{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,9]],"date-time":"2025-11-09T05:16:22Z","timestamp":1762665382153,"version":"build-2065373602"},"reference-count":15,"publisher":"MDPI AG","issue":"11","license":[{"start":{"date-parts":[[2025,11,7]],"date-time":"2025-11-07T00:00:00Z","timestamp":1762473600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100008532","name":"Federal University of Rio Grande do Norte","doi-asserted-by":"crossref","award":["23077.110294\/2025-55"],"award-info":[{"award-number":["23077.110294\/2025-55"]}],"id":[{"id":"10.13039\/501100008532","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJERPH"],"abstract":"<jats:p>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.<\/jats:p>","DOI":"10.3390\/ijerph22111686","type":"journal-article","created":{"date-parts":[[2025,11,7]],"date-time":"2025-11-07T09:14:45Z","timestamp":1762506885000},"page":"1686","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Guidance for Home Enzyme Replacement Therapy in Children and Adolescents Diagnosed with Mucopolysaccharidoses: A Scoping Review Protocol"],"prefix":"10.3390","volume":"22","author":[{"given":"Vin\u00edcius Rodrigues de","family":"Oliveira","sequence":"first","affiliation":[{"name":"Departament of Nursing, Federal University of Rio Grande do Norte, Natal 59078-970, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yarytizza Nycoly Fernandes","family":"Martins","sequence":"additional","affiliation":[{"name":"Departament of Nursing, Federal University of Rio Grande do Norte, Natal 59078-970, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Juliana Iscarlaty Freire de","family":"Ara\u00fajo","sequence":"additional","affiliation":[{"name":"Departament of Public Health, Federal University of Rio Grande do Norte, Natal 59078-970, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0837-095X","authenticated-orcid":false,"given":"Eunice Fernandes da","family":"Silva","sequence":"additional","affiliation":[{"name":"Pediatric Outpatient Clinic, Onofre Lopes University Hospital, Natal 59010-054, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Margarita Baeza","family":"Fuentes","sequence":"additional","affiliation":[{"name":"Departament of Nursing, University of Santiago de Chile, Santiago 9170022, Chile"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jonas S\u00e2mi Albuquerque de","family":"Oliveira","sequence":"additional","affiliation":[{"name":"Departament of Nursing, Federal University of Rio Grande do Norte, Natal 59078-970, Brazil"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2025,11,7]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"2249","DOI":"10.4103\/ijo.IJO_425_22","article-title":"Mucopolysaccharidosis: A broad review","volume":"70","author":"Nagpal","year":"2022","journal-title":"Indian J. Ophthalmol."},{"key":"ref_2","doi-asserted-by":"crossref","unstructured":"Celik, B., Tomatsu, S.C., Tomatsu, S., and Khan, S.A. (2021). Epidemiology of Mucopolysaccharidoses Update. Diagnostics, 11.","DOI":"10.3390\/diagnostics11020273"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1016\/j.revmed.2019.11.010","article-title":"Mucopolysaccharidoses: Quand y penser ? [Mucopolysaccharidosis: A review]","volume":"41","author":"Michaud","year":"2020","journal-title":"Rev. Med. Interne"},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"e07830","DOI":"10.1016\/j.heliyon.2021.e07830","article-title":"Mucopolysaccharidoses diagnosis in the era of enzyme replacement therapy in Egypt","volume":"7","author":"Fateen","year":"2021","journal-title":"Heliyon"},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1186\/s13052-018-0562-1","article-title":"Enzyme replacement therapy: Efficacy and limitations","volume":"44","author":"Concolino","year":"2018","journal-title":"Ital. J. Pediatr."},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"338","DOI":"10.1186\/s13023-023-02919-8","article-title":"Safety outcomes and patients\u2019 preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond","volume":"18","author":"Toscano","year":"2023","journal-title":"Orphanet J. Rare Dis."},{"key":"ref_7","first-page":"100971","article-title":"The patients\u2019 perspective on homebased infusion: A longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy","volume":"35","author":"Heinrich","year":"2023","journal-title":"Mol. Genet. Metab."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"953","DOI":"10.11124\/JBIES-21-00242","article-title":"Best practice guidance and reporting items for the development of scoping review protocols","volume":"20","author":"Peters","year":"2022","journal-title":"JBI Evid. Synth."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/2046-4053-4-1","article-title":"Preferred reporting items for systematic review and meta-analysis protocols (PRISMA-P) 2015 statement","volume":"4","author":"Moher","year":"2015","journal-title":"Syst. Rev."},{"key":"ref_10","doi-asserted-by":"crossref","unstructured":"Aromataris, E., Lockwood, C., Porritt, K., Pilla, B., and Jordan, Z. (2024). Scoping Reviews. JBI Manual for Evidence Synthesis, JBI.","DOI":"10.46658\/JBIMES-24-01"},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"467","DOI":"10.7326\/M18-0850","article-title":"PRISMA Extension for Scoping Reviews (PRISMA-ScR): Checklist and Explanation","volume":"169","author":"Tricco","year":"2018","journal-title":"Ann. Intern. Med."},{"key":"ref_12","first-page":"457","article-title":"Scoping reviews: The PAGER framework for improving the quality of reporting","volume":"25","author":"Aveyard","year":"2021","journal-title":"Int. J. Soc. Res. Methodol."},{"key":"ref_13","unstructured":"Esposito, A.C. (2016). A Descentraliza\u00e7\u00e3o do Tratamento Medicamentoso (Terapia de Reposi\u00e7\u00e3o Enzim\u00e1tica) nas Mucopolissacaridoses. [Master\u2019s Thesis, Funda\u00e7\u00e3o Oswaldo Cruz]."},{"key":"ref_14","doi-asserted-by":"crossref","unstructured":"Kie\u0107-Wilk, B., Guijt, P., Dan, M., Abdelwahab, M., Revel-Vilk, S., and Serratrice, C. (2025). Home Enzyme Replacement Therapy in Gaucher Disease: A Review. J. Clin. Med., 14.","DOI":"10.3390\/jcm14030842"},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"g7647","DOI":"10.1136\/bmj.g7647","article-title":"Preferred reporting items for systematic review and meta-analysis protocols (PRISMA-P) 2015: Elaboration and explanation","volume":"349","author":"Shamseer","year":"2015","journal-title":"BMJ"}],"container-title":["International Journal of Environmental Research and Public Health"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1660-4601\/22\/11\/1686\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,9]],"date-time":"2025-11-09T05:12:53Z","timestamp":1762665173000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1660-4601\/22\/11\/1686"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,11,7]]},"references-count":15,"journal-issue":{"issue":"11","published-online":{"date-parts":[[2025,11]]}},"alternative-id":["ijerph22111686"],"URL":"https:\/\/doi.org\/10.3390\/ijerph22111686","relation":{},"ISSN":["1660-4601"],"issn-type":[{"type":"electronic","value":"1660-4601"}],"subject":[],"published":{"date-parts":[[2025,11,7]]}}}