Evolocumab for severe refractory hypercholesterolemia in a child with steroid-resistant nephrotic syndrome and membranous nephropathy.

A 12-year-old girl presented with steroid-resistant nephrotic syndrome and was found on kidney biopsy to have membranous nephropathy with a full-house immunofluorescence pattern. At presentation, she had marked hypercholesterolemia, with an LDL-C level of 589 mg/dL, and a family history suggestive of familial hypercholesterolemia. Multiple immunosuppressive therapies resulted in only transient or insufficient improvement in proteinuria, while conventional lipid-lowering agents failed to control hypercholesterolemia. On hospital day 98, evolocumab was initiated for refractory hypercholesterolemia. LDL-C levels declined rapidly, and proteinuria subsequently improved during the clinical course. Corticosteroids were tapered and discontinued. Evolocumab was administered for three doses, and nephrotic remission was achieved during the subsequent clinical course and maintained for nearly 1 year. This case illustrates that evolocumab may be an effective lipid-lowering option for selected pediatric patients with nephrotic syndrome complicated by severe refractory hypercholesterolemia. The subsequent improvement in proteinuria should be interpreted cautiously as a temporal association.