For patients with acute myeloid leukemia who have achieved complete remission from their disease, the European Commission has approved the first oral maintenance therapy, capable of increasing both progression-free survival and overall survival. The new molecule, azacitidine, can be prescribed after induction therapy, with or without consolidation treatment, to patients who are not candidates for haematopoietic stem cell transplantation or who have chosen not to.
The benefits of this therapy have been shown to be significant across a broad spectrum of acute myeloid leukemia subtypes, demonstrated in the Quazar AML-001 clinical trial, upon which the European approval was based. “Responses to induction therapy can be short-lived and the risk of relapse is high, especially for patients ineligible for stem cell transplantation,” explained Andrew Wei of Alfred Hospital and Monash University in Melbourne, Australia. and principal investigator of the study: “The approval of oral azacitidine by the European Commission offers the possibility of bringing clinical benefit and changing the therapeutic paradigm of patients with acute myeloid leukemia”.
Approximately half of the patients will recover within a year
Acute myeloid leukemia (AML) is one of the most common forms of acute leukemia in adults. The estimated overall incidence is 350,000 cases and the estimated 5-year survival rate in Europe is 17%. It is characterized by rapid growth of abnormal cells in the bone marrow, which interferes with the normal production of blood cells and their function. Due to the alteration in the production of red blood cells, platelets and white blood cells, it presents with anemia, bleeding and infections. It is a heterogeneous disease associated with various genetic mutations and can progress rapidly and lead to death if not treated promptly. The response of acute myeloid leukemia to first-line treatment can be short-lived, meaning that after patients’ initial response to therapy, about half relapse within one year.
The new drug
Azacitidine is a hypomethylating drug of DNA: that is, it induces a “chemical reaction”, hypomethylation, which can restore the normal function of the fundamental genes in cell differentiation and proliferation. The Quazar AML-001 study involved patients aged 55 years or older, with a recent diagnosis of acute myeloid leukemia, with intermediate or poor prognosis, in first complete remission or in complete remission with incomplete recovery of the blood count, after intensive induction chemotherapy with or without consolidation treatment. Median overall survival was greater than two years (24.7 months) in the azacitidine arm compared to 14.8 months in the placebo arm. The mean duration of treatment was 12 cycles (1 – 82) for azacitidine and 6 cycles with placebo (1 – 76). Median relapse-free survival was significantly longer with azacitidine than with placebo (10.2 months and 4.8 months, respectively). The overall health-related quality of life was preserved.
“The approval of oral azacitidine represents a significant advance for patients in the European Union with acute myeloid leukemia, with an urgent need for maintenance therapy for this aggressive blood cancer,” said Noah Berkowitz, senior vice president, Hematology Development. by Bristol Myers Squibb: “We are committed to helping improve long-term outcomes and significantly extend survival of patients with difficult-to-treat diseases – he concludes – by collaborating with EU Member States to make oral azacitidine available as soon as possible for eligible patients “.