2023-05-03 15:27:25
Anemia, fatigue, paleness, bleeding and bruising, linked to platelet deficiency, and infections are the main symptoms of acute myeloid leukemia, an aggressive blood cancer that affects around 3,300 people in Italy every year. For newly diagnosed patients unsuitable for standard intensive chemotherapy, potentially 40% of the total, AIFA 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. Recognition reserved only for molecules which demonstrate, on the basis of solid scientific evidence, that they satisfy important therapeutic needs and bring an added therapeutic value, first of all, in the case of oncological drugs, the increase in survival. Venetoclax in combination with azacitidine has in fact demonstrated a longer overall survival, higher remission rates and rapid and durable responses. This is what emerges from the press conference “Acute myeloid leukemia: new perspectives in the management of the disease”, promoted today in Rome.
“Acute myeloid leukemia is an aggressive hematological disease – says Maria Teresa Voso, full professor of Hematology at the Tor Vergata University and head of the Advanced Oncohaematological Diagnostics laboratory of the Tor Vergata Polyclinic – extremely heterogeneous, characterized by the uncontrolled proliferation of immature bone marrow cells bone, the blasts, with a very rapid development. Diagnosis usually occurs following evaluation of blood count abnormalities and is confirmed with more specific investigations that require evaluation of the bone marrow through bone marrow aspiration. The majority of cases present in old age and the mean age at diagnosis is 69 years. Elderly or frail patients, due to other pathologies, are unable 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”.
“Venetoclax – continues Voso – induces apoptosis, the ‘programmed’ death of acute myeloid leukemia cells, through a selective and potent inhibition of Bcl-2, a protein that allows the survival of cancer cells. Venetoclax also performs synergistic activity with hypomethylating agents, indirectly increasing sensitivity to Bcl-2 inhibition.” Hypomethylating agents also interfere with other mechanisms that drive disease development, and prevent the growth of abnormal cells in the bone marrow.
The 5-year survival from diagnosis today is about 28% – reports a note -. 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 – explains Giovanni 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, it confirmed this survival advantage, with a 42% reduction in the risk of death. Complete remission achieved with venetoclax plus azacitidine was two times greater (66%) than with azacitidine alone (28.3%). It should be emphasized 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”.
“These results – remarked Martinelli – have a significant clinical significance: for the first time it is possible to obtain complete remissions without resorting to chemotherapy. Furthermore, the data have been confirmed in ‘real life’, i.e. in daily clinical practice, as evidenced 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 of 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”.
“More than 50% of these patients are over 65 years of age and about a third are over 75 – underlines Felicetto Ferrara, director of hematology at the Cardarelli hospital in Naples -. The disease in the elderly has poor prognostic characteristics compared to young adults because they are more resistant to chemotherapy. Hence the difficulties of achieving complete remission and long-term survival in this population. Thanks to ‘early access’ tools, i.e. early access to treatments, this innovative therapy was made available in Italy even a year before European approval and three before reimbursement in our country”.
“In fact, Aifa – underlines Ferrara – in March 2020, at the request of the scientific community and patient associations, included venetoclax in the list of drugs that can be paid entirely by the NHS pursuant to law n.648 of 1996, for the treatment of acute myeloid leukemia, in patients unsuitable for induction chemotherapy. In this way, a possibility of treatment was offered in a particularly delicate population, to cope with the lack of therapeutic alternatives and respond to unmet clinical needs. Thanks to the ‘early access’ over 2,000 patients were treated before reimbursement, with results comparable to those obtained in the pivotal study”.
“The moment of communication of the diagnosis – says Giuseppe Toro, national president of Ail – is characterized by emotions such as fear, discouragement, anger, concern. For this reason, increasingly integrated and multidisciplinary assistance is needed which, together with the figures of the hematologist and of the nurse, also includes the psychologist – a central figure in patient support which, like Ail, we intend to strengthen more and more – the nutritionist, the palliative care specialist and the infectious disease specialist.Furthermore, it is important to contribute to guaranteeing rapid and uniform access to innovation and social-health support services in the area”.
“We are facing a virtuous case of access to therapeutic innovation in our country, made possible by the concrete contribution of all the actors involved – highlights Fabrizio Greco, CEO of AbbVie – In 2020, Aifa’s decision to accept the community’s request scientific and patient associations to make venetoclax available in Italy in advance of the European approval, has allowed more than 2,000 patients with acute myeloid leukemia to benefit from increased survival. 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 and individual center level. 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 allow the value of therapeutic innovation to be recognized and to make it available quickly and widely to all those who they need it”. Finally, Tilde Minasi, president of the parliamentary intergroup ‘Oncology: prevention, research and innovation’ and member of the Senate’s Social Affairs Commission: “Our universal healthcare system guarantees access to treatment for all patients. For people affected by acute myeloid leukemia, the strengthening of home care is essential, also envisaged 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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