New biologics in the treatment of rare glomerular diseases of childhood

- Anti-CD20 chimeric antibody rituximab has been shown to safely improve natural history of patients with multirelapsing or steroid-dependent nephrotic syndrome.
- Recent evidence supports the use of humanized anti-CD20 antibody ofatumumab to treat children with steroid-resistant nephrotic syndrome refractory to rituximab.
- Available data do not support the use of CTLA4-Ig, anti-TNFα, or anti-TGFβ antibodies to treat nephrotic syndrome in children.

Minimal change disease and focal segmental glomerulosclerosis are rare but important causes of end-stage kidney disease in children. Though their pathogenesis is still unclear, evidence of immune abnormalities provided the background for the use of immunosuppressive drugs, such as corticosteroids, calcineurin inhibitors, antiproliferative and alkylating agents. Unfortunately, these treatments fail to achieve a sustained remission in a significant portion of patients and are burdened by significant toxicities. Recent developments of new biologics, including anti-CD20 monoclonal antibodies rituximab and ofatumumab, offered the opportunity to selectively target immune cell subsets or activation pathways, leading to more effective and safer hypothesis-driven treatments.