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...
Հիմնական հեղինակներ: | , , , , , |
---|---|
Ձևաչափ: | Journal article |
Լեզու: | English |
Հրապարակվել է: |
BioMed Central
2017
|
_version_ | 1826268803223257088 |
---|---|
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. |
first_indexed | 2024-03-06T21:15:12Z |
format | Journal article |
id | oxford-uuid:3f8e9e9d-ae13-435c-99db-ced87d438dcf |
institution | University of Oxford |
language | English |
last_indexed | 2024-03-06T21:15:12Z |
publishDate | 2017 |
publisher | BioMed Central |
record_format | dspace |
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 |
work_keys_str_mv | AT dahalp statisticalmethodstoderiveefficacyestimatesofantimalarialsforuncomplicatedplasmodiumfalciparummalariapitfallsandchallenges AT simpsonj statisticalmethodstoderiveefficacyestimatesofantimalarialsforuncomplicatedplasmodiumfalciparummalariapitfallsandchallenges AT dorseyg statisticalmethodstoderiveefficacyestimatesofantimalarialsforuncomplicatedplasmodiumfalciparummalariapitfallsandchallenges AT guerinp statisticalmethodstoderiveefficacyestimatesofantimalarialsforuncomplicatedplasmodiumfalciparummalariapitfallsandchallenges AT pricer statisticalmethodstoderiveefficacyestimatesofantimalarialsforuncomplicatedplasmodiumfalciparummalariapitfallsandchallenges AT stepniewskak statisticalmethodstoderiveefficacyestimatesofantimalarialsforuncomplicatedplasmodiumfalciparummalariapitfallsandchallenges |