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

ABSTRACT: 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.

SUBMITTER: De Vries MC

PROVIDER: S-EPMC7383489 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature

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

De Vries Maaike C MC Brown David A DA Allen Mitchell E ME Bindoff Laurence L Gorman Gráinne S GS Karaa Amel A Keshavan Nandaki N Lamperti Costanza C McFarland Robert R Ng Yi Shiau YS O'Callaghan Mar M Pitceathly Robert D S RDS Rahman Shamima S Russel Frans G M FGM Varhaug Kristin N KN Schirris Tom J J TJJ Mancuso Michelangelo M

Journal of inherited metabolic disease 20200207 4

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 countr ...[more]

PMID: 32030781

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Project description:BackgroundThe lack of agreement regarding what constitutes successful treatment for periprosthetic joint infections (PJI) makes it difficult to compare the different strategies of management that are used in clinical practice and in research studies.Questions/purposesThe aims of this study were to create a consensus definition for success after PJI treatment, and to provide a universal, multidimensional framework for reporting of studies regarding PJI treatment.MethodsA two-round basic Delphi method was used to reach a consensus definition. We invited 159 international experts (orthopaedic surgeons, infectious disease specialists, and clinical researchers) from 17 countries to participate; 59 participated in the first round, and 42 participated in the second round. The final definition consisted of all statements that achieved strong agreement (80% or greater of participants considering a criterion relevant for defining success).ResultsThe consensus definition of a successfully treated PJI is: (1) infection eradication, characterized by a healed wound without fistula, drainage, or pain, and no infection recurrence caused by the same organism strain; (2) no subsequent surgical intervention for infection after reimplantation surgery; and (3) no occurrence of PJI-related mortality (by causes such as sepsis, necrotizing fasciitis). The Delphi panel agreed to defining midterm results as those reported 5 or more years after the definitive PJI surgery, and long-term results as those reported 10 or more years after surgery. Although no consensus was reached on the definition of short-term results, 71% of the participants agreed that 2 years after the definitive PJI surgery is acceptable to define it.ConclusionsThis multidimensional definition of success after PJI treatment may be used to report and compare results of treatment of this catastrophic complication.Level of evidenceLevel V, therapeutic study. See Guidelines for Authors for a complete description of levels of evidence.

Project description:The availability of over-the-counter (OTC) proton pump inhibitors (PPIs) for the short-term (2 weeks) management of frequent heartburn (?2 days/week) has increased markedly, yet evidence-based recommendations have not been developed. A panel of nine international experts in gastroesophageal reflux disease developed consensus statements regarding the risks and benefits of OTC PPIs using a modified Delphi process. Consensus (based on ?80% approval) was reached through multiple rounds of remote voting and a final round of live voting. To identify relevant data, the available literature was searched and summarized. Grading of Recommendations, Assessment, Development and Evaluation (GRADE) system terminology was used to rate the quality of evidence and strength of recommendations; consensus was based on ?2/3 agreement. After 4 rounds of review, consensus was achieved for 18 statements. Notably, the available data did not directly reflect OTC use, but instead, prescription use; therefore, extrapolations to the OTC setting were often necessary. This limitation is regrettable, but it justifies performing this exercise to provide evidence-based expert opinion on a widely used class of drugs. The panel determined that using OTC PPIs according to label instructions is unlikely to mask the symptoms of esophageal or gastric cancer or adversely impact the natural history of related precursor conditions. OTC PPIs are not expected to substantially affect micronutrient absorption or bone mineral density or cause community-acquired pneumonia, Clostridium difficile infection, or cardiovascular adverse events. However, OTC PPI use may be associated with slightly increased risks for infectious diarrhea, certain idiosyncratic reactions, and cirrhosis-related spontaneous bacterial peritonitis. The available evidence does not suggest that OTC PPI use consistent with label instructions is associated with substantial health risks. To minimize potential risks, healthcare professionals and consumers must actively participate in decision making when managing reflux-related symptoms in the self-care setting.

Project description:BACKGROUND: Problem use of illicit drugs (i.e. drug abuse or dependence) is associated with considerable health and social harms, highlighting the need for early intervention and engagement with health services. Family members, friends and colleagues play an important role in supporting and assisting individuals with problem drug use to seek professional help, however there are conflicting views about how and when such support should be offered. This paper reports on the development of mental health first aid guidelines for problem drug use in adults, to help inform community members on how to assist someone developing problem drug use or experiencing a drug-related crisis. METHODS: A systematic review of the scientific and lay literature was conducted to develop a 228-item survey containing potential first-aid strategies to help someone developing a drug problem or experiencing a drug-related crisis. Three panels of experts (29 consumers, 31 carers and 27 clinicians) were recruited from Australia, Canada, New Zealand, the United Kingdom, and the United States. Panel members independently rated the items over three rounds, with strategies reaching consensus on importance written into the guidelines. RESULTS: The overall response rate across three rounds was 80% (86% consumers, 81% carers, 74% clinicians). 140 first aid strategies were endorsed as essential or important by 80% or more of panel members. The endorsed strategies provide information and advice on what is problem drug use and its consequences, how to approach a person about their problem drug use, tips for effective communication, what to do if the person is unwilling to change their drug use, what to do if the person does (or does not) want professional help, what are drug-affected states and how to deal with them, how to deal with adverse reactions leading to a medical emergency, and what to do if the person is aggressive. CONCLUSIONS: The guidelines provide a consensus-based resource for community members who want to help someone with a drug problem. It is hoped that the guidelines will lead to better support and understanding for those with problem drug use and facilitate engagement with professional help.

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

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

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