Therapies and Treatment Responses for Hemophagocytic Lymphohistiocytosis in Children: A Single-center Retrospective Study

Abstract

Abstract Hemophagocytic lymphohistiocytosis (HLH) is a severe and life-threatening systemic inflammatory disorder. This retrospective study aims to evaluate the effectiveness of different therapies and identify early treatment responses maybe for indicators to overall survival. The study involved 102 patients from January 1, 2012, to December 31, 2022, using Cox regression to identify prognostic risk factors. Causal mediation analysis assessed the impact of various treatments on overall survival through the mediator of complete remission at 4 or 8 weeks. Achieving complete remission at 4 or 8 weeks suggests a favorable prognosis. However central nervous system involvement, high lactate dehydrogenase levels, and blood purification alone indicate poor prognosis (P < 0.05). HLH-94/04 protocol treatments had higher survival rates at 81.3% and 76.6%, compared to blood purification alone or combined with HLH-94/04 protocol treatments at 23.4% and 15.4%. Complete remission at 4 or 8 weeks resulted in higher survival rates of 90.7% and 92.3% respectively, compared to 2.9% and 3.4% for partial or no response. Compared to HLH-94 protocol treatment, blood purification alone has a 33.28% effect mediated by inducing complete remission at 4 weeks, which decreases to 26.56% at 8 weeks. Blood purification combined with HLH-94/04 protocol treatment with a higher mediation effect was 79.88% at 4 weeks compared to 51.95% at 8 weeks. HLH-94/04 protocol treatments led to complete remission and improved survival rates than Blood purification alone or combined with HLH-94/04 protocol treatment. Complete remission at 4 weeks may be a better mediator of overall survival than that at 8 weeks.