Longitudinal analysis of the neurological features of ataxia telangiectasia

Aim: To assess the relationship between genotype and neurological progression in ataxia‐telangiectasia (A‐T). Methods: Clinical and laboratory data were extracted retrospectively from the records of patients attending the UK National Ataxia‐Telangiectasia Clinic. Neurological assessments were perfo...

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Main Authors: Jackson, Thomas J., Chow, Gabriel, Suri, Mohnish, Byrd, Philip, Taylor, Malcolm R., Whitehouse, William P.
Format: Article
Published: Wiley 2016
Online Access:https://eprints.nottingham.ac.uk/51341/
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author Jackson, Thomas J.
Chow, Gabriel
Suri, Mohnish
Byrd, Philip
Taylor, Malcolm R.
Whitehouse, William P.
author_facet Jackson, Thomas J.
Chow, Gabriel
Suri, Mohnish
Byrd, Philip
Taylor, Malcolm R.
Whitehouse, William P.
author_sort Jackson, Thomas J.
building Nottingham Research Data Repository
collection Online Access
description Aim: To assess the relationship between genotype and neurological progression in ataxia‐telangiectasia (A‐T). Methods: Clinical and laboratory data were extracted retrospectively from the records of patients attending the UK National Ataxia‐Telangiectasia Clinic. Neurological assessments were performed using the A‐T Index (Crawford Score) and the A‐T Neurological Examination Scale Toolkit (A‐T NEST). Variables influencing phenotype were identified by using an information‐theoretic approach starting from a maximal model to generate estimates of coefficients for each variable. Per‐individual progression was assessed for patients with three or more clinic attendances. Results: The genotype could be determined for 125/135 patients. Crawford and A‐T NEST scores were well correlated. For both scoring systems the estimated coefficients were significantly positive for Age x kinase activity but not Age x protein expression. Unlike the per–genotype analysis, the individual progression of neurological scores in the 34 patients that attended on three or more occasions was not smooth and linear (and in some cases improved over time). Interpretation: Residual kinase activity confers a milder phenotype but there is no difference between kinase‐dead and protein‐null genotypes. The non‐linear progression of individual patients’ neurological scores may reflect biological complexity, day‐to‐day variability, limitations of the assessment methods or a combination of all three.
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spelling nottingham-513412020-05-04T17:58:54Z https://eprints.nottingham.ac.uk/51341/ Longitudinal analysis of the neurological features of ataxia telangiectasia Jackson, Thomas J. Chow, Gabriel Suri, Mohnish Byrd, Philip Taylor, Malcolm R. Whitehouse, William P. Aim: To assess the relationship between genotype and neurological progression in ataxia‐telangiectasia (A‐T). Methods: Clinical and laboratory data were extracted retrospectively from the records of patients attending the UK National Ataxia‐Telangiectasia Clinic. Neurological assessments were performed using the A‐T Index (Crawford Score) and the A‐T Neurological Examination Scale Toolkit (A‐T NEST). Variables influencing phenotype were identified by using an information‐theoretic approach starting from a maximal model to generate estimates of coefficients for each variable. Per‐individual progression was assessed for patients with three or more clinic attendances. Results: The genotype could be determined for 125/135 patients. Crawford and A‐T NEST scores were well correlated. For both scoring systems the estimated coefficients were significantly positive for Age x kinase activity but not Age x protein expression. Unlike the per–genotype analysis, the individual progression of neurological scores in the 34 patients that attended on three or more occasions was not smooth and linear (and in some cases improved over time). Interpretation: Residual kinase activity confers a milder phenotype but there is no difference between kinase‐dead and protein‐null genotypes. The non‐linear progression of individual patients’ neurological scores may reflect biological complexity, day‐to‐day variability, limitations of the assessment methods or a combination of all three. Wiley 2016-07-31 Article PeerReviewed Jackson, Thomas J., Chow, Gabriel, Suri, Mohnish, Byrd, Philip, Taylor, Malcolm R. and Whitehouse, William P. (2016) Longitudinal analysis of the neurological features of ataxia telangiectasia. Developmental Medicine and Child Neurology, 58 (7). pp. 690-697. ISSN 0012-1622 https://onlinelibrary.wiley.com/doi/abs/10.1111/dmcn.13052 doi:10.1111/dmcn.13052 doi:10.1111/dmcn.13052
spellingShingle Jackson, Thomas J.
Chow, Gabriel
Suri, Mohnish
Byrd, Philip
Taylor, Malcolm R.
Whitehouse, William P.
Longitudinal analysis of the neurological features of ataxia telangiectasia
title Longitudinal analysis of the neurological features of ataxia telangiectasia
title_full Longitudinal analysis of the neurological features of ataxia telangiectasia
title_fullStr Longitudinal analysis of the neurological features of ataxia telangiectasia
title_full_unstemmed Longitudinal analysis of the neurological features of ataxia telangiectasia
title_short Longitudinal analysis of the neurological features of ataxia telangiectasia
title_sort longitudinal analysis of the neurological features of ataxia telangiectasia
url https://eprints.nottingham.ac.uk/51341/
https://eprints.nottingham.ac.uk/51341/
https://eprints.nottingham.ac.uk/51341/