Pellegrin, S;
Baldeweg, T;
Pujar, S;
D'Arco, F;
Cantalupo, G;
Varadkar, S;
Cross, JH;
(2021)
Immunomodulation With Azathioprine Therapy in Rasmussen Syndrome: A Multimodal Evaluation.
Neurology
, 96
(2)
e267-e279.
10.1212/WNL.0000000000011004.
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Abstract
OBJECTIVE: To verify safety and efficacy of the corticosteroid-sparing drug Azathioprine (AZA) in Rasmussen syndrome (RS), we retrospectively analyzed a cohort of RS patients recruited in a single pediatric neuroscience center. METHODS: We compared outcomes in 30 RS patients 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 one patient discontinued treatment due to pancytopenia. In 27/30 AZA patients, all of whom were corticosteroid responders, corticosteroid therapy could be weaned or reduced without worsening of seizures in 89%. AZA patients 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 (of about 2 years, Exp(B)=0.295, 95%CI[0.108, 0.807];p=0.017), 2) hemiparesis (about one year, Exp(B)=0.315, 95%CI[0.137, 0.724];p=0.007), and 3) surgery (about 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 grey matter atrophy on serial MRI scans. CONCLUSION: AZA treatment appears to slow clinical progression of Rasmussen syndrome in steroid responders; this will give most 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 RS patients AZA is well tolerated and slows hemiparesis and appearance of EPC.
| Type: | Article |
|---|---|
| Title: | Immunomodulation With Azathioprine Therapy in Rasmussen Syndrome: A Multimodal Evaluation |
| Location: | United States |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1212/WNL.0000000000011004 |
| Publisher version: | https://doi.org/10.1212/WNL.0000000000011004 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| Keywords: | Autoimmune diseases, Class III, Volumetric MRI use in epilepsy |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept |
| URI: | https://discovery-pp.ucl.ac.uk/id/eprint/10113003 |
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