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Standards in the treatment of leukemia have been nearly the same over the past half a century. Today – apart from chemotherapy – we also have targeted therapy at our disposal. This improves the effectiveness of treatment – explains prof. UM dr hab. n. med. Lidia Gil, head of the Department of Hematology and Bone Marrow Transplantation at the Medical University of Poznań.
- Leukemias are malignant diseases of the hematopoietic system that occur at any age, and the risk of their occurrence increases after the age of 60.
- Under the slogan “leukemia” there are several dozen different types of this disease – chronic and acute leukemias, myeloid and lymphoblastic leukemias
- Thanks to the currently available studies, especially genetic studies, it has become possible to define the subtypes of leukemia, which allows to determine the prognosis and select the therapy – for example, to decide whether the patient should only be scheduled for chemotherapy or if treatment with bone marrow transplantation is necessary
- A novelty in the treatment of leukemia is targeted therapy. It means that when we have a patient with acute leukemia, we give him chemotherapy, but if we find that leukemia cells have a specific genetic trait, we can add a drug to the treatment that accurately destroys this population of cancer cells. Drugs taken orally are also new.
The subject of leukemias is a very extensive issue, as it covers both chronic and acute conditions. Leukemias are hematopoietic neoplastic diseases that occur at any age, and the risk of their occurrence increases as the body ages. Population statistics show that leukemia is much more common in adults than in children, and the incidence is highest in people over 60 years of age. Acute myeloid leukemia (AML) is the most common disease in adults. acute myeloid leukemia). While in some subtypes of these cancers it may happen that men are affected more often, they generally affect both genders with the same frequency.
Diagnostics and treatment adapted to the patient’s needs
Chronic and acute leukemias are typical haematological entities. According to the WHO classification, several dozen subtypes are distinguished among acute myeloid leukemias. The method of treatment and the standard of care vary depending on the patient’s age, general condition and comorbidities – this applies to both acute myeloid leukemias and lymphoblastic leukemias: patients up to 60-65 are treated differently. years of age, otherwise the elderly sick. Specialized medical scales are helpful in qualifying patients for treatment. Thanks to this, we can decide how to safely treat the patient and what treatment goals should be set.
Confirmation of the diagnosis of leukemia is based on a fairly wide diagnostic profile. Bone marrow examination is essential: aspiration method and trepanobiopsy (especially in the diagnosis of chronic leukemias), as well as genetic tests – cytogenetics, molecular tests and flow cytometric testing.
The type of leukemia matters
Thanks to the currently available research, especially genetic research, it has become possible to determine the molecular subtypes of leukemias as well as the characteristics of these types. Depending on the type of leukemia, we can change the treatment and management. The prognosis is also different. On the one hand, genetic diagnosis confirms the diagnosis and enables the initiation of treatment, and on the other, it enables the prognosis to be determined and the question of whether the patient should only receive chemotherapy, or whether treatment with bone marrow transplantation is necessary.
The results of molecular tests indicate the possibility of using targeted therapy, usually in combination with standard chemotherapy, which significantly improves the effectiveness of the treatment. Recently, immunotherapy has been very successful in hematology, especially in the treatment of lymphoid neoplasms, such as acute lymphoblastic leukemia.
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Extending overall survival – the primary goal of treatment
In the last few years, new drugs for acute myeloid leukemia have been approved worldwide – for the first time in 50 years. And these are the drugs that really affect the prognosis and the effectiveness of the treatment. Treatment standards have been nearly the same over the past half a century. Today, in addition to chemotherapy, we also have targeted therapy. This means that when we have a patient with acute leukemia, we give him chemotherapy, but if we find that the leukemia cells have a specific genetic trait, we can add a drug to the treatment that accurately destroys this particular population of cancer cells.
This improves the effectiveness of the entire treatment. By the effectiveness of treatment, we mean that, firstly, we will eliminate leukemia, and secondly, that we will permanently eliminate it. In medicine, this is called complete remission. Our primary goal is to extend overall survival.
Currently, there are several drugs available for which we, doctors, are fighting a lot because they are registered in Poland and in Europe, but there is no funding for treatment with these drugs in our country. Their use is possible thanks to special early access programs offered by pharmaceutical companies that manufacture these drugs – the patient then receives such a drug for free.
- Leukemia. Find out about the causes of this disease, its symptoms, course and treatment
Oral drugs – comfort and safety of therapy
Currently, the most frequently used targeted drug in the treatment of acute myeloid leukemias is midostaurin, used in the form of a disease with an unfavorable change in the FLT3 gene. It is a drug that, in registration trials and in practice, has an impact on survival in patients with this type of molecular disorder. It is used orally in combination with standard chemotherapy. In patients with the FLT3 mutation, we can also use a second oral drug – gilteritinib, approved for the treatment of refractory and relapsed forms of AML. There are also other drugs – already registered in the United States and successfully used there – for the treatment of selected patients. We are still waiting for registration by the European Medicines Agency (EMA). European Medicines Agency) and therefore these therapies are not available in Poland yet. We are waiting longingly for many drugs.
The important thing: the current pandemic will permanently change the way people think about certain treatments. The related situation in medicine also showed us that the use of oral forms of drugs gives us time and comfort, because we know that the patient is protected. This is very important in the treatment of cancer, and in the treatment of acute leukemia in particular, because any delay, even several days, in the administration of the drug may have serious consequences for the patient’s health.
FLT3 mutation and its consequences
The FLT3 mutation is the most common mutation in acute myeloid leukemia in adults. It is noted in 30% of patients with acute leukemia – this is what the literature says and I confirm it based on my own experience, because in our center 1/3 of patients with leukemia show this mutational change.
In patients with the FLT3 mutation, the prognosis is worse – even if the standard treatment is successful in achieving remission of the disease, the disease recurs very quickly and relapses are usually resistant to treatment. So far, patients with this mutation have been intensively treated with chemotherapy and quickly qualified for hematopoietic cells transplantation. Unfortunately, also in these patients – more often than in others – the disease also recurs after transplantation, and in some patients we cannot transplant, because we cannot carry them out to the transplant phase due to the disease that is active all the time.
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Thanks to the possibility of combining chemotherapy with targeted drugs, a greater percentage of patients respond to treatment, remission is more permanent and we have time to find a donor and prepare both the transplant procedure and the patient for transplantation.
Continuation of pharmacological treatment after the end of therapy, during the so-called maintenance period, is very important and extends the survival of patients. And it gives a better prognosis – especially in relation to patients who cannot undergo bone marrow transplantation due to their age or the presence of comorbidities.
What influences the prognosis?
In patients up to 60-65. years of age, whom we can treat intensively, who qualify for attachment, the survival rate on a scale of 3 to 5 years is about 40-60%. Much better results are obtained in patients of the same age, but from the group of the so-called good prognosis, i.e. treated only with chemotherapy, in whom there is no need for transplantation, because the disease has a good prognosis for them. With proper treatment, 80% or more patients can be cured; many patients survive for several years.
On the other hand, the results of older patients, who cannot be treated intensively because age or coexisting diseases do not allow it, are much worse. Here, the effectiveness of the therapy is estimated at about 20-30% percent.
- The article comes from the campaign “Hematology – learn about blood diseases” prepared by Warsaw Press. All materials can be found on http://hematologia.warsawpress.com/
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