How is primary Focal segmental glomerulosclerosis treated?
Angiotensin-converting enzyme (ACE) inhibitor/angiotensin receptor blocker (ARB) therapy has been shown to be beneficial in reducing proteinuria and slowing the decline in kidney function in proteinuric kidney diseases.
There is only one randomized controlled trial of ACE therapy in adults with FSGS and one in children with steroid-resistant nephrotic syndrome. These studies document a reduction in urinary protein excretion by approximately 30%. Long-term data to assess a benefit in preventing a decline in kidney function are not available.
Regardless, ACE inhibitor/ARB therapy is considered standard care for proteinuria control and hypertension management.
Therapy targeted at a presumed immunologic basis of FSGS has been used clinically and in controlled and uncontrolled trials.
Corticosteroids induce remission in 20% to 30% of patients. Treatment may begin with daily dosing and then progress to alternate-day therapy for primary FSGS. The duration of treatment may affect response to therapy with a typical initial course up to 3 months in children and 6 months in adults.
Cyclosporine, tacrolimus, and mycophenolate mofetil have been reported to be effective in case reports. In a few small, randomized trials, calcineurin inhibitors, cyclosporine and tacrolimus, show combined complete and partial remission rates of 50% to 60% in primary FSGS. Mycophenolate appears to be less effective, with a combined complete and partial remission rate of approximately 33%. However, mycophenolate has less kidney toxicity than calcineurin inhibitors.
There are several case reports and small case series of other therapeutic agents. Rituximab, a CD20 monoclonal antibody, adalimumab, a tumor necrosis factor (TNF) inhibitor, adrenocorticotropin hormone with potential action through corticosteroid-associated avenues, as well as melanocortin receptor agonism, and perfenidone as an antifibrotic agent have all been used in the treatment of FSGS.
Plasmapheresis can be used to treat FSGS recurrence after kidney transplant. The mechanism of action is postulated to be removal of circulating factors that increase glomerular permeability.
Therapy for dyslipidemia is also a component of standard therapy in patients with nephrotic syndrome, with a goal to improve nephrotic syndrome–associated dyslipidemia.
Diuretics are indicated for control of edema in patients with nephrotic syndrome. Intravenous albumin infusion with diuretic therapy may be beneficial to control severe edema in select patients with nephrotic syndrome, but it has not been shown to have significant additive benefit in controlled trials and carries with it the risk of pulmonary edema and hypertensive crisis.
