Definitions for the levels of evidence (I–IV) can be found at the end of the "Major Recommendations" field.
Guidelines
No recommendations possible based on Level I or II evidence
Suggestions for Clinical Care
(Suggestions are based on Level III and IV sources)
There have been a number of case series using mycophenolate mofetil in patients with resistant focal segmental glomerulosclerosis (FSGS). Most demonstrate that although mycophenolate mofetil can induce some reduction of proteinuria, complete remission of proteinuria is rare. No data on long-term follow-up evaluation with this drug are currently available.
- One group of authors performed an open-label, 6-month trial of mycophenolate mofetil in 18 patients with biopsy-proven FSGS who were resistant to corticosteroid therapy. Seventy-five percent had also failed to respond to a cytotoxic agent and/or a cyclosporin. A substantial improvement in proteinuria was seen in 44% (8/18) of patients by 6 months. However, no patient achieved complete remission. In addition, relapses were common after therapy was discontinued.
- One group of authors previously reported the use of mycophenolate mofetil in 7 patients, in whom a substantial improvement in proteinuria was also observed.
- One group of authors investigated the effect of mycophenolate mofetil in 7 children with a resistant nephrotic syndrome (6 of whom had minimal change disease and one with FSGS). In this patient, mycophenolate mofetil resulted in complete remission for a follow-up of 28 months.
Other therapies have been used in patients with FSGS who prove resistant to standard treatment:
- Partial remission has been observed in a few case reports using tacrolimus.
- Vincristine has also been used for the treatment of steroid- and cyclophosphamide-resistant nephrotic syndrome. In a series of eight cases presented by one group of authors, two children treated with vincristine achieved complete remission associated with preserved renal function. Another experienced transient relapses. Although studied in primary FSGS, there may be particular advantages of vincristine in secondary forms of nephrotic syndrome associated with malignancy (see Guideline titled "FSGS: cytotoxic therapy" in the original guideline document).
- Plasma exchange, lipid apheresis and immunoadsorption have also been reported to induce remission of proteinuria in selected patients.
Definitions:
Levels of Evidence
Level I: Evidence obtained from a systematic review of all relevant randomized controlled trials (RCTs)
Level II: Evidence obtained from at least one properly designed RCT
Level III: Evidence obtained from well-designed pseudo-randomized controlled trials (alternate allocation or some other method); comparative studies with concurrent controls and allocation not randomized, cohort studies, case-control studies, interrupted time series with a control group; comparative studies with historical control, two or more single arm studies, interrupted time series without a parallel control group
Level IV: Evidence obtained from case series, either post-test or pretest/post-test
