Risk- and Response-Adapted Treatment in Pediatric Epstein-Barr Virus–Associated Hemophagocytic Lymphohistiocytosis

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BACKGROUND: Hemophagocytic lymphohistiocytosis (HLH) is a rapidly progressive and often fatal disorder. This study aimed to evaluate the stratification and treatment of pediatric Epstein-Barr virus-associated HLH (EBV-HLH) based on cytokine levels and clinical condition. METHODS: Pediatric patients newly diagnosed with EBV-HLH (n = 108) were risk stratified by cytokine and clinical criteria. Six were excluded for protocol violations. RESULTS: Among the 102 evaluable patients, 61 and 41 were classified as low and high risk and initially received dexamethasone treatment and HLH-94/04 regimens, respectively. Notably, 32.4% of children (33/102) were cured by dexamethasone monotherapy. The 12-month overall survival (OS) for the entire cohort was 86.3% (95% CI, 79.6%-93.0%), with 91.8% (95% CI, 84.9%-98.7%) and 78.0% (95% CI, 65.3%-90.7%) for patients at low and high risk, respectively (P = .037). Patients at low risk whose treatment shifted from dexamethasone to the HLH-94/04 regimen presented similar 12-month OS as those initially treated with the HLH-94/04 regimen (82.1% [95% CI, 68.0%-96.2%] vs 78.0% [95% CI, 65.3%-90.7%]; P = .589). In multivariate Cox regression model, risk group and response to initial treatment at 48-72 hours thereafter were associated with OS. CONCLUSIONS: A cytokine- and clinical-based risk stratification system, combined with dynamic response assessment, enables tailored treatment for pediatric patients with EBV-HLH.

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