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

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dc.contributor.authorDe Vries, Maaike C.en
dc.contributor.authorBrown, David A.en
dc.contributor.authorAllen, Mitchell E.en
dc.contributor.authorBindoff, Laurenceen
dc.contributor.authorGorman, Grainne S.en
dc.contributor.authorKaraa, Amelen
dc.contributor.authorKeshavan, Nandakien
dc.contributor.authorLamperti, Costanzaen
dc.contributor.authorMcFarland, Roberten
dc.contributor.authorNg, Yi Shiauen
dc.contributor.authorO'Callaghan, Maren
dc.contributor.authorPitceathly, Robert D. S.en
dc.contributor.authorRahman, Shamimaen
dc.contributor.authorRussel, Frans G. M.en
dc.contributor.authorVarhaug, Kristin N.en
dc.contributor.authorSchirris, Tom J. J.en
dc.contributor.authorMancuso, Michelangeloen
dc.date.accessioned2020-12-21T15:40:13Zen
dc.date.available2020-12-21T15:40:13Zen
dc.date.issued2020-07en
dc.description.abstractClinical 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.en
dc.description.notesEuropean Molecular Biology Organization, Grant/Award Number: ALTF 268-2016; Fondazione Telethon, Grant/Award Numbers: GUP09004, GSP16001; Instituto de salud carlos, Grant/Award Number: ISCIII-FIS PI17/00109; Medical Research Council (GB), Grant/Award Number: MR/S002065/1; National Institute Food and Agriculture, Grant/Award Number: 1017927; UK Medical Research Council, Grant/Award Number: 13/NE/0326; Wellcome Trust, Grant/Award Numbers: 203105/Z/16/Z, 204709/Z/16/Z; European Molecular Biology Organisation; Prinses Beatrix Spierfonds, Grant/Award Number: WO.R17-19; National Institute for Health Research, Grant/Award Number: CL2016-01-003; Newcastle upon Tyne Hospitals NHS Foundation Trust; Biotechnology and Biological Sciences Research Council; Newcastle University; Wellcome; European Union; Instituto de Salud Carlos III; Virginia Tech; National Institute of Food and Agriculture; National Institutes of Health, Grant/Award Number: HL123647; Medical Research Council, Grant/Award Number: L016354; Action Medical Research; Biomedical Research Centre; Muscular Dystrophy UKen
dc.description.sponsorshipEuropean Molecular Biology OrganizationEuropean Molecular Biology Organization (EMBO) [ALTF 268-2016]; Fondazione TelethonFondazione Telethon [GUP09004, GSP16001]; Instituto de salud carlosInstituto de Salud Carlos III [ISCIII-FIS PI17/00109]; Medical Research Council (GB) [MR/S002065/1]; National Institute Food and AgricultureUnited States Department of Agriculture (USDA)National Institute of Food and Agriculture [1017927]; UK Medical Research CouncilMedical Research Council UK (MRC) [13/NE/0326]; Wellcome TrustWellcome Trust [203105/Z/16/Z, 204709/Z/16/Z]; European Molecular Biology Organisation; Prinses Beatrix Spierfonds [WO.R17-19]; National Institute for Health ResearchNational Institute for Health Research (NIHR) [CL2016-01-003]; Newcastle upon Tyne Hospitals NHS Foundation Trust; Biotechnology and Biological Sciences Research CouncilBiotechnology and Biological Sciences Research Council (BBSRC); Newcastle University; Wellcome; European UnionEuropean Union (EU); Instituto de Salud Carlos IIIInstituto de Salud Carlos III; Virginia Tech; National Institute of Food and AgricultureUnited States Department of Agriculture (USDA)National Institute of Food and Agriculture; National Institutes of HealthUnited States Department of Health & Human ServicesNational Institutes of Health (NIH) - USA [HL123647]; Medical Research CouncilMedical Research Council UK (MRC) [L016354]; Action Medical Research; Biomedical Research Centre; Muscular Dystrophy UKen
dc.format.mimetypeapplication/pdfen
dc.identifier.doihttps://doi.org/10.1002/jimd.12196en
dc.identifier.eissn1573-2665en
dc.identifier.issn0141-8955en
dc.identifier.issue4en
dc.identifier.pmid32030781en
dc.identifier.urihttp://hdl.handle.net/10919/101558en
dc.identifier.volume43en
dc.language.isoenen
dc.rightsCreative Commons Attribution 4.0 Internationalen
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/en
dc.subjectdrugsen
dc.subjectin vitro studiesen
dc.subjectin vivo studiesen
dc.subjectmitochondrial diseasesen
dc.subjectmitochondrial toxicityen
dc.subjectsafetyen
dc.titleSafety of drug use in patients with a primary mitochondrial disease: An international Delphi-based consensusen
dc.title.serialJournal of Inherited Metabolic Diseaseen
dc.typeArticle - Refereeden
dc.type.dcmitypeTexten
dc.type.dcmitypeStillImageen

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