Imaging-based measures of disease progression in clinical trials of disease-modifying drugs for Alzheimer disease

Brandy Matthews, Eric R. Siemers, P. David Mozley

Research output: Contribution to journalArticle

32 Citations (Scopus)

Abstract

The authors review and assess imaging-based strategies for measuring the rate of progression of Alzheimer disease (AD). Such techniques may be useful in addition to the behavioral instruments typically used in these studies and may be more sensitive to treatment-related change. MEDLINE searches obtained relevant published literature. Articles were reviewed with particular attention to assessments of rate of disease progression and the effects of investigational drugs. Authors studied a variety of techniques, including volumetric magnetic resonance imaging, functional MRI, fluorodeoxyglucose positron emission tomography, and several target-specific radiopharmaceuticals. In cross-sectional as well as small longitudinal trials, many of these show promise not only in diagnosis, but also as measures of disease progression. The effects of drugs that provide symptomatic relief on these measures have not been fully characterized. Even less is known about the effects of investigational drugs that may slow disease progression. Several neuroimaging techniques have been studied that could improve the ability of clinical trials to quantify the rate of progression of AD. Clinical trials of investigational drugs would benefit from more systematic validation of image-based outcome measures. Several choices of imaging techniques are available. An understanding of the relationship between a statistically significant effect size in an imaging marker and a clinically significant change in rate of disease progression will require additional studies.

Original languageEnglish (US)
Pages (from-to)146-159
Number of pages14
JournalAmerican Journal of Geriatric Psychiatry
Volume11
Issue number2
DOIs
StatePublished - 2003
Externally publishedYes

Fingerprint

Investigational Drugs
Disease Progression
Alzheimer Disease
Clinical Trials
Pharmaceutical Preparations
Magnetic Resonance Imaging
Aptitude
Radiopharmaceuticals
Neuroimaging
MEDLINE
Positron-Emission Tomography
Outcome Assessment (Health Care)

ASJC Scopus subject areas

  • Geriatrics and Gerontology
  • Psychiatry and Mental health

Cite this

Imaging-based measures of disease progression in clinical trials of disease-modifying drugs for Alzheimer disease. / Matthews, Brandy; Siemers, Eric R.; Mozley, P. David.

In: American Journal of Geriatric Psychiatry, Vol. 11, No. 2, 2003, p. 146-159.

Research output: Contribution to journalArticle

@article{042c39a24b4c49b2982489b03c412df1,
title = "Imaging-based measures of disease progression in clinical trials of disease-modifying drugs for Alzheimer disease",
abstract = "The authors review and assess imaging-based strategies for measuring the rate of progression of Alzheimer disease (AD). Such techniques may be useful in addition to the behavioral instruments typically used in these studies and may be more sensitive to treatment-related change. MEDLINE searches obtained relevant published literature. Articles were reviewed with particular attention to assessments of rate of disease progression and the effects of investigational drugs. Authors studied a variety of techniques, including volumetric magnetic resonance imaging, functional MRI, fluorodeoxyglucose positron emission tomography, and several target-specific radiopharmaceuticals. In cross-sectional as well as small longitudinal trials, many of these show promise not only in diagnosis, but also as measures of disease progression. The effects of drugs that provide symptomatic relief on these measures have not been fully characterized. Even less is known about the effects of investigational drugs that may slow disease progression. Several neuroimaging techniques have been studied that could improve the ability of clinical trials to quantify the rate of progression of AD. Clinical trials of investigational drugs would benefit from more systematic validation of image-based outcome measures. Several choices of imaging techniques are available. An understanding of the relationship between a statistically significant effect size in an imaging marker and a clinically significant change in rate of disease progression will require additional studies.",
author = "Brandy Matthews and Siemers, {Eric R.} and Mozley, {P. David}",
year = "2003",
doi = "10.1097/00019442-200303000-00005",
language = "English (US)",
volume = "11",
pages = "146--159",
journal = "American Journal of Geriatric Psychiatry",
issn = "1064-7481",
publisher = "Lippincott Williams and Wilkins",
number = "2",

}

TY - JOUR

T1 - Imaging-based measures of disease progression in clinical trials of disease-modifying drugs for Alzheimer disease

AU - Matthews, Brandy

AU - Siemers, Eric R.

AU - Mozley, P. David

PY - 2003

Y1 - 2003

N2 - The authors review and assess imaging-based strategies for measuring the rate of progression of Alzheimer disease (AD). Such techniques may be useful in addition to the behavioral instruments typically used in these studies and may be more sensitive to treatment-related change. MEDLINE searches obtained relevant published literature. Articles were reviewed with particular attention to assessments of rate of disease progression and the effects of investigational drugs. Authors studied a variety of techniques, including volumetric magnetic resonance imaging, functional MRI, fluorodeoxyglucose positron emission tomography, and several target-specific radiopharmaceuticals. In cross-sectional as well as small longitudinal trials, many of these show promise not only in diagnosis, but also as measures of disease progression. The effects of drugs that provide symptomatic relief on these measures have not been fully characterized. Even less is known about the effects of investigational drugs that may slow disease progression. Several neuroimaging techniques have been studied that could improve the ability of clinical trials to quantify the rate of progression of AD. Clinical trials of investigational drugs would benefit from more systematic validation of image-based outcome measures. Several choices of imaging techniques are available. An understanding of the relationship between a statistically significant effect size in an imaging marker and a clinically significant change in rate of disease progression will require additional studies.

AB - The authors review and assess imaging-based strategies for measuring the rate of progression of Alzheimer disease (AD). Such techniques may be useful in addition to the behavioral instruments typically used in these studies and may be more sensitive to treatment-related change. MEDLINE searches obtained relevant published literature. Articles were reviewed with particular attention to assessments of rate of disease progression and the effects of investigational drugs. Authors studied a variety of techniques, including volumetric magnetic resonance imaging, functional MRI, fluorodeoxyglucose positron emission tomography, and several target-specific radiopharmaceuticals. In cross-sectional as well as small longitudinal trials, many of these show promise not only in diagnosis, but also as measures of disease progression. The effects of drugs that provide symptomatic relief on these measures have not been fully characterized. Even less is known about the effects of investigational drugs that may slow disease progression. Several neuroimaging techniques have been studied that could improve the ability of clinical trials to quantify the rate of progression of AD. Clinical trials of investigational drugs would benefit from more systematic validation of image-based outcome measures. Several choices of imaging techniques are available. An understanding of the relationship between a statistically significant effect size in an imaging marker and a clinically significant change in rate of disease progression will require additional studies.

UR - http://www.scopus.com/inward/record.url?scp=0037371280&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=0037371280&partnerID=8YFLogxK

U2 - 10.1097/00019442-200303000-00005

DO - 10.1097/00019442-200303000-00005

M3 - Article

C2 - 12611744

AN - SCOPUS:0037371280

VL - 11

SP - 146

EP - 159

JO - American Journal of Geriatric Psychiatry

JF - American Journal of Geriatric Psychiatry

SN - 1064-7481

IS - 2

ER -