From the Guidelines
Maintenance treatment for retroperitoneal fibrosis is not explicitly addressed in the provided evidence, but based on the context of Erdheim-Chester disease, which can involve retroperitoneal fibrosis, treatment with targeted therapies such as BRAF-inhibitors or MEK-inhibitors may be considered, with maintenance treatment in the setting of metabolic remission with low-dose therapy as tolerated. The provided evidence focuses on Erdheim-Chester disease, a condition that can involve retroperitoneal fibrosis among other manifestations. According to the evidence [ 1 ], for patients with BRAF-V600E ECD who have cardiac/neurologic disease or end-organ dysfunction, BRAF-inhibitor therapy with vemurafenib or dabrafenib should be implemented as first-line therapy.
- Key points to consider in the management of retroperitoneal fibrosis in the context of ECD include:
- The use of systemic corticosteroids, surgery, and radiation therapy may be used to relieve edema or acute symptoms, but are not recommended as monotherapies for ECD [ 1 ].
- For ECD patients without BRAF-V600E and cardiac/neurologic disease or end-organ dysfunction, empiric treatment with MEK-inhibitor should be strongly considered as first-line therapy [ 1 ].
- Optimal duration and dosing of targeted therapies is not known, although relapse has been observed in the majority of cases following complete cessation of BRAF-inhibitors; maintenance treatment in the setting of metabolic remission with low-dose therapy as tolerated may be considered [ 1 ].
- Regular monitoring with full-body FDG-PET-CT and organ-specific imaging is recommended for response assessment and to guide treatment decisions [ 1 ]. Given the complexity and variability of ECD, including its potential to involve retroperitoneal fibrosis, a personalized approach to maintenance treatment, considering the specific characteristics of the disease in each patient, is essential. This may involve collaboration with specialists in rheumatology, oncology, and other relevant fields to optimize patient outcomes.
From the Research
Maintenance Treatment for Retroperitoneal Fibrosis
The maintenance treatment for retroperitoneal fibrosis involves several approaches, including:
- Glucocorticoids, such as prednisone, which are considered the mainstay of treatment 2
- Steroid-sparing agents, like tamoxifen, cyclophosphosphate, or azathioprine, which can be used to reduce the dosage of glucocorticoids and minimize their side effects 3
- Combination therapy with rituximab, cyclophosphamide, and dexamethasone, followed by rituximab and dexamethasone maintenance, which has shown promising results in achieving complete remission 4
- Treatment response may depend on the association with IgG4-related disease, with cases associated with IgG4-RD showing a more dramatic response to steroid therapy 5
Treatment Approaches
Different treatment approaches have been suggested, including:
- Mfficulous glucocorticoid therapy based on that for immunoglobulin G4-related disease 6
- Ureteral obstruction management with conservative procedures, such as ureteral stenting or percutaneous nephrostomy 6
- Aggressive surgical treatment, such as ureterolysis, which can achieve favorable outcomes but is associated with high morbidity 6
- Combination therapies, such as prednisone and tamoxifen, or prednisone and azathioprine, which can be used in cases that do not respond to steroid monotherapy 5