A landmark paper published online on December 23, 2020 in the New England Journal of Medicine describes the results from a global trial across 148 sites in 23 countries, showing a 30 per cent improvement in survival in patients with acute myeloid leukemia (AML). The Phase 3 clinical trial called QUAZAR, showed that a drug, called CC-486 (oral azacytidine) (image), significantly improved survival in older patients, over the age of 55, with the disease. AML is the most acute blood cancer in adults and its incidence increases with age, with a poor prognosis. With current treatments, the majority of older patients will die of their disease within 2 years of diagnosis. Approximately 20,000 people in the United States are diagnosed with AML every year. The global trial, led by Professor Andrew Wei from Monash University's Australian Centre for Blood Diseases and a hematologist at Alfred Health, focused on people with AML over 55 years of age, "because of an unmet need to identify new agents able to improve outcome in patients after completing chemotherapy," he said. "After intensive chemotherapy, the risk of AML relapse is high. Many older patients are not eligible to receive a stem cell transplant and so a less toxic option to reduce disease recurrence is desirable, rather than just being monitored and waiting for the disease to come back," he said. "Based on the results of the QUAZAR study, it is very exciting to think that, by taking a tablet that is relatively well-tolerated, we can help reduce relapse risk and improve survival." The NEJM article was published online on December 24, 2020 and is titled “Oral Azacitidine Maintenance Therapy for Acute Myeloid Leukemia in First Remission.” The trial involved 472 patients, with an average age 68 years, who were either given CC-486 or a placebo.
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