Doença falciforme: um estudo sobre os custos de três eixos terapêuticos no Brasil

Abstract

Sickle Cell Disease (SCD) is the most prevalent single-gene disease in the world, with a great impact on public health. The pathology has high morbidity and mortality. As it presents heterogeneous clinical manifestations, according to severity, SCD requires treatments with a variable impact on the budget of the Unified Health System (SUS). The main scope of follow-up and treatment of sickle cell patients revolves around basic care in the first years of life, which includes specialized medical follow-up, infectious prophylaxis with antibiotics and vaccines, continuous use of Folic Acid, complementary exams, a protocol for the prevention of stroke (Transcrian Doppler), among other actions. In addition, it is important to mention the transfusional support, which – is fundamental for the disease control and the hospitalizations, since SCD patients are hospitalized several times throughout their lives. This research aims to determine the direct medical costs of these three therapeutic axes of SCD in Brazil. In order to accomplish this objective, we use different methodologies and databases for data analysis. We only used data from public sources for the measurement of expenses related to basic prophylactic care in children with sickle cell disease up to five years of age and those related to hospitalizations. For the analysis of transfusion costs, we used the basis of the Recipient Epidemiology and Donor Evaluation-III (REDS-III) Brazil SCD Cohort. This is a cohort created to define the prevalence of clinical complications with a focus on transfusion outcomes in a large sample of the Brazilian population with SCD and to create the infrastructure to carry out targeted studies within the cohort. In all three investigations, the cost perspective adopted was that of the financing SUS (payer). These costs are paid by the health system to the service providers and the referral centers for the treatment of SCD, as it is the case of Blood Centers. Direct medical costs (consultations, exams, medication, etc.) were included in these analyses. Direct non-medical costs (companions, rehabilitation, etc.) and indirect costs (loss of productivity) were not included. We conclude that antibiotic prophylaxis, extended active immunization, multidisciplinary follow-up and the Transcranial Doppler Protocol are examples of effective and already implemented public policies that have a positive impact on the quality of life of children with SCD. We observed that morbidity and mortality reduce, the costs are considerably lower than those of transfusion therapy and expenses with hospital admissions due to disease complications. There was a huge gap in the scientific literature regarding costs related mainly to prophylactic care and transfusion therapy in SCD, making it difficult to compare results. Given the lack of studies on the subject in question, the data generated here can be used to calibrate the criteria adopted in future studies and help to encourage discussion about the important costs related to SCD.