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 Elsone; Joanna Kitley; Sebastian Luppe; Daniel Lythgoe; Kerry Mutch; Saiju Jacob; Rachel Brown; Kathryn Moss; Benjamin McNeillis; Yee Yen Goh; M Isabel Leite; Neil Robertson; Jackie Palace; Anu Jacob

doi:10.1177/1352458514525870

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 Elsone, Joanna Kitley, Sebastian Luppe, Daniel Lythgoe, Kerry Mutch, Saiju Jacob, Rachel Brown, Kathryn Moss, Benjamin McNeillis, Yee Yen Goh, M Isabel Leite, Neil Robertson, Jackie Palace, Anu Jacob

Immunology and Immunotherapy

Research output: Contribution to journal › Article › peer-review

76 Citations (Scopus)

Abstract

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

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 treatment.

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.

Original language	English
Pages (from-to)	1533-1540
Number of pages	8
Journal	Multiple Sclerosis
Volume	20
Issue number	11
Early online date	19 Mar 2014
DOIs	https://doi.org/10.1177/1352458514525870
Publication status	Published - 1 Oct 2014

Keywords

Adult
Aged
Aquaporin 4/immunology
Azathioprine/therapeutic use
Female
Humans
Immunosuppressive Agents/therapeutic use
Male
Middle Aged
Neuromyelitis Optica/drug therapy
Recurrence
Retrospective Studies
Time
Treatment Outcome
United Kingdom
NMO
tolerability
retention

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Elsone, L., Kitley, J., Luppe, S., Lythgoe, D., Mutch, K., Jacob, S., Brown, R., Moss, K., McNeillis, B., Goh, Y. Y., Leite, M. I., Robertson, N., Palace, J., & Jacob, A. (2014). 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. Multiple Sclerosis, 20(11), 1533-1540. https://doi.org/10.1177/1352458514525870

@article{431d30124bb4471cadfc663c604cae58,

title = "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",

abstract = "BACKGROUND: Azathioprine (AZA) is a common immunosuppressive drug used for relapse prevention in neuromyelitis optica (NMO).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 treatment.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.",

keywords = "Adult, Aged, Aquaporin 4/immunology, Azathioprine/therapeutic use, Female, Humans, Immunosuppressive Agents/therapeutic use, Male, Middle Aged, Neuromyelitis Optica/drug therapy, Recurrence, Retrospective Studies, Time, Treatment Outcome, United Kingdom, NMO, tolerability, retention",

author = "Liene Elsone and Joanna Kitley and Sebastian Luppe and Daniel Lythgoe and Kerry Mutch and Saiju Jacob and Rachel Brown and Kathryn Moss and Benjamin McNeillis and Goh, {Yee Yen} and Leite, {M Isabel} and Neil Robertson and Jackie Palace and Anu Jacob",

year = "2014",

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day = "1",

doi = "10.1177/1352458514525870",

language = "English",

volume = "20",

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journal = "Multiple Sclerosis",

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Elsone, L, Kitley, J, Luppe, S, Lythgoe, D, Mutch, K, Jacob, S, Brown, R, Moss, K, McNeillis, B, Goh, YY, Leite, MI, Robertson, N, Palace, J & Jacob, A 2014, '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', Multiple Sclerosis, vol. 20, no. 11, pp. 1533-1540. https://doi.org/10.1177/1352458514525870

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. / Elsone, Liene; Kitley, Joanna; Luppe, Sebastian et al.
In: Multiple Sclerosis, Vol. 20, No. 11, 01.10.2014, p. 1533-1540.

Research output: Contribution to journal › Article › peer-review

TY - JOUR

T1 - Long-term efficacy, tolerability and retention rate of azathioprine in 103 aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder patients

T2 - a multicentre retrospective observational study from the UK

AU - Elsone, Liene

AU - Kitley, Joanna

AU - Luppe, Sebastian

AU - Lythgoe, Daniel

AU - Mutch, Kerry

AU - Jacob, Saiju

AU - Brown, Rachel

AU - Moss, Kathryn

AU - McNeillis, Benjamin

AU - Goh, Yee Yen

AU - Leite, M Isabel

AU - Robertson, Neil

AU - Palace, Jackie

AU - Jacob, Anu

PY - 2014/10/1

Y1 - 2014/10/1

N2 - BACKGROUND: Azathioprine (AZA) is a common immunosuppressive drug used for relapse prevention in neuromyelitis optica (NMO).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 treatment.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.

AB - BACKGROUND: Azathioprine (AZA) is a common immunosuppressive drug used for relapse prevention in neuromyelitis optica (NMO).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 treatment.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.

KW - Adult

KW - Aged

KW - Aquaporin 4/immunology

KW - Azathioprine/therapeutic use

KW - Female

KW - Humans

KW - Immunosuppressive Agents/therapeutic use

KW - Male

KW - Middle Aged

KW - Neuromyelitis Optica/drug therapy

KW - Recurrence

KW - Retrospective Studies

KW - Time

KW - Treatment Outcome

KW - United Kingdom

KW - NMO

KW - tolerability

KW - retention

U2 - 10.1177/1352458514525870

DO - 10.1177/1352458514525870

M3 - Article

C2 - 24647557

SN - 1352-4585

VL - 20

SP - 1533

EP - 1540

JO - Multiple Sclerosis

JF - Multiple Sclerosis

IS - 11

ER -

Elsone L, Kitley J, Luppe S, Lythgoe D, Mutch K, Jacob S et al. 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. Multiple Sclerosis. 2014 Oct 1;20(11):1533-1540. Epub 2014 Mar 19. doi: 10.1177/1352458514525870