Outcome measures for children with mitochondrial disease: consensus recommendations for future studies from a Delphi-based international workshop.
J Inherit Metab Dis
; 41(6): 1267-1273, 2018 11.
Article
em En
| MEDLINE
| ID: mdl-30027425
ABSTRACT
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.
Texto completo:
1
Eixos temáticos:
Pesquisa_clinica
Base de dados:
MEDLINE
Assunto principal:
Técnica Delphi
/
Miopatias Mitocondriais
/
Encefalomiopatias Mitocondriais
Tipo de estudo:
Clinical_trials
/
Guideline
Limite:
Child
/
Humans
Idioma:
En
Ano de publicação:
2018
Tipo de documento:
Article