New treatments for mitochondrial disease-no time to drop our standards
| Autor: | Anu Suomalainen, Kari Majamaa, Patrick F. Chinnery, Michael G. Hanna, Douglas M. Turnbull, Valerio Carelli, Saskia Koene, Laurence A. Bindoff, Patrick Yu-Wai-Man, Robert McFarland, Rita Horvath, Michio Hirano, Jan A.M. Smeitink, Massimo Zeviani, Vamsi K. Mootha, Thomas Klopstock, Gerald Pfeffer |
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| Přispěvatelé: | G. Pfeffer, R. Horvath, T. Klopstock, V. K. Mootha, A. Suomalainen, S. Koene, M. Hirano, M. Zeviani, L. A. Bindoff, P. Y. Wai-Man, M. Hanna, V. Carelli, R. McFarland, K. Majamaa, D. M. Turnbull, J. Smeitink, P. F. Chinnery |
| Rok vydání: | 2013 |
| Předmět: |
medicine.medical_specialty
Pathology Mitochondrial Diseases Mitochondrial disease Alternative medicine MEDLINE Article Bias (Epidemiology) Humans Mitochondrial Disease 03 medical and health sciences Cellular and Molecular Neuroscience 0302 clinical medicine Bias medicine Humans Clinical significance Intensive care medicine 030304 developmental biology 0303 health sciences business.industry Reproducibility of Results Mitochondrial medicine Energy and redox metabolism [IGMD 8] Publication bias medicine.disease 3. Good health Multisystem disease Mitochondrial medicine [IGMD 8] epidemiology/therapy Reproducibility of Results Biomarker (medicine) Neurology (clinical) business 030217 neurology & neurosurgery |
| Zdroj: | Nature Reviews. Neurology, 9, 474-81 Nature Reviews Neurology; Vol 9 Nature Reviews. Neurology, 9, 8, pp. 474-81 |
| ISSN: | 1759-4758 |
| Popis: | Item does not contain fulltext Mitochondrial dysfunction is a common cause of inherited multisystem disease that often involves the nervous system. Despite major advances in our understanding of the pathophysiology of mitochondrial diseases, clinical management of these conditions remains largely supportive. Using a systematic approach, we identified 1,039 publications on treatments for mitochondrial diseases, only 35 of which included observations on more than five patients. Reports of a positive outcome on the basis of a biomarker of unproven clinical significance were more common in nonrandomized and nonblinded studies, suggesting a publication bias toward positive but poorly executed studies. Although trial design is improving, there is a critical need to develop new biomarkers of mitochondrial disease. In this Perspectives article, we make recommendations for the design of future treatment trials in mitochondrial diseases. Patients and physicians should no longer rely on potentially biased data, with the associated costs and risks. 01 augustus 2013 |
| Databáze: | OpenAIRE |
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