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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Promising finding for possible future myelofibrosis treatment

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Adelaide researchers have discovered a possible new method to treat myelofibrosis (MF), that could have the potential to greatly extend lifespan without the side effects caused by current treatments.

While MF symptoms can often be controlled with the drug Ruxolitinib, side effects may be problematic and currently, the only cure for MF is an allogeneic stem cell transplant.

Dr Daniel Thomas, leader of the Myeloid Metabolism Lab at the South Australian Health and Medical Research Institute, (SAHMRI) and Associate Professor of Medicine at the University of Adelaide, led the study in collaboration with Professor Angel Lopez at SA Pathology.

Dr Thomas says it was a stroke of luck that led to the extraordinary find.
“We were actually trying to make a tool to study myelofibrosis. We didn’t realise the antibody we made would have therapeutic properties,” Dr Thomas said.
“Our drug blocked the growth of cancer cells in a very aggressive live model of the disease, significantly increasing survival rate without noticeable negative side effects,” Dr Thomas said.

Co-lead author and biochemist Dr Denis Tvorogov generated antibodies using a peptide fragment called ‘neoepitope’ that’s only present within the cancer and not on any normal tissues. What he didn’t expect was for the antibody to kill cancer cells when he tested it on patient samples, working with an early career scientist Dr Chloe Thompson-Peach.
“What is really exciting is that many other cancers have similar peptide fragments that we could also target by harnessing the immune system,” Dr Tvorogov said.
“These fragments are created by the insertion or deletion mutations within the cancer. We’ve found they not only drive cancer growth but also vulnerable for targeting without side effects.”

The new antibody is currently being prepared for early phase clinical trials set to run in South Australia later this year, supported by local biotech company, AusHealth.  Pre-clinical models have shown the drug is effective at shrinking tumours and Dr Thomas is confident the antibodies will prove to be safe and effective in humans.
“We estimate there are at least 12,000 Australians living with cancer or having had cancer that express a recurrent neoepitope similar to what is found in myelofibrosis, that could be curable with
an immunotherapeutic approach,” Dr Thomas said.

“This discovery brings us a fresh perspective. We need to build cell therapies and antibody therapies against these fragments as fast as possible.”

The full media release is available:  SAHMRI media release – Lucky find could hold key to beating rare blood cancer —

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Momelotinib: an emerging treatment for myelofibrosis patients with anaemia

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A phase 3 clinical trial has been underway for myelofibrosis patients who are symptomatic, suffer from anaemia and have been previously treated with a Jak inhibitor. The trial was using the drug Momelotinib.

Findings just released on 25 January this year are very encouraging.  Momelotinib achieved statistically significant benefit on symptoms, anaemia and spleen size.
The trial, undertaken by drug company Sierra Oncology, is a global, randomized, double-blind clinical trial evaluating momelotinib in 195 myelofibrosis patients.

Access to the full report of the trial is available HERE. The full data set will be presented at an upcoming medical meeting which we will also publish once available.

Ruben Mesa, MPN specialist  and co-principal investigator of the study said that “As a clinician, I am thrilled to see data that confirm the potential of momelotinib as a treatment option for myelofibrosis patients who are anaemic or at risk of becoming anaemic,”  “Anaemia of myelofibrosis is strongly correlated with reduced quality of life and a decrease in overall survival. Half of all myelofibrosis patients present with anaemia at diagnosis and virtually all become anaemic over time. With currently approved therapies being myelosuppressive, it’s wonderful to know that we may soon have such an effective treatment option for these patients.”

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Expert interview with Professor Andrew Perkins

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The latest Leukaemia Foundation’s MPN newsletter features a detailed and wide-ranging interview with MPN expert, Professor Andrew Perkins.
As well as his ongoing care for MPN patients as a clinical haematologist, Professor Perkins and team are focussed on improving diagnostics and treatment for MPN patients, especially those with myelofibrosis.

His interview is available HERE.

 

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Protection against COVID for people with blood cancers

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Norman Swan’s Health Report has featured an item about people with blood malignancies and their response to Covid-19 vaccines.
The item relates to new research published in the Lancet Haematology.  This research found that people with blood malignancies may not produce as many protective antibodies to the vaccines as others.
This is because in blood cancers, the immune system itself is affected.

Norman interviewed haematologist, Associate Professor Kate Burbury, about this research.  A brief extract is below as well as the link to the article.

Kate Burbury:  Most of us [haematologists and oncologists] would be advocating some protection is better than none. And the only concern we would have is that they [the blood cancer patient] might not mount an adequate antibody response and therefore advocating that they continue to pursue the usual protective mechanisms such as keeping themselves safe and avoiding people that might have infective symptoms.
The interview transcript, podcast and the research in the Lancet Haematology are linked HERE.

