What is the role of monoclonal antibodies in the treatment of pediatric nephrotic syndrome?

Updated: Mar 04, 2020
  • Author: Jerome C Lane, MD; Chief Editor: Craig B Langman, MD  more...
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Ofatumumab (OFA), a new humanized anti-CD20 antibody that depletes B cells in a similar manner to rituximab, is currently under investigation for the treatment of childhood INS.

In one study, 4 of 5 children with rituximab-resistant SRNS achieved complete remission over 12 months of follow-up after treatment with OFA (300 mg/1.73 m2 for the first week, followed by 5 weekly infusions [2 g/1.73 m2 each]). [97]

In another study, 4 children with SRNS unresponsive to multiple drugs, including rituximab, were treated with OFA (300 + 700 mg/1.73 m2, 2 weeks apart). One patient achieved stable remission at 12 months of follow-up, whereas another child had only transient remittance of proteinuria; the other 2 children had no response. [98] In a later study, the same group treated 2 patients allergic to rituximab and induced stable remission after 12 months of follow-up following treatment with a single dose (750 mg/1.73 m2) of OFA. [99]  

Wang et al treated 5 childhood INS patients (4 with SRNS and one with post-transplant recurrence of FSGS) with OFA (300 mg/1.73 m2 for the first week, followed by 4 or 5 weekly infusions of 2 g/1.73 m2). Three patients achieved complete remission, and 1 patient had partial remission. [100]

A double-blind, randomized, controlled superiority trial is underway to compare OFA with rituximab in children who have SDNS and CNI-dependent NS. [101]

Other biologic therapies that have been investigated include the following:

  • Abatacept (an inhibitor of the T-cell costimulatory molecule B7-1 [CD80])
  • Adalimumab (a monoclonal antibody against tumor necrosis factor alpha)
  • Fresolimumab (a monoclonal antibody against transforming growth factor beta)

There are no data to support the efficacy or use of these agents at this time. [102]

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