Methotrexate as a Steroid-sparing Agent in Idiopathic Retroperitoneal Fibrosis: a Randomised, Multicenter Trial

Summary

Chronic periaortitis is a clinico-pathological entity encompassing idiopathic retroperitoneal fibrosis and perianeurysmal retroperitoneal fibrosis. The treatment of this disease is generally based on the use of glucocorticoids, which are often effective. However, prolonged steroid treatments are usually needed to achieve a sustained remission; additionally, patients frequently develop disease relapses following treatment discontinuation, therefore they may be exposed to high cumulative doses of glucocorticoids. Preliminary data reported in the literature show that methotrexate may be effective in combination with prednisone for retroperitoneal fibrosis. In addition, methotrexate is often used as a steroid-sparing agent in different inflammatory diseases. The aim of this study is to evaluate whether a treatment with low-dose prednisone plus methotrexate is non-inferior to conventional dose-prednisone in achieving remission in retroperitoneal fibrosis patients.

Timeline

Start

2007-05

Primary completion

2012-05

Completion

2013-12

Outcome

Met primary endpoint

paper The difference between remission rates was 9.1 % (95 %CI -9.9 %-27.3 %), meeting the criterion for non-inferiority. PMID 40974866 ↗

Publications

• Background van Bommel EF, Siemes C, Hak LE, van der Veer SJ, Hendriksz TR. Long-term renal and patient outcome in idiopathic retroperitoneal fibrosis treated with prednisone. Am J Kidney Dis. 2007 May;49(5):615-25. doi: 10.1053/j.ajkd.2007.02.268.
• Background Vaglio A, Palmisano A, Corradi D, Salvarani C, Buzio C. Retroperitoneal fibrosis: evolving concepts. Rheum Dis Clin North Am. 2007 Nov;33(4):803-17, vi-vii. doi: 10.1016/j.rdc.2007.07.013.
• Background Vaglio A, Salvarani C, Buzio C. Retroperitoneal fibrosis. Lancet. 2006 Jan 21;367(9506):241-51. doi: 10.1016/S0140-6736(06)68035-5.
• Background Warnatz K, Keskin AG, Uhl M, Scholz C, Katzenwadel A, Vaith P, Peter HH, Walker UA. Immunosuppressive treatment of chronic periaortitis: a retrospective study of 20 patients with chronic periaortitis and a review of the literature. Ann Rheum Dis. 2005 Jun;64(6):828-33. doi: 10.1136/ard.2004.029793.
• Peyronel F, Palmisano A, Maritati F, Alberici F, Urban ML, Gianfreda D, Rossi GM, Fenaroli P, Bettiol A, Moroni G, Vaglio A. Methotrexate and low-dose prednisone in idiopathic retroperitoneal fibrosis: a randomised clinical trial. J Autoimmun. 2025 Dec;157:103487. doi: 10.1016/j.jaut.2025.103487. Epub 2025 Sep 19.

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