Encouraging responses for MPN patients to Covid vaccines

Subsequent to the above article, the Leukaemia and Lymphoma society in the US has outlined the response of  different blood cancer patients to Covid vaccines and while a small sample, the results are very encouraging for MPN patients, showing some 97% of patients had detectable Covid-19 antibodies.
The graphic is below and the full article is linked HERE.

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Swab a cheek and save a life

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The Australian Bone Marrow Donor Registry is urging Australians to consider becoming a bone marrow donor.  It’s a very simple process through its ‘Strength to Give’ campaign.

Please ask your family, friends and their friends to consider becoming a donor.

For a small number of  MPN patients, the only possibility of prolonging survival is through a blood stem cell transplant. Many other blood cancer patients also benefit from stem cell transplants.

The Strength to Give donor recruitment campaign is targeted towards younger male donors (18 to 30 years old) as young male donors typically result in better outcomes for patients and increases their overall chance at achieving a full recovery (unfortunately only 4% of the registry are young male donors). It is also important to attract donors from different ethnic backgrounds as patients are more likely to find a match with a donor from the same ethnic background.

Some heart-warming stories from donors and more information is available from the Strength to Give website at strengthtogive.org.au.

 

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MPN awareness day September 2020- video of patient webinar

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If you were not able to join the inspiring and informative Australian MPN Awareness Day webinar, you can watch it HERE.
The MPNAA is grateful to Professor Andrew Perkins, Professor Wendy Erber, Professor Peter Baade as well as the Leukaemia Foundation and MPN patient Helen Williams for making this event possible.
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MPN Alliance Australia donates $15,000 towards groundbreaking MPN research at University of Western Australia

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MPN AA is very proud to announce that with the help of our supporters, we have donated $15,000 for MPN research in Australia to Professor Wendy Erber and her colleagues, Dr Kathryn Fuller, Dr Henry Hui and Dr Belinda Guo at the University of Western Australia. The team are undertaking research on a new technique to see if they can detect which MPN patients may be at risk of progressing to leukaemia or marrow fibrosis. It is hoped this may ultimately provide an alternative to the current method of utilising bone marrow biopsies for the purpose of detecting the first signs of disease escape or progression. Although only a minority of MPN patients are affected, it is not currently known who this might affect or when this will occur. By the time symptoms appear, it is generally difficult to cure. Hence a predictor of progression may assist in providing treatment earlier and possibly providing cures.

The method the team will use is their own invention, and the one that won a Eureka Award (affectionately known as the “Oscars of Science”!) in 2018. As you will see from the Youtube video it is a flow cytometry method that enables the study thousands of cells to see if the cell-of-interest has the chromosome change that may predict progression.

We wish Prof Erber, and Drs Kathy Fuller, Henry Hui and Belinda Guo every success with their research and look forward to sharing their progress in the future.

 

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New international website – MPN Hub

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We are pleased to discover that several European and American hematologists with a particular interest in MPNs have collaborated and established a website and twitter presence solely devoted to MPNs called MPN Hub. A facebook page and instagram account also complete the social media presence of the MPN Hub.

According to French hematologist Professor Jean-Jacques Kiladjian, MPN Hub ‘will be an open-access and unbiased educational website for all healthcare professionals …… providing the latest medical information, live congress coverage, trial updates, drug approvals, case studies as well as expert opinions.’
The twitter introduction states: ‘Lifting levels of knowledge in myeloproliferative neoplasms (MPN).  A global online platform providing latest treatment options and medical information.’

While patients are not MPN Hub’s primary audience, we believe this information is valuable for MPN patients to keep up to date about with developments in the MPN field.

For example, MPN Hub has been showcasing MPN findings from the European Hematology Association’s 2020 conference held ‘virtually’ in June this year.

Practice changing abstracts for MPN at the 2020 European Haematology Association conference

  • This is a 4 minute video overview by Professor Kiladjian.

Some of those practice changing abstracts relating to PV mentioned by Professor Kiladjian are at the links below.

  1. Should ropeginterferon become standard of care (SOC) for all patients with low-risk PV?
    While Ropeginterferon is not available in Australia, another form of pegylated interferon is available (called Pegasys).
    These findings are likely to be of particular interest to PV patients who are currently considered ‘low risk’.
    This study showed that while low risk patients are usually treated by phlebotomy only and aspirin, there were better results with interferon alpha compared with phlebotomy/aspirin only.
  2. PROUD-PV phase III results on ropeginterferon alpha-2b for polycythemia vera
    It is known that Ropeginterferon is able to reduce the Jak2 v617F allele burden. This article shows that it can also reduce associated mutations such as in TET2.
  3. TET2  mutations predict thrombotic risk in patients with polycythemia vera

Articles about drug developments/combinations in myelofibrosis presented at the EHA. This includes promising developments for MF patients refractory to ruxolitinib.

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