Acute myeloid leukemia: Aifa approves new therapy that extends survival by 15 months
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Every year in Italy there are about 3,300 new cases of acute myeloid leukemia, the majority of which concern elderly patients who are unable to tolerate intensive chemotherapy. A new possibility arrives for them thanks to Aifa’s approval of a new therapy that gives these patients 15 months of extra life.
An aggressive blood cancer
Anemia, tiredness, paleness, bleeding and bruising, related to platelet deficiency, and infections are the main symptoms of acute myeloid leukemia. “It is an aggressive, extremely heterogeneous hematological disease, characterized by the uncontrolled proliferation of immature bone marrow cells, the blasts, with a very rapid development – says Maria Theresa Voso, full professor of Hematology at the Tor Vergata University and head of the Advanced Oncohaematological Diagnostics laboratory at the Tor Vergata Polyclinic in Rome. “The diagnosis usually occurs following the evaluation of blood count abnormalities and is confirmed with more specific investigations that require evaluation of the bone marrow through bone marrow aspiration”.
Elderly patients and with multiple pathologies
The majority of cases present in old age and the mean age at diagnosis is 69 years. “Elderly or frail patients, because they are affected by other pathologies – continues Voso – are not able to tolerate standard intensive chemotherapy, followed by allogeneic stem cell transplantation, if indicated. In these cases, the therapy is based on hypomethylating agents which, however, have been shown to induce low response rates, in no more than 20% of cases, and a survival of around 10-12 months”. To underline the particular condition in which the elderly patient is also Felicetto Ferrara, Director of Hematology at the Cardarelli Hospital in Naples: “More than 50% of patients affected by acute myeloid leukemia are over 65 years of age and about a third are over 75. The disease in the elderly has unfavorable prognostic characteristics compared to the young – adult because more resistant to chemotherapy. Hence the difficulties of achieving complete remission and long-term survival in this population.
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The new therapy
For newly diagnosed patients unsuitable for standard intensive chemotherapy, potentially 40% of the total, the Italian Medicines Agency has approved the reimbursement of venetoclax in combination with a hypomethylating agent (azacitidine). The molecule has received the designation of full innovation from Aifa, which allows the inclusion of innovative drugs in the Fund, a recognition reserved only for those molecules that demonstrate, on the basis of solid scientific evidence, to satisfy, among other dimensions, important needs therapeutics, and to bring an added therapeutic value, first of all, in the case of oncological drugs, the increase in survival.
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The mechanism of action
Venetoclax in combination with azacitidine has in fact demonstrated a longer overall survival, higher remission rates and rapid and durable responses. “Venetoclax – continues Voso – induces apoptosis, ie the ‘programmed’ death of acute myeloid leukemia cells, through a selective and potent inhibition of BCL-2, a protein that allows the survival of tumor cells. Venetoclax also performs a synergistic activity with hypomethylating agents, indirectly increasing the sensitivity to BCL-2 inhibition”. Hypomethylating agents also interfere with other mechanisms that determine the development of the disease, and prevent the growth of abnormal cells in the bone marrow.
A longer survival
The 5-year survival after diagnosis is now about 28%. The rates are lower for those who are ineligible for intensive chemotherapy, of whom only 5% are alive at 5 years. In the pivotal VIALE-A study of over 400 newly diagnosed patients ineligible for intensive chemotherapy, the combination treatment venetoclax plus azacitidine was more effective than azacitidine alone. “The median overall survival was 14.7 months compared to 9.6 months,” he explains John Martinelli, scientific director of the Romagna Institute for the Study of Tumors ‘Dino Amadori’ – IRST 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. Not only. Complete remission achieved with venetoclax plus azacitidine was two times greater (66%) than with azacitidine alone (28.3%). It should also be underlined that the responses were rapid and lasting. In fact, approximately half of the patients treated with venetoclax plus azacitidine achieved complete remission already before the start of the second cycle, with a median duration of response of 17.5 months”.
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The Italian study
The enthusiasm of clinicians and patients is strong because for the first time it is possible to obtain complete remissions without resorting to chemotherapy. “The data – continues Martinelli – have been confirmed in ‘real life’, ie in daily clinical practice, as highlighted in an all-Italian study, AVALON, on 190 patients. With a follow-up of 20.9 months, the median survival was 12.7 months in newly diagnosed patients, similar to that emerging from VIALE-A. AVALON is the demonstration of the role of Italian hematology in improving the life expectancy of patients affected by one of the most serious blood cancers”.
The need for integrated assistance
If the availability of new therapeutic opportunities is a great step forward, the timely management of the haematological patient remains fundamental. “Acute myeloid leukemia is one of the most difficult blood cancers to treat and taking it is critical. “The moment when the diagnosis is communicated – he underlines Joseph Bull, national president of AIL (Italian Association against Leukemia, Lymphoma and Myeloma) – is characterized by emotions such as fear, discouragement, anger, worry. This requires increasingly integrated and multidisciplinary assistance which, together with the figures of the haematologist and the nurse, also includes the psychologist – a central figure in patient support which, as AIL, we intend to strengthen more and more – the nutritionist, the palliative care specialist and infectious disease specialist. Furthermore, it is important to help ensure rapid and uniform access to innovation and social-health support services in the area”.
Over 2,000 patients treated before reimbursement
Thanks to ‘early access’ tools, i.e. early access to treatments, this innovative therapy was made available in Italy one year before European approval and three before reimbursement in our country. “Aifa – continues Ferrara – in March 2020, at the request of the scientific community and patient associations, included venetoclax in the list of drugs that can be supplied at the total expense of the National Health Service pursuant to law n.648 of 1996, for the treatment acute myeloid leukemia in patients unsuitable for induction chemotherapy. In this way, a possibility of treatment was offered in a particularly delicate population, to address the lack of therapeutic alternatives and respond to unmet clinical needs”. Thanks to the ‘early access’ programme, over 2,000 patients were treated before reimbursement, with results comparable to those obtained in the pivotal study. “We are facing a virtuous case of access to therapeutic innovation in our country, made possible by the concrete contribution of all the players involved – he says Fabrizio Greco, chief executive officer of AbbVie. “The recent decision by AIFA to recognize venetoclax, together with the reimbursement by the NHS, the status of innovativeness with the relative inclusion in the Fund for innovative drugs, will guarantee rapid and uniform access at regional level and at single center. We must continue to collaborate with Institutions, Scientific Societies and Patient Associations to overcome individual virtuous cases and define a system of rules and resources that allows the value of therapeutic innovation to be recognized and to make it available quickly and widely to all who need it”.
“Our universal healthcare system guarantees access to treatment for all patients – he concludes Tilde Minasi, President of the Parliamentary Intergroup ‘Oncology: prevention, research and innovation’ and member of the Senate Social Affairs Commission -. Institutions must listen to the needs of patients. For people affected by acute myeloid leukemia, the strengthening of home care is essential, also foreseen by the National Oncological Plan 2023-2027. Bringing treatments closer to people also facilitates their accessibility, impacts on life expectancy and promotes savings for the system and for patients”.
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