Methodological approaches for analysing data from therapeutic efficacy studies
Abstract Several anti-malarial drugs have been evaluated in randomized clinical trials to treat acute uncomplicated Plasmodium falciparum malaria. The outcome of anti-malarial drug efficacy studies is classified into one of four possible outcomes defined by the World Health Organization: adequate cl...
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ftdoajarticles:oai:doaj.org/article:1f2841539cc6438db138c9b5819ccd59 2023-05-15T15:11:24+02:00 Methodological approaches for analysing data from therapeutic efficacy studies Solange Whegang Youdom Leonardo K. Basco 2021-05-01T00:00:00Z https://doi.org/10.1186/s12936-021-03768-1 https://doaj.org/article/1f2841539cc6438db138c9b5819ccd59 EN eng BMC https://doi.org/10.1186/s12936-021-03768-1 https://doaj.org/toc/1475-2875 doi:10.1186/s12936-021-03768-1 1475-2875 https://doaj.org/article/1f2841539cc6438db138c9b5819ccd59 Malaria Journal, Vol 20, Iss 1, Pp 1-5 (2021) Plasmodium falciparum Drug resistance Artemisinin Ordinal outcome Multiple comparison Network meta-analysis Arctic medicine. Tropical medicine RC955-962 Infectious and parasitic diseases RC109-216 article 2021 ftdoajarticles https://doi.org/10.1186/s12936-021-03768-1 2022-12-31T12:38:57Z Abstract Several anti-malarial drugs have been evaluated in randomized clinical trials to treat acute uncomplicated Plasmodium falciparum malaria. The outcome of anti-malarial drug efficacy studies is classified into one of four possible outcomes defined by the World Health Organization: adequate clinical and parasitological response, late parasitological failure, late clinical failure, early treatment failure. These four ordered categories are ordinal data, which are reduced to either a binary outcome (i.e., treatment success and treatment failure) to calculate the proportions of treatment failure or to time-to-event outcome for Kaplan–Meier survival analysis. The arbitrary transition from 4-level ordered categories to 2-level type categories results in a loss of statistical power. In the opinion of the authors, this outcome can be considered as ordinal at a fixed endpoint or at longitudinal endpoints. Alternative statistical methods can be applied to 4-level ordinal categories of therapeutic response to optimize data exploitation. Furthermore, network meta-analysis is useful not only for direct comparison of drugs which were evaluated together in a randomized design, but also for indirect comparison of different artemisinin-based combinations across different clinical studies using a common drug comparator, with the aim to determine the ranking order of drug efficacy. Previous works conducted in Cameroonian children served as data source to illustrate the feasibility of these novel statistical approaches. Data analysis based on ordinal end-point may be helpful to gain further insight into anti-malarial drug efficacy. Article in Journal/Newspaper Arctic Directory of Open Access Journals: DOAJ Articles Arctic Meier ENVELOPE(-45.900,-45.900,-60.633,-60.633) Malaria Journal 20 1 |
institution |
Open Polar |
collection |
Directory of Open Access Journals: DOAJ Articles |
op_collection_id |
ftdoajarticles |
language |
English |
topic |
Plasmodium falciparum Drug resistance Artemisinin Ordinal outcome Multiple comparison Network meta-analysis Arctic medicine. Tropical medicine RC955-962 Infectious and parasitic diseases RC109-216 |
spellingShingle |
Plasmodium falciparum Drug resistance Artemisinin Ordinal outcome Multiple comparison Network meta-analysis Arctic medicine. Tropical medicine RC955-962 Infectious and parasitic diseases RC109-216 Solange Whegang Youdom Leonardo K. Basco Methodological approaches for analysing data from therapeutic efficacy studies |
topic_facet |
Plasmodium falciparum Drug resistance Artemisinin Ordinal outcome Multiple comparison Network meta-analysis Arctic medicine. Tropical medicine RC955-962 Infectious and parasitic diseases RC109-216 |
description |
Abstract Several anti-malarial drugs have been evaluated in randomized clinical trials to treat acute uncomplicated Plasmodium falciparum malaria. The outcome of anti-malarial drug efficacy studies is classified into one of four possible outcomes defined by the World Health Organization: adequate clinical and parasitological response, late parasitological failure, late clinical failure, early treatment failure. These four ordered categories are ordinal data, which are reduced to either a binary outcome (i.e., treatment success and treatment failure) to calculate the proportions of treatment failure or to time-to-event outcome for Kaplan–Meier survival analysis. The arbitrary transition from 4-level ordered categories to 2-level type categories results in a loss of statistical power. In the opinion of the authors, this outcome can be considered as ordinal at a fixed endpoint or at longitudinal endpoints. Alternative statistical methods can be applied to 4-level ordinal categories of therapeutic response to optimize data exploitation. Furthermore, network meta-analysis is useful not only for direct comparison of drugs which were evaluated together in a randomized design, but also for indirect comparison of different artemisinin-based combinations across different clinical studies using a common drug comparator, with the aim to determine the ranking order of drug efficacy. Previous works conducted in Cameroonian children served as data source to illustrate the feasibility of these novel statistical approaches. Data analysis based on ordinal end-point may be helpful to gain further insight into anti-malarial drug efficacy. |
format |
Article in Journal/Newspaper |
author |
Solange Whegang Youdom Leonardo K. Basco |
author_facet |
Solange Whegang Youdom Leonardo K. Basco |
author_sort |
Solange Whegang Youdom |
title |
Methodological approaches for analysing data from therapeutic efficacy studies |
title_short |
Methodological approaches for analysing data from therapeutic efficacy studies |
title_full |
Methodological approaches for analysing data from therapeutic efficacy studies |
title_fullStr |
Methodological approaches for analysing data from therapeutic efficacy studies |
title_full_unstemmed |
Methodological approaches for analysing data from therapeutic efficacy studies |
title_sort |
methodological approaches for analysing data from therapeutic efficacy studies |
publisher |
BMC |
publishDate |
2021 |
url |
https://doi.org/10.1186/s12936-021-03768-1 https://doaj.org/article/1f2841539cc6438db138c9b5819ccd59 |
long_lat |
ENVELOPE(-45.900,-45.900,-60.633,-60.633) |
geographic |
Arctic Meier |
geographic_facet |
Arctic Meier |
genre |
Arctic |
genre_facet |
Arctic |
op_source |
Malaria Journal, Vol 20, Iss 1, Pp 1-5 (2021) |
op_relation |
https://doi.org/10.1186/s12936-021-03768-1 https://doaj.org/toc/1475-2875 doi:10.1186/s12936-021-03768-1 1475-2875 https://doaj.org/article/1f2841539cc6438db138c9b5819ccd59 |
op_doi |
https://doi.org/10.1186/s12936-021-03768-1 |
container_title |
Malaria Journal |
container_volume |
20 |
container_issue |
1 |
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1766342263810555904 |