دورية أكاديمية
Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease
| العنوان: | Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease |
|---|---|
| المؤلفون: | Hughes, D, Gonzalez, D, Maegawa, G, Bernat, JA, Holida, M, Giraldo, P, Atta, MG, Chertkoff, R, Alon, S, Almon, EB, Rocco, R, Goker-Alpan, O |
| المصدر: | Genetics in Medicine , 25 (12) , Article 100968. (2023) (In press). |
| بيانات النشر: | Elsevier BV |
| سنة النشر: | 2023 |
| المجموعة: | University College London: UCL Discovery |
| مصطلحات موضوعية: | Fabry disease, anti-drug antibodies, enzyme replacement therapy, estimated glomerular filtration rate, globotriaosylsphingosine, immunogenicity, long-term, pegunigalsidase alfa, safety |
| الوصف: | Purpose: Fabry disease (FD) is a rare lysosomal storage disorder caused by pathogenic variants in the GLA gene encoding α-galactosidase (α-Gal)-A. We evaluated long-term safety/efficacy of pegunigalsidase alfa, a novel PEGylated α-Gal-A enzyme replacement therapy (ERT) now approved for FD. Methods: In a phase-1/2 dose-ranging study, 15 ERT-naive adults with FD completed 12 months of pegunigalsidase alfa and enrolled in this 60-month open-label extension of 1 mg/kg pegunigalsidase alfa infusions every 2 weeks. Results: Fifteen patients enrolled (8 males; 7 females); 10 completed ≥48 months (60 months total treatment), and 2 completed 60 months (72 months total treatment). During treatment, most treatment-emergent adverse events were mild/moderate in severity and all infusion-related reactions were mild/moderate in severity. Four patients were transiently positive for anti-pegunigalsidase alfa IgG. Patients showed continuous reduction in plasma lyso-Gb3 concentrations with mean (standard error) reduction of 76.1 [25.1] ng/mL from baseline to month 24. At 60 months, the estimated glomerular filtration rate slope was comparable to that observed in patients treated with other ERTs. Cardiac function assessments revealed stability; no cardiac fibrosis was observed. Conclusion: In this first long-term assessment of pegunigalsidase alfa administration in patients with FD, we found favorable safety/efficacy. Our data suggest long-term continuous benefits of pegunigalsidase alfa treatment in adults with FD. |
| نوع الوثيقة: | article in journal/newspaper |
| وصف الملف: | application/pdf |
| اللغة: | English |
| العلاقة: | https://discovery.ucl.ac.uk/id/eprint/10181024/1/1-s2.0-S109836002300984X-main.pdfTest; https://discovery.ucl.ac.uk/id/eprint/10181024Test/ |
| الإتاحة: | https://discovery.ucl.ac.uk/id/eprint/10181024/1/1-s2.0-S109836002300984X-main.pdfTest https://discovery.ucl.ac.uk/id/eprint/10181024Test/ |
| حقوق: | open |
| رقم الانضمام: | edsbas.2816EE31 |
| قاعدة البيانات: | BASE |
| الوصف غير متاح. |