Promising future for MF targeted therapies

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A review article dated 2 May 2022 in the American Cancer Society Journal ‘Cancer’ titled ‘Defining disease modification in myelofibrosis in the era of targeted therapy’ has outlined a promising way forward for myelofibrosis (MF) targeted therapies.

The authors are several renowned MPN specialists from around the world and their article refers to ‘exciting developments’. Their conclusions are extremely encouraging for all MPN patients.

Currently, the only cure for MF is an allogeneic stem cell transplant and treatment criteria for MF have mainly focussed on relieving symptoms rather than survival benefit.  However in this review article, the authors outline several promising clinical trials able to modify the disease course in MF.  While these trials have had relatively small patient numbers, the authors’ view is that the treatment landscape for MF is ‘set to evolve rapidly as understanding of the molecular pathogenesis of MF sheds light on novel therapeutic targets and the possibility of selectively depleting the malignant HSC compartment.’

The article concludes:
‘In summary, the possibility of disease modification has the potential to revolutionize clinical practice and treatment decision-making for patients with MF. As novel end points begin to emerge, it will be important to re-evaluate clinical trial designs, and potentially redefine disease modification, adding new end points to survival outcomes, to ensure the true potential for disease modification and MF therapy is realized. Standardized definitions and assessments are needed across clinical trials, along with the inclusion of patients with newly diagnosed disease, where the greater potential for disease modification may lie.’

The full article is unlocked and able to be read HERE.

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Updated European LeukemiaNet recommendations for cytoreduction in polycythemia vera

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European LeukemiaNet recommendations for cytoreduction for polycythemia vera (PV) patients under 60 have just been updated and now published in The Lancet Haematology.

The expert panel of European LeukemiaNet (ELN) investigators recommends that “patients with polycythaemia vera who are younger than 60 years and have not had previous thrombotic events should start cytoreductive drug therapy if at least one of the following criteria are fulfilled:

  • strictly defined intolerance to phlebotomy,
  • symptomatic progressive splenomegaly,
  • persistent leukocytosis (>15×109 white blood cells per L),
  • progressive leukocytosis (at least 100% increase if baseline count is <10×109 cells per L or at least 50% increase if baseline count is >10×109 cells per L),
  • extreme thrombocytosis (>1500×109 platelets per L),
  • inadequate haematocrit control requiring phlebotomies,
  • persistently high cardiovascular risk, and
  • persistently high symptom burden.

Recombinant interferon alfa, either in the form of ropeginterferon alfa-2b or pegylated interferon alfa-2a, is the recommended cytoreductive treatment for these patients. The expert panel suggested that either interferon alfa or ruxolitinib should be considered for patients who are being treated with hydroxyurea but require a therapy change.”

Please note that while ropeginterferon alfa-2b is not available in Australia, pegylated interferon alfa-2a (Pegasys) is equally recommended and is available on the PBS.

The abstract from the Lancet with references is available HERE.

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Myelofibrosis treatments – real world findings from the UK

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Some of the UK’s leading MPN haematologists have published results of a retrospective real-world study of current treatment pathways for myelofibrosis (MF) in the United Kingdom.
The study is published in the Journal ‘Therapeutic Advances in Hematology’.

The abstract summary explains:
‘These results provide insight into real-world clinical practice for patients with MF in the United Kingdom. Despite the known high prevalence of disease-associated symptoms in MF, symptoms were poorly documented. Most patients were initially observed or received hydroxycarbamide, and ruxolitinib was used as first-line management strategy in only a minority of patients.’
And, further…..

‘The results of this study suggest that medical records can be missing key information, which is needed to decide which is the best way to treat a patient with myelofibrosis. They also suggest that clinicians in the UK prefer observation to treatment for a large number of patients with myelofibrosis. This could mean that the approach used for many patients with myelofibrosis does not help them to control symptoms that have an impact on their daily lives.’

The full paper is available HERE.

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Detailed MPN information for GPs now available

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MPNs have been featured in an article in the Australian Journal of General Practice titled ‘Myeloproliferative neoplasms: Classifications and an approach to diagnosis’.

The aim of the article is to provide general practitioners (GPs) insight into the classification and clinical approach to diagnosis of MPNs.

The full article is available HERE.

