Skip to main content
Advertisement
  • Neurology.org
  • Journals
    • Neurology
    • Clinical Practice
    • Genetics
    • Neuroimmunology & Neuroinflammation
  • Specialty Sites
    • COVID-19
    • Practice Current
    • Practice Buzz
    • Without Borders
    • Equity, Diversity and Inclusion
    • Innovations in Care Delivery
  • Collections
    • Topics A-Z
    • Residents & Fellows
    • Infographics
    • Patient Pages
    • Null Hypothesis
    • Translations
  • Podcast
  • CME
  • About
    • About the Journals
    • Contact Us
    • Editorial Board
  • Authors
    • Submit a Manuscript
    • Author Center

Advanced Search

Main menu

  • Neurology.org
  • Journals
    • Neurology
    • Clinical Practice
    • Genetics
    • Neuroimmunology & Neuroinflammation
  • Specialty Sites
    • COVID-19
    • Practice Current
    • Practice Buzz
    • Without Borders
    • Equity, Diversity and Inclusion
    • Innovations in Care Delivery
  • Collections
    • Topics A-Z
    • Residents & Fellows
    • Infographics
    • Patient Pages
    • Null Hypothesis
    • Translations
  • Podcast
  • CME
  • About
    • About the Journals
    • Contact Us
    • Editorial Board
  • Authors
    • Submit a Manuscript
    • Author Center
  • Home
  • Latest Articles
  • Current Issue
  • Past Issues
  • Residents & Fellows

User menu

  • Subscribe
  • My Alerts
  • Log in

Search

  • Advanced search
Neurology
Home
The most widely read and highly cited peer-reviewed neurology journal
  • Subscribe
  • My Alerts
  • Log in
Site Logo
  • Home
  • Latest Articles
  • Current Issue
  • Past Issues
  • Residents & Fellows

Share

January 12, 2021; 96 (2) Article

Immunomodulation With Azathioprine Therapy in Rasmussen Syndrome

A Multimodal Evaluation

View ORCID ProfileSerena Pellegrin, View ORCID ProfileTorsten Baldeweg, View ORCID ProfileSuresh Pujar, View ORCID ProfileFelice D'Arco, View ORCID ProfileGaetano Cantalupo, View ORCID ProfileSophia Varadkar, View ORCID ProfileJ. Helen Cross
First published October 12, 2020, DOI: https://doi.org/10.1212/WNL.0000000000011004
Serena Pellegrin
From the Developmental Neurosciences Programme (S.P., T.B., J.H.C.), Great Ormond Street Institute of Child Health, London, UK; Child Neuropsychiatry Unit (S.P., G.C.), University of Verona, Italy; and Great Ormond Street Hospital for Children NHS Foundation Trust (S.P., F.D., S.V., J.H.C.), London, UK.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Serena Pellegrin
Torsten Baldeweg
From the Developmental Neurosciences Programme (S.P., T.B., J.H.C.), Great Ormond Street Institute of Child Health, London, UK; Child Neuropsychiatry Unit (S.P., G.C.), University of Verona, Italy; and Great Ormond Street Hospital for Children NHS Foundation Trust (S.P., F.D., S.V., J.H.C.), London, UK.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Torsten Baldeweg
Suresh Pujar
From the Developmental Neurosciences Programme (S.P., T.B., J.H.C.), Great Ormond Street Institute of Child Health, London, UK; Child Neuropsychiatry Unit (S.P., G.C.), University of Verona, Italy; and Great Ormond Street Hospital for Children NHS Foundation Trust (S.P., F.D., S.V., J.H.C.), London, UK.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Suresh Pujar
Felice D'Arco
From the Developmental Neurosciences Programme (S.P., T.B., J.H.C.), Great Ormond Street Institute of Child Health, London, UK; Child Neuropsychiatry Unit (S.P., G.C.), University of Verona, Italy; and Great Ormond Street Hospital for Children NHS Foundation Trust (S.P., F.D., S.V., J.H.C.), London, UK.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Felice D'Arco
Gaetano Cantalupo
From the Developmental Neurosciences Programme (S.P., T.B., J.H.C.), Great Ormond Street Institute of Child Health, London, UK; Child Neuropsychiatry Unit (S.P., G.C.), University of Verona, Italy; and Great Ormond Street Hospital for Children NHS Foundation Trust (S.P., F.D., S.V., J.H.C.), London, UK.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Gaetano Cantalupo
Sophia Varadkar
From the Developmental Neurosciences Programme (S.P., T.B., J.H.C.), Great Ormond Street Institute of Child Health, London, UK; Child Neuropsychiatry Unit (S.P., G.C.), University of Verona, Italy; and Great Ormond Street Hospital for Children NHS Foundation Trust (S.P., F.D., S.V., J.H.C.), London, UK.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for Sophia Varadkar
J. Helen Cross
From the Developmental Neurosciences Programme (S.P., T.B., J.H.C.), Great Ormond Street Institute of Child Health, London, UK; Child Neuropsychiatry Unit (S.P., G.C.), University of Verona, Italy; and Great Ormond Street Hospital for Children NHS Foundation Trust (S.P., F.D., S.V., J.H.C.), London, UK.
  • Find this author on Google Scholar
  • Find this author on PubMed
  • Search for this author on this site
  • ORCID record for J. Helen Cross
Full PDF
Short Form
Citation
Immunomodulation With Azathioprine Therapy in Rasmussen Syndrome
A Multimodal Evaluation
Serena Pellegrin, Torsten Baldeweg, Suresh Pujar, Felice D'Arco, Gaetano Cantalupo, Sophia Varadkar, J. Helen Cross
Neurology Jan 2021, 96 (2) e267-e279; DOI: 10.1212/WNL.0000000000011004

