APOA4 as a novel predictor of prognosis in Stevens-Johnson syndrome/toxic epidermal necrolysis: a proteomics analysis from two prospective cohorts.

BACKGROUND: Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) are rare but life-threatening adverse drug reactions. Conventional systemic therapies are of limited efficacy and often exhibit strong side effects.

OBJECTIVE: To assess the efficacy and safety of the combination treatment with a tumor necrosis factor (TNF) - α antagonist adalimumab and delineate the underlying mechanisms.

METHODS: We evaluated the efficacy and safety of the combination therapy with adalimumab by comparing two treatment cohorts of SJS/TEN patients. Patient plasma samples were collected for proteomics analysis.

RESULTS: The combination therapy with adalimumab significantly shortened the time to mucocutaneous reepithelization and healing, with reduced side effects caused by corticosteroids. Plasma proteomic profiling showed that apolipoprotein A-Ⅳ (APOA4) was one of the most significant differentially expressed proteins. Multivariate regression analysis revealed that APOA4 level was significantly associated with prognosis parameter of SJS/TEN (P=0.004), but not with disease severity score (SCORTEN) (P=0.118). Thus further research will be helpful to effectively incorporate APOA4 into current SCORTEN-driven protocols.

LIMITATIONS: The cohort size is relatively small. Both cohorts had low overall SCORTEN scores.

CONCLUSION: Adalimumab in combination with corticosteroids demonstrates significant clinical benefits over corticosteroids alone in SJS/TEN patients. Moreover, APOA4 may serve as a novel prognostic marker of SJS/TEN.