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

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 ca...

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Հիմնական հեղինակներ: Dahal, P, Simpson, J, Dorsey, G, Guérin, P, Price, R, Stepniewska, K
Ձևաչափ: Journal article
Լեզու:English
Հրապարակվել է: BioMed Central 2017
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author Dahal, P
Simpson, J
Dorsey, G
Guérin, P
Price, R
Stepniewska, K
author_facet Dahal, P
Simpson, J
Dorsey, G
Guérin, P
Price, R
Stepniewska, K
author_sort Dahal, P
collection OXFORD
description 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.
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spelling oxford-uuid:3f8e9e9d-ae13-435c-99db-ced87d438dcf2022-03-26T14:32:45ZStatistical methods to derive efficacy estimates of anti-malarials for uncomplicated Plasmodium falciparum malaria: pitfalls and challengesJournal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:3f8e9e9d-ae13-435c-99db-ced87d438dcfEnglishSymplectic Elements at OxfordBioMed Central2017Dahal, PSimpson, JDorsey, GGuérin, PPrice, RStepniewska, KThe 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.
spellingShingle Dahal, P
Simpson, J
Dorsey, G
Guérin, P
Price, R
Stepniewska, K
Statistical methods to derive efficacy estimates of anti-malarials for uncomplicated Plasmodium falciparum malaria: pitfalls and challenges
title Statistical methods to derive efficacy estimates of anti-malarials for uncomplicated Plasmodium falciparum malaria: pitfalls and challenges
title_full Statistical methods to derive efficacy estimates of anti-malarials for uncomplicated Plasmodium falciparum malaria: pitfalls and challenges
title_fullStr Statistical methods to derive efficacy estimates of anti-malarials for uncomplicated Plasmodium falciparum malaria: pitfalls and challenges
title_full_unstemmed Statistical methods to derive efficacy estimates of anti-malarials for uncomplicated Plasmodium falciparum malaria: pitfalls and challenges
title_short Statistical methods to derive efficacy estimates of anti-malarials for uncomplicated Plasmodium falciparum malaria: pitfalls and challenges
title_sort statistical methods to derive efficacy estimates of anti malarials for uncomplicated plasmodium falciparum malaria pitfalls and challenges
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