Trial reportBritish journal of clinical pharmacology2026
Infusion rate adjustment in enzyme replacement therapy with pabinafusp alfa for mucopolysaccharidosis II.
Trial report in British journal of clinical pharmacology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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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.
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.
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Who cites it
3 citing papers in PubMed.
- Infusion rate adjustment in enzyme replacement therapy with pabinafusp alfa for mucopolysaccharidosis II.British journal of clinical pharmacology · 2026Trial
- Antibody-Based Biologics for CNS Disorders.Antibodies (Basel, Switzerland) · 2026Review
- Biologic Therapies for Alleviating Neurodegeneration in Lysosomal Storage Diseases.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2026Review
Corrections and comments
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Authors and funding
7 authors.
Funding
Abstract
aimsEnzyme replacement therapy (ERT) for mucopolysaccharidosis II (MPS II) requires long-term, weekly intravenous infusions often lasting over 3 h each time, which can burden paediatric patients and caregivers and negatively affect their quality of life and treatment compliance. The aim of this study was to assess whether shortening infusion duration impacts the long-term efficacy and safety of ERT.
methodsA post hoc analysis was conducted using 260 weeks of clinical data from 27 Japanese patients with MPS II who received pabinafusp alfa at a dose of 2.0 mg/kg/week during a Phase II/III clinical trial and an extension study. Individual adjustments to the weekly infusion duration were allowed only during the extension study. Safety was assessed through the incidence of adverse events and infusion-associated reactions, while efficacy was evaluated by measuring heparan sulfate and dermatan sulfate levels in the cerebrospinal fluid (CSF), serum and urine, as well as assessing liver and spleen volumes and neurocognitive development scores.
resultsShorter infusion times were not associated with increased infusion-associated reactions or other adverse events. Heparan sulfate and dermatan sulfate levels in the CSF, serum and urine, as well as liver and spleen volumes were not affected by changes in infusion rates, indicating sustained therapeutic efficacy.
conclusionsInfusion rate adjustments were not associated with notable changes in the safety or efficacy of ERT with pabinafusp alfa in patients with MPS II. Clinicians may safely consider decreasing infusion times on a case-by-case basis to improve patients' quality of life and treatment compliance.
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