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2547 Long-term response to treatment and disease recurrence in a prospective cohort of morphea patients

OBJECTIVES/SPECIFIC AIMS: Morphea (localized scleroderma) is an autoimmune disease characterized that is widely thought to have a monophasic course, in which an initial period of inflammation (activity) ultimately results in scarring, atrophy, and functional impairment (damage). Understanding the lo...

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Autores principales: Florez-Pollack, Stephanie, O’Brien, Jack C., Jacobe, Heidi T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cambridge University Press 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6798764/
http://dx.doi.org/10.1017/cts.2018.170
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author Florez-Pollack, Stephanie
O’Brien, Jack C.
Jacobe, Heidi T.
author_facet Florez-Pollack, Stephanie
O’Brien, Jack C.
Jacobe, Heidi T.
author_sort Florez-Pollack, Stephanie
collection PubMed
description OBJECTIVES/SPECIFIC AIMS: Morphea (localized scleroderma) is an autoimmune disease characterized that is widely thought to have a monophasic course, in which an initial period of inflammation (activity) ultimately results in scarring, atrophy, and functional impairment (damage). Understanding the long-term clinical course of morphea is important for the planning of future interventional studies, and as a tool for clinicians in determining risk for poor disease outcomes. METHODS/STUDY POPULATION: We conducted a prospective cohort study of 130 participants enrolled in the Morphea in Children and Adults Cohort over a median follow-up time of 4.3 years, to determine the rates of response to treatment and disease recurrence as measured by the Localized Scleroderma Cutaneous Assessment Tool (LoSCAT). To determine risk factors for recurrence of disease activity, survival analysis using the log-rank test was used to compare subgroups by morphea type, therapy, and age at disease onset. RESULTS/ANTICIPATED RESULTS: Within a 1-year follow-up period, 66% of patients treated with methotrexate and 46% of patients with UVA1 phototherapy had achieved complete response to treatment. In patients who had achieved response to treatment, 29% experienced disease recurrence at an average of 1.7 years after documented disease inactivity. Patients with generalized morphea experienced higher recurrence rates than those with linear morphea (HR: 3.03, 95% CI: 1.48–6.22), and those treated with UVA1 phototherapy had higher recurrence over those treated with methotrexate (HR: 2.33, 95% CI: 1.03–5.31). In patients with follow-up periods longer than 5 years (n=50), disease recurrence was observed in 44% of patients and the majority of recurrence represented new activity in an area of pre-existing morphea (82%). DISCUSSION/SIGNIFICANCE OF IMPACT: This study highlights the previously under-studied dynamic long-term course of morphea, and identifies the clinical characteristics that predispose patients to having a relapsing-remitting course. We conclude that patients with morphea should be followed closely over time even in the absence of disease activity due to the potential for disease recurrence.
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spelling pubmed-67987642019-10-28 2547 Long-term response to treatment and disease recurrence in a prospective cohort of morphea patients Florez-Pollack, Stephanie O’Brien, Jack C. Jacobe, Heidi T. J Clin Transl Sci Basic/Translational Science/Team Science OBJECTIVES/SPECIFIC AIMS: Morphea (localized scleroderma) is an autoimmune disease characterized that is widely thought to have a monophasic course, in which an initial period of inflammation (activity) ultimately results in scarring, atrophy, and functional impairment (damage). Understanding the long-term clinical course of morphea is important for the planning of future interventional studies, and as a tool for clinicians in determining risk for poor disease outcomes. METHODS/STUDY POPULATION: We conducted a prospective cohort study of 130 participants enrolled in the Morphea in Children and Adults Cohort over a median follow-up time of 4.3 years, to determine the rates of response to treatment and disease recurrence as measured by the Localized Scleroderma Cutaneous Assessment Tool (LoSCAT). To determine risk factors for recurrence of disease activity, survival analysis using the log-rank test was used to compare subgroups by morphea type, therapy, and age at disease onset. RESULTS/ANTICIPATED RESULTS: Within a 1-year follow-up period, 66% of patients treated with methotrexate and 46% of patients with UVA1 phototherapy had achieved complete response to treatment. In patients who had achieved response to treatment, 29% experienced disease recurrence at an average of 1.7 years after documented disease inactivity. Patients with generalized morphea experienced higher recurrence rates than those with linear morphea (HR: 3.03, 95% CI: 1.48–6.22), and those treated with UVA1 phototherapy had higher recurrence over those treated with methotrexate (HR: 2.33, 95% CI: 1.03–5.31). In patients with follow-up periods longer than 5 years (n=50), disease recurrence was observed in 44% of patients and the majority of recurrence represented new activity in an area of pre-existing morphea (82%). DISCUSSION/SIGNIFICANCE OF IMPACT: This study highlights the previously under-studied dynamic long-term course of morphea, and identifies the clinical characteristics that predispose patients to having a relapsing-remitting course. We conclude that patients with morphea should be followed closely over time even in the absence of disease activity due to the potential for disease recurrence. Cambridge University Press 2018-11-21 /pmc/articles/PMC6798764/ http://dx.doi.org/10.1017/cts.2018.170 Text en © The Association for Clinical and Translational Science 2018 http://creativecommons.org/licenses/by/4.0/ This is an Open Access article, distributed under the terms of the Creative Commons Attribution licence (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Basic/Translational Science/Team Science
Florez-Pollack, Stephanie
O’Brien, Jack C.
Jacobe, Heidi T.
2547 Long-term response to treatment and disease recurrence in a prospective cohort of morphea patients
title 2547 Long-term response to treatment and disease recurrence in a prospective cohort of morphea patients
title_full 2547 Long-term response to treatment and disease recurrence in a prospective cohort of morphea patients
title_fullStr 2547 Long-term response to treatment and disease recurrence in a prospective cohort of morphea patients
title_full_unstemmed 2547 Long-term response to treatment and disease recurrence in a prospective cohort of morphea patients
title_short 2547 Long-term response to treatment and disease recurrence in a prospective cohort of morphea patients
title_sort 2547 long-term response to treatment and disease recurrence in a prospective cohort of morphea patients
topic Basic/Translational Science/Team Science
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6798764/
http://dx.doi.org/10.1017/cts.2018.170
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