Citation Manager Formats

  • BibTeX
  • Bookends
  • EasyBib
  • EndNote (tagged)
  • EndNote 8 (xml)
  • Medlars
  • Mendeley
  • Papers
  • RefWorks Tagged
  • Ref Manager
  • RIS
  • Zotero
Permissions

Make Comment

See Comments

Downloads
0

Share

  • Article
  • Figures & Data
  • Info & Disclosures
Loading

This article requires a subscription to view the full text. If you have a subscription you may use the login form below to view the article. Access to this article can also be purchased.

Abstract

Objective To verify safety and efficacy of the corticosteroid-sparing drug azathioprine (AZA) in Rasmussen syndrome (RS), we retrospectively analyzed a cohort of patients with RS recruited in a single pediatric neuroscience center.

Methods We compared outcomes in 30 patients with RS who received AZA with 23 patients who were not treated with this drug. We used a multimodal approach to correlate therapy with clinical features (seizures, epilepsia partialis continua [EPC], hemiparesis) and neuroimaging markers of progressive brain atrophy.

Results AZA was well tolerated; only 1 patient discontinued treatment due to pancytopenia. In 27 of 30 patients receiving AZA, all of whom were corticosteroid responders, corticosteroid therapy could be weaned or reduced without worsening of seizures in 89%. Patients receiving AZA had a lower prevalence of EPC (42% vs 67% in controls) and hemiparesis (64% vs 92%, respectively). Cox regression showed for the AZA group compared to controls a delayed time to (1) EPC (≈2 years, exp[B] = 0.295, 95% confidence interval [CI] 0.108–0.807; p = 0.017), (2) hemiparesis (≈1 year, exp[B] = 0.315, 95% CI 0.137–0.724; p = 0.007), and (3) surgery (≈2 years, exp[B] = 2.068, 95% CI 1.012–4.227; p = 0.046). However, there were no group differences in cognitive decline over time (IQ change per year) or in hemispheric gray matter atrophy on serial MRI scans.

Conclusion AZA treatment appears to slow clinical progression of RS in steroid responders; this will give the greatest advantage in patients in the early stages of the disease in whom surgical decision-making may require further time.

Classification of Evidence This study provides Class III evidence that for pediatric patients with RS AZA is well tolerated and slows hemiparesis and appearance of EPC.

Glossary

AH=
affected hemisphere;
AZA=
azathioprine;
CI=
confidence interval;
EPC=
epilepsia partialis continua;
FSIQ=
Full-Scale IQ;
GAI=
General Ability Index;
GM=
gray matter;
GM-HRvol=
GM interhemispheric ratio;
RS=
Rasmussen syndrome;
SE=
standard error;
UH=
unaffected hemisphere;
WISC=
Wechsler Intelligence Scale for Children;
WPPSI=
Wechsler Preschool and Primary Scale of Intelligence

Footnotes

  • Go to Neurology.org/N for full disclosures. Funding information and disclosures deemed relevant by the authors, if any, are provided at the end of the article.

  • ↵* These authors contributed equally to this work.

