Long-term efficacy and tolerability of mycophenolate mofetil therapy in diffuse scleroderma skin disease

Daniel Boulos, Gene-Siew Ngian, Anton Rajadurai, Kathleen Elford, Wendy Stevens, Susanna M Proudman, Claire Owen, Janet Roddy, Mandana Nikpour, Peter Paul Youssef, Catherine Hill, Joanne Sahhar

Research output: Contribution to journalArticleResearchpeer-review

Abstract

Objectives: To assess the long-term efficacy and tolerability of mycophenolate mofetil (MMF) in patients with diffuse cutaneous systemic sclerosis (dcSSc). Methods: Patients enrolled in the Australian Scleroderma Cohort study with dcSSc and baseline modified Rodnan skin score (mRSS) ≥ 12 who were treated for a minimum of 12 months with MMF for the primary indication of skin disease were included and their prospectively collected data retrieved. Change in mRSS, the proportion with a clinically significant improvement (reduction in mRSS ≥ 5 from baseline) and adverse effects due to therapy were determined. Results: Seventy-four participants treated with MMF were identified and of these, 42 met inclusion criteria. The mean age was 53 ± 12 years, with mean disease duration at MMF commencement of 4.8 ± 4.3 years. Twenty-one participants (50%) commenced MMF within 2 years of disease onset and the mean duration of therapy was 2.7 ± 1.7 years. The mean mRSS at baseline was 25.9 ± 9.2 with a reduction of 3.7 ± 7.1 (P = 0.07) after 1 year of therapy, 7.6 ± 8.3 after 2 years (P = 0.01) and 10.5 ± 10.3 after 5 years (P < 0.01). Response to treatment was not affected by disease duration at MMF commencement or baseline skin score. Eighteen participants (43%) demonstrated clinically significant improvement after 1 year, increasing to 92% after 4 years. Two participants (5%) ceased MMF due to adverse effects. Conclusion: MMF was associated with a modest improvement in mRSS and was well tolerated in the treatment of dcSSc. Given the natural history of dcSSc where skin involvement can spontaneously improve, randomized, placebo-controlled studies are required to confirm whether improvement can be attributed to MMF therapy.

Original languageEnglish
Pages (from-to)481-488
Number of pages8
JournalInternational Journal of Rheumatic Diseases
Volume20
Issue number4
DOIs
Publication statusPublished - 1 Apr 2017

Keywords

  • diffuse
  • mycophenolate mofetil
  • scleroderma
  • systemic sclerosis

Cite this

Boulos, Daniel ; Ngian, Gene-Siew ; Rajadurai, Anton ; Elford, Kathleen ; Stevens, Wendy ; Proudman, Susanna M ; Owen, Claire ; Roddy, Janet ; Nikpour, Mandana ; Youssef, Peter Paul ; Hill, Catherine ; Sahhar, Joanne. / Long-term efficacy and tolerability of mycophenolate mofetil therapy in diffuse scleroderma skin disease. In: International Journal of Rheumatic Diseases. 2017 ; Vol. 20, No. 4. pp. 481-488.
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title = "Long-term efficacy and tolerability of mycophenolate mofetil therapy in diffuse scleroderma skin disease",
abstract = "Objectives: To assess the long-term efficacy and tolerability of mycophenolate mofetil (MMF) in patients with diffuse cutaneous systemic sclerosis (dcSSc). Methods: Patients enrolled in the Australian Scleroderma Cohort study with dcSSc and baseline modified Rodnan skin score (mRSS) ≥ 12 who were treated for a minimum of 12 months with MMF for the primary indication of skin disease were included and their prospectively collected data retrieved. Change in mRSS, the proportion with a clinically significant improvement (reduction in mRSS ≥ 5 from baseline) and adverse effects due to therapy were determined. Results: Seventy-four participants treated with MMF were identified and of these, 42 met inclusion criteria. The mean age was 53 ± 12 years, with mean disease duration at MMF commencement of 4.8 ± 4.3 years. Twenty-one participants (50{\%}) commenced MMF within 2 years of disease onset and the mean duration of therapy was 2.7 ± 1.7 years. The mean mRSS at baseline was 25.9 ± 9.2 with a reduction of 3.7 ± 7.1 (P = 0.07) after 1 year of therapy, 7.6 ± 8.3 after 2 years (P = 0.01) and 10.5 ± 10.3 after 5 years (P < 0.01). Response to treatment was not affected by disease duration at MMF commencement or baseline skin score. Eighteen participants (43{\%}) demonstrated clinically significant improvement after 1 year, increasing to 92{\%} after 4 years. Two participants (5{\%}) ceased MMF due to adverse effects. Conclusion: MMF was associated with a modest improvement in mRSS and was well tolerated in the treatment of dcSSc. Given the natural history of dcSSc where skin involvement can spontaneously improve, randomized, placebo-controlled studies are required to confirm whether improvement can be attributed to MMF therapy.",
keywords = "diffuse, mycophenolate mofetil, scleroderma, systemic sclerosis",
author = "Daniel Boulos and Gene-Siew Ngian and Anton Rajadurai and Kathleen Elford and Wendy Stevens and Proudman, {Susanna M} and Claire Owen and Janet Roddy and Mandana Nikpour and Youssef, {Peter Paul} and Catherine Hill and Joanne Sahhar",
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Long-term efficacy and tolerability of mycophenolate mofetil therapy in diffuse scleroderma skin disease. / Boulos, Daniel; Ngian, Gene-Siew; Rajadurai, Anton; Elford, Kathleen; Stevens, Wendy; Proudman, Susanna M; Owen, Claire; Roddy, Janet; Nikpour, Mandana; Youssef, Peter Paul; Hill, Catherine; Sahhar, Joanne.

