Oliveira, Jonas Sami Albuquerque deOliveira, Vinicius Rodrigues de2025-12-292025-12-292025-10-10OLIVEIRA, Vinicius Rodrigues de. Validação de um conjunto de orientações para guiar a atuação da enfermeira na terapia de reposição enzimática domiciliar administrada em crianças e adolescentes com mucopolissacaridose. Orientador: Jonas Sâmi Albuquerque de Oliveira. 2025. 192 f. Tese (Doutorado em Enfermagem na Atenção à Saúde) - Centro de Ciências da Saúde, Universidade Federal do Rio Grande do Norte, Natal, 2025.https://repositorio.ufrn.br/handle/123456789/67460Enzyme replacement therapy is widely used for the treatment of mucopolysaccharidoses and should be started early, during childhood. This therapy is performed weekly or biweekly at infusion centers, with an average duration of more than three hours, which directly impacts the routine of the patient and their family, with school and work absences and long commutes. Home treatment is a promising alternative, bringing benefits that result 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 guidelines to guide the nurse's performance in home enzyme replacement therapy administered to children and adolescents diagnosed with mucopolysaccharidosis. The philosophical framework of Emmanuel Levinas was used to promote discussion about comprehensive care throughout the study. A methodological study was conducted based on the assumptions of Pasquali's psychometrics (2010) and operationalized in three phases, namely: (1) development of the set of guidelines; (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 with opinion number: 7,633,703 and Certificate of Ethical Review: 87097225.2.0000.5537. In the first phase, a scoping review was conducted, analyzing 12 publications, which guided, using the PAGER strategy, the development of five categories that provided theoretical support for the preparation of the set of guidelines. The recommendations were then analyzed and adapted to the recommendations of the Appraisal of Guidelines for Research and Evaluation II tool. The second and third phases took place in two cycles (Delphi I and Delphi II). In the first cycle, 10 judges participated, evaluating 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 Cohen's 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 evaluation per item and also overall. The final version of the set of guidelines consisted of 14 items. The set of guidelines was considered valid by the expert judges and stands out for its innovative nature at the national level. It is hoped that, when applied in practice, this material will promote positive changes in the health of children and adolescents with mucopolysaccharidosis and in the work of nurses. Furthermore, this study promoted a discussion about comprehensive care, highlighting the leading role of nursespt-BRAcesso AbertoTerapia de reposição de enzimasMucopolissacaridosesCriançaAdolescenteEnfermagem domiciliardoctoralThesisCIENCIAS DA SAUDE::ENFERMAGEM