Safety of drug use in patients with a primary mitochondrial disease: An international Delphi-based consensus.

Autor: De Vries MC; Radboudumc Amalia Children's Hospital, Radboud Center for Mitochondrial Medicine, Nijmegen, The Netherlands., Brown DA; Department of Human Nutrition, Foods, and Exercise and the Virginia Tech Center for Drug Discovery, Virginia Tech, Blacksburg, Virginia., Allen ME; Department of Human Nutrition, Foods, and Exercise and the Virginia Tech Center for Drug Discovery, Virginia Tech, Blacksburg, Virginia., Bindoff L; Department of Clinical Medicine, University of Bergen, Bergen, Norway.; Department of Neurology, Haukeland University Hospital, Bergen, Norway., Gorman GS; Wellcome Centre for Mitochondrial Research, Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, UK.; The Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Karaa A; Genetics Unit, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts., Keshavan N; Mitochondrial Research Group, UCL Great Ormond Street Institute of Child Health, London, UK.; Metabolic Unit, Great Ormond Street Hospital NHS Foundation Trust, London, UK., Lamperti C; Unit of Medical Genetics and Neurogenetics, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., McFarland R; Wellcome Centre for Mitochondrial Research, Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, UK.; The Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., Ng YS; Wellcome Centre for Mitochondrial Research, Institute of Neuroscience, Newcastle University, Newcastle upon Tyne, UK.; The Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK., O'Callaghan M; Department of Neurology, Metabolic Unit, Hospital Sant Joan de Déu, Barcelona, Spain.; CIBERER, Instituto de Salud Carlos III, Barcelona, Spain., Pitceathly RDS; Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology and The National Hospital for Neurology and Neurosurgery, London, UK., Rahman S; Mitochondrial Research Group, UCL Great Ormond Street Institute of Child Health, London, UK.; Metabolic Unit, Great Ormond Street Hospital NHS Foundation Trust, London, UK., Russel FGM; Department of Pharmacology and Toxicology, Radboud Institute for Molecular Life Sciences, Radboud Center for Mitochondrial Medicine, Radboudumc, Nijmegen, The Netherlands., Varhaug KN; Department of Clinical Medicine, University of Bergen, Bergen, Norway.; Department of Neurology, Haukeland University Hospital, Bergen, Norway., Schirris TJJ; Department of Pharmacology and Toxicology, Radboud Institute for Molecular Life Sciences, Radboud Center for Mitochondrial Medicine, Radboudumc, Nijmegen, The Netherlands., Mancuso M; Department of Clinical and Experimental Medicine, Neurological Institute, University of Pisa, Pisa, Italy.
Jazyk: angličtina
Zdroj: Journal of inherited metabolic disease [J Inherit Metab Dis] 2020 Jul; Vol. 43 (4), pp. 800-818. Date of Electronic Publication: 2020 Feb 07.
DOI: 10.1002/jimd.12196
Abstrakt: Clinical guidance is often sought when prescribing drugs for patients with primary mitochondrial disease. Theoretical considerations concerning drug safety in patients with mitochondrial disease may lead to unnecessary withholding of a drug in a situation of clinical need. The aim of this study was to develop consensus on safe medication use in patients with a primary mitochondrial disease. A panel of 16 experts in mitochondrial medicine, pharmacology, and basic science from six different countries was established. A modified Delphi technique was used to allow the panellists to consider draft recommendations anonymously in two Delphi rounds with predetermined levels of agreement. This process was supported by a review of the available literature and a consensus conference that included the panellists and representatives of patient advocacy groups. A high level of consensus was reached regarding the safety of all 46 reviewed drugs, with the knowledge that the risk of adverse events is influenced both by individual patient risk factors and choice of drug or drug class. This paper details the consensus guidelines of an expert panel and provides an important update of previously established guidelines in safe medication use in patients with primary mitochondrial disease. Specific drugs, drug groups, and clinical or genetic conditions are described separately as they require special attention. It is important to emphasise that consensus-based information is useful to provide guidance, but that decisions related to drug prescribing should always be tailored to the specific needs and risks of each individual patient. We aim to present what is current knowledge and plan to update this regularly both to include new drugs and to review those currently included.
(© 2020 The Authors. Journal of Inherited Metabolic Disease published by John Wiley & Sons Ltd on behalf of SSIEM.)
Databáze: MEDLINE
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