Coalition Against Major
Diseases/European Medicines Agency biomarker qualification of hippocampal
volume for enrichment of clinical trials in predementia stages of Alzheimer's
disease
Regulatory
qualification of a biomarker for a defined context of use provides
scientifically robust assurances to sponsors and regulators that accelerate
appropriate adoption of biomarkers into drug development.
The Coalition Against Major Diseases submitted a dossier to the
Scientific Advice Working Party of the European Medicines Agency requesting a
qualification opinion on the use of hippocampal volume as a biomarker for
enriching clinical trials in subjects with mild cognitive impairment,
incorporating a scientific rationale, a literature review and a de novo analysis
of Alzheimer's Disease Neuroimaging Initiative data.
p. 421-429
Robustness of automated hippocampal
volumetry across magnetic resonance field strengths and repeat images
Low HCV has recently been qualified by the European Medicines
Agency as a biomarker for enrichment of clinical trials in predementia stages
of Alzheimer's disease. For automated methods to meet the necessary regulatory
requirements, it is essential they be standardized and their performance be
well characterized.
p. 430-438
The prevalence of mild cognitive
impairment and its etiological subtypes in elderly Chinese
Background
Epidemiologic studies on mild cognitive impairment (MCI) are
limited in China.
Methods
Using a multistage cluster sampling design, a total of 10,276
community residents (6096 urban, 4180 rural) aged 65 years or older
were evaluated and diagnosed with normal cognition, MCI, or dementia.
Conclusions
The prevalence of MCI in elderly Chinese is higher in rural than
in urban areas. Vascular-related MCI (MCI-CVD and MCI-VRF) was most common.
p. 439-447
Addition of MHPG to Alzheimer's disease
biomarkers improves differentiation of dementia with Lewy bodies from
Alzheimer's disease but not other dementias
Overlapping clinical features make it difficult to distinguish
dementia with Lewy bodies (DLB) from Alzheimer's disease (AD) and other
dementia types. In this study we aimed to determine whether the combination of
cerebrospinal fluid (CSF) biomarkers, amyloid-β42 (Aβ42),
total tau protein (t-tau), and phosphorylated tau protein (p-tau), in
combination with 3-methoxy-4-hydroxyphenylethyleneglycol (MHPG), could be
useful in discriminating DLB from vascular dementia (VaD) and frontotemporal
dementia (FTD), as we previously demonstrated for differentiation of DLB from
AD.
p. 448-455
Automatic temporal lobe atrophy
assessment in prodromal AD: Data from the DESCRIPA study
In the framework of the clinical validation of research tools,
this investigation presents a validation study of an automatic medial temporal
lobe atrophy measure that is applied to a naturalistic population sampled from
memory clinic patients across Europe.
p. 456-467
Features of the Japanese national
dementia strategy in comparison with international dementia policies: How
should a national dementia policy interact with the public health- and
social-care systems?
The Ministry of Health, Labour, and Welfare of the Japanese
national government announced a “Five-Year Plan for Promotion of Measures
Against Dementia (Orange Plan)” in September 2012. This article described
features of the Japanese dementia strategy in comparison with international
dementia policies.
p. 468-476
A platform for discovery: The University
of Pennsylvania Integrated Neurodegenerative Disease Biobank
Neurodegenerative diseases (NDs) are defined by the accumulation
of abnormal protein deposits in the central nervous system (CNS), and only
neuropathological examination enables a definitive diagnosis. Brain banks and
their associated scientific programs have shaped the actual knowledge of NDs,
identifying and characterizing the CNS deposits that define new diseases,
formulating staging schemes, and establishing correlations between
neuropathological changes and clinical features. However, brain banks have
evolved to accommodate the banking of biofluids as well as DNA and RNA samples.
Moreover, the value of biobanks is greatly enhanced if they link all the
multidimensional clinical and laboratory information of each case, which is
accomplished, optimally, using systematic and standardized operating procedures,
and in the framework of multidisciplinary teams with the support of a flexible
and user-friendly database system that facilitates the sharing of information
of all the teams in the network. We describe a biobanking system that is a
platform for discovery research at the Center for Neurodegenerative Disease
Research at the University of Pennsylvania.
p. 477-484
Plasma nutrient status of patients with
Alzheimer's disease: Systematic review and meta-analysis
Alzheimer disease (AD) patients are at risk of nutritional
insufficiencies because of physiological and psychological factors. Nutritional
compounds are postulated to play a role in the pathophysiological processes
that are affected in AD. We here provide the first systematic review and
meta-analysis that compares plasma levels of micronutrients and fatty acids in
AD patients to those in cognitively intact elderly controls. A secondary
objective was to explore the presence of different plasma nutrient levels
between AD and control populations that did not differ in measures of
protein/energy nourishment.
p. 485-502
Justifying reimbursement for Alzheimer's
diagnostics and treatments: Seeking alignment on evidence
The increasing cost of health care combined with expensive new
drugs and diagnostics is leading to more frequent gaps between regulatory and
subsequent reimbursement approval decisions. As a result, persons with
Alzheimer's disease may have difficulty accessing the benefit of medical
advances. In contrast to the long history and established structure for drug
approval, payer decision making is dispersed, not standardized, and
perspectives on necessary evidence and the evaluation of this evidence differ
and are often poorly defined. Particularly challenging is how to demonstrate
the value of drugs and diagnostics for patients who do not yet have significant
functional decline. Although discussions to develop consensus continue,
clinical trials should begin to incorporate health system and patient-oriented
outcomes. In some situations, additional studies designed to demonstrate value
and comparative effectiveness will be needed. Such studies should examine
outcomes of representative populations in community settings. To assure
scientific advances in diagnosis and treatment benefit in patients, developing
evidence to support reimbursement will become as important as obtaining
regulatory approval.
p. 503-508
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