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Juvenile Dermatomyositis: what comes next? Long-term outcomes in childhood myositis from a patient perspective

BACKGROUND: To describe long-term outcomes in JDM using patient questionnaires and link to longitudinal, prospectively collected data for each patient within the Juvenile Dermatomyositis Cohort and Biomarker Study, UK and Ireland (JDCBS) to determine outcome predictors.  METHODS: JDCBS participants...

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Autores principales: Boros, C., McCann, L., Simou, S., Cancemi, D., Ambrose, N., Pilkington, C. A., Cortina-Borja, M., Wedderburn, L. R
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9670456/
https://www.ncbi.nlm.nih.gov/pubmed/36384526
http://dx.doi.org/10.1186/s12969-022-00754-y
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author Boros, C.
McCann, L.
Simou, S.
Cancemi, D.
Ambrose, N.
Pilkington, C. A.
Cortina-Borja, M.
Wedderburn, L. R
author_facet Boros, C.
McCann, L.
Simou, S.
Cancemi, D.
Ambrose, N.
Pilkington, C. A.
Cortina-Borja, M.
Wedderburn, L. R
author_sort Boros, C.
collection PubMed
description BACKGROUND: To describe long-term outcomes in JDM using patient questionnaires and link to longitudinal, prospectively collected data for each patient within the Juvenile Dermatomyositis Cohort and Biomarker Study, UK and Ireland (JDCBS) to determine outcome predictors.  METHODS: JDCBS participants aged ≥ 16y completed the SF36, HAQ and a questionnaire regarding current disease features, medications, education and employment. Data collected from the JDCBS included disease subtype, demographics, clinical and laboratory features. Intensity indices were calculated for physician VAS, modified skin DAS, CMAS and MMT8 by dividing area under the curve (AUC) from longitudinal score trajectories by duration of study follow-up (y). Relationships between questionnaire and JDCBS clinical / laboratory data were investigated fitting statistical models appropriate for cross sectional and longitudinal data. RESULTS: Of 190 questionnaires sent, 84 (44%) were returned. Average age of respondents was 20.6 years (SD 3.9), time since diagnosis was 12.4 years (SD 5.0), age at onset was 9.2 years (SD 4.3), female to male ratio 4.25:1. Forty-nine (59%) self-reported persistently active disease, 54 (65%) were still taking immunosuppressive medication. 14/32 at school/higher education reported myositis adversely affecting academic results. 18–24 year-olds were twice as likely to be unemployed compared the UK population (OR = 0.456, 95% CI 0.24, 0.84, p = 0.001). Participants ≥ 18 years were three times as likely to be living with a parent/guardian (OR = 3.39, p < 0.001). SF36 MCS and MMT8 intensity index scores were significantly correlated (ρ = 0.328, p = 0.007). CONCLUSIONS: After 12.4 years, questionnaire responders reported self-perceived high rates of persistently active disease and medication use, reduced rates of employment and were more likely to live with a parent/guardian. Perceived persistently active muscle disease appeared to affect quality of life in these patients and was the most significant contributor to long-term outcomes. Our findings highlight the importance of including the patient perspective in the assessment of long term outcomes, so that that we can start to target initial management strategies more effectively based on a combination of clinical and patient-reported data. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s12969-022-00754-y.
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spelling pubmed-96704562022-11-18 Juvenile Dermatomyositis: what comes next? Long-term outcomes in childhood myositis from a patient perspective Boros, C. McCann, L. Simou, S. Cancemi, D. Ambrose, N. Pilkington, C. A. Cortina-Borja, M. Wedderburn, L. R Pediatr Rheumatol Online J Research Article BACKGROUND: To describe long-term outcomes in JDM using patient questionnaires and link to longitudinal, prospectively collected data for each patient within the Juvenile Dermatomyositis Cohort and Biomarker Study, UK and Ireland (JDCBS) to determine outcome predictors.  METHODS: JDCBS participants aged ≥ 16y completed the SF36, HAQ and a questionnaire regarding current disease features, medications, education and employment. Data collected from the JDCBS included disease subtype, demographics, clinical and laboratory features. Intensity indices were calculated for physician VAS, modified skin DAS, CMAS and MMT8 by dividing area under the curve (AUC) from longitudinal score trajectories by duration of study follow-up (y). Relationships between questionnaire and JDCBS clinical / laboratory data were investigated fitting statistical models appropriate for cross sectional and longitudinal data. RESULTS: Of 190 questionnaires sent, 84 (44%) were returned. Average age of respondents was 20.6 years (SD 3.9), time since diagnosis was 12.4 years (SD 5.0), age at onset was 9.2 years (SD 4.3), female to male ratio 4.25:1. Forty-nine (59%) self-reported persistently active disease, 54 (65%) were still taking immunosuppressive medication. 14/32 at school/higher education reported myositis adversely affecting academic results. 18–24 year-olds were twice as likely to be unemployed compared the UK population (OR = 0.456, 95% CI 0.24, 0.84, p = 0.001). Participants ≥ 18 years were three times as likely to be living with a parent/guardian (OR = 3.39, p < 0.001). SF36 MCS and MMT8 intensity index scores were significantly correlated (ρ = 0.328, p = 0.007). CONCLUSIONS: After 12.4 years, questionnaire responders reported self-perceived high rates of persistently active disease and medication use, reduced rates of employment and were more likely to live with a parent/guardian. Perceived persistently active muscle disease appeared to affect quality of life in these patients and was the most significant contributor to long-term outcomes. Our findings highlight the importance of including the patient perspective in the assessment of long term outcomes, so that that we can start to target initial management strategies more effectively based on a combination of clinical and patient-reported data. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s12969-022-00754-y. BioMed Central 2022-11-16 /pmc/articles/PMC9670456/ /pubmed/36384526 http://dx.doi.org/10.1186/s12969-022-00754-y Text en © The Author(s) 2022 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Research Article
Boros, C.
McCann, L.
Simou, S.
Cancemi, D.
Ambrose, N.
Pilkington, C. A.
Cortina-Borja, M.
Wedderburn, L. R
Juvenile Dermatomyositis: what comes next? Long-term outcomes in childhood myositis from a patient perspective
title Juvenile Dermatomyositis: what comes next? Long-term outcomes in childhood myositis from a patient perspective
title_full Juvenile Dermatomyositis: what comes next? Long-term outcomes in childhood myositis from a patient perspective
title_fullStr Juvenile Dermatomyositis: what comes next? Long-term outcomes in childhood myositis from a patient perspective
title_full_unstemmed Juvenile Dermatomyositis: what comes next? Long-term outcomes in childhood myositis from a patient perspective
title_short Juvenile Dermatomyositis: what comes next? Long-term outcomes in childhood myositis from a patient perspective
title_sort juvenile dermatomyositis: what comes next? long-term outcomes in childhood myositis from a patient perspective
topic Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9670456/
https://www.ncbi.nlm.nih.gov/pubmed/36384526
http://dx.doi.org/10.1186/s12969-022-00754-y
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