Cargando…
What is a minimal clinically important difference for clinical trials in patients with disorders of consciousness? a novel probabilistic approach
Over the last 30 years, there has been a growing trend in clinical trials towards assessing novel interventions not only against the benchmark of statistical significance, but also with respect to whether they lead to clinically meaningful changes for patients. In the context of Disorders of Conscio...
Autores principales: | , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2023
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10449161/ https://www.ncbi.nlm.nih.gov/pubmed/37616196 http://dx.doi.org/10.1371/journal.pone.0290290 |
_version_ | 1785094889225060352 |
---|---|
author | Monti, Martin M. Spivak, Norman M. Edlow, Brian L. Bodien, Yelena G. |
author_facet | Monti, Martin M. Spivak, Norman M. Edlow, Brian L. Bodien, Yelena G. |
author_sort | Monti, Martin M. |
collection | PubMed |
description | Over the last 30 years, there has been a growing trend in clinical trials towards assessing novel interventions not only against the benchmark of statistical significance, but also with respect to whether they lead to clinically meaningful changes for patients. In the context of Disorders of Consciousness (DOC), despite a growing landscape of experimental interventions, there is no agreed standard as to what counts as a minimal clinically important difference (MCID). In part, this issue springs from the fact that, by definition, DOC patients are either unresponsive (i.e., in a Vegetative State; VS) or non-communicative (i.e., in a Minimally Conscious State; MCS), which renders it impossible to assess any subjective perception of benefit, one of the two core aspects of MCIDs. Here, we develop a novel approach that leverages published, international diagnostic guidelines to establish a probability-based minimal clinically important difference (pMCID), and we apply it to the most validated and frequently used scale in DOC: the Coma Recovery Scale–Revised (CRS-R). This novel method is objective (i.e., based on published criteria for patient diagnosis) and easy to recalculate as the field refines its agreed-upon criteria for diagnosis. We believe this new approach can help clinicians determine whether observed changes in patients’ behavior are clinically important, even when patients cannot communicate their experiences, and can align the landscape of clinical trials in DOC with the practices in other medical fields. |
format | Online Article Text |
id | pubmed-10449161 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Public Library of Science |
record_format | MEDLINE/PubMed |
spelling | pubmed-104491612023-08-25 What is a minimal clinically important difference for clinical trials in patients with disorders of consciousness? a novel probabilistic approach Monti, Martin M. Spivak, Norman M. Edlow, Brian L. Bodien, Yelena G. PLoS One Research Article Over the last 30 years, there has been a growing trend in clinical trials towards assessing novel interventions not only against the benchmark of statistical significance, but also with respect to whether they lead to clinically meaningful changes for patients. In the context of Disorders of Consciousness (DOC), despite a growing landscape of experimental interventions, there is no agreed standard as to what counts as a minimal clinically important difference (MCID). In part, this issue springs from the fact that, by definition, DOC patients are either unresponsive (i.e., in a Vegetative State; VS) or non-communicative (i.e., in a Minimally Conscious State; MCS), which renders it impossible to assess any subjective perception of benefit, one of the two core aspects of MCIDs. Here, we develop a novel approach that leverages published, international diagnostic guidelines to establish a probability-based minimal clinically important difference (pMCID), and we apply it to the most validated and frequently used scale in DOC: the Coma Recovery Scale–Revised (CRS-R). This novel method is objective (i.e., based on published criteria for patient diagnosis) and easy to recalculate as the field refines its agreed-upon criteria for diagnosis. We believe this new approach can help clinicians determine whether observed changes in patients’ behavior are clinically important, even when patients cannot communicate their experiences, and can align the landscape of clinical trials in DOC with the practices in other medical fields. Public Library of Science 2023-08-24 /pmc/articles/PMC10449161/ /pubmed/37616196 http://dx.doi.org/10.1371/journal.pone.0290290 Text en © 2023 Monti et al https://creativecommons.org/licenses/by/4.0/This is an open access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/) , which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. |
spellingShingle | Research Article Monti, Martin M. Spivak, Norman M. Edlow, Brian L. Bodien, Yelena G. What is a minimal clinically important difference for clinical trials in patients with disorders of consciousness? a novel probabilistic approach |
title | What is a minimal clinically important difference for clinical trials in patients with disorders of consciousness? a novel probabilistic approach |
title_full | What is a minimal clinically important difference for clinical trials in patients with disorders of consciousness? a novel probabilistic approach |
title_fullStr | What is a minimal clinically important difference for clinical trials in patients with disorders of consciousness? a novel probabilistic approach |
title_full_unstemmed | What is a minimal clinically important difference for clinical trials in patients with disorders of consciousness? a novel probabilistic approach |
title_short | What is a minimal clinically important difference for clinical trials in patients with disorders of consciousness? a novel probabilistic approach |
title_sort | what is a minimal clinically important difference for clinical trials in patients with disorders of consciousness? a novel probabilistic approach |
topic | Research Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10449161/ https://www.ncbi.nlm.nih.gov/pubmed/37616196 http://dx.doi.org/10.1371/journal.pone.0290290 |
work_keys_str_mv | AT montimartinm whatisaminimalclinicallyimportantdifferenceforclinicaltrialsinpatientswithdisordersofconsciousnessanovelprobabilisticapproach AT spivaknormanm whatisaminimalclinicallyimportantdifferenceforclinicaltrialsinpatientswithdisordersofconsciousnessanovelprobabilisticapproach AT edlowbrianl whatisaminimalclinicallyimportantdifferenceforclinicaltrialsinpatientswithdisordersofconsciousnessanovelprobabilisticapproach AT bodienyelenag whatisaminimalclinicallyimportantdifferenceforclinicaltrialsinpatientswithdisordersofconsciousnessanovelprobabilisticapproach |