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Statistical methods to derive efficacy estimates of anti-malarials for uncomplicated Plasmodium falciparum malaria: pitfalls and challenges

Abstract:
The Kaplan-Meier (K-M) method is currently the preferred approach to derive an efficacy estimate from anti-malarial trial data. In this approach event times are assumed to be continuous and estimates are generated on the assumption that there is only one cause of failure. In reality, failures are captured at pre-scheduled time points and patients can fail treatment due to a variety of causes other than the primary endpoint, commonly termed competing risk events. Ignoring these underlying assumptions can potentially distort the derived efficacy estimates and result in misleading conclusions. This review details the evolution of statistical methods used to derive anti-malarial efficacy for uncomplicated Plasmodium falciparum malaria and assesses the limitations of the current practices. Alternative approaches are explored and their implementation is discussed using example data from a large multi-site study.
Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1186/s12936-017-2074-7

Authors


More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
NDM; Tropical Medicine
Role:
Author
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
NDM; Tropical Medicine
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
NDM
Sub department:
Tropical Medicine
Role:
Author


Publisher:
BioMed Central
Journal:
Malaria journal More from this journal
Volume:
16
Issue:
1
Pages:
430
Publication date:
2017-10-26
Acceptance date:
2017-10-19
DOI:
ISSN:
1475-2875
Pmid:
29073901


Language:
English
Keywords:
Pubs id:
pubs:742523
UUID:
uuid:3f8e9e9d-ae13-435c-99db-ced87d438dcf
Local pid:
pubs:742523
Source identifiers:
742523
Deposit date:
2017-11-04

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