Journal of inherited metabolic disease · 2018
Outcome measures for children with mitochondrial disease: consensus recommendations for future studies from a Delphi-based international workshop.
Consensus StatementСтатьяПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Koene Saskia, van Bon Lara, Bertini Enrico, Jimenez-Moreno Cecilia, van der Giessen Lianne, de Groot Imelda, McFarland Robert, Parikh Sumit, Rahman Shamima, Wood Michelle, Zeman Jiri, Janssen Anjo, Smeitink Jan
Аннотация
Although there are no effective disease-modifying therapies for mitochondrial diseases, an increasing number of trials are being conducted in this rare disease group. The use of sensitive and valid endpoints is essential to test the effectiveness of potential treatments. There is no consensus on which outcome measures to use in children with mitochondrial disease. The aims of this two-day Delphi-based workshop were to (i) define the protocol for an international, multi-centre natural history study in children with mitochondrial myopathy and (ii) to select appropriate outcome measures for a validation study in children with mitochondrial encephalopathy. We suggest two sets of outcome measures for a natural history study in children with mitochondrial myopathy and for a proposed validation study in children with mitochondrial encephalopathy.