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Related Concept Videos

Blood Studies I: ABG and VBG01:26

Blood Studies I: ABG and VBG

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Blood studies are critical in the medical field, enabling healthcare professionals to assess a patient's health status accurately. This page will focus on two significant blood studies: Arterial Blood Gas (ABG) and Venous Blood Gas (VBG).
Arterial Blood Gas (ABG)
Arterial Blood Gas (ABG) studies are crucial for assessing the lungs' ability to supply oxygen and remove carbon dioxide, reflecting the patient's ventilation status. They also help understand the kidneys' capacity to...
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Viability Assays for Cells in Culture
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Plasma neurofilament analysis in VITALITY-ALS.

Tyrell J Simkins1, Stuart Kupfer1, Fady I Malik1

  • 1Cytokinetics, Incorporated, South San Francisco, CA, USA.

Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration
|November 8, 2024
PubMed
Summary

Neurofilament light chain (NfL) levels at baseline correlate with disease progression in people with ALS. These findings suggest NfL can serve as a prognostic biomarker, especially for rapid disease progression.

Keywords:
Amyotrophic lateral sclerosisbiomarkersneurofilament heavy chainneurofilament light chainneurofilaments

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Area of Science:

  • Biochemistry
  • Neurology
  • Clinical Trials

Background:

  • Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease.
  • Biomarkers are needed to track disease progression and predict outcomes in ALS patients.
  • Neurofilaments (Nf) are proteins released into the bloodstream upon neuronal damage.

Purpose of the Study:

  • To investigate the correlation between neurofilament (Nf) concentrations and clinical characteristics in ALS.
  • To assess Nf concentrations as potential markers for disease progression in a large longitudinal ALS cohort.
  • To analyze Nf levels in relation to tirasemtiv treatment in the VITALITY-ALS trial.

Main Methods:

  • Longitudinal analysis of plasma Nf concentrations from the VITALITY-ALS trial (NCT02496767).
  • Correlation analysis (Pearson's r) between Nf levels and clinical measures like slow vital capacity (SVC) and ALSFRS-R score.
  • Evaluation of Nf concentrations against pre-study and in-study rates of disease progression (psRDP/isRDP).

Main Results:

  • Baseline Nf light chain (NfL) concentration significantly correlated with both pre-study (r=0.50) and in-study (r=0.53) rates of disease progression.
  • Nf concentrations showed correlations with changes in SVC and ALSFRS-R scores over time.
  • Plasma phosphorylated Nf heavy chain (pNfH) decreased over time, while NfL showed no significant change during the study period.

Conclusions:

  • Baseline NfL concentration is a significant prognostic marker for disease progression in people with ALS.
  • Neurofilaments show promise as prognostic biomarkers, particularly for identifying rapid disease progression in ALS patients.
  • The study highlights the utility of Nf measurements in understanding ALS disease dynamics.