Patients with β-thalassemia major (Mβ-th) are transfusion-dependent, which affects their quality of life . To maintain a safe level of iron in the body, patients with Mβ-th require adequate regular therapy with chelation drugs (CP). Currently, for the correction of iron overload in patients with Mβ-th, along with oral CP, parenteral CP continues to be used. However, oral and parenteral CP are perceived by patients ambiguously.

Comparative assessment of the quality of life of transfusion-dependent children with Mβ-th receiving various CPs: parenteral deferoxamine and oral deferasirox.

For 2 years, a survey and clinical observation of 201 children with Mβ-th aged 2 to 18 years (boys 128, girls 73) was conducted. The control group consisted of apparently healthy children from preschool and school institutions (n=30). Patients with Mβ-th underwent a quality of life study (PedsQL- Pediatrics Quality of Life Inventory, Generic Core Scales and PedsQLTM4.0) and a psychological examination.The survey was conducted after obtaining the informed consent of the parents of older children at the beginning and at the end of the study. Once a month, the necessary clinical and biochemical analyzes were carried out.Patients with Mβ-th regularly prescribed various CP regimens: deferoxamine subcutaneously; deferasirox, orally.

All studied patients with Mβ-th were divided into four age groups: group 1 - children under 4 years old according to parents (n=41); group 2 - children 5-7 years old according to the assessment of children and parents separately (n=62); group 3 - children 8-12 years old according to the assessment of children and parents separately (n= 47); Group 4 - children aged 13-18 years old according to the assessment of children and parents separately (n=51). Each of the 4 groups of Mβ-th patients was divided into a subgroup taking only deferiprone and a subgroup taking only deferasirox.

According to the Results of the survey, the indicators of the quality of life and the psychological state of children with Mβ-th receiving parenteral and oral CP differed. So, in sick children with Mβ-th of different age groups, when taking parenteral CP in comparison with those taking oral CP, the quality of life was reduced, and the psychological state worsened significantly. This was especially impacted patients in the group of 8-13 years. In this group, there were more complex relationships with peers, parents, there was an increase in anxiety and aggressiveness, which is associated with the need for hours of use of the pump for subcutaneous injection of the drug, the presence of pathology that limits the use of oral chelators. In children of 4 different age groups, there is a significant difference in the values given by patients and their parents to the quality of life in patients receiving parenteral and enteral chelator therapy.