Guidance for Home Enzyme Replacement Therapy in Children and Adolescents Diagnosed with Mucopolysaccharidoses: A Scoping Review Protocol

Vinícius Rodrigues de Oliveira (), Yarytizza Nycoly Fernandes Martins, Juliana Iscarlaty Freire de Araújo, Eunice Fernandes da Silva, Margarita Baeza Fuentes and Jonas Sâmi Albuquerque de Oliveira
Additional contact information
Vinícius Rodrigues de Oliveira: Departament of Nursing, Federal University of Rio Grande do Norte, Natal 59078-970, Brazil
Yarytizza Nycoly Fernandes Martins: Departament of Nursing, Federal University of Rio Grande do Norte, Natal 59078-970, Brazil
Juliana Iscarlaty Freire de Araújo: Departament of Public Health, Federal University of Rio Grande do Norte, Natal 59078-970, Brazil
Eunice Fernandes da Silva: Pediatric Outpatient Clinic, Onofre Lopes University Hospital, Natal 59010-054, Brazil
Margarita Baeza Fuentes: Departament of Nursing, University of Santiago de Chile, Santiago 9170022, Chile
Jonas Sâmi Albuquerque de Oliveira: Departament of Nursing, Federal University of Rio Grande do Norte, Natal 59078-970, Brazil

IJERPH, 2025, vol. 22, issue 11, 1-10

Abstract: Enzyme replacement therapy is one of the main alternatives in the treatment of mucopolysaccharidosis. Conventionally, this therapy is administered in an outpatient setting, but this can be carried out at home, where the patient feels most comfortable. However, there is still a need for guidance on how to implement the use of this therapy in the home setting. This study aims to present the protocol of a scoping review that aims to identify and map the main existing guidance for the implementation of home-based enzyme replacement therapy in children and adolescents with mucopolysaccharidosis. The review protocol was developed based on the methodological guidance proposed by Peters and the PRISMA-P checklist and was registered on the Open Science Framework platform, available at OSF.IO/QUSB6. The searches will be carried out in the following databases: CINAHL, Cochrane Library, Embase, LILACS, MEDLINE, Science Direct, Scopus, and Web of Science. Gray literature will be searched in the CAPES/Brazilian Theses and Dissertations Catalog, the Brazilian Ministry of Health website, Google Scholar, the National Institute for Health and Care Excellence, and the WHO IRIS. Articles will be selected according to predefined eligibility criteria and extracted using a standardized collection form. The results of this review are expected to provide valuable insights for researchers regarding the main guidance for the implementation of home-based ERT in children and adolescents with mucopolysaccharidosis.

Keywords: mucopolysaccharidosis; enzyme replacement therapy; therapy; home infusion; child; adolescent (search for similar items in EconPapers)
JEL-codes: I I1 I3 Q Q5 (search for similar items in EconPapers)
Date: 2025
References: Add references at CitEc
Citations:

Downloads: (external link)
https://www.mdpi.com/1660-4601/22/11/1686/pdf (application/pdf)
https://www.mdpi.com/1660-4601/22/11/1686/ (text/html)

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:gam:jijerp:v:22:y:2025:i:11:p:1686-:d:1789392

Access Statistics for this article

IJERPH is currently edited by Ms. Jenna Liu

More articles in IJERPH from MDPI
Bibliographic data for series maintained by MDPI Indexing Manager ().