Acquired aplastic anemia
The disease occurs in all age groups; in the age group of 0-17 years, 1-2 cases per 1 million children are registered. The pathogenesis of PAA is based on immuno-mediated depletion of the hematopoietic stem cell pool. In most cases, it is not possible to determine the presence of a specific etiological factor that caused the development of the disease. In such cases, we are talking about idiopathic (more than 70% of all cases) acquired aplastic anemia. Possible etiological factors include certain medications, exposure to chemical / toxic agents. Some viruses also play a role in the development of PAA: hepatitis a, B, and C viruses, cytomegalovirus, parvovirus B19, and some others. PAA can develop as a complication of diseases associated with immune disorders.
The clinical manifestations of the disease may develop slowly and are presented anemia by reducing red blood cells and hemoglobin, hemorrhagic syndrome due to the decrease in platelet count and the violation of their function and infectious complications by reducing the content, the total number of leukocytes. Therefore, the complaints that the patient addresses to the doctor are very diverse. Performing such a publicly available laboratory study as a General blood test with the calculation of the leukocyte formula allows you to suspect aplastic anemia and refer the patient to a specialist. There are also cases when the patient is not concerned about anything, and changes in the General blood test were detected during a preventive examination.
PAA should differentiate from other conditions that lead to pancytopenia, including congenital aplastic anemia (for example, Fanconi anemia, Blackfan-diamond anemia). Differential diagnosis of PAA and hypoplastic variant of myelodysplastic syndrome is particularly difficult.
Until recently, PAA treatment was symptomatic. The annual survival rate of patients receiving symptomatic therapy did not exceed 10-20%. Death occurred from infectious complications or hemorrhages in vital structures (the brain).
Currently, there are highly effective methods of treating АAA. The optimal method of treatment for children is hematopoietic stem cell transplantation (bone marrow transplantation) from a related fully compatible donor, which is effective in 90% of cases. Unfortunately, less than 25% of patients have a related compatible donor.
Patients with АAA who do not have a related compatible donor, in first-line therapy, imunosuppressive therapy is performed using antithymocytic globulin (rabbit or horse) and cyclosporin. The effectiveness of therapy is good. According to various studies, its effectiveness is 55-85%. Patients who have not received a good hematological response by a certain time, a second course of immunosuppressive therapy is performed, while antithymocytic globulin from another manufacturer is used or hematopoietic stem cells are transplanted from an unrelated donor.
Diagnosis and treatment of АAA for children who are citizens of the Republic of Belarus is carried out only in the state institution "Republican scientific and practical center of children's Oncology, Hematology and immunology". During the ten-year period from 2003 to 2012, 70 children aged 0-17 years were diagnosed with АAA in the Republic of Belarus. Treatment was carried out according to the principles described above. The overall survival rate of these patients was 81%.
Diagnosis and treatment of АAA for children who are citizens of the Republic of Belarus is carried out in the state institution "Republican scientific and practical center of children's Oncology, Hematology and immunology", regardless of the address of residence (registration) at the time of diagnosis. Diagnostics, treatment and follow-up in the center's consulting and polyclinic Department for citizens of the Republic of Belarus are free of charge. Patients who do not need intensive follow-up can undergo most of the treatment in the day care unit, which makes the treatment more comfortable for the patient.
For foreign citizens, treatment is carried out on a paid basis in accordance with the current Legislation. The cost of АAA treatment is the cost of a bed-day in the Department, the cost of medicines, blood products, reagents, consumables, for the examination of a hematopoietic stem cell donor and the procedure for cell sampling.
The absence of a language barrier is an important advantage for CIS citizens who are being treated at the state institution "Republican scientific and practical center for children's Oncology, Hematology and immunology".