Título:	Análise temporal da mortalidade por leucemia linfoblástica aguda no Distrito Federal - Brasil e avaliação global de custo-efetividade do tisagenlecleucel
Autor(es):	Andrade, Aurélio Matos
Orientador(es):	Carvalho, Juliana Lott de
Coorientador(es):	Figueiredo, Ana Claudia Morais Godoy
Assunto:	Leucemia linfoblástica aguda (LLA) Terapia celular Câncer - tratamento
Data de publicação:	29-Jun-2026
Data de defesa:	26-Mar-2024
Referência:	ANDRADE, Aurélio Matos. Análise temporal da mortalidade por leucemia linfoblástica aguda no Distrito Federal - Brasil e avaliação global de custo-efetividade do tisagenlecleucel. 2024. 162 f., il Tese (Doutorado em Ciências Médicas) — Universidade de Brasília, Brasília, 2024.
Abstract:	Acute Lymphoblastic Leukemia (ALL) is an aggressive hematological neoplasm characterized by the uncontrolled proliferation of lymphocyte precursor cells. The disorder promotes the accumulation of immature cells in the blood, substantially compromising hematopoiesis in the bone marrow and potentially leading to death. Although this pathology is more prevalent in children, its incidence also extends to the adult population group. Currently, there are efficient therapeutic options for some patients. However, refractory and recurrent cases of this neoplasm impose a clinical, economic and social dilemma, requiring innovative and costly therapeutic approaches. With this in mind, this thesis was structured with the objective of analyzing mortality from acute lymphoblastic leukemia in the Federal District - Brazil and the overall cost effectiveness ratio of advanced therapy Tisagenlecleucel versus conventional therapies for Refractory and Refractory B-cell Acute Lymphoblastic Leukemia. Relapsed R/R-B ALL. The methodological path was outlined in two methods: the first was an ecological time series study using data from the Mortality Information System classified by code C91.0 of the International Classification of Diseases. The results showed a predominantly male profile (51.70%), single (60.41%), white race/color (52.16%), with four to seven years of education (27.37%). The mortality trend due to ALL was increasing for the age groups of 0-4 years and 50-59 years, with increasing rates of 9.0% and 8.7%. It is concluded that it was possible to determine the profile of people who die most from ALL in the Federal District (DF), in addition to understanding temporal changes over 20 years in different age groups, warning that scientific discussions and reallocation of resources must be prioritized in the direction and applicability of public policies, highlighting the gaps in diagnosis and treatment for children up to 4 years old and for adults who are aging. In the second article, the method consisted of a systematic review that followed the parameters of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA). The systematic review highlighted the Incremental Cost-Effectiveness Ratio with discount per Quality-Adjusted Life Year (QALY) gained after the use of Tisagenlecleucel compared to the analysis of the averages for Combined Clofarabine (Clo-C) was US$38,837.00 and the Blinatumomab (Blina) US$25,569.00. Regarding the cost of the medicine, the average for Tisagenlecleucel was around 4.3 times; 10.8 times; 4.7 times respectively higher than the average Monotherapeutic Clofarabine (Clo-M); Clo C; and Blina. The global cost-effectiveness assessment played a crucial role in this thesis for children and young adults with R/R-B ALL, considering Tisagenlecleucel is a cost-effective option. Therefore, it should be considered and financed by public policy makers and by the Unified Health System (SUS) as an alternative for treating patients in the Federal District, Brazil and around the world.
Unidade Acadêmica:	Faculdade de Medicina (FM)
Informações adicionais:	Tese (doutorado) — Universidade de Brasília, Faculdade de Medicina, Programa de Pós-Graduação em Ciências Médicas, 2024.
Programa de pós-graduação:	Programa de Pós-Graduação em Ciências Médicas
Agência financiadora:	Fundação para o Desenvolvimento Científico e Tecnológico em Saúde (FIOTEC)
Aparece nas coleções:	Teses, dissertações e produtos pós-doutorado

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