Clinical outcomes of adults with hemophagocytic lymphohistiocytosis treated with the HLH-04 protocol: a retrospective analysis. Issue 7 (6th June 2020)

• Record Type: Journal Article
• Authors: Bubik, Rachel J.; Barth, Dylan M.; Hook, Christopher; Wolf, Robert C.; Muth, Jessica M.; Mara, Kristin; Patnaik, Mrinal S.; Pruthi, Rajiv K.; Marshall, Ariela L.; Litzow, Mark R.; Elliott, Michelle A.; Hogan, William J.; Shah, Mithun V.; Begna, Kebede H.; Alkhateeb, Hassan; Pardanani, Animesh; Ashrani, Aneel A.; Call, Timothy G.; Rivera, Candido E.; Camoriano, John K.; Go, Ronald S.; Wolanskyj-Spinner, Alexandra P.; Parikh, Sameer A.
• Is Part Of: Leukemia & lymphoma. Volume 61:Issue 7(2020)
• Page Start: 1592
• Page End: 1600
• Publication Date: 2020-06-06
• Subjects: Hemophagocytic lymphohistiocytosis (HLH) -- immune activation -- etoposide -- corticosteroids; Leukemia -- Periodicals; Lymphomas -- Periodicals; 616.99419
• DOI: 10.1080/10428194.2020.1737684
• Languages: English
• ISSNs: 1042-8194

Abstract

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 × 10 9 /L (HR 4.06, p = 0.027), ANC <1 × 10 9 /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.