Background Retroperitoneal fibrosis (RPF) is a rare disorder characterized by the development of fibrotic tissue in the retroperitoneum involving the abdominal aorta, iliac arteries and ureters. Erdheim-Chester disease (ECD) is a rare, non-Langerhans histiocytosis. Symptoms of ECD often resemble retroperitoneal fibrosis.
Objectives Evaluation of standard treatment efficacy in 13 consecutive RPF patients. Revision of the diagnosis in patients unresponsive to standard therapy.
Methods A retrospective analysis of 13 consecutive RPF patients (10 males and 3 females) treated in our department since 2008 was performed. All patients were treated with tapered dose of steroids combined with an immunosuppressive agent (cyclophosphamide in 4, azathioprine in 9, methotrexate in 6, hydroxychloroquine in 5 patients). Urologic interventions were undertaken as necessary.
Results The treatment was effective in 10 patients: reduction of the retroperitoneal mass in computed tomography (CT) and normalization of the laboratory tests, including markers of inflammation and creatinine concentration. No improvement was found in 3 patients. However, in a further diagnostic work-up the diagnosis of RPF was maintained only in one of these 3 patients. In the second pateint an ANCA-asssotiated vasculitus was ultimately diagnosed. The third patient was treated with a combination of steroids and cyclophosphamide without any radiological improvement. However, the revision of CTs revealed the presence of changes typical of Erdheim-Chester disease.
Conclusions The standard treatment (based on a combination of steroids and immunosuppressive drugs) is efficient in most partients with RPF. In refractory cases an alternative diagnosis, such as systemic vasculitis or Erdheim-Chester disease, should be taken into account.
Diamond El et al. Consensus guidelines for the diagnosis and clinical management of Erdheim-Chester disease. Blood. 2014 Jul 24;124(4):483–92.
Kermani TA et al. Idiopathic retroperitoneal fibrosis: a retrospective review of clinical presentation, treatment, and outcomes. Mayo Clin Proc. 2011 Apr;86(4):297–303.
Disclosure of Interest None declared