ClinicSpeak: off-label use of rituximab in children with MS

Off label use of rituximab in children with MS is effective and safe. #OffLabel #ClinicSpeak #MSBlog #MSResearch

"The study below should allow many parents of children with MS sigh with relief. The report describes the off-label use of rituximab, a depleting anti-CD20 antibody, in paediatric-onset MS. There is no surprise that children and adolescents tolerate B cell depletion very well and their disease goes into long-term remission. EDSS scores flat-lined or improved. Importantly, rituximab was safe and well tolerated."

"It is clear that anti-CD20 therapies when given early in both relapsing and progressive MS (PPMS) have an effect on both relapses and focal MRI activity and on disease progression. The real 'killer attribute' of anti-CD20 therapies in MS is that it looks like they won't need blood and/or urine monitoring. Can you imagine starting a treatment that does not need blood monitoring every 2 weeks (teriflunomide), monthly (alemtuzumab), 3  monthly (DMF and possibly fingolimod) or 6 monthly (interferon beta), etc.? When I recently gave my 'Back-to-the-Future' talk at the MS Trust Congress in Windsor and stated that we had to do something amount the monitoring requirements of our DMTs the audience broke out into spontaneous applause. Why? Simply because our MS clinical nurse specialists (CNSs) are sick and tired of doing pharmacovigilance; in short they have become pharmacovigilance nurses. Pharmacovigilance is not what CNSs are trained to do; they want to, and need to, look after pwMS; all people with MS not only those on DMTs. Therefore it is not difficult to see why anti-CD20 therapies (rituximab, ocrelizumab and ofatumumab) are going to become the CNSs' DMT of choice; high-efficacy, infrequent administration, relatively good safety profile and most importantly minimal monitoring."

"Who said the field is not innovating? I still have nagging concerns that anti-CD20 although good news doesn't get to the plasma cell pool within the brains and spinal cords of pwMS. Plasma cells don't express CD20 on their surface and continue to make antibodies for decades. In addition, anti-CD20 therapy is not the complete answer for people with progressive MS; despite it slowing down progression it does not stop it. We therefore have plenty more work to do in the MS DMT space."


Epub: Salzeret al. Rituximab in paediatric onset multiple sclerosis: a case series. J Neurol. 2015 Nov 24.

Background: Paediatric onset multiple sclerosis (POMS) is characterized by high inflammatory activity. No disease modifying treatment has been approved for POMS. 

Objective: The objective of this report was to report the use of rituximab, a B cell depleting monoclonal anti-CD20-antibody, in POMS. 

Methods: This is a retrospective case series at four specialized MS centres in Sweden. Participants were identified through the Swedish MS-registry and our own patient stocks. Data were collected through medical charts review. 

Results: We identified 14 POMS patients treated with i.v. rituximab 500-1000 mg every 6th to 12th months. Median age at disease onset was 14.7 years, median age at rituximab treatment initiation was 16.5 years, and median treatment duration was 23.6 months. No relapses were reported, and the EDSS scores remained stable or decreased in 13 of 14 cases during rituximab treatment. Beyond 6 months from initiating rituximab treatment, only one new lesion was detected on MRI. No serious AEs were reported. The drug survival was 86 %. 

Conclusions: Our data indicate that rituximab treatment is safe, effective and well tolerated in children with MS. Nine POMS cases treated with rituximab have previously been published. They had higher disease activity pre-rituximab, but similar safety and efficacy outcomes after treatment. An RCT of rituximab in POMS is warranted.

CoI: multiple

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