightly different way, and I think you do it in the best way, so tell us how you do that in Hospital Saint-Louis.”

Jean-Jacques Kiladjian: “I’m not sure it’s better, but, yeah, it’s different. Well, we have a quite aggressive and proactive attitude regarding cardiovascular assessment of all patients with PV regardless of age. Obviously the older they are, they’re usually already followed by a cardiologist sometimes or they already had some cardiovascular events.

But even in younger patients with absolutely no history of cardiovascular events, we frequently refer them to a cardiologist, not any cardiologist, but a cardiologist that knows what we are looking for, that these patients are at high risk of cardiovascular problems in the future, that they will be followed for decades and we need this proper assessment.

At least an echocardiogram, doppler of arteries, peripheral arteries, is performed. We ask to perform that for almost every patient at baseline and then regularly every 2 to 3 years in patients without any risk factors, more frequently if needed. So yes, we have a quite very proactive attitude against these additional factors because we also noticed that—and maybe this is a field for the future as Alessandro said—that it’s not exactly maybe the same risk factors for arterial versus venous thrombotic events.

Maybe some characteristics of the disease, of the patients, may predispose more to arterial events that are more dangerous for the patients like myocardial infarction, stroke, et cetera, and these risk factors may be different. And the cardiovascular, let’s say landscape of the patient at baseline is very important to avoid and to prevent these arterial events.”

The full presentation with transcript is available HERE.

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