Introduction and Aims: Focal segmental glomerulosclerosis (FSGS) is one of the most common causes of nephrotic syndrome (NS) in children and adults. It often displays a complicated course due to resistance to steroids and other immunosuppressants. The anti-CD20 monoclonal antibody Rituximab (RTX) has been suggested in the therapy of difficult-to-treat NS secondary to minimal-change disease and FSGS; however the evidence of efficacy in FSGS is actually still poor.
Methods: We reviewed our experience in FSGS patients treated with RTX as rescue therapy in our Department in the time-interval 2009-2014. We identified 5 male patients affected by long-lasting biopsy-proven FSGS, with NS resistant to steroids and calcineurin inhibitors (persistence of NS). The mean age of patients at nephropathy diagnosis was 26.2 (2- 38) years. Patients received a RTX-based rescue therapy schedule (1 gram, twice, in 2 weeks). Improvement in proteinuria and glomerular filtration rate (GFR) were considered as outcomes of our evaluation.
Results: Mean follow-up (FU) time was 18.8 (9-29) months. At the end of FU, only 1 patient (the one with the highest GFR) showed a complete remission (urinary protein excretion < 0.3 g/day), whereas in 4 patients NS persisted. GFR increased in 2/5 patients (+ 11.7 and + 10.7 ml/min respectively) (Table 1). No correlation was found between GFR at T0 and ∆ proteinuria (T0 - FUend) or GFR at FUend. No adverse events to RTX were reported and the drug was well tolerated.
Conclusions: In our experience RTX treatment for resistant FSGS in adult patients showed controversial results. Nevertheless, we think RTX should be considered in any case as rescue therapy since the prognosis of patients with this clinical picture would be really poor without any other therapeutic option. The observation of a very good response in proteinuria in the patient with the highest GFR at baseline could suggest that RTX therapy in difficult-to-treat FSGS should not be delayed and administered as far as renal function is still preserved.
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