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 and 4 others
PMID 42467880WHAT IT FOUND
Home enzyme replacement therapy for children and adolescents with mucopolysaccharidosis can be nurse-led, but guidelines are sparse and safety details are missing.
Key findings
01The mapped guidelines apply only to MPS types I, II, IV-A, and VI.
02Nurses are responsible for home visits, family guidance, preparing and administering ERT, monitoring infusions, and managing adverse reactions.
03The mapped criteria for starting home ERT include preferably age five or older, at least six months of outpatient or hospital ERT without adverse infusion reactions, suitable venous access, a favorable home, and no significant respiratory disease.
STILL TO COME
How it was doneWhat they foundWhat it means for RNs
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What it does not show
The review included only 13 articles, and several were experience reports or expert consensus rather than controlled trials. It searched only children and adolescents with MPS, so it may miss guidance for other ages. Some articles were not freely available, which may have excluded relevant evidence. No publications from 2021 to 2024 were found, so the mapped guidance may be outdated.
Declared interests
The paper reports financial support from Universidade Federal do Rio Grande do Norte. It does not report commercial funding or author conflicts of interest.
The easy way to misread this
Do not read this as proof that home ERT works for all children and adolescents with MPS. It is a scoping review of 13 articles, including experience reports and expert consensus, and it maps nurse-led protocols for selected patients after prior hospital treatment.