Friday, 21 February 2014

Efficacy and safety of natalizumab after 5-10 years

Are we ready for the early highly-effective paradigm shift? #MSBlog #MSResearch 

"I have been discussing the TOP results for several years; they show that MSers derive the most benefit from natalizumab when it is started early and as a first-line therapy. In addition the lower the disability at baseline the greater your chances of improving or recovering; low disability implies reserve capacity to allow recovery. It will be interesting to see if the EMA are swayed by this data and allow MSers who are JCV-ve access to this drug as a first-line therapy. At present it is mainly used as a second-line drug in those with highly-active or severe rapidly-evolving MS. 


"I don't think these results will be unique to natalizumab, but simply represent the impact of highly-effective therapies when used early. The question is whether or not the wider MS community are ready for a paradigm shift to this approach."

"Please note that although the EDSS flat-lined over 5 years this is an average and that some MSers will have improved, some will have stayed the same and some would have deteriorated. However, the observation of flat-lining over a 5 year period is remarkable; I am keen  to see if this will continue over the next 5 years and whether or not those started early on natalizumab will be protected from developing SPMS."

"Please note that I have numerous conflicts of interest in relation to this study and my commentary above."



Epub: Butzkueven et al. Efficacy and safety of natalizumab in multiple sclerosis: interim observational programme results. J Neurol Neurosurg Psychiatry. 2014 Feb

BACKGROUND: Clinical trials established the efficacy and safety of natalizumab. Data are needed over longer periods of time and in the clinical practice setting.


OBJECTIVE: To evaluate long-term safety of natalizumab and its impact on annualised relapse rate and Expanded Disability Status Scale (EDSS) progression in RRMSers. 

METHODS: The Tysabri (natalizumab) Observational Program (TOP) is an open-label, multinational, 10-year prospective study in clinical practice settings.

RESULTS: In this 5-year interim analysis, 4821 RRMSers were enrolled. Follow-up for at least 4 years from natalizumab commencement in 468 MSers and at least 2 years in 2496 MSers revealed no new safety signals. There were 18 cases of progressive multifocal leucoencephalopathy reported, following 11-44 natalizumab infusions. Mean annualised relapse rate decreased from 1.99 in the 12 months prior to baseline to 0.31 on natalizumab therapy (p<0.0001), remaining low at 5 years. Lower annualised relapse rates were observed in MSers who used natalizumab as first MS therapy, in MSers with lower baseline EDSS scores, and in MSers with lower pre-natalizumab relapse rates. Mean EDSS scores remained unchanged up to 5 years.

CONCLUSIONS: Interim TOP data confirm natalizumab's overall safety profile and the low relapse rate and stabilised disability levels in natalizumab-treated MSers with RRMS in clinical practice.

CoI: multiple

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