Evidence map›Paper›PMID 42467880›Full record

ArticleRevista da Escola de Enfermagem da U S P2026

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

Vinicius Rodrigues de Oliveira, Bárbara Letícia de Queiroz Xavier, Carlos Vinícius de Souza Felipe, Isabele Gouveia Muniz de Alencar, Hallyson Leno Lucas da Silva, Francisca Marta de Lima Costa Souza, Jonas Sâmi Albuquerque de Oliveira

Abstract readScoping Review
In one paragraph

Article in Revista da Escola de Enfermagem da U S P, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

Vinicius Rodrigues de OliveiraUniversidade Federal do Rio Grande do Norte, Programa de Pós-Graduação em Enfermagem, Natal, RN, Brazil.ORCID http://orcid.org/0000-0002-9915-0062
Bárbara Letícia de Queiroz XavierUniversidade Federal do Rio Grande do Norte, Programa de Pós-Graduação em Saúde Coletiva, Natal, RN, Brazil.ORCID http://orcid.org/0000-0003-1622-9128
Carlos Vinícius de Souza FelipeUniversidade Federal do Rio Grande do Norte, Curso de Medicina, Natal, RN, Brazil.ORCID http://orcid.org/0009-0004-0821-7519
Isabele Gouveia Muniz de AlencarUniversidade Federal do Rio Grande do Norte, Programa de Pós-Graduação em Enfermagem, Natal, RN, Brazil.ORCID http://orcid.org/0000-0001-9626-3830
Hallyson Leno Lucas da SilvaUniversidade Federal do Rio Grande do Norte, Programa de Pós-Graduação em Enfermagem, Natal, RN, Brazil.ORCID http://orcid.org/0000-0001-7237-1706
Francisca Marta de Lima Costa SouzaUniversidade Federal do Rio Grande do Norte, Departamento de Enfermagem, Natal, RN, Brazil.ORCID http://orcid.org/0009-0000-0401-8310
Jonas Sâmi Albuquerque de OliveiraUniversidade Federal do Rio Grande do Norte, Programa de Pós-Graduação em Enfermagem, Natal, RN, Brazil.ORCID http://orcid.org/0000-0003-0303-409X

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

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

methodA 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.

resultsThe 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.

conclusionDespite 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.

Indexed as

Enzyme Replacement TherapyHome Care ServicesMucopolysaccharidosesPractice Guidelines as TopicAdolescentChildHumans

Identifiers

PMID42467880
PMCPMC13398687

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.