Banca de DEFESA: VINICIUS RODRIGUES DE OLIVEIRA

Uma banca de DEFESA de DOUTORADO foi cadastrada pelo programa.
STUDENT : VINICIUS RODRIGUES DE OLIVEIRA
DATE: 10/10/2025
TIME: 10:00
LOCAL: Departamento de Enfermagem
TITLE:

Validation of a set of guidances to support nurses in home-based
enzyme replacement therapy administered to children and adolescents diagnosed
with mucopolysaccharidosis.

 

KEY WORDS:

Mucopolysaccharidoses; Home-based Enzyme Replacement Therapy; Child; Adolescent; Nursing.

 

PAGES: 182
BIG AREA: Ciências da Saúde
AREA: Enfermagem
SUMMARY:

Enzyme replacement therapy is widely used for the treatment of mucopolysaccharidoses and should be started early after diagnosis, still in childhood. This therapy is performed weekly or biweekly in infusion centers, with an average duration of more than three hours, which directly impacts the routine of the patient and family, with school and work absences and long commutes. Home treatment is emerging as a promising alternative, bringing benefits that culminate in greater well-being and comfort for children, adolescents, and their families. However, in Brazil, there are few guidelines for this modality, especially regarding nursing care. Thus, the present study aimed to validate a set of guidances to support nurses in home-based enzyme replacement therapy administered to children and adolescents diagnosed with mucopolysaccharidosis. This is a methodological study guided by the assumptions of Pasquali's psychometrics (2010) and operationalized in three phases, namely: (1) scoping review; (2) content validation with expert judges; (3) statistical analyses to confirm the validity of the material. The study was approved by the research ethics committee of the Federal University of Rio Grande do Norte with opinion number: 7,633,703 and Certificate of Ethical Review: 87097225.2.0000.5537. In the first phase (scoping review), 12 publications were analyzed, which, using the PAGER strategy, led to the creation of five categories that provided theoretical support for the set of guidelines. The second (content validation) and third phases (statistical analyses) took place in two cycles (Delphi I and Delphi II). Ten judges participated in the first cycle, most of whom were women (80%), residents of the northeast region of the country (80%), nurses (80%), with up to seven years of experience (70%), working in a university hospital (40%). These judges evaluated the clarity, relevance, and accuracy of the material and made 33 suggestions for improving the content. Regarding the statistical analysis of the evaluations, in this first cycle, the content validity coefficient exceeded 0.80, but 54% of the items did not reach the recommended value (≥0.80) in the Kappa coefficient test. Given this, a new cycle of evaluations was undertaken. In the second cycle, nine judges reevaluated the material, four of whom made observations about the writing of the item. In the statistical analysis, there was an increase in the content validity coefficient and in the Kappa test; in both tests, the values were above 0.80 in the item-by-item and overall evaluations. The final version of the set of guidelines consisted of 14 items distributed across five categories. The set of guidelines was considered valid by the expert judges. It is hoped that, when applied in practice, this material will promote positive changes in the health context of children and adolescents with mucopolysaccharidosis and in the work of nurses. In addition, this study promoted a discussion about comprehensive care, the vulnerabilities and rights of children and adolescents with rare diseases, highlighting the leading role of nurses.

 

COMMITTEE MEMBERS:
Externa à Instituição - Margarita Del Rosario Baeza Fuentes
Interna - 1299781 - ALEXSANDRA RODRIGUES FEIJAO
Interna - 3149773 - DANDARA NAYARA AZEVEDO DANTAS
Presidente - 2696718 - JONAS SAMI ALBUQUERQUE DE OLIVEIRA
Externa à Instituição - Rosângela Aparecida Pimenta Ferrari