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Systemic sclerosis (scleroderma): remaining challenges
Despite progress in treating internal organ involvement in systemic sclerosis (scleroderma) (SSc), such as pulmonary disease, effective treatments for the hallmark of the disease, cutaneous fibrosis, remain elusive. None of the disease-modifying antirheumatic drugs (DMARDS) have shown proven efficac...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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AME Publishing Company
2021
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8033370/ https://www.ncbi.nlm.nih.gov/pubmed/33842659 http://dx.doi.org/10.21037/atm-20-5449 |
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author | Connolly, Mary Karin |
author_facet | Connolly, Mary Karin |
author_sort | Connolly, Mary Karin |
collection | PubMed |
description | Despite progress in treating internal organ involvement in systemic sclerosis (scleroderma) (SSc), such as pulmonary disease, effective treatments for the hallmark of the disease, cutaneous fibrosis, remain elusive. None of the disease-modifying antirheumatic drugs (DMARDS) have shown proven efficacy for SSc skin fibrosis, and there remain no FDA-approved medications, all of which are off-label, for cutaneous fibrosis in SSc. This review article will briefly summarize conventional therapies, biologics and hematopoietic stem cell transplants and select ongoing clinical trials in SSc. The gold standard for measuring skin fibrosis in SSc is the modified Rodnan skin score (MRSSS). This is a validated test that measures skin thickness (0 to 3) at 17 locations for a total score of 51. Improvements in skin score over time are used in clinical trials to quantitate skin fibrosis. Although recording the Rodnan skin score is technically straightforward, requiring no special equipment, and noninvasive, the fluctuating natural history of the disease includes improvement over time without interventions, rendering meaningful trials difficult to assess. Understanding of the basic molecular mechanisms driving pathologic fibrosis in SSc remains lacking, and underpins the often empiric nature and likely the lack of efficacy of many therapeutics that have been tried. Although repeated skin biopsies might be a more precise way to follow disease progression and regression, this is necessarily invasive and requires special tools. Here, this review will look at conventional therapies, biologics, autologous hematopoietic stem cell transplantation, and catalog some of the ongoing clinical trials in SSc with a focus on cutaneous fibrosis. |
format | Online Article Text |
id | pubmed-8033370 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | AME Publishing Company |
record_format | MEDLINE/PubMed |
spelling | pubmed-80333702021-04-09 Systemic sclerosis (scleroderma): remaining challenges Connolly, Mary Karin Ann Transl Med Review Article on Rheumatologic Skin Disease Despite progress in treating internal organ involvement in systemic sclerosis (scleroderma) (SSc), such as pulmonary disease, effective treatments for the hallmark of the disease, cutaneous fibrosis, remain elusive. None of the disease-modifying antirheumatic drugs (DMARDS) have shown proven efficacy for SSc skin fibrosis, and there remain no FDA-approved medications, all of which are off-label, for cutaneous fibrosis in SSc. This review article will briefly summarize conventional therapies, biologics and hematopoietic stem cell transplants and select ongoing clinical trials in SSc. The gold standard for measuring skin fibrosis in SSc is the modified Rodnan skin score (MRSSS). This is a validated test that measures skin thickness (0 to 3) at 17 locations for a total score of 51. Improvements in skin score over time are used in clinical trials to quantitate skin fibrosis. Although recording the Rodnan skin score is technically straightforward, requiring no special equipment, and noninvasive, the fluctuating natural history of the disease includes improvement over time without interventions, rendering meaningful trials difficult to assess. Understanding of the basic molecular mechanisms driving pathologic fibrosis in SSc remains lacking, and underpins the often empiric nature and likely the lack of efficacy of many therapeutics that have been tried. Although repeated skin biopsies might be a more precise way to follow disease progression and regression, this is necessarily invasive and requires special tools. Here, this review will look at conventional therapies, biologics, autologous hematopoietic stem cell transplantation, and catalog some of the ongoing clinical trials in SSc with a focus on cutaneous fibrosis. AME Publishing Company 2021-03 /pmc/articles/PMC8033370/ /pubmed/33842659 http://dx.doi.org/10.21037/atm-20-5449 Text en 2021 Annals of Translational Medicine. All rights reserved. https://creativecommons.org/licenses/by-nc-nd/4.0/Open Access Statement: This is an Open Access article distributed in accordance with the Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International License (CC BY-NC-ND 4.0), which permits the non-commercial replication and distribution of the article with the strict proviso that no changes or edits are made and the original work is properly cited (including links to both the formal publication through the relevant DOI and the license). See: https://creativecommons.org/licenses/by-nc-nd/4.0 (https://creativecommons.org/licenses/by-nc-nd/4.0/) . |
spellingShingle | Review Article on Rheumatologic Skin Disease Connolly, Mary Karin Systemic sclerosis (scleroderma): remaining challenges |
title | Systemic sclerosis (scleroderma): remaining challenges |
title_full | Systemic sclerosis (scleroderma): remaining challenges |
title_fullStr | Systemic sclerosis (scleroderma): remaining challenges |
title_full_unstemmed | Systemic sclerosis (scleroderma): remaining challenges |
title_short | Systemic sclerosis (scleroderma): remaining challenges |
title_sort | systemic sclerosis (scleroderma): remaining challenges |
topic | Review Article on Rheumatologic Skin Disease |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8033370/ https://www.ncbi.nlm.nih.gov/pubmed/33842659 http://dx.doi.org/10.21037/atm-20-5449 |
work_keys_str_mv | AT connollymarykarin systemicsclerosissclerodermaremainingchallenges |