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Published on: December 20, 2017
Home-based infusion therapy for patients with Fabry disease
1Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK.
Insights
Home infusion therapy for Fabry disease using agalsidase beta offers a safe and practical alternative to hospital treatments. This patient-centered approach improves quality of life and optimizes hospital resources.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Fabry disease is a progressive, life-threatening inherited disorder requiring lifelong enzyme replacement therapy.
- Hospital-based agalsidase therapy is often perceived as inconvenient by patients and healthcare providers.
- Home-based infusion therapy presents a patient-preferred alternative for managing Fabry disease.
Purpose of the Study:
- To review clinical experiences with agalsidase beta home infusion therapy.
- To outline the organization of safe, patient-centered homecare for Fabry disease.
- To assess the feasibility and impact of home infusion therapy.
Main Methods:
- Review of collective clinical experiences with agalsidase beta home infusion.
- Identification of prerequisites for safe home therapy (stable patient, suitable environment).
- Discussion of nurse-assisted and self-care infusion models.
Main Results:
- Home infusion therapy is safe and practical, significantly improving patient quality of life.
- Home therapy reduces constraints on hospital resources.
- Eligible patients can benefit from home infusion therapy without delay.
Conclusions:
- Home-based agalsidase beta infusion therapy is a viable and beneficial treatment for Fabry disease.
- Patient-centered homecare models enhance treatment convenience and adherence.
- The principles of home infusion therapy can be applied to other enzyme replacement therapies.
Abstract:
Fabry disease is an inherited, progressive, life-threatening disease; therefore, lifelong therapy is needed. By replacing the deficient enzyme, disease progression may be delayed or halted, thereby avoiding serious complications. Hospital-based agalsidase therapy is generally perceived as inconvenient and home-based infusion therapy is greatly appreciated by patients, their families and healthcare professionals. Patients can get familiar with infusion therapy in a hospital setting and, if specific requirements are fulfilled, routine nurse-assisted infusion, or self-care, at the patient's home can be organized. A stable patient who tolerates the infusion and a suitable home environment are prerequisites for home therapy. The authors' clinical experiences underscore the safety and practicality of home therapy. In addition to a major positive impact on the patient's quality of life, home infusion therapy may reduce the constraints of hospital resources. This article reviews the collective experiences with agalsidase beta home infusion therapy and outlines how safe, patient-centred homecare can be organized. Home infusion therapy with Fabrazyme should not be withheld from patients considered eligible according to the proposed criteria. Similar approaches to other enzyme therapies are also possible.
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