Emapalumab use in malignancy-associated hemophagocytic lymphohistiocytosis in the United States: the REAL-HLH study
Year: 2025
Type: article
Study design: Observational Study
Abstract: ABSTRACT: Malignancy-associated hemophagocytic lymphohistiocytosis (mHLH), a hyperinflammatory syndrome, has poor prognosis and no standard therapy. Emapalumab, a fully human monoclonal antibody that neutralizes the proinflammatory cytokine interferon gamma, is approved for treating primary hemophagocytic lymphohistiocytosis (pHLH). REAL-HLH, a retrospective chart review conducted across 33 US hospitals, evaluated real-world treatment patterns and outcomes in patients treated with ≥1 dose of emapalumab between 20 November 2018 and 31 October 2021. Data are presented for the subset of patients with mHLH. Overall, 51 of 105 (48.6%) patients were categorized as having secondary HLH, of which 17 of 51 patients had HLH associated with an underlying malignancy (mHLH). At HLH diagnosis, the median age (range) was 15.0 (3.0-27.0) years, 6 of 14 (42.9%) patients with available data had a positive Optimized HLH Inflammatory index, indicating pathologic inflammation; 9 of 17 (52.9%) had infections; and 10 of 17 (58.8%) received emapalumab in an intensive care unit. Emapalumab was primarily initiated for treating refractory (10/17; 58.8%) or progressive (3/17, 17.7%) disease. Most patients received HLH-related therapies before (16/17; 94.1%) and/or concurrent with (15/17; 88.2%) emapalumab. Treatment with emapalumab-containing regimens normalized most key laboratory parameters and maintained normal values for others (fibrinogen [11/13; 84.6%], absolute neutrophil count [6/10; 60%], and chemokine ligand 9 [7/8; 87.5%]) per physician assessment. Overall survival at the end of follow-up and 12-month survival probability from emapalumab initiation were 23.5% and 22.1%, respectively. In conclusion, emapalumab-containing regimens normalized, improved, or maintained normal values for most laboratory parameters in patients with mHLH. Future studies are warranted to establish appropriate emapalumab dosing and utility in this high-risk population.
Source: Blood Advances
Authors: Adi Zoref-Lorenz, Carl E. Allen, Edward M. Behrens, Shanmuganathan Chandrakasan, May Chien, Michelle L. Hermiston, Ashley P. Hinson, Stefanos Intzes, Michael S. Isakoff, Jennifer W. Leiding, Abiola Oladapo, Arun Panigrahi, Sachit A. Patel, Priti Pednekar, Anish K. Ray, Jennifer A. Rothman, Blachy Dávila Saldaña, Kelly J. Walkovich, Joanna Weinstein, John D. Yee, Michael B. Jordan, Joanna Weinstein, Ashley P. Hinson, Carl E. Allen, Olive S. Eckstein, Nitya Gulati, Shanmuganathan Chandrakasan, Hilary Haines, Taizo A. Nakano, Sachit A. Patel, Edward M. Behrens, Stefanos Intzes, Allyson Hays, Blachy Dávila Saldaña, Michael B. Jordan, Adi Zoref-Lorenz, Rabi Hanna, Michael Isakoff, Anish K. Ray, Jennifer A. Rothman, Anand Tandra, Robert Cooper, Jennifer W. Leiding, May Chien, Susmita Sarangi, Vinod K Gidvani-Diaz, Prakash Satwani, John Carter, Michael Henry, Nicholas Gloude, Sima Bhatt, Deepika Bhatla, Lauren Draper, Arun Panigrahi, Michelle L. Hermiston, Renee Modica, Mona Riskalla, Ashley Baker, Brant Ward
Institutions: Cincinnati Children's Hospital Medical Center, Tel Aviv University, Meir Medical Center, Baylor College of Medicine, Children's Hospital of Philadelphia, Emory University, Children's Healthcare of Atlanta, Lucile Packard Children's Hospital, Stanford University, University of California, San Francisco, VinUniversity, Levine Children's Hospital, Providence Sacred Heart Medical Center, Connecticut Children's Medical Center, Johns Hopkins University, Johns Hopkins All Children's Hospital, University of California Davis Medical Center, University of California, Davis, University of Nebraska Medical Center, Cook Children's Medical Center, Duke University, Duke Medical Center, Children's National, University of Michigan, Michigan Medicine, Northwestern University, Lurie Children's Hospital, Robert H. Lurie Comprehensive Cancer Center of Northwestern University, University of Cincinnati
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $3,800
Funder: Swedish Orphan Biovitrum
OpenAlex ID: W4416969005