Article type
Abstract
"Background:Spinal muscular atrophy (SMA) is a rare disease and the most common among the group of hereditary autosomal recessive neuromuscular diseases. The most prevalent form of the disease is type I SMA, which has an early onset and a more severe prognosis. Due to rapid muscle deterioration and inability to breathe or swallow, SMA care places a great burden on patients, caregivers and health systems, making it essential to develop Clinical Practice Guidelines (CPG) that can guide SMA patients’ treatment.
Objective: To describe the SMA CPG in Brazilian Unified Health System (SUS).
Methods:Descriptive study of the SMA CPG development and its updates by the National Committee for Health Technology Incorporation (Conitec).
Results: The first version of SMA CPG was published in 2019, due to nusinersen incorporation for type I SMA treatment. In addition to drug treatment, CPG guides clinical and genetic diagnosis and non-pharmacological care. Its public consultation (PC) received more than 1,500 contributions from civil society.
In 2021, the CPG was updated to recommend nusinersen use in type II SMA patients, diagnosed up to 18 months age. This update PC received more than 500 contributions.
In 2022, the CPG was updated again to recommend risdiplam use for types I and II SMA patients. Its PC received approximately 200 contributions.
In December 2022, onasemnogen abeparvoveque was incorporated for type I SMA treatment in patients aged up to six months, who are not on invasive ventilation for more than 16 hours a day. Thus, the CPG was updated in 2023. In the PC 1,110 contributions were received, including patient caregivers, those interested in the topic, health professionals, patients, civil society organizations and companies.
Conclusions: The main treatments for SMA are incorporated into SUS and their use is subject to scientific evidence-based criteria. SMA Clinical Practice Guidelines promote access and the proper use of technologies by Brazilian patients. Also, during all CPG development process, Brazilian citizens could participate and contribute with technical and non-technical information."
Objective: To describe the SMA CPG in Brazilian Unified Health System (SUS).
Methods:Descriptive study of the SMA CPG development and its updates by the National Committee for Health Technology Incorporation (Conitec).
Results: The first version of SMA CPG was published in 2019, due to nusinersen incorporation for type I SMA treatment. In addition to drug treatment, CPG guides clinical and genetic diagnosis and non-pharmacological care. Its public consultation (PC) received more than 1,500 contributions from civil society.
In 2021, the CPG was updated to recommend nusinersen use in type II SMA patients, diagnosed up to 18 months age. This update PC received more than 500 contributions.
In 2022, the CPG was updated again to recommend risdiplam use for types I and II SMA patients. Its PC received approximately 200 contributions.
In December 2022, onasemnogen abeparvoveque was incorporated for type I SMA treatment in patients aged up to six months, who are not on invasive ventilation for more than 16 hours a day. Thus, the CPG was updated in 2023. In the PC 1,110 contributions were received, including patient caregivers, those interested in the topic, health professionals, patients, civil society organizations and companies.
Conclusions: The main treatments for SMA are incorporated into SUS and their use is subject to scientific evidence-based criteria. SMA Clinical Practice Guidelines promote access and the proper use of technologies by Brazilian patients. Also, during all CPG development process, Brazilian citizens could participate and contribute with technical and non-technical information."