A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium. - 2023
A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium.
De Bleecker, Jan L; Claeys, Kristl G; DELSTANCHE, Stéphanieet al.
2023 • In Acta Neurologica Belgica, 123 (3), p. 1029 - 1037
A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium.pdf
[en] [en] INTRODUCTION: Hereditary transthyretin-mediated (hATTR) amyloidosis, a genetic disease caused by mutations in the transthyretin gene, leads to progressive sensory and autonomic neuropathy and/or cardiomyopathy and is associated with renal and ophthalmologic manifestations and a poor prognosis.
METHODS: This is a retrospective study based on data collected from the medical records of patients with hATTR amyloidosis treated with patisiran between 01 July 2018 and 01 February 2021. Six Belgian neuromuscular reference centers participated, covering all patisiran-treated hATTR amyloidosis patients at the study time. This study was conducted to collect data requested in the context of the reimbursement of patisiran in Belgium.
RESULTS: Thirty-one patients were diagnosed with hATTR amyloidosis with polyneuropathy, Coutinho stage 1 or 2, and eligible for active treatment during the data collection period. Of the hATTR amyloidosis patients treated with patisiran (n = 12), seven and five had polyneuropathy stages 1 and 2, respectively. Six patients had cardiac symptoms (New York Heart Association class 2 or above). Follow-up information was available for nine patients. Following patisiran treatment, eight patients showed stable or improved assessments for most neurological or cardiological parameters. Only one patient presented with worsening statuses at the end of the data collection period.
CONCLUSIONS: The patients with hATTR amyloidosis in Belgium have similar baseline demographics and disease characteristics to those studied in the patisiran APOLLO study and show a similar therapeutic response in the real-world, altering the expected disease progression in most patients.
Disciplines :
Neurology
Author, co-author :
De Bleecker, Jan L ; Department of Neurology, University Hospital Ghent, Ghent, Belgium
Claeys, Kristl G ; Department of Neurology, University Hospitals Leuven, Laboratory for Muscle Diseases and Neuropathies, KU Leuven, Leuven, Belgium
DELSTANCHE, Stéphanie ; Centre Hospitalier Universitaire de Liège - CHU > > Service de neurologie (CHR)
Van Parys, Vinciane; Department of Neurology, Cliniques Universitaires Saint-Luc, Brussels, Belgium
Baets, Jonathan ; Department of Neurology, Antwerp University Hospital, Faculty of Medicine and Health Sciences, Translational Neurosciences, UAntwerpen, Antwerp, Belgium
Remiche, Gauthier ; Department of Neurology, Hôpital Erasme, Université Libre de Bruxelles (ULB), Brussels, Belgium
Language :
English
Title :
A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium.
Publication date :
June 2023
Journal title :
Acta Neurologica Belgica
ISSN :
0300-9009
eISSN :
2240-2993
Publisher :
Springer Science and Business Media Deutschland GmbH, Italy
The authors would like to thank all study staff for their contribution. The authors also thank Xavier Ducarme, Karen Riks, and Erol Gabor from Modis Life Sciences for developing the study protocol and performing data collection and analysis, and Lies Schoonaert and Sebastian Vermeersch from Hict NV for editorial assistance. Jan L. De Bleecker, Kristl. G. Claeys, Stéphanie Delstanche, Vinciane Van Parys, Jonathan Baets, and Gauthier Remiche are member of the neuromuscular reference center in their hospital and of the European Reference Network for Rare Neuromuscular Diseases (ERN EURO-NMD). Alnylam Belgium BVBA (Schumanplein 6, 1040 Brussels, Belgium) covered all costs associated with the development and publication of the manuscript.
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