by Vera Martinella
The drug in tablet form, now available in Italy, is able to prolong the overall survival of patients in remission and the duration of response to therapy, with a good quality of life
After 30 years in which only chemotherapy was available, unfortunately only partially effective, several innovative treatments have arrived in the last five years which are radically changing the way of treating acute myeloid leukemia, which still remains an aggressive neoplasm (which was fatal for the ‘former footballer Sinisa Mihajlovic), and difficult to fight also because, not infrequently, he faces a recurrence. Precisely to reduce the risk of relapse, the Italian Medicines Agency (Aifa) has approved a new maintenance therapy, azacitidine, capable of lengthening the survival of patients who cannot undergo a transplant and reducing the risk of the tumor returning .
Symptoms
Every year there are about 3600 new cases of acute myeloid leukemia in Italy, which originates in the stem cells present in the bone marrow and develops very rapidly. It is most likely to affect people over 65, but it can also occur in children and younger people. Current treatments can bring the disease to remission, but after the initial response to standard treatment, in about 50% of cases the disease returns within a year. So, unfortunately, 5 years after diagnosis, depending on age, survival fluctuates between 20% and 40-45% of patients and does not exceed 12 months for those with relapsing or refractory disease, i.e. that does not respond to treatment. The symptoms depend on the progressive infiltration of leukemia cells into the bone marrow, which loses the ability to perform its functions and produce blood cells – explains Adriano Venditti, director of hematology at the University of Rome Tor Vergata -. A condition of bone marrow insufficiency occurs which leads to anemia, tiredness and pallor. The number of platelets decreases, with a tendency to haemorrhage. Furthermore, there is a reduction in white blood cells which determines a greater probability of developing infections, precisely because the defenses constituted by white blood cells are lessened. The alterations of the blood count values lead to the diagnosis, which also passes through the bone marrow sampling.
Therapy
To date, the most effective therapy for healing remains donor marrow transplantation, but the majority of patients (also due to advanced age) are not candidates for this heavy procedure to tolerate. Furthermore, it is essential that the treatment is differentiated on the basis of the type of molecular alterations present in the individual case: for this reason it is essential that all patients, before starting therapy, do a very sophisticated test on the blood and marrow cells, to identify quickly and precisely the targets for the drugs to be associated with the traditional treatment. We generally proceed with intensive chemotherapy which aims to achieve complete remission of the disease, and then move on to consolidation or maintenance therapy, the aim of which is to eliminate all residual tumor cells – explains Fabrizio Pane, Full Professor of Hematology and Director of the Hematology and Marrow Transplant Operational Unit at the Federico II University of Naples -. If possible, in patients who achieve complete remission and who can tolerate it, continue with stem cell transplantation. Responses to intensive chemotherapy may be short-lived and the risk of recurrence high, especially for people who are not transplant candidates . It is in this context that the new therapy takes place.
The new cure
The new drug approved by Aifa, oral azacitidine, is able to improve survival by reducing the probability of recurrence. Treatment now available as maintenance therapy in patients who have achieved complete remission (or complete remission with incomplete recovery of blood counts) after induction therapy with or without consolidation treatment and who are ineligible for stem cell transplantation hematopoietic. Azacitidine is the first oral maintenance therapy that has been shown to increase overall survival and has shown a recurrence-free survival benefit in patients with acute myeloid leukemia. The drug belongs to the class of hypomethylants, because it reduces DNA methylation: in this way the normal function of the genes fundamental in cell differentiation and proliferation compromised by the disease is restored. In the international QUAZAR AML-001 study, published in the New England Journal of Medicine, which enrolled 472 patients, median overall survival was greater than two years (24.7 months) in patients treated with oral azacitidine compared with 14.8 months with placebo. Median recurrence-free survival was also significantly longer with oral azacitidine, reaching 10.2 months compared with 4.8 months in the control arm.
Improving the quality of life
The availability of innovative therapies such as oral azacitidine reopens the “chapter” of maintenance therapy, for which interest had waned for at least twenty years and which is now starting to play an important role again, not only for elderly patients – underlines Venditti – . Younger people should also be considered and the general conditions of the patient analyzed who, during intensive chemotherapy, may develop complications that contraindicate allogeneic transplantation. In fact, it is estimated that only 20-30% of patients eligible for a transplant are then able to actually access this complex procedure. There is therefore a large number of patients who, regardless of age, can find benefit from oral azacitidine. Furthermore, the fact that the treatment is in tablets makes it easier to take for people who are in complete remission and therefore they can and must be able to have a good quality of life. Chronic myeloid leukemia is one of the most insidious and difficult haematological diseases to treat, which often has an insidious onset – explains Marco Vignetti, vice president of AIL (Italian Association against leukemia, lymphoma and myeloma) -. In many cases, the patient progresses from a state of well-being to a condition of severely impaired health within a short time. The emotional impact of a sudden diagnosis of acute myeloid leukemia is devastating for both patients and family members. The most common reaction is a sense of profound distress and concern, which subsides as complete remission is achieved. In patients in remission, it occurs due to the uncertainty due to the possibility that the disease may recur. These elements underline the need to provide innovative therapies capable of reducing the risk of recurrence and structured psychological support. Fragility and uncertainty, combined with anxiety, are the keywords that accompany the lives of patients and caregivers on a daily basis. Innovation must be combined with the multidisciplinary nature of the treatment path, which also includes the figure of the psycho-oncologist.
July 17, 2023 (change July 17, 2023 | 06:56)
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