Rituximab use in pediatric central demyelinating disease

Pediatr Neurol. 2014 Jul;51(1):114-8. doi: 10.1016/j.pediatrneurol.2014.02.007. Epub 2014 Feb 15.


Background: Rituximab is a B-cell therapy used off-label to reduce relapses in adult demyelinating diseases. There is limited knowledge of its clinical use in pediatric neuromyelitis optica and multiple sclerosis. Demyelinating diseases in children can have high morbidity, and B-cell therapies hold promise for those with a severe course. Our study investigates the clinical experience of safety and efficacy with rituximab in children with demyelinating diseases of the central nervous system.

Methods: This is a retrospective case series of 11 patients with pediatric neuromyelitis optica and multiple sclerosis who received at least one rituximab infusion at the Pediatric Multiple Sclerosis Clinic, University of California, San Francisco. Each patient was infused up to 1000 mg twice 2 weeks apart. Patients were monitored prospectively, and relapse events, laboratories, and adverse reactions were recorded.

Results: Eight children with neuromyelitis optica, two with relapsing-remitting multiple sclerosis and one with secondary-progressive multiple sclerosis received rituximab treatment. The median number of cycles was 3. Most patients (82%, n = 9) experienced reduction of relapses after initiating rituximab. There were no serious infections. Infusion reactions were reported in three patients and managed successfully in subsequent infusions with increased pretreatment (dexamethasone and diphenhydramine) and use of slower infusion rates. Rituximab was not discontinued in any child because of side effects; two switched treatment therapy after 4.5 and 11 months because of relapses.

Conclusions: The use of rituximab in our pediatric neuromyelitis optica and multiple sclerosis cohort was overall safe and effective. Larger studies should confirm our observations.

Keywords: demyelinating diseases; devic disease; multiple sclerosis; neuromyelitis optica; rituximab; treatment.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antibodies / blood
  • Antibodies, Monoclonal, Murine-Derived / therapeutic use*
  • Aquaporin 4 / immunology
  • Child
  • Child, Preschool
  • Demyelinating Autoimmune Diseases, CNS / blood
  • Demyelinating Autoimmune Diseases, CNS / drug therapy*
  • Female
  • Humans
  • Immunologic Factors / therapeutic use*
  • Male
  • Retrospective Studies
  • Rituximab
  • Treatment Outcome
  • Young Adult


  • AQP4 protein, human
  • Antibodies
  • Antibodies, Monoclonal, Murine-Derived
  • Aquaporin 4
  • Immunologic Factors
  • Rituximab