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• W4383961965 abstract "Parkin et al.1Parkin G.M. Thomas E.A. Corey-Bloom J. Plasma NfL as a prognostic biomarker for enriching HD-ISS stage 1 categorisation: a cross-sectional study.eBioMedicine. 2023; 93: 104646https://doi.org/10.1016/j.ebiom.2023.104646Summary Full Text Full Text PDF Scopus (0) Google Scholar propose the use of blood neurofilament light (NfL) levels as an enrichment biomarker for individuals with Huntington's disease (HD) who may be classified as Huntington's Disease Integrated Staging System (HD-ISS) stage 1. The HD-ISS is a recently developed staging system for HD that offers the advantage of describing the entire disease course from birth to death, dividing it into four stages based on robust evidentiary criteria.2Tabrizi S.J. Schoble S. Gantman E.C. et al.A biological classification of Huntington's disease: the integrated staging system.Lancet Neurol. 2022; 21: 632-644Summary Full Text Full Text PDF PubMed Google Scholar While this staging system describes clear metrics for transition from one stage to the next, there are no metrics to indicate where within a given stage an HD individual may be, and predicting when the transition to the next stage will occur would benefit from having additional easily obtainable biomarkers. The criteria chosen for describing the staging system had to meet two key requirements: they must be prognostic, and their prognostic value had to be established in at least two independent models that used large (n > 99), longitudinal datasets. The use of blood NfL levels as an enrichment biomarker should also meet stringent criteria. The validation of an enrichment biomarker requires the demonstration it is prognostic or diagnostic for the state it is enriching for;3FDA-NIH Biomarker Working GroupBEST (biomarkers, EndpointS, and other tools) resource [internet]. Silver Spring (MD): Food and Drug Administration (US).https://www.ncbi.nlm.nih.gov/books/NBK326791/Co-published by National Institutes of Health (US), Bethesda (MD)Date: 2016Google Scholar in the Parkin et al.1Parkin G.M. Thomas E.A. Corey-Bloom J. Plasma NfL as a prognostic biomarker for enriching HD-ISS stage 1 categorisation: a cross-sectional study.eBioMedicine. 2023; 93: 104646https://doi.org/10.1016/j.ebiom.2023.104646Summary Full Text Full Text PDF Scopus (0) Google Scholar paper that state is HD-ISS stage 1. The canonical criteria for classifying individuals as HD-ISS stage 1 are based on the presence of CAG >39, the degree of caudate or putamen atrophy above the reference cut-off, and not meeting the criteria for stage 2 that are based on scores on the total motor score or Single Digit Modality test (SDMT).2Tabrizi S.J. Schoble S. Gantman E.C. et al.A biological classification of Huntington's disease: the integrated staging system.Lancet Neurol. 2022; 21: 632-644Summary Full Text Full Text PDF PubMed Google Scholar However, the necessity of conducting volumetric MRI to establish classification in HD-ISS stage 1 is considered a relatively high burden when screening candidates for a clinical study. Volumetric MRI is not commonly used in clinical practice, requiring an expensive and specialized imaging segmentation and quantification pipeline. When screening potential participants for a clinical trial aimed at individuals in HD-ISS stage 1, there is a retrospective cost associated with conducting MRIs on all candidates who ultimately do not meet the criteria. However, for those who do meet the criteria, the cost of volumetric MRI is of lesser concern, as it will always be necessary to evaluate this particular population. Incorporating an enrichment criterion based on a blood measurement that effectively filters out participants unlikely to be classified as stage 1 in a clinical trial targeting that specific population holds significant appeal. The findings presented by Parkin et al.,1Parkin G.M. Thomas E.A. Corey-Bloom J. Plasma NfL as a prognostic biomarker for enriching HD-ISS stage 1 categorisation: a cross-sectional study.eBioMedicine. 