Infusion rate adjustment in enzyme replacement therapy with pabinafusp alfa for mucopolysaccharidosis II.
Br J Clin PharmacolMay 17, 2026 (epub)
Kimitoshi Nakamura, Norio Sakai, Hideaki Hirai et al.
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In 30 seconds
This study evaluated the impact of shortening infusion durations on the safety and efficacy of enzyme replacement therapy (ERT) with pabinafusp alfa in 27 Japanese patients with mucopolysaccharidosis II (MPS II). A post hoc analysis of 260 weeks of clinical data revealed that shorter infusion times did not lead to increased adverse events or affect therapeutic efficacy, indicating that adjustments can be made safely.
Key findings
- Shorter infusion times did not increase infusion-associated reactions or adverse events.
- Heparan sulfate and dermatan sulfate levels remained stable despite changes in infusion rates.
- Liver and spleen volumes were unaffected by infusion duration adjustments.
Why it matters
Understanding the safety and efficacy of adjusting infusion rates in ERT can enhance patient comfort and adherence to treatment, ultimately improving quality of life for those with MPS II.