Increased intervals in enzyme replacement therapy for stable type 1 Gaucher disease: A non-inferiority sequential trial emulation.

Key Points

• The study aims to determine if longer intervals for enzyme replacement therapy are effective for patients with stable type 1 Gaucher disease.
• Conducted a non-inferiority sequential trial emulation
• Compared ERT administration every 3-4 weeks to the standard biweekly regimen
• Evaluated patient quality of life and healthcare costs
• Extending ERT intervals to every 3-4 weeks was found to be non-inferior to the standard biweekly regimen
• Personalized ERT spacing may enhance quality of life
• Potential reduction in healthcare costs associated with less frequent treatments