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The MAJIC-PV trial  studied ruxolitinib for PV patients against best available therapy (BAT) which in this case was mainly hydroxycarbamide (hydroxyurea).  It concluded that ‘ruxolitinib treatment benefits hydroxyurea intolerant or resistant patients with superior complete [haematological] response, and event free survival as well as molecular response; importantly also demonstrating for the first time, to our knowledge, that molecular response is linked to event free survival, progression free survival, and overall survival.’

Watch Dr Claire Harrison MD, FRCP, FRCPath, Guy’s and St Thomas’s NHS Foundation Trust discuss the results from the MAJIC-PV trial: ruxolitinib vs BAT for hydroxyurea resistant/intolerant PV.

Jean-Jacques Kiladjian further explains in a recent talk about ‘State of the art in PV’, that the MAJIC study also showed that ‘Patients having a complete haematological response during the first year of treatment had a significantly better event free survival than those who did not.  So this is for the first time, an evidence that normalising the blood count, results in benefits in terms of less complications during the follow up. And the other point is …… that this event free survival was also better in patients who achieved a molecular response (reduction in allele burden) compared to those who did not. …….
So to summarise, …….we have to optimise [PV] management by recognising resistance in tolerance to first line therapy, and switch quickly to second line therapy because….achieving complete haematological response within 12 months results in better long term outcomes.’

Image: screenshot of the MAJIC trial outcomes showing: superiority of ruxolitinib in event free survival for patients intolerant or resistant to HU; and also the benefits to PV patients of achieving a complete haematological response within 12 months

While not specifically addressed in the MAJIC study, pegylated interferon is also an existing alternative first line treatment to hydroxycarbamide for PV patients. However about 20% of patients are unable to tolerate interferon and so a need remains for effective additional treatments. Of interest is that the MITHRIDATE trial now underway in the UK and France is comparing ruxolitinib with hydroxyurea and with pegylated interferon.

The October edition of ‘Leukemia’ features a letter to the editor about life expectancy for polycythaemia vera (PV) patients.

The US study noted that, while historically, thrombosis has been the major contributor to early mortality and the leading cause of death in PV, vigilant phlebotomy to a target hematocrit, low dose aspirin, and myelosuppressive therapy have all reduced fatal cardiovascular events and major thromboses.

The study then considered how much a diagnosis of PV alters life expectancy of patients receiving available treatment, and whether a normal life expectancy is possible.

Findings were that treatment with both hydroxyurea and interferon were associated with improved survival. This was independent of age, thrombosis history, sex, and white blood count. However the study also found that early diagnosis and treatment of PV with effective therapies, such as interferon, may prevent or delay disease progression.  ‘Because PV progression to MF or AML is among the leading causes of late morbidity and mortality, it may be time to reconsider and improve upon PV risk assessment and stratification.’

The letter concludes that while this large population-based and single-centre study shows that PV survival remains shortened, encouragingly, normal life expectancy for PV patients is possible with available care. The discrepancy between survival nationally and at a specialized centre, highlights the need for greater community outreach and education regarding treatment standards. The authors hope this study motivates the development of new PV risk stratification and treatment recommendations that focus not only on hematologic control and reducing thromboses, but also on preventing progression, improving overall survival, and restoring normal life expectancy.

The full letter is available HERE.

An exciting new change has occurred to the way Australians can register to become blood stem cell donors. A simple cheek swab is now all you need to do to register through the Australian Bone Marrow Donor Registry’s ‘Strength to Give’ campaign.

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

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. 

More information is available from the Strength to Give website at strengthtogive.org.au.