A plea to provide best evidence in trials under sample-size restrictions: the example of pioglitazone to resolve leukoplakia and erythroplakia in Fanconi anemia patients

A plea to provide best evidence in trials under sample-size restrictions: the example of pioglitazone to resolve leukoplakia and erythroplakia in Fanconi anemia patients

Abstract In planning a clinical trial for demonstrating the efficacy of pioglitazone to resolve leukoplakia and erythroplakia in Fanconi anemia patients we had to discuss the need for a randomized controlled trial particularly under sample-size restrictions as very promising results were available f...

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Bibliographic Details
Main Authors: Florian Lasch, Kristina Weber, Mwe Mwe Chao, Armin Koch
Format: Article
Language:English
Published: BMC 2017-05-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Randomization
Single-arm trial
RCT
Rare disease
Historical control
Fanconi anemia
Online Access:http://link.springer.com/article/10.1186/s13023-017-0655-8

Internet

http://link.springer.com/article/10.1186/s13023-017-0655-8

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