A new therapy arrives for the most fragile patients with acute myeloid leukemia. The Italian Medicines Agency (Aifa) gives the green light to the reimbursement of the drug venetoclax in combination with azacitidine, in patients with acute myeloid leukemia not suitable for intensive chemotherapy. The combination reduces the risk of death by 42%. This was reported during the meeting ‘acute myeloid leukemia: new perspectives in the management of the disease’. AIFA has also granted the designation of full innovation, which allows innovative medicines to be included in the Fund.
Acute myeloid leukemia is an aggressive blood cancer that affects around 3,300 people every year in Italy. The majority of cases present in old age and the mean age at diagnosis is 69 years. Elderly or frail patients affected by other pathologies cannot tolerate intensive chemotherapy, explains Maria Teresa Voso, of Tor Vergata University. In these cases, the therapy is based on hypomethylating agents (capable of interfering with the epigenetic alterations) which, however, induce responses in no more than 20% of cases, and a survival of around 10-12 months.
“Venetoclax – says Voso – induces the ‘programmed’ death of tumor cells. Venetoclax also performs a synergistic activity with hypomethylating agents”. Survival 5 years after diagnosis today is about 28%, but of patients unsuitable for intensive chemotherapy only 5% are alive at 5 years. In the Viale-A study of over 400 newly diagnosed patients ineligible for intensive chemotherapy, the venetoclax-azacitidine combination treatment was more effective than azacitidine alone.
“The median overall survival was 14.7 months compared to 9.6 months – explains Giovanni Martinelli, Scientific Director of the Romagna Institute for the Study of Tumors ‘Dino Amadori’ Irccs, Meldola -. The long-term follow up of the study, at 43.2 months, confirmed this survival advantage, with a 42% reduction in the risk of death.
In addition, complete remission achieved with venetoclax plus azacitidine was two times higher (66%) than with azacitidine alone (28.3%). About half of the patients treated with venetoclax plus azacitidine achieved complete remission of the disease already before the start of the second cycle, with an average duration of response of 17.5 months”. “These results – concludes Martinelli – have a significant significance clinical, because for the first time it is possible to obtain complete remissions without resorting to chemotherapy”.
“Acute myeloid leukemia is one of the most difficult blood cancers to treat and timely management of the hematological patient is essential – says Giuseppe Toro, National President of AIL (Italian Association against Leukemia, Lymphoma and Myeloma) -. Scientific research makes innovative therapies are available in a tangible way, capable of improving both survival and quality of life. The moment of communication of the diagnosis is characterized by emotions such as fear, discouragement, anger, concern. For this reason, increasingly integrated and multidisciplinary which, together with the figures of the haematologist and the nurse, also includes the psychologist – a central figure in patient support that as AIL we intend to strengthen more and more – the nutritionist, the palliative care specialist and the infectious disease specialist. to ensure rapid and uniform access to innovation and social-health support services in the area”.