Australian researchers report breakthrough in treatment of a chronic form of leukaemia

21 February 2018

Australian medical researchers have reported a breakthrough in the treatment of a chronic form of leukaemia. They hope, the development will help improve the quality of life for many people living with the disease.

A Phase 2 clinical trial, published in journal Annals of Internal Medicine, has shown that it is possible to achieve treatment-free remission (TFR) in patients with chronic myeloid leukaemia (CML) who were treated with a "potent" new drug called nilotinib.

According to the lead investigator of the trial, professor, Tim Hughes of the South Australian Health and Medical Research Institute, the results will have an impact on the way doctors treat the disease.

With the help of drugs known as tyrosine kinase inhibitors (TKIs), that target the enzyme causing the leukaemia, dramatically improved outcomes for patients with CML, have been reported. However, the majority (80 per cent) must remain on medication for life even when clinically in remission, according to professor Hughes.

This causes many side-effects and greatly affects their health.

"About a quarter of these patients tell us the side-effects are in some cases unbearable, another 25 per cent say that the side effects are significant enough to impair their quality of life," said professor Hughes, who works as a consultant haematologist at Royal Adelaide Hospital, Australian Associated Press reported.

The Phase 2, open-label study assessed treatment-free remission (TFR) after second-line nilotinib therapy was terminated. 

Researchers studied 163 patients who had reached sustained deep molecular response (DMR) following a change from imatinib to nilotinib to see if these patients could stay in remission without tyrosine kinase inhibitor (TKI) therapy. 

Researchers found that 58 per cent of patients were able to maintain TFR at the primary end point of 48 weeks. 

At 96 weeks, 53 per cent of patients maintained TFR.  According to experts, the results indicate that TFR can be attained by CML patients who have reached DMR after ceasing second-line nilotinib treatment.

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