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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 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
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