New trial helps high-risk blood cancer patients live longer

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Health news
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The University of Leeds has collaborated on new research that helps high-risk cancer patients live longer.

Patients with the most aggressive forms of the blood cancer – multiple myeloma – can live significantly longer when treatment is tailored to their disease, according to new long-term results from a major UK-led clinical trial. 

The findings come from extended follow up of the OPTIMUM (MUKnine) trial, led by scientists at The Institute of Cancer Research, London, alongside colleagues at Leeds, The Royal Marsden NHS Foundation Trust and hospitals across the UK. 

Study results 

Results of the study, published in The Lancet Oncology, show that a personalised, risk-adapted treatment strategy improves survival for patients with high-risk myeloma – a group historically associated with rapid relapse and poor outcomes. 

At around six years, almost seven out of 10 patients (69.1%) on the OPTIMUM trial were still alive, compared with around 40% on standard therapy, showing a clear survival benefit. 

Multiple myeloma is a cancer of the plasma cells, with around 5,900 new cases diagnosed each year in the UK. Although modern treatments mean more than half of patients now survive at least five years, around 20–25% have aggressive disease that responds poorly to conventional treatment. 

Earlier analyses from OPTIMUM trial, which was funded by Myeloma UK and The David Forbes Nixon Foundation, with support from The Royal Marsden Cancer Charity and the National Institute for Health and Care Research, showed promising early outcomes for this group of high-risk patients. The new long-term data now confirms that these benefits are durable. 

More than half of patients (53.8%) on the trial remained progression-free at six years, compared with fewer than one in five (17.6%) patients in a matched external control group from the Myeloma XI trial.  

By the end of the follow-up period, median survival had not been reached for OPTIMUM, meaning more than half of patients were still alive. In contrast, the median survival for the patients on standard therapy was about 57 months (just under five years). 

The OPTIMUM trial is a powerful example of what can be achieved through collaborative academic research. Bringing together the NHS, academic researchers and charities from across the UK enables ambitious studies that generate the evidence needed to improve care for patients. Continued investment in UK academic clinical research is essential if we are to deliver more advances like this.

Dr Andrew Hall, Principal Statistician at The University of Leeds

Personalised approach

The researchers also examined outcomes within specific high-risk subgroups to understand which patients benefited most from the personalised approach. 
One subgroup comprised patients who had been identified as high-risk by gene expression profiling using the MMProfiler SKY92 test, who would not otherwise have been identified as high risk.  

The results showed that 62.3% of these patients who were given personalised risk-adapted treatment were still alive and progression-free at six years, compared with 20.3% of patients who had received conventional treatment.

Gene expression profiling is not routinely available in the NHS, meaning that many of these patients are currently diagnosed as having “standard-risk” myeloma and may not receive optimal treatment from the outset. 

The findings highlight the importance of modern molecular diagnostics to ensure that patients receive the most appropriate therapy when they are diagnosed.   

Martin Kaiser, Professor of Molecular Haematology at The Institute of Cancer Research, London, and Consultant Haematologist at The Royal Marsden NHS Foundation Trust, said:  “High-risk myeloma has traditionally been one of the toughest challenges we face, with patients often relapsing early despite the best available treatments. These long-term results show that when we adapt treatment to the biology of the disease, we can significantly extend survival for many patients who previously had very limited options. 

“This study also shows that some patients with aggressive disease are currently being missed because the necessary molecular tests are not routinely available. Identifying these patients earlier could allow us to tailor treatment from the start and change the course of their disease.”

Further information

Email media enquiries to the University of Leeds press office via pressoffice@leeds.ac.uk.