Longitudinal study to assess the quantitative use of fundus autofluorescence for monitoring disease progression in choroideremia
<strong>Background:</strong> Characterisation of preserved autofluorescence (PAF) area in choroideremia (CHM) and its validity for monitoring disease progression in clinical trials is of importance. <strong>Methods:</strong> Eighty patients with molecularly confirmed CHM wer...
| Main Authors: | , , , , , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
MDPI
2021
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| _version_ | 1826306553509052416 |
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| author | Dubis, AM Lim, WS Jolly, JK Toms, M MacLaren, RE Webster, AR Moosajee, M |
| author_facet | Dubis, AM Lim, WS Jolly, JK Toms, M MacLaren, RE Webster, AR Moosajee, M |
| author_sort | Dubis, AM |
| collection | OXFORD |
| description | <strong>Background:</strong> Characterisation of preserved autofluorescence (PAF) area in choroideremia (CHM) and its validity for monitoring disease progression in clinical trials is of importance.
<strong>Methods:</strong> Eighty patients with molecularly confirmed CHM were recruited. PAF area was measured manually by 2 graders and half-life was calculated based on exponential decay model.
<strong>Results:</strong> Mean age at baseline and follow-up examination was 38.1 (range, 10–69) and 40.7 (range, 11–70) years. Mean follow-up interval was 29 months (range, 6–104). The median LogMAR visual acuity was 0.10 (OD) and 0.18 (OS). Interobserver repeatability for PAF area was −0.99 to 1.03 mm2 (−6.46 to 6.49% of area). There was a statistically significant relationship between age and rate of PAF area loss (r2 = 0.28, p = 0.012). The half-life for PAF area was 13.7 years (range, 1.7–216.0 years). The correlation between half-life and age was stronger than between half-life and log transformed baseline PAF area, although neither was statistically significant.
<strong>Conclusions:</strong> The intra- and inter-observer PAF area measurement variability provides a baseline change, which must be overcome in a clinical trial if this metric were to be used. Treatments must slow progression to alter the exponential decay in a timely manner accounting for naturally slow progression patterns. |
| first_indexed | 2024-03-07T06:49:41Z |
| format | Journal article |
| id | oxford-uuid:fc1c3cea-7eb5-4dff-858c-bdddb2c254cd |
| institution | University of Oxford |
| language | English |
| last_indexed | 2024-03-07T06:49:41Z |
| publishDate | 2021 |
| publisher | MDPI |
| record_format | dspace |
| spelling | oxford-uuid:fc1c3cea-7eb5-4dff-858c-bdddb2c254cd2022-03-27T13:18:30ZLongitudinal study to assess the quantitative use of fundus autofluorescence for monitoring disease progression in choroideremiaJournal articlehttp://purl.org/coar/resource_type/c_dcae04bcuuid:fc1c3cea-7eb5-4dff-858c-bdddb2c254cdEnglishSymplectic ElementsMDPI2021Dubis, AMLim, WSJolly, JKToms, MMacLaren, REWebster, ARMoosajee, M<strong>Background:</strong> Characterisation of preserved autofluorescence (PAF) area in choroideremia (CHM) and its validity for monitoring disease progression in clinical trials is of importance. <strong>Methods:</strong> Eighty patients with molecularly confirmed CHM were recruited. PAF area was measured manually by 2 graders and half-life was calculated based on exponential decay model. <strong>Results:</strong> Mean age at baseline and follow-up examination was 38.1 (range, 10–69) and 40.7 (range, 11–70) years. Mean follow-up interval was 29 months (range, 6–104). The median LogMAR visual acuity was 0.10 (OD) and 0.18 (OS). Interobserver repeatability for PAF area was −0.99 to 1.03 mm2 (−6.46 to 6.49% of area). There was a statistically significant relationship between age and rate of PAF area loss (r2 = 0.28, p = 0.012). The half-life for PAF area was 13.7 years (range, 1.7–216.0 years). The correlation between half-life and age was stronger than between half-life and log transformed baseline PAF area, although neither was statistically significant. <strong>Conclusions:</strong> The intra- and inter-observer PAF area measurement variability provides a baseline change, which must be overcome in a clinical trial if this metric were to be used. Treatments must slow progression to alter the exponential decay in a timely manner accounting for naturally slow progression patterns. |
| spellingShingle | Dubis, AM Lim, WS Jolly, JK Toms, M MacLaren, RE Webster, AR Moosajee, M Longitudinal study to assess the quantitative use of fundus autofluorescence for monitoring disease progression in choroideremia |
| title | Longitudinal study to assess the quantitative use of fundus autofluorescence for monitoring disease progression in choroideremia |
| title_full | Longitudinal study to assess the quantitative use of fundus autofluorescence for monitoring disease progression in choroideremia |
| title_fullStr | Longitudinal study to assess the quantitative use of fundus autofluorescence for monitoring disease progression in choroideremia |
| title_full_unstemmed | Longitudinal study to assess the quantitative use of fundus autofluorescence for monitoring disease progression in choroideremia |
| title_short | Longitudinal study to assess the quantitative use of fundus autofluorescence for monitoring disease progression in choroideremia |
| title_sort | longitudinal study to assess the quantitative use of fundus autofluorescence for monitoring disease progression in choroideremia |
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