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Doctoral Thesis
DOI
https://doi.org/10.11606/T.5.2022.tde-15062022-101526
Document
Author
Full name
Renata Aparecida dos Santos Lobo
E-mail
Institute/School/College
Knowledge Area
Date of Defense
Published
São Paulo, 2022
Supervisor
Committee
Rocha, Vanderson Geraldo (President)
Gualandro, Sandra Fatima Menosi
Peregrino, Antonio Augusto de Freitas
Silva, Roberto Carlos Lyra da
Title in Portuguese
Avaliação econômica do tratamento convencional versus o transplante de células-tronco hematopoiéticas na doença falciforme grave (SS e SBeta0)
Keywords in Portuguese
Anemia falciforme
Custos de cuidados de saúde
Hidroxiureia
Saúde
Transfusão de sangue
Transplante de células-tronco hematopoiéticas
Abstract in Portuguese
INTRODUÇÃO e OBJETIVO: Avaliações econômicas são relevantes e requeridas para o planejamento e sustentabilidade dos programas assistenciais do sistema de saúde. A doença falciforme (DF) é frequente no Brasil devido sua miscigenação populacional. DF desenvolve distúrbios multissistêmicos, com complicações agudas e crônicas graves ao longo da vida, como as crises vasooclusivas, acidentes vasculares encefálicos e insuficiência renal que reduzem a qualidade e expectativa de vida requerendo tratamentos de alto custo. A evolução da coorte com DF grave, portando genótipos SS e SBeta0 e >16 anos, foi estudada por 9 anos no Hospital das Clínicas da Universidade de São Paulo. Este estudo é o primeiro do Brasil a avaliar o impacto econômico da DF grave. MÉTODOS: Avaliação econômica com dados de ressarcimento dos recursos assistenciais prestados aos pacientes entre 2008 e 2016. As estratégias terapêuticas permitiram classificá-los em quatro subgrupos: 1. Seguimento Clínico; 2. Hidroxiureia; 3. Terapia Transfusional associada a Terapia Quelante; 4. Hidroxiureia, Terapia Transfusional associada a Terapia Quelante. A matriz relacional obtida por linkage, permitiu consolidar dados administrativos e clínicos para estimar modelos econômicos representativos de cada subgrupo de pacientes. Os ressarcimentos da jurisdição estadual de São Paulo permitiram validá-los e estimar o modelo comparativo com os dados contemporâneos dos Transplantes de Células-Tronco Hematopoiéticas realizados em pacientes com Anemia Aplástica. RESULTADOS: Avaliamos 333 pacientes, 267 do genótipo SS (80,18%) e 66 do genótipo SBeta0 (19,82%), com média de 39 anos de idade. Analisamos 387.672 AIHs referentes a 9.065 dias de internação. O ressarcimento total foi de R$3.315.910,60 entre 2008 e 2016, sendo 57% (R$1.891.949,49) referentes a tratamento ambulatorial e 43% (R$1.427.639,49) às hospitalizações. Os subgrupos foram comparados sob modelagem econômica tipo Árvore de Decisão em três cenários: o modelo referente com os ressarcimentos SUS e dois outros sob perspectivas mistas, considerando custos reais máximos e mínimos das estratégias terapêuticas adotadas. O custo do paciente falciforme grave foi de R$10.090,50 por ano. Pacientes DF-SS tiveram custo de R$11.075,48 e os DF-SBeta0 de R$ 6.149,38 por ano de tratamento. Aqueles em Terapia Transfusional, especificamente a ERTX e que vieram a óbito, tiveram maiores custos com valor médio de R$30.705,25 por ano. CONCLUSÃO: Nesta coorte, pacientes entre 20 e 39 anos foram os maiores direcionadores no impacto econômico na DF Grave, com maior frequência de internações de emergência e elevadas médias de permanência hospitalar, e representaram 70% dos potenciais pacientes elegíveis ao TCTH. O ressarcimento médio anual para o tratamento convencional da doença foi de R$ 10.090,50. O tratamento é por toda a vida em comparação com TCTH que pode curar a DF e que o SUS ressarce R$71.602,25/TCTH. Um TCTH, portanto, corresponde a sete anos de tratamento convencional e que deverá ser mantido por toda a vida, dessa forma, a realização do TCTH em tempo adequado pode aumentar a qualidade e expectativa de vida e diminuir os custos a médio e longo prazos para o tratamento da doença no sistema de saúde
