Evidence-based Management of Rapidly Progressing Systemic Sclerosis

Overview

Journal Best Pract Res Clin Rheumatol

Publisher Elsevier

Specialty Rheumatology

Date 2010 Jun 11

PMID 20534372

Citations 30

Authors

Dinesh Khanna

Christopher P Denton

Affiliations

Soon will be listed here.

Abstract

Systemic sclerosis has the highest case-specific mortality of any of the auto-immune rheumatic diseases, as well as causing major morbidity. It is a major clinical challenge and one that has previously provoked substantial nihilism due to the limited therapeutic options available and the perceived lack of evidence for clinical effectiveness of those treatments that are currently in use. However, this situation is changing; there are emerging data supporting efficacy for some treatment approaches for this patient group together with a growing number of exciting potential novel approaches to treatment that are moving into the clinical arena. Some of the recent clinical trials are reviewed and discussed in detail.

Citing Articles

Does regression of skin thickening predict improvement of internal organ involvement and survival in patients with diffuse cutaneous systemic sclerosis? A EUSTAR analysis.

Wyss A, Jordan S, Graf N, Carreira P, Distler J, Cerinic M Arthritis Res Ther. 2024; 26(1):187.

PMID: 39482761 PMC: 11526720. DOI: 10.1186/s13075-024-03418-2.

The Relationship between Smoking, Raynaud's Phenomenon, Digital Ulcers, and Skin Thickness in the Waikato Systemic Sclerosis Cohort.

Silva C, Solanki K, White D Rheumatol Immunol Res. 2022; 3(2):84-89.

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Patient preferences for the treatment of systemic sclerosis-associated interstitial lung disease: a discrete choice experiment.

Bruni C, Heidenreich S, Duenas A, Hoffmann-Vold A, Gabrielli A, Allanore Y Rheumatology (Oxford). 2022; 61(10):4035-4046.

PMID: 35238334 PMC: 9536797. DOI: 10.1093/rheumatology/keac126.

New risk model is able to identify patients with a low risk of progression in systemic sclerosis.

van Leeuwen N, Maurits M, Liem S, Ciaffi J, Ajmone Marsan N, Ninaber M RMD Open. 2021; 7(2).

PMID: 34059523 PMC: 8169494. DOI: 10.1136/rmdopen-2020-001524.

Association of Anti-Topoisomerase I Antibodies of the IgM Isotype With Disease Progression in Anti-Topoisomerase I-Positive Systemic Sclerosis.

Boonstra M, Bakker J, Grummels A, Ninaber M, Ajmone Marsan N, Wortel C Arthritis Rheumatol. 2020; 72(11):1897-1904.

PMID: 32840062 PMC: 7702063. DOI: 10.1002/art.41403.

References

Mukerjee D, St George D, Coleiro B, Knight C, Denton C, Davar J . Prevalence and outcome in systemic sclerosis associated pulmonary arterial hypertension: application of a registry approach. Ann Rheum Dis. 2003; 62(11):1088-93. PMC: 1754353. DOI: 10.1136/ard.62.11.1088. View

Williams M, E Handler C, Akram R, Smith C, Das C, Smee J . Role of N-terminal brain natriuretic peptide (N-TproBNP) in scleroderma-associated pulmonary arterial hypertension. Eur Heart J. 2006; 27(12):1485-94. DOI: 10.1093/eurheartj/ehi891. View

Khurma V, Meyer C, Park G, McMahon M, Lin J, Singh R . A pilot study of subclinical coronary atherosclerosis in systemic sclerosis: coronary artery calcification in cases and controls. Arthritis Rheum. 2008; 59(4):591-7. DOI: 10.1002/art.23540. View

Penn H, Howie A, Kingdon E, Bunn C, Stratton R, Black C . Scleroderma renal crisis: patient characteristics and long-term outcomes. QJM. 2007; 100(8):485-94. DOI: 10.1093/qjmed/hcm052. View

Clements P, Hurwitz E, Wong W, Seibold J, Mayes M, White B . Skin thickness score as a predictor and correlate of outcome in systemic sclerosis: high-dose versus low-dose penicillamine trial. Arthritis Rheum. 2000; 43(11):2445-54. DOI: 10.1002/1529-0131(200011)43:11<2445::AID-ANR11>3.0.CO;2-Q. View

