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Aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder: long-term efficacy, tolerability and retention rate of Azathioprine


Azathioprine ( Imuran ) is a common immunosuppressive drug used for relapse prevention in neuromyelitis optica ( NMO ).

The objective of this study was to assess efficacy, tolerability and retention of Azathioprine in a large neuromyelitis optica cohort.

Researchers have conducted a retrospective review of medical records of 103 aquaporin-4 antibody-positive NMO and NMO spectrum disorder ( NMOSD ) patients treated with Azathioprine.

This is the largest reported cohort of AQP4-Ab positive patients treated with Azathioprine. Eighty-nine per cent ( n=92 ) had reduction in median annualised relapse rates from 1.5 to 0, p
Sixty-one per cent ( n=63 ) remained relapse free at a median follow-up of 18 months. Neurological function improved or stabilised in 78%.

At last follow-up, treatment was discontinued in 46% ( n=47 ). Of these, 62% ( n=29 ) were because of side effects, 19% ( n=9 ) because of death, 15% ( n=7 ) because of ongoing disease activity, and 2% ( n=1 ) because of pregnancy.

Using Kaplan-Meyer curves, researchers have estimated that 73%, 58%, 47% and 33% of patients will remain on Azathioprine for longer than one, three, five and 10 years, respectively, after initiation of treatment.

In conclusion, Azathioprine is a modestly effective treatment for neuromyelitis optica. However, many patients discontinue Azathioprine over time and this seems to reflect poor tolerability more than lack of efficacy. ( Xagena )

Elsone L et al, Mult Scler 2014; Published online before print

XagenaMedicine_2014



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