Background S econdary hemophagocytic lymphohistiocytosis (HLH) is a rare hyperinflammatory syndrome that requires prompt diagnosis and appropriate treatment. A risk-stratification model that could be used to identify high-risk pediatric patients with HLH who should be considered for second-line therapies, including salvage regimens and allogeneic hematopoietic cell transplantation (HCT), was developed. Methods The medical records of 88 pediatric patients (median age 1.4 years, range 0.2–15 years) with non-malignancy associated secondary HLH were retrospectively reviewed. Treatment strategies included dexamethasone, etoposide, and cyclosporine. Results Survival analysis showed HLH patients with infections other than Epstein-Barr virus (EBV) and unknown causes experienced better 5-year overall survival (OS) than patients with HLH due to autoimmune disease, EBV or immunodeficiency (76% vs. 65, 33.3, 11%, p < 0.001). On multivariate analysis, among all patients, non-response at 8 weeks was the most powerful predictor of poor OS. When treatment response was excluded, hemoglobin < 60 g/L and albumin < 25 g/L at diagnosis were associated with poor OS. In patients with EBV-HLH, hemoglobin < 60 g/L at diagnosis was associated with poor OS. A prognostic risk score was established and weighted based on hazard ratios calculated for three parameters measured at diagnosis: hemoglobin < 60 g/L (2 points), platelets < 30 × 10 9 /L (1 point), albumin < 25 g/L (2 points). Five-year OS of low-risk (score 0–1), intermediate-risk (score 2), and poor-risk (score ≥ 3) patients were 88, 38, and 22%, respectively ( p < 0.001). Conclusions These findings indicate that clinicians should be aware of predictive factors at diagnosis and consider 8-week treatment response to identify patients with high-risk of disease progression and the need for second-line therapy and allogeneic HCT.
【저자키워드】 hemophagocytic lymphohistiocytosis, Prognostic factor, risk stratification, 【초록키워드】 Dexamethasone, Treatment, therapy, Infection, immunodeficiency, Diagnosis, risk, virus, Disease progression, Autoimmune disease, survival, Hyperinflammatory syndrome, Patient, albumin, Platelet, Allogeneic, hemoglobin, prognostic, patients, HLH, EBV, Therapies, Analysis, survival analysis, regimen, Predictive, medical record, cell transplantation, Factor, Pediatric patient, Treatment response, clinician, hazard ratio, median age, parameter, HCT, Result, identify, calculated, cause, excluded, 【제목키워드】 children, outcome, secondary hemophagocytic lymphohistiocytosis, malignancy, Prognostic factor,