Evidence map›Paper›PMID 42477279›Full record

ArticleBioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy2026

Home Infusion With Recombinant Human α-Glucosidase in Children With Pompe Disease: The Dutch Experience Over 20 Years Across the Spectrum From Classic Infantile to Late-Onset Phenotypes.

Imke A M Ditters, Hidde H Huidekoper, Jacqueline F Hardon, Michelle E Kruijshaar, Ans T van der Ploeg, Johanna M P van den Hout

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Article in BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

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2 · The registry

The trial behind it

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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

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5 · Who and what money

Authors and funding

6 authors.

Imke A M DittersDepartment of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, PO Box 2060, 3000 CB, Rotterdam, The Netherlands.ORCID http://orcid.org/0000-0002-2377-3108
Hidde H HuidekoperDepartment of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, PO Box 2060, 3000 CB, Rotterdam, The Netherlands.ORCID http://orcid.org/0000-0003-1276-0931
Jacqueline F HardonDepartment of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, PO Box 2060, 3000 CB, Rotterdam, The Netherlands.
Michelle E KruijshaarDepartment of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, PO Box 2060, 3000 CB, Rotterdam, The Netherlands.ORCID http://orcid.org/0009-0009-8700-0591
Ans T van der PloegDepartment of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, PO Box 2060, 3000 CB, Rotterdam, The Netherlands.ORCID http://orcid.org/0000-0002-3359-1324
Johanna M P van den HoutDepartment of Pediatrics, Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, PO Box 2060, 3000 CB, Rotterdam, The Netherlands. j.vandenhout@erasmusmc.nl.ORCID http://orcid.org/0000-0001-8091-263X

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionEnzyme replacement therapy (ERT) is the standard treatment for patients with Pompe disease, a hereditary metabolic myopathy. While ERT in the home situation is increasingly common in adults with Pompe disease, experience in children remains limited due to higher dosing requirements and increased risk of infusion-associated reactions (IARs). We analysed the results of the in-hospital and home-based infusion programme applied in the Netherlands to children since 1999 to provide guidance.

methodsWe studied hospital and home-based infusions administered to children with Pompe disease (i.e., classic infantile, atypical infantile, and childhood onset phenotypes) who started ERT between 1999 and 2022 and analysed the characteristics of patients and IARs. The IARs were graded by healthcare providers.

resultsA total of 11,898 infusions with recombinant human

conclusionRecombinant human α-glucosidase can be safely administered at home using our protocol in children with Pompe disease, including those with classic infantile Pompe disease who are more likely to develop IAR, provided that in-hospital treatment has been shown to be safe, and the appropriate infrastructure and clinical support are in place.

Indexed as

alpha-GlucosidasesEnzyme Replacement TherapyGlycogen Storage Disease Type IIHome Infusion TherapyAdolescentAge of OnsetChildChild, PreschoolFemaleHumansInfantInfusions, IntravenousMaleNetherlandsPhenotypeRecombinant Proteinsalpha-GlucosidasesGAA protein, humanRecombinant Proteins

Identifiers

PMID42477279
PMCPMC13570904

What Socratic holds

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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 Socratic graph.