Banca de QUALIFICAÇÃO: SUELY NILSA GUEDES DE SOUSA ESASHIKA

Uma banca de QUALIFICAÇÃO de DOUTORADO foi cadastrada pelo programa.
STUDENT : SUELY NILSA GUEDES DE SOUSA ESASHIKA
DATE: 12/05/2025
TIME: 14:30
LOCAL: Plataforma Teams
TITLE:

Monitoring of Genetic Therapies Incorporated by the Unified Health System (SUS) for the Treatment of Spinal Muscular Atrophy (SMA) 5q Types I and II: A Retrospective Analysis of Medication Dispensation.

KEY WORDS:

Spinal Muscular Atrophy; Medications; Nusinersen; Risdiplam; Zolgensma; Dispensation; Monitoring; Information System; Unified Health System (SUS)

PAGES: 100
BIG AREA: Ciências da Saúde
AREA: Saúde Coletiva
SUMMARY:

“_Introduction: Spinal Muscular Atrophy (SMA) is characterized as a neuromuscular, autosomal recessive disease caused by the deletion or mutation of the SMN1 gene. It is a neurodegenerative disease, characterized by the progressive loss of motor neurons, resulting in muscle weakness. The most prevalent form of the disease, occurring in more than 95% of cases, is SMA type I, which has an early onset and a more severe prognosis, followed by SMA type II, which represents 27% of the cases. For the treatment of SMA, the Brazilian Unified Health System (SUS) provides the following medications: Nusinersen and Risdiplam, and recently incorporated the drug Zolgensma (onasemnogene abeparvovec-xioi). These incorporations began in 2019, and require periodic evaluation regarding their use, proper dispensation of the drugs, and monitoring that includes effective access to the incorporated technology by the population, as well as observed effects after use. General Objective: (1) To evaluate the importance of monitoring the incorporation, implementation (access to technology by the population), and dispensation of the medications Nusinersen and Risdiplam for use by patients with SMA 5q Types I or II, from 2019 to 2024, according to the criteria established in the SUS Clinical Protocols and Therapeutic Guidelines. Specific Objective: (1) To analyze the information generated by the SUS Information System that supports the incorporation, substitution, evaluation, and implementation of technologies for the treatment of SMA types I and II, and to identify factors that may compromise the effectiveness of incorporating these technologies into SUS. Specific Objective: (2) To analyze the socio-epidemiological profile of the population with SMA Types I and II, according to demographic distribution (age, sex, ethnicity, state etc.) of the affected population, during the same period, that encompasses the incorporation of these technologies. Method: The research project consists of two methodological stages: (I) the development of a literature review on the technological advances in SMA treatment, and (II) the analysis of existing records in the Ministry of Health's Information Systems, regarding the dispensation and monitoring of the treatment progress of patients with SMA types I and II in Brazil. Preliminary Results: Considering the complexity of the information generated by the Ministry of Health's Information System, and its relevance for public health actions and policies, a preliminary analysis of the data revealed important aspects regarding the criteria established for the dispensation of medications, such as the absence of information in some fields that support distribution and the lack of case registration in some states. These issues could compromise treatment and increase the burden on the health system. ”

COMMITTEE MEMBERS:
Presidente - 404501 - DIRCE BELLEZI GUILHEM
Externa à Instituição - FLAVIA CASELLI PACHECO - MS
Externa à Instituição - MARTA DA CUNHA LOBO SOUTO MAIOR - MS
Externo à Instituição - Ricardo Gadelha de Abreu - MS