Long-term efficacy,tolerability and
retention rate of azathioprine in
103 aquaporin-4 antibody-positive
neuromyelitis optica spectrum disorder
patients: a multicentre retrospective
observational study from the UK
Liene Elsone1, Joanna Kitley2, Sebastian Luppe3, Daniel Lythgoe4,
Kerry Mutch1, Saiju Jacob5, Rachel Brown2, Kathryn Moss6,
Benjamin McNeillis2, Yee Yen Goh1, M Isabel Leite2,
Neil Robertson3, Jackie Palace2 and Anu Jacob1
Background: Azathioprine (AZA) is a common immunosuppressive drug used for relapse prevention in neuromyelitis
Objectives: The objective of this paper is to assess efficacy, tolerability and retention of AZA in a large NMO cohort.
Methods: We conducted a retrospective review of medical records of 103 aquaporin-4 antibody-positive NMO and
NMO spectrum disorder (NMOSD) patients treated with AZA.
Results: This is the largest reported cohort of AQP4-Ab positive patients treated with AZA. Eighty-nine per cent
(n = 92) had reduction in median annualised relapse rates from 1.5 (IQR 0.6–4.0) to 0 (IQR 0–0.27, p < 0.00005)
with treatment. 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, we estimate that 73%, 58%, 47% and 33%
of patients will remain on AZA for longer than one, three, five and 10 years, respectively, after initiation of
Conclusions: AZA is a modestly effective treatment for NMO. However, many patients discontinue AZA over time
and this seems to reflect poor tolerability more than lack of efficacy.
Neuromyelitis optica, NMO, azathioprine, tolerability, retention, aquaporin-4
Date received: 26 October 2013; revised: 24 January 2014; accepted: 5 February 2014
1The Walton Centre NHS Foundation Trust, UK.
2Nuffield Department of Clinical Neurosciences, John Radcliffe Hospital,
3Cardiff and Vale University Health Board, University Hospital of Wales, UK
4University of Liverpool, CRUK Liverpool Cancer Trials Unit, UK.
5Queen Elizabeth Neuroscience Centre, University Hospitals of
6Royal Liverpool University Hospital, UK.
525870 MSJ0010.1177/1352458514525870Multiple Sclerosis JournalELsone et al.
Anu Jacob, The Walton Centre NHS Foundation Trust, Lower Lane,
Liverpool, L9 7LJ, UK.
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Long-term efficacy, tolerability and retention rate of azathioprine in 103 aquaporin-4 antibody-positive
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