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World rare disease day

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Today is RARE DISEASE DAY 2022.

WATCH MPN expert Professor Andrew Perkins and the MPNAA’s Ken Young speaking about MPNs.

 

 

 

 

 

 

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Congratulations to Nathalie Cook OAM

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The MPN AA is absolutely thrilled to learn of an Australia Day award for our team member, Nathalie Cook.

Nathalie won the award for ‘service to dietetics’. Nathalie is an Accredited Practising Dietitian and has worked tirelessly for her patients.

However we know her best for her work in a volunteer capacity for MPN patients, playing a key role in the listing of pegylated interferon, Pegasys® onto the Pharmaceutical Benefits Scheme in 2018.
And alongside this advocacy work, she has been a team member of MPN AA, as well as presenting at Australian and international MPN conferences.

For Nathalie’s advice to MPN patients in her role as an Accredited Practising Dietitian, please see her videos and presentations HERE.

For more information about Nathalie’s extraordinary life achievements so far, see Nathalie Cook OAM.

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Second versus first wave of COVID in MPN patients

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European haematologists have been undertaking ongoing studies of MPN patients who have contracted COVID.  This study covers the second COVID wave of 304 MPN COVID cases from 39 different hematology centres.  Their findings are outlined in just released (January 2022) correspondence in the International Journal ‘Leukemia’.  Importantly, they found a relatively higher risk from COVID for patients over 70, more thromboses in ET patients and a need for permanent surveillance of MPN patients who have survived the acute phase of a COVID infection.

‘Patients of the second wave presented, compared to those of the first, with a less severe disease, including a lower degree of inflammation, leading to hospitalization in a smaller percentage of cases. Overall, the mortality rate was significantly lower, likely due to early COVID-19 diagnosis, facilitated by the greater availability of swabs than in the first wave, more efficient management of infected patients, better prepared health systems and preferential protection of older and higher-risk MPN vulnerable subjects.’

‘However, patients over 70 years still presented with an excess of mortality, particularly when associated with comorbidities and an MF phenotype. Unfortunately, no data are available so far in our series to support a role of vaccinations. The high thrombosis rate in patients with ET was confirmed, suggesting that in this MPN phenotype regimens of antithrombotic prophylaxis in addition to heparin should be explored. Also in the second wave, but to a lesser extent than in the first, the health consequences of COVID-19 protracted far beyond acute infection, suggesting careful and permanent surveillance of patients with MPN who have survived the acute phase of SARS-CoV-2 virus infection.’

The full article has been made available HERE.

 

 

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Exploring real life with blood cancer – a mind and body experience

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This excellent webinar, organised by the Leukaemia Foundation, features patient stories as well as presentations by:

  • clinical haematologist Cecily Forsyth who has a special interest in MPNs,
  • health psychologist Jane Fletcher whose presentation is incredibly encouraging and motivating, and
  • physiotherapist Julie Allen, herself a lymphoma survivor who provides a wealth of knowledge about how to manage fatigue and the physical impacts of blood cancer.

It is a long webinar but we cannot recommend it highly enough.  It is available HERE.

 

 

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October 2021 ATAGI advice for third primary COVID-19 vaccine dose for blood cancer patients

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Many of you will have noted the October 2021 advice from ATAGI which recommends a third primary dose of COVID-19 vaccine for severely immunocompromised patients. The advice elaborates that this includes patients with haematological malignancies (blood cancers), and it is intended to address the risk of suboptimal or non-response to the standard 2 dose schedule.

There is advice about particular vaccines and the timing of a third dose, which should be exactly within 2-6 months of your second dose. If you have queries, we suggest that you reach out to your GP or specialist.

 

 

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Reported incidence and survival of myeloproliferative neoplasms in Australia

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An update to the Australian Cancer Atlas was released earlier this year and for the very first time it included MPNs. This was such an encouraging development for MPN research.

Since that time, researchers and epidemiologists have been exploring MPN incidence further and a paper has just been released in the journal ‘Pathology’.

MPN AA thanks the authors for their thoughtful analysis and is proud to have been able to support this research through donations from the Australian MPN community.

Spatial disparities in the reported incidence and survival of myeloproliferative neoplasms in Australia

Due to the restrictions around access to medical journals, free access to the full article will be available until 3 November 2021. 

 

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