Denton C, Merkel P, Furst D, Khanna D, Emery P, Hsu V . Recombinant human anti-transforming growth factor beta1 antibody therapy in systemic sclerosis: a multicenter, randomized, placebo-controlled phase I/II trial of CAT-192. Arthritis Rheum. 2006; 56(1):323-33. DOI: 10.1002/art.22289. View

Khanna D, Merkel P . Outcome measures in systemic sclerosis: an update on instruments and current research. Curr Rheumatol Rep. 2007; 9(2):151-7. DOI: 10.1007/s11926-007-0010-5. View

Robertson L, Pignone A, Kowal-Bielecka O, Fiori G, Denton C, Matucci-Cerinic M . Pulmonary arterial hypertension in systemic sclerosis: diagnostic pathway and therapeutic approach. Ann Rheum Dis. 2005; 64(6):804-7. PMC: 1755513. DOI: 10.1136/ard.2004.026427. View

Daniels C, Wilkes M, Edens M, Kottom T, Murphy S, Limper A . Imatinib mesylate inhibits the profibrogenic activity of TGF-beta and prevents bleomycin-mediated lung fibrosis. J Clin Invest. 2004; 114(9):1308-16. PMC: 524221. DOI: 10.1172/JCI19603. View

10.

Tashkin D, Elashoff R, Clements P, Goldin J, Roth M, Furst D . Cyclophosphamide versus placebo in scleroderma lung disease. N Engl J Med. 2006; 354(25):2655-66. DOI: 10.1056/NEJMoa055120. View

11.

Korn J, Mayes M, Cerinic M, Rainisio M, Pope J, Hachulla E . Digital ulcers in systemic sclerosis: prevention by treatment with bosentan, an oral endothelin receptor antagonist. Arthritis Rheum. 2004; 50(12):3985-93. DOI: 10.1002/art.20676. View

12.

Seibold J, Korn J, Simms R, Clements P, Moreland L, Mayes M . Recombinant human relaxin in the treatment of scleroderma. A randomized, double-blind, placebo-controlled trial. Ann Intern Med. 2000; 132(11):871-9. DOI: 10.7326/0003-4819-132-11-200006060-00004. View

13.

Herrick A, Lunt M, Whidby N, Ennis H, Silman A, McHugh N . Observational study of treatment outcome in early diffuse cutaneous systemic sclerosis. J Rheumatol. 2009; 37(1):116-24. DOI: 10.3899/jrheum.090668. View

14.

Khanna D, Yan X, Tashkin D, Furst D, Elashoff R, Roth M . Impact of oral cyclophosphamide on health-related quality of life in patients with active scleroderma lung disease: results from the scleroderma lung study. Arthritis Rheum. 2007; 56(5):1676-84. DOI: 10.1002/art.22580. View

15.

Gerbino A, Goss C, Molitor J . Effect of mycophenolate mofetil on pulmonary function in scleroderma-associated interstitial lung disease. Chest. 2007; 133(2):455-60. DOI: 10.1378/chest.06-2861. View

16.

Denton C, Humbert M, Rubin L, Black C . Bosentan treatment for pulmonary arterial hypertension related to connective tissue disease: a subgroup analysis of the pivotal clinical trials and their open-label extensions. Ann Rheum Dis. 2006; 65(10):1336-40. PMC: 1798307. DOI: 10.1136/ard.2005.048967. View

17.

Condliffe R, Kiely D, Peacock A, Corris P, Gibbs J, Vrapi F . Connective tissue disease-associated pulmonary arterial hypertension in the modern treatment era. Am J Respir Crit Care Med. 2008; 179(2):151-7. DOI: 10.1164/rccm.200806-953OC. View

18.

Khanna D, Clements P, Furst D, Korn J, Ellman M, Rothfield N . Recombinant human relaxin in the treatment of systemic sclerosis with diffuse cutaneous involvement: a randomized, double-blind, placebo-controlled trial. Arthritis Rheum. 2009; 60(4):1102-11. PMC: 3711466. DOI: 10.1002/art.24380. View

19.

Distler J, Distler O . Imatinib as a novel therapeutic approach for fibrotic disorders. Rheumatology (Oxford). 2008; 48(1):2-4. DOI: 10.1093/rheumatology/ken431. View

20.

Clements P, Lachenbruch P, Seibold J, Zee B, Steen V, Brennan P . Skin thickness score in systemic sclerosis: an assessment of interobserver variability in 3 independent studies. J Rheumatol. 1993; 20(11):1892-6. View