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Long-term safety and efficacy of agalsidase beta in Japanese patients with Fabry disease: aggregate data from two post-authorization safety studies
- Publication Year :
- 2021
- Publisher :
- Taylor & Francis, 2021.
-
Abstract
- Enzyme replacement therapy in Fabry disease has been available in Japan since 2004. Two post-authorization safety studies were conducted to evaluate agalsidase beta in Japanese patients with Fabry disease in real-world practice. The Special Drug Use Investigation monitored the long-term safety and efficacy of agalsidase beta, and the Drug Use Investigation monitored safety in patients not participating in the Special Drug Use Investigation. Safety and efficacy evaluations included adverse drug reactions (ADRs), infusion-associated reactions and hypersensitivity reactions, and change in blood GL-3 level over time. Of 396 patients in the aggregated data set, safety and efficacy analysis sets comprised 307 and 196 patients, respectively. ADRs occurred in 93 (30.3%) patients and serious ADRs occurred in 25 (8.1%) patients, with general disorders and administration site conditions (n=55, 17.9%), nervous system disorders (n=30, 9.8%) and skin and subcutaneous tissue disorders (n=23, 7.5%) the most common. Reductions in blood GL-3 levels occurred over the study, irrespective of age or disease phenotype. Agalsidase beta demonstrated acceptable safety and tolerability, with sustained reductions in blood GL-3 levelsin Japanese patients with Fabry disease in real-world clinical practice NCT00233870/AGAL03004 (Special Drug Use Investigation of Agalsidase beta)
- Subjects :
- Adult
Male
Pediatrics
medicine.medical_specialty
Adolescent
030204 cardiovascular system & hematology
Young Adult
03 medical and health sciences
0302 clinical medicine
Japan
Product Surveillance, Postmarketing
Humans
Medicine
Enzyme Replacement Therapy
Pharmacology (medical)
Aged
Safety studies
business.industry
Trihexosylceramides
Authorization
General Medicine
Enzyme replacement therapy
Middle Aged
medicine.disease
Fabry disease
AGALSIDASE BETA
Isoenzymes
Treatment Outcome
alpha-Galactosidase
030220 oncology & carcinogenesis
Fabry Disease
Female
Aggregate data
Long term safety
business
Subjects
Details
- ISSN :
- 00233870
- Database :
- OpenAIRE
- Accession number :
- edsair.doi.dedup.....ba7654fe57d139a950627f52e78ddd2f
- Full Text :
- https://doi.org/10.6084/m9.figshare.14191347