Background
Immune effector cell-associated neurotoxicity syndrome (ICANS) is a serious, potentially life-threatening complication of CAR T-cell therapy. Management of corticosteroid-refractory ICANS remains limited to supportive care and case reports.
Aim
To characterise and evaluate the use of ruxolitinib for Grade 2-4 (G2-4) corticosteroid- and anakinra-refractory ICANS in CAR-T patients at a quaternary teaching hospital.
Method
A retrospective review identified patients with G2-4 ICANS refractory to corticosteroids (≥24h of dexamethasone 10mg QID or equivalent) and anakinra (≥24h of ≥200mg/day) treated with ruxolitinib, via Individual Patient Approvals using dispensing software, December 2024 to June 2026. Data included demographics, CAR-T product, disease status, prior lines of therapy, CRS and ICANS grade, ruxolitinib dose/duration, time to symptom resolution (resolution of G3/4 or ICE-score 9-10), and adverse events (i.e. cytopenias, bloodstream infections, CMV reactivation).
Results
Four patients were identified (2 male, 2 female; age range 24-79, median 70 years). CAR-T products were heterogeneous (Axi-cel, Tisa-cel 1, Tecartus 1, Clinical trial 1). All experienced G2 CRS; three developed G3/4 ICANS and one had persistent (>21 days) G2 ICANS. The first patient received ruxolitinib as fourth-line therapy after 14 days of maximum-dose corticosteroids, anakinra (ceased due to prolonged infections), and intrathecal triple therapy. Ruxolitinib was used as third-line therapy, after maximum-dose corticosteroids and anakinra, in the remaining three patients. Initial dosing was 5mg twice daily in three patients and 10mg twice daily for one patient. All patients achieved resolution of ICANS, with median time to symptom resolution of 10 days. One patient developed G4 neutropenia (duration of 6 days) and two BSIs; CMV reactivation occurred in two patients.
Conclusion
Although small patient numbers, this series suggests ruxolitinib may be an effective option for corticosteroid- and Anakinra-refractory G2-4 ICANS. Larger, prospective, multi-centre data is needed before its use can be supported in guidelines.