Guidelines for home-based enzyme replacement therapy in children and adolescents with mucopolysaccharidosis: a scoping review.

OBJECTIVE

To identify and map the main guidelines for carrying out home-based enzyme replacement therapy (ERT) administered to children and adolescents diagnosed with mucopolysaccharidosis.

METHOD: A scoping review, conducted according to JBI Manual for Evidence Synthesis (Edition 2024). Twelve data sources were consulted using specific search strategies. Data were extracted using a specific form and analyzed using simple descriptive statistics and a qualitative PAGER approach.

RESULTS

The final sample consisted of 13 articles published between 2007 and 2020, mostly conducted in European countries, with a predominance of authors affiliated with the Royal Manchester Children's Hospital. Five patterns were identified for consolidating qualified and safe care in the context of homebased ERT administered to children and adolescents with types I, II, IV-A, and VII mucopolysaccharidoses. The nurse emerges as an essential professional in all stages and activities presented.

CONCLUSION

Despite the progress made regarding guidelines for the decentralization of ERT, challenges to its implementation still persist, among which the need for dissemination of technical and scientific knowledge stands out.