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Infusion rate adjustment in enzyme replacement therapy with pabinafusp alfa for mucopolysaccharidosis II
Kimitoshi Nakamura1, Norio Sakai2, Hideaki Hirai3
1Graduate School of Medical Science, Kumamoto University, Kumamoto, Japan.
Aims:
Enzyme 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.
Methods:
A 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.
Results:
Shorter 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.
Conclusions:
Infusion 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.
Insights
Shortening enzyme replacement therapy (ERT) infusions for mucopolysaccharidosis II (MPS II) did not impact safety or efficacy. This approach may improve quality of life and treatment compliance for MPS II patients.
Area of Science:
- Biochemistry
- Pharmacology
- Genetics
Background:
- Enzyme replacement therapy (ERT) for mucopolysaccharidosis II (MPS II) involves lengthy weekly infusions, impacting patient quality of life and compliance.
- Optimizing ERT administration is crucial for long-term pediatric patient management.
Purpose of the Study:
- To evaluate the impact of shortened infusion durations on the long-term safety and efficacy of pabinafusp alfa in MPS II patients.
- To determine if reducing infusion time affects treatment outcomes and patient experience.
Main Methods:
- A post hoc analysis of 260 weeks of clinical data from 27 Japanese MPS II patients receiving pabinafusp alfa (2.0 mg/kg/week).
- Infusion durations were individually adjusted during an extension study.
- Safety assessed via adverse events and infusion reactions; efficacy by glycosaminoglycan levels (CSF, serum, urine), organ volumes, and neurocognitive scores.
Main Results:
- Shortened infusion times showed no increase in adverse events or infusion-associated reactions.
- Therapeutic efficacy was sustained, with no significant changes in heparan and dermatan sulfate levels in CSF, serum, or urine.
- Liver and spleen volumes remained unaffected by altered infusion rates.
Conclusions:
- Adjusting infusion rates for pabinafusp alfa in MPS II patients did not compromise safety or efficacy.
- Decreasing infusion times can be safely considered to enhance patient quality of life and treatment adherence.
- This strategy offers a practical approach to improve the management of MPS II patients undergoing ERT.