In: International Journal of Rheumatic Diseases, Vol. 20, No. 4, 01.04.2017, p. 481-488.

Research output: Contribution to journalArticleResearchpeer-review

TY - JOUR

T1 - Long-term efficacy and tolerability of mycophenolate mofetil therapy in diffuse scleroderma skin disease

AU - Boulos, Daniel

AU - Ngian, Gene-Siew

AU - Rajadurai, Anton

AU - Elford, Kathleen

AU - Stevens, Wendy

AU - Proudman, Susanna M

AU - Owen, Claire

AU - Roddy, Janet

AU - Nikpour, Mandana

AU - Youssef, Peter Paul

AU - Hill, Catherine

AU - Sahhar, Joanne

PY - 2017/4/1

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N2 - Objectives: To assess the long-term efficacy and tolerability of mycophenolate mofetil (MMF) in patients with diffuse cutaneous systemic sclerosis (dcSSc). Methods: Patients enrolled in the Australian Scleroderma Cohort study with dcSSc and baseline modified Rodnan skin score (mRSS) ≥ 12 who were treated for a minimum of 12 months with MMF for the primary indication of skin disease were included and their prospectively collected data retrieved. Change in mRSS, the proportion with a clinically significant improvement (reduction in mRSS ≥ 5 from baseline) and adverse effects due to therapy were determined. Results: Seventy-four participants treated with MMF were identified and of these, 42 met inclusion criteria. The mean age was 53 ± 12 years, with mean disease duration at MMF commencement of 4.8 ± 4.3 years. Twenty-one participants (50%) commenced MMF within 2 years of disease onset and the mean duration of therapy was 2.7 ± 1.7 years. The mean mRSS at baseline was 25.9 ± 9.2 with a reduction of 3.7 ± 7.1 (P = 0.07) after 1 year of therapy, 7.6 ± 8.3 after 2 years (P = 0.01) and 10.5 ± 10.3 after 5 years (P < 0.01). Response to treatment was not affected by disease duration at MMF commencement or baseline skin score. Eighteen participants (43%) demonstrated clinically significant improvement after 1 year, increasing to 92% after 4 years. Two participants (5%) ceased MMF due to adverse effects. Conclusion: MMF was associated with a modest improvement in mRSS and was well tolerated in the treatment of dcSSc. Given the natural history of dcSSc where skin involvement can spontaneously improve, randomized, placebo-controlled studies are required to confirm whether improvement can be attributed to MMF therapy.

AB - Objectives: To assess the long-term efficacy and tolerability of mycophenolate mofetil (MMF) in patients with diffuse cutaneous systemic sclerosis (dcSSc). Methods: Patients enrolled in the Australian Scleroderma Cohort study with dcSSc and baseline modified Rodnan skin score (mRSS) ≥ 12 who were treated for a minimum of 12 months with MMF for the primary indication of skin disease were included and their prospectively collected data retrieved. Change in mRSS, the proportion with a clinically significant improvement (reduction in mRSS ≥ 5 from baseline) and adverse effects due to therapy were determined. Results: Seventy-four participants treated with MMF were identified and of these, 42 met inclusion criteria. The mean age was 53 ± 12 years, with mean disease duration at MMF commencement of 4.8 ± 4.3 years. Twenty-one participants (50%) commenced MMF within 2 years of disease onset and the mean duration of therapy was 2.7 ± 1.7 years. The mean mRSS at baseline was 25.9 ± 9.2 with a reduction of 3.7 ± 7.1 (P = 0.07) after 1 year of therapy, 7.6 ± 8.3 after 2 years (P = 0.01) and 10.5 ± 10.3 after 5 years (P < 0.01). Response to treatment was not affected by disease duration at MMF commencement or baseline skin score. Eighteen participants (43%) demonstrated clinically significant improvement after 1 year, increasing to 92% after 4 years. Two participants (5%) ceased MMF due to adverse effects. Conclusion: MMF was associated with a modest improvement in mRSS and was well tolerated in the treatment of dcSSc. Given the natural history of dcSSc where skin involvement can spontaneously improve, randomized, placebo-controlled studies are required to confirm whether improvement can be attributed to MMF therapy.

KW - diffuse

KW - mycophenolate mofetil

KW - scleroderma

KW - systemic sclerosis

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