Title in English
Economic evaluation of conventional treatment versus hematopoietic stem cell transplantation in severe sickle cell disease (SS and SBeta0)
Keywords in English
Anemia sickle cell
Blood transfusion
Health-care costs
Hematopoietic stem cell transplantation
Hydroxyurea
Public health
Abstract in English
INTRODUCTION AND OBJECTIVE: Economic evaluations are relevant and required for the planning and sustainability of assistance programs in the healthcare system. Sickle cell disease (SCD) is frequent in Brazil due to its population miscegenation. SCD develops multisystem disorders, with severe acute and chronic complications throughout life, such as vaso-occlusive crises, strokes and renal failure that reduce quality and life expectancy, requiring highcost treatments. The severe SCD cohort evolution, carrying SS and SBeta0 genotypes and >16 years, was studied for 9 years at Hospital das Clínicas of the São Paulo University Medical School. This is the first study to assess the economic impact of severe SCD in Brazil. METHODS: Economic evaluation with data on reimbursement of healthcare resources provided to patients between 2008 and 2016. The therapeutic strategies allowed to classify the patients into four subgroups: 1. Clinical follow-up; 2. Hydroxyurea; 3. Transfusion Therapy associated with Chelation Therapy; 4. Hydroxyurea, Transfusion Therapy associated with Chelation Therapy. The relational matrix obtained by linkage allowed the consolidation of administrative and clinical data to estimate representative economic models for each patient subgroup. The reimbursements from the São Paulo state jurisdiction allowed to validate them and estimate the comparative model with contemporary data on Hematopoietic Stem Cell Transplants, HSCT, performed for aplastic anemia patients. RESULTS: We evaluated the cohort of 333 patients, 267 of the SS genotype (80.18%) and 66 of the SBeta0 genotype (19.82%), with a mean age of 39 years. We analyzed 387,672 admissions authorized, AIHs, referring to 9,065 days of hospitalization. The total reimbursement was R$3,315,910.60 between 2008 and 2016, with 57% (R$1,891,949.49) referring to outpatient treatment and 43% (R$1,427,639.49) to hospitalizations. The subgroups were compared under Decision Tree economic modeling in three scenarios: the first model referred to the Brazilian healthcare system, SUS, reimbursements and two others under mixed perspectives, considering the maximum and minimum real institutional costs of the adopted therapeutic strategies. The cost of the severe sickle cell patient was R$10,090.50 per year. DF (SS) patients had a cost of R$11,075.48 and DF (SBeta0) of R$ 6,149.38 per year of treatment. Those with Transfusion Therapy, specifically with erythrocytaphereses, ERTX, and who died, had the highest costs of R$30,705.25 average per year. CONCLUSION: In this cohort, patients aged between 20 and 39 years were the main drivers of the economic impact in Severe SCD, with a higher frequency of emergency admissions and elevated length of hospital stay, and they represented 70% of the patients potentially eligible for HSCT. The average annual reimbursement for the conventional treatment of the disease was R$10,090.50. The treatment is lifelong compared to HSCT. HSCT can cure SCD and the SUS reimburses R$71,602.25/HSCT. An HSCT, therefore, corresponds to seven years of conventional treatment. Since conventional treatment must be maintained for life, offering HSCT at an appropriated stage can increase the patients quality and expectancy of life and can also, at medium and long term, reduce the costs for the treatment of this disease for the Brazilian healthcare system
 
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Publishing Date
2022-06-23
 
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