2023; 93: 104646https://doi.org/10.1016/j.ebiom.2023.104646Summary Full Text Full Text PDF Scopus (0) Google Scholar where individuals in HD-ISS stage 1 demonstrate a median blood NfL value of 48.7 pg/ml, represent an advancement in this regard. The findings are based on data from a cohort that is substantial (n = 290), and the NFL assay has demonstrated reasonable robustness at the authors' site. However, before plasma neurofilament light can be systematically used for HD-ISS stage 1 enrichment, several key requirements need to be met. First, the employed assay must undergo clinical validation, as different platforms can yield variable measurements and affect the defined cutoff value. Second, it is necessary to validate the cutoff values in a cohort where HD-ISS stages were prospectively established. Last, the cutoff value must be determined for the specific endpoint of interest, such as classification in HD-ISS stage 1. With ongoing research advancements, these concerns are likely to be addressed. It is crucial that any enrichment biomarker intended to facilitate clinical trial population screening is highly sensitive, as reflected in a very high negative predictive value (NPV). Sensitivity, specificity, or positive/negative predictive values for the classification of HD-ISS stage 1 based on a given level of NFL in plasma are not yet available. A high NPV is necessary because, in this context, the enrichment biomarker is operating as a diagnostic of the HD-ISS stage 1. A high NPV minimizes the risk of erroneously excluding potential participants who possess NFL plasma levels below the designated cutoff but are genuinely in HD-ISS stage 1.4Pencina M.J. D'Agostino R.B. Vasan R.S. Statistical methods for assessment of added usefulness of new biomarkers.Clin Chem Lab Med. 2010; 48: 1703-1711https://doi.org/10.1515/CCLM.2010.340Crossref PubMed Scopus (269) Google Scholar,5Kerr K.F. Roth J. Zhu K. et al.Evaluating biomarkers for prognostic enrichment of clinical trials.Clin Trials. 2017; 14: 629-638https://doi.org/10.1177/1740774517723588Crossref PubMed Scopus (20) Google Scholar Failing to attain a robust negative predictive value would lead to the unnecessary rejection of numerous authentic HD-ISS stage 1 participants, thereby impeding the recruitment process for the clinical trial. The progressive recognition by regulatory authorities like the FDA of the HD-ISS as the reference staging system foreshadows its wide adoption in the coming years. Establishing blood-based biomarkers as tools for drug development has the potential to be transformative, as demonstrated by the significant impact of the blood β-amyloid assay in Alzheimer's disease research and clinical care.6Hansson O. Blennow K. Zetterberg H. Dage J. Blood biomarkers for Alzheimer's disease in clinical practice and trials.Nat Aging. 2023; 3: 506-519https://doi.org/10.1038/s43587-023-00403-3Crossref Scopus (0) Google Scholar Prior publications report on efforts to investigate plasma neurofilament light as a biomarker for disease monitoring in Huntington's disease (e.g., Byrne et al.7Byrne L.M. Rodrigues F.B. Blennow K. et al.Neurofilament light protein in blood as a potential biomarker of neurodegeneration in Huntington's disease: a retrospective cohort analysis.Lancet Neurol. 2017; 16 (Erratum in: Lancet Neurol. 2017 Sep;16(9):683): 601-609https://doi.org/10.1016/S1474-4422(17)30124-2Summary Full Text Full Text PDF PubMed Scopus (208) Google Scholar and Parkin et al.8Parkin G.M. Corey-Bloom J. Snell C. Castleton J. Thomas E.A. Plasma neurofilament light in Huntington's disease: a marker for disease onset, but not symptom progression.Parkinsonism Relat Disord. 2021; 87: 32-38https://doi.org/10.1016/j.parkreldis.2021.04.017Summary Full Text Full Text PDF PubMed Scopus (12) Google Scholar). Notably, the study conducted by Parkin et al.1Parkin G.M. Thomas E.A. Corey-Bloom J. Plasma NfL as a prognostic biomarker for enriching HD-ISS stage 1 categorisation: a cross-sectional study.eBioMedicine. 2023; 93: 104646https://doi.org/10.1016/j.ebiom.2023.104646Summary Full Text Full Text PDF Scopus (0) Google Scholar serves as a stepping stone in recognizing plasma NfL as an enrichment biomarker, thereby expanding its potential utility. C.S. wrote the first draft. C.S. and H.W. revised and finalized the submitted version. The authors used an AI assisted tool (ChatGPT) to edit for readability some of the sentences. Authors revised and re-edited for style consistency the text produced by ChatGPT. The authors take full responsibility for the content of the publication. C.S. is the senior author of the paper describing the HD-ISS and its development referenced in 2. The authors do not have other conflicts of interest to disclose. Plasma NfL as a prognostic biomarker for enriching HD-ISS stage 1 categorisation: a cross-sectional studyOur findings suggest that plasma NfL levels may have use in enriching Stage 1 membership into sub-groups that are less than, and within, predicted 10 years until CMD. Full-Text PDF Open Access" @default.
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• W4383961965 title "Facilitating Huntington's disease research: plasma neurofilament levels as a promising enrichment biomarker for HD-ISS stage 1" @default.
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