Clinical outcomes of adults with hemophagocytic lymphohistiocytosis treated with the HLH-04 protocol: a retrospective analysis
Rachel J. Bubik, Dylan Barth, C. Christopher Hook, Robert Christian Wolf, Jessica M. Muth, Kristin C. Mara, Mrinal M. Patnaik, Rajiv K. Pruthi, Ariela L. Marshall, Mark R. Litzow, Michelle A. Elliott, William J. Hogan, Mithun Vinod Shah, Kebede H. Begna, Hassan B. Alkhateeb, Animesh Pardanani, Aneel A. Ashrani, Timothy G. Call, Candido E. Rivera, John Camoriano, Ronald S. Go, Alexandra P. Wolanskyj, Sameer A. Parikh
Abstract
Hemophagocytic lymphohistiocytosis (HLH) is a rare syndrome of pathologic immune activation in children that is increasingly being recognized in adults. Efficacy data for the HLH-04 protocol in adults is lacking. This study retrospectively analyzed 31 adult patients, median age 46 years, who received HLH-04 from 1/1/2004 to 5/1/2018. HLH etiology included malignancy (n = 9), autoimmune (n = 8), infection (n = 8), and idiopathic (n = 6). Eighteen patients were evaluable for response at week 4 with 7 having no response, 11 reaching partial response, and 0 reaching complete response (CR). Six patients eventually achieved CR at a median 195 days. The 1-year overall survival (OS) was 35% and median OS was 3.2 months. Univariate analysis showed shorter survival for hemoglobin <9 g/dL (HR 4.29, p = 0.003), platelets <100 × 109/L (HR 4.06, p = 0.027), ANC <1 × 109/L (HR 5.24, p = 0.001), and total bilirubin >1.2 mg/dL (HR 3.30, p = 0.022). Outcomes of adults treated with HLH-04 remain dismal and newer treatment modalities are needed.