Clinical Profile Radiologic Features and Outcomes in Children with Acute Necrotizing Encephalopathy: A Comparative Study of Tocilizumab Use
Lokesh Saini1, Harshvi Jobanputra2, Ashwini Chityala1, Sarbesh Tiwari3, Ankit Meena1, Rahul Gupta1
1Pediatric Neurology, 2Pediatrics, 3Diagnostic and Intervention Radiology, AIIMS Jodhpur
Objective:
To evaluate the impact of Tocilizumab in moderate and high-risk ANE Severity score patients, with outcomes measured using standardized scoring systems
Background:

Acute necrotizing encephalopathy (ANE), a life-threatening neurological emergency presents as a clinico-radiological syndrome characterized by bilateral thalamic lesions. Patients develop neurological sequelae following viral prodrome.

Design/Methods:

This ambispective study was conducted from 2022 to 2025 in our department. Following ethical clearance, children aged 1 month to 10 years were enrolled.

Baseline demographic and clinical data were documented in a predesigned proforma. Laboratory investigations included liver function tests, serum ammonia, and inflammatory markers. All patients underwent neuroimaging. Treatment protocols were tailored to disease severity and clinical response, including immunotherapy with pulse methylprednisolone, IVIG, and early initiation of tocilizumab. Treatment responses were evaluated using standardized tools- Pediatric Cerebral Performance Category (PCPC) scale at baseline and 6-month follow-up, and the modified Rankin Scale (MRS) at 6-month follow-up.

Results:

Twenty-eight patients were enrolled. The median (IQR) age was 48 months (36–69 months), with a female predominance (61%). Common clinical characteristics included encephalopathy (100%), seizures (100%) and dystonia (100%). ANE severity scores (SS) indicated medium (32%) and high risk (67%) among patients. All children had bilateral symmetrical thalamic involvement on MRI with varying involvement of other areas.

In both the tocilizumab group (n=12) and the non-tocilizumab group(N=16)- at baseline, all patients had severe disability (PCPC >4). At 6-month follow-up, 80% of the tocilizumab group achieved complete recovery and in non-tocilizumab group, 56% achieved complete recovery. The relative risk for disability with tocilizumab was 0.47 (95% CI 0.11–1.98), indicating a beneficial trend. 58% children in Tocilizumab group had mRS score of 0 compared to 25% in non-tocilizumab group at 6 months follow up.
Conclusions:

Early initiation of tocilizumab in moderate- to high-risk cases is potentially transformative, improving survival and reducing long-term disability. 

10.1212/WNL.0000000000216235
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