A new drug sharply improves the survival rate of patients with a bone-marrow disorder that often develops into acute myeloid leukaemia (AML), according to a study released Wednesday.
Up until now, there has been no known treatment for the disorder -- called myelodysplastic syndrome (MDS) -- besides bone-marrow transplant, which is suitable for only a small percentage of patients.
MDS is condition in which immature blood cells in the bone marrow known as blasts fail to develop properly into adult blood cells. This leads to a lack of while blood cells to fight infection, red blood cells to carry oxygen, and platelets to stop bleeding.
In clinical trials led by Pierre Fenaux at the Universite Paris 13 north of Paris, 358 patients with high-risk MDS were divided into two groups.
One received azacitidine injections seven days each month for six months. The other 179 patients were randomly given one of several conventional treatments, including so called "best supportive" or palliative care, and intensive chemotherapy.
Follow-up continued for an average of just over 21 months.
Overall, patients who took azacitidine survived more than nine months longer than those who received conventional care.
The benefits of the new drug became apparent after only three months, and researchers estimated that after two years of treatment, the survival rate with the new drug would be doubled.
The drug also delayed the onset of acute myeloid leukaemia by six months, and improved the chances of complete and partial remission compared to standard treatments.
As with other therapies, there were side-effects. The frequency of severe blood-related problems was slightly higher than in patients who received best care, but lower than in those who underwent chemotherapy.
"The results of this study indicate that azacitidine significantly lengthens overall survival," Fenaux said in a statement, adding that the drug "should become the reference treatment in most patients with this condition."
The study was published in the British medical journal The Lancet Oncology.