Tofacitinib Rescue for Refractory Drug‐Induced Liver Injury With Severe DRESS : A Prospective Proof‐of‐Concept Study
Jingyan Gu, Yuan Wang, Xiaoyu Wang, Yingying Yan, Fei Pei, Tianyu He, Chunlei Zhang, Jun Li, Wenhui Wang
Source abstract
ABSTRACT Drug‐induced liver injury (DILI) with severe DRESS carries high mortality, yet rescue therapies for corticosteroid‐refractory cases remain undefined. We hypothesized that JAK inhibition could reverse refractory liver injury by targeting the underlying cytokine storm. In this prospective proof‐of‐concept study, consecutive patients with severe DRESS‐associated DILI refractory to corticosteroids (persistent/worsening liver injury) received oral tofacitinib rescue. Key assessments were the normalisation of liver biochemistries. Despite initial corticosteroids, all four patients exhibited progressive severe injury (median peak ALT 1126 U/L). Tofacitinib rapidly reversed liver injury in all cases. Transaminases normalised or near‐normalised (median: 48 days), total bilirubin normalised, and cutaneous symptoms resolved completely. Notably, baseline positive autoantibodies seroconverted to negative. No severe adverse events occurred (median follow‐up: 274 days). Tofacitinib shows promise as a rescue strategy for corticosteroid‐refractory DILI with severe DRESS. The rapid recovery underscores the JAK–STAT pathway's pathogenic role in this setting.
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