Comparative Efficacy of Immunosuppressive Therapies in the Treatment of Diffuse Cutaneous Systemic Sclerosis

Objectives The RESOLVE‐1 trial of lenabasum in diffuse cutaneous systemic sclerosis (dcSSc) allowed background immunosuppressive therapy (IST) at the discretion of individual investigators, and no significant differences were observed between treatment arms. This provides a powerful opportunity to c...

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Main Authors: Barbara White, Daniel E. Furst, Tracy M. Frech, Masataka Kuwana, Laura Hummers, Wendy Stevens, Suzanne Kafaja, Eun Bong Lee, Oliver Distler, Dinesh Khanna, Christopher P. Denton, Robert Spiera, for the RESOLVE‐1 investigators
Format: Article
Language:English
Published: Wiley 2025-03-01
Series:ACR Open Rheumatology
Online Access:https://doi.org/10.1002/acr2.70004
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author Barbara White
Daniel E. Furst
Tracy M. Frech
Masataka Kuwana
Laura Hummers
Wendy Stevens
Suzanne Kafaja
Eun Bong Lee
Oliver Distler
Dinesh Khanna
Christopher P. Denton
Robert Spiera
for the RESOLVE‐1 investigators
author_facet Barbara White
Daniel E. Furst
Tracy M. Frech
Masataka Kuwana
Laura Hummers
Wendy Stevens
Suzanne Kafaja
Eun Bong Lee
Oliver Distler
Dinesh Khanna
Christopher P. Denton
Robert Spiera
for the RESOLVE‐1 investigators
author_sort Barbara White
collection DOAJ
description Objectives The RESOLVE‐1 trial of lenabasum in diffuse cutaneous systemic sclerosis (dcSSc) allowed background immunosuppressive therapy (IST) at the discretion of individual investigators, and no significant differences were observed between treatment arms. This provides a powerful opportunity to compare the relative efficacy of different ISTs in a well‐defined large cohort of patients with dcSSc. Methods Prespecified IST categories, efficacy end points, baseline disease characteristics likely to influence efficacy outcomes, the definition of interstitial lung disease, definitions of IST use, and categories of IST use by which efficacy outcomes were evaluated were. Descriptive statistics are used to present results. Results For skin, change in modified Rodnan skin score (mRSS) was numerically greatest with mycophenolate mofetil (MMF) treatment in patients with the earliest disease, reaching −10.8 points in the MMF group versus −4.8 points in the no IST group in patients with a disease duration ≤2 years. Other ISTs had improvements intermediate between that seen in the MMF and no IST groups. Forced vital capacity (mL) was stable over 52 weeks in patients in the MMF group compared to an around 160‐mL decline over 52 weeks in no IST group. Differences in outcome were observed between antinuclear antibody subgroups, with greater difference in favor of MMF for skin and lungs being observed in anti–topoisomerase 1 autoantibody–positive patients. In contrast, anti–RNA polymerase III autoantibody–positive patients in both the no IST and MMF groups improved rapidly, with a decrease in mRSS. Conclusion Taken together, our findings robustly support routine use of MMF in dcSSc and show benefit especially in early‐stage disease. Those patients with high‐risk antibodies for lung fibrosis might be especially suitable for MMF treatment.
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spelling doaj-art-c03aa9d584dd4db7860d35fe19b905122025-08-20T03:03:49ZengWileyACR Open Rheumatology2578-57452025-03-0173n/an/a10.1002/acr2.70004Comparative Efficacy of Immunosuppressive Therapies in the Treatment of Diffuse Cutaneous Systemic SclerosisBarbara White0Daniel E. Furst1Tracy M. Frech2Masataka Kuwana3Laura Hummers4Wendy Stevens5Suzanne Kafaja6Eun Bong Lee7Oliver Distler8Dinesh Khanna9Christopher P. Denton10Robert Spiera11for the RESOLVE‐1 investigatorsCorbus Pharmaceuticals Holdings Inc, Norwood, Massachusetts, USA (current address: SFJ Pharmaceuticals, Inc, Pleasanton, California, USA)University of California Los AngelesVanderbilt University Nashville TennesseeNippon Medical School Tokyo JapanJohns Hopkins University Baltimore MarylandUniversity of Melbourne Melbourne Victoria AustraliaUniversity of California Los AngelesSeoul National University Seoul South KoreaUniversity Hospital Zürich Zürich SwitzerlandUniversity of Michigan Ann ArborUniversity College London London United KingdomHospital for Special Surgery New York New YorkObjectives The RESOLVE‐1 trial of lenabasum in diffuse cutaneous systemic sclerosis (dcSSc) allowed background immunosuppressive therapy (IST) at the discretion of individual investigators, and no significant differences were observed between treatment arms. This provides a powerful opportunity to compare the relative efficacy of different ISTs in a well‐defined large cohort of patients with dcSSc. Methods Prespecified IST categories, efficacy end points, baseline disease characteristics likely to influence efficacy outcomes, the definition of interstitial lung disease, definitions of IST use, and categories of IST use by which efficacy outcomes were evaluated were. Descriptive statistics are used to present results. Results For skin, change in modified Rodnan skin score (mRSS) was numerically greatest with mycophenolate mofetil (MMF) treatment in patients with the earliest disease, reaching −10.8 points in the MMF group versus −4.8 points in the no IST group in patients with a disease duration ≤2 years. Other ISTs had improvements intermediate between that seen in the MMF and no IST groups. Forced vital capacity (mL) was stable over 52 weeks in patients in the MMF group compared to an around 160‐mL decline over 52 weeks in no IST group. Differences in outcome were observed between antinuclear antibody subgroups, with greater difference in favor of MMF for skin and lungs being observed in anti–topoisomerase 1 autoantibody–positive patients. In contrast, anti–RNA polymerase III autoantibody–positive patients in both the no IST and MMF groups improved rapidly, with a decrease in mRSS. Conclusion Taken together, our findings robustly support routine use of MMF in dcSSc and show benefit especially in early‐stage disease. Those patients with high‐risk antibodies for lung fibrosis might be especially suitable for MMF treatment.https://doi.org/10.1002/acr2.70004
spellingShingle Barbara White
Daniel E. Furst
Tracy M. Frech
Masataka Kuwana
Laura Hummers
Wendy Stevens
Suzanne Kafaja
Eun Bong Lee
Oliver Distler
Dinesh Khanna
Christopher P. Denton
Robert Spiera
for the RESOLVE‐1 investigators
Comparative Efficacy of Immunosuppressive Therapies in the Treatment of Diffuse Cutaneous Systemic Sclerosis
ACR Open Rheumatology
title Comparative Efficacy of Immunosuppressive Therapies in the Treatment of Diffuse Cutaneous Systemic Sclerosis
title_full Comparative Efficacy of Immunosuppressive Therapies in the Treatment of Diffuse Cutaneous Systemic Sclerosis
title_fullStr Comparative Efficacy of Immunosuppressive Therapies in the Treatment of Diffuse Cutaneous Systemic Sclerosis
title_full_unstemmed Comparative Efficacy of Immunosuppressive Therapies in the Treatment of Diffuse Cutaneous Systemic Sclerosis
title_short Comparative Efficacy of Immunosuppressive Therapies in the Treatment of Diffuse Cutaneous Systemic Sclerosis
title_sort comparative efficacy of immunosuppressive therapies in the treatment of diffuse cutaneous systemic sclerosis
url https://doi.org/10.1002/acr2.70004
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