The therapy of this disease requires an integrated approach of medical and surgical therapies. The medical therapy includes an empirical therapy (corticosteroids, tamoxifen and azathioprine) and an experimental therapy (azathioprine, cyclophosphamide, mycophenolatemofetil, cyclosporin, medroxyprogesterone acetate, and progesterone). Glucocorticoids and azathioprine are most useful in patients with signs of inflammation. Temporizing maneuvers in the form of percutaneous nephrostomy or ureteral stenting are recomended in the presence of obstructive uropathy.
About the medical therapy of retroperitoneal fibrosis exist only one randomized trial from our Parma Group (Vaglio et al. Lancet. 2011 Jul 23;378(9788):338-46. doi: 10.1016/S0140-6736(11)60934-3.) showing that Prednisone is more effective in prevention of relapses than is tamoxifen in patients with idiopathic retroperitoneal fibrosis. Therefore, prednisone should be considered as first-line treatment for patients with newly diagnosed idiopathic retroperitoneal fibrosis. Recently we had some interesting experience with Rituximab (Maritati et al.,Ann Rheum Dis. 2012 Jul;71(7):1262-4)
IRPF falls under the newly termed disease entity of IgG4-related diseases. Therefore immunosuppressive drugs make sense. Our experience is that steroids and azathioprine. Is most effective. We tend to avoid colchicine of the GFR is low due to risk of a neuromyopathy.
In the past, Treatment used to focus on relieving the obstruction with percutaneous or cystoscopic assisted placement of ureteral stents followed by more conclusive resolution of ureteric obstruction with open or laparoscopic ureterolysis.
primarily a surgical approach followed by an immunosuppressive-based therapy( METHYLEPREDNISOLONE, AZATHIOPRINE, AND PENICILLAMINE etc) could be a better option.
One of the problems in treating this disease is the need of long-term therapy with related adverse events. I feel that different drugs may be used (including azathioprine and mycophenolate) but prednisone remains the most effective agent. I am wondering why not to use an mTOR inhibitor (everolimus or sirolimus) as an alternative treatment. These drugs not only exert immunosuppressive effects but can also have anti-fibrotic effects. My experience with everolimus in IRPF is very limited but I would be interested to know if someone else used mTOR inhibibitors in IRPF. The possibility of rotating different drugs may prevent iatrogenic toxicity and increase the efficacy.
The cost of mTOR inhibitors is probably the sole factor. In my country everolimus is about 1000 TIMES more expensive than steroids.
I believe it is now crucial to differentiate idiopathic forms with those linked to IgG4-RD, by systematic biopsies. This will allow in the future to decide the best therapeutic strategies based on the disease's etiology. Actually, prednisone should be considered as first-line treatment for patients with idiopathic retroperitoneal fibrosis. I agree with the Professor Ponticelli: mTOR inibhitors should be considered as third-line therapy in patients unresponsive to mycophenolate or azathioprine. The rationale exists because these immunosoppressive drugs have the proprieties to down-regulated pro-fibrogenic genes in various diseases. Will need further studies to understand their usefulness in retroperitoneal fibrosis.
Some reports of case series include tamoxifen in the treatment scheme of idiopatic retroperitoneal fibrosis. i included this drug with succes in some patients, drug you can follow in yhe long term, in opposition with CE.