  • Class of Evidence: NPub.org/coe

  • Received April 20, 2020.
  • Accepted in final form August 25, 2020.
  • © 2020 American Academy of Neurology
View Full Text

AAN Members

We have changed the login procedure to improve access between AAN.com and the Neurology journals. If you are experiencing issues, please log out of AAN.com and clear history and cookies. (For instructions by browser, please click the instruction pages below). After clearing, choose preferred Journal and select login for AAN Members. You will be redirected to a login page where you can log in with your AAN ID number and password. When you are returned to the Journal, your name should appear at the top right of the page.

Google Safari Microsoft Edge Firefox

Click here to login

AAN Non-Member Subscribers

Click here to login

Purchase access

For assistance, please contact:
AAN Members (800) 879-1960 or (612) 928-6000 (International)
Non-AAN Member subscribers (800) 638-3030 or (301) 223-2300 option 3, select 1 (international)

Sign Up
Information on how to subscribe to Neurology and Neurology: Clinical Practice can be found here 

Purchase
Individual access to articles is available through the Add to Cart option on the article page.  Access for 1 day (from the computer you are currently using) is US$ 39.00.  Pay-per-view content is for the use of the payee only, and content may not be further distributed by print or electronic means.  The payee may view, download, and/or print the article for his/her personal, scholarly, research, and educational use.  Distributing copies (electronic or otherwise) of the article is not allowed.

Disputes & Debates: Rapid online correspondence

No comments have been published for this article.
Comment

NOTE: All authors' disclosures must be entered and current in our database before comments can be posted. Enter and update disclosures at http://submit.neurology.org. Exception: replies to comments concerning an article you originally authored do not require updated disclosures.

  • Stay timely. Submit only on articles published within the last 8 weeks.
  • Do not be redundant. Read any comments already posted on the article prior to submission.
  • 200 words maximum.
  • 5 references maximum. Reference 1 must be the article on which you are commenting.
  • 5 authors maximum. Exception: replies can include all original authors of the article.
  • Submitted comments are subject to editing and editor review prior to posting.

More guidelines and information on Disputes & Debates

Compose Comment

More information about text formats

Plain text

  • No HTML tags allowed.
  • Web page addresses and e-mail addresses turn into links automatically.
  • Lines and paragraphs break automatically.
Author Information
NOTE: The first author must also be the corresponding author of the comment.
First or given name, e.g. 'Peter'.
Your last, or family, name, e.g. 'MacMoody'.
Your email address, e.g. higgs-boson@gmail.com
Your role and/or occupation, e.g. 'Orthopedic Surgeon'.
Your organization or institution (if applicable), e.g. 'Royal Free Hospital'.
Publishing Agreement
NOTE: All authors, besides the first/corresponding author, must complete a separate Disputes & Debates Submission Form and provide via email to the editorial office before comments can be posted.
CAPTCHA
This question is for testing whether or not you are a human visitor and to prevent automated spam submissions.

Vertical Tabs

You May Also be Interested in

Back to top
  • Article
    • Abstract
    • Glossary
    • Methods
    • Results
    • Discussion
    • Study Funding
    • Disclosure
    • Acknowledgment
    • Appendix Authors
    • Footnotes
    • References
  • Figures & Data
  • Info & Disclosures
Advertisement

Related Articles

  • No related articles found.

Topics Discussed

  • Volumetric MRI use in epilepsy
  • All Epilepsy/Seizures
  • All Pediatric
  • Autoimmune diseases
  • Class III

Alert Me

  • Alert me when eletters are published
Neurology: 96 (2)

Articles

  • Ahead of Print
  • Current Issue
  • Past Issues
  • Popular Articles
  • Translations

About

  • About the Journals
  • Ethics Policies
  • Editors & Editorial Board
  • Contact Us
  • Advertise

Submit

  • Author Center
  • Submit a Manuscript
  • Information for Reviewers
  • AAN Guidelines
  • Permissions

Subscribers

  • Subscribe
  • Activate a Subscription
  • Sign up for eAlerts
  • RSS Feed
Site Logo
  • Visit neurology Template on Facebook
  • Follow neurology Template on Twitter
  • Visit Neurology on YouTube
  • Neurology
  • Neurology: Clinical Practice
  • Neurology: Genetics
  • Neurology: Neuroimmunology & Neuroinflammation
  • AAN.com
  • AANnews
  • Continuum
  • Brain & Life
  • Neurology Today

Wolters Kluwer Logo

Neurology | Print ISSN:0028-3878
Online ISSN:1526-632X

© 2021 American Academy of Neurology

  • Privacy Policy
  • Feedback
  • Advertise