Emapalumab use in malignancy-associated hemophagocytic lymphohistiocytosis in the United States: the REAL-HLH study

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.

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Publication Details

Journal
Blood Advances
Published
2025-12-03
DOI
https://doi.org/10.1182/bloodadvances.2024013690
Citations
1
Primary Topic
Autoimmune and Inflammatory Disorders Research
Type
article
Field-Weighted Citation Impact
1.77

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article

Emapalumab use in malignancy-associated hemophagocytic lymphohistiocytosis in the United States: the REAL-HLH study

Michelle L. Hermiston, Carl E. Allen, Abiola Oladapo, Joanna Weinstein et al.
1 citations
Blood Advances
Autoimmune and Inflammatory Disorders Research
1.77
article

Emapalumab use in malignancy-associated hemophagocytic lymphohistiocytosis in the United States: the REAL-HLH study

Michelle L. Hermiston, Carl E. Allen, Abiola Oladapo, Joanna Weinstein, Jennifer Rothman, Adi Zoref‐Lorenz, May Chien, Michael S. Isakoff, Shanmuganathan Chandrakasan, Sachit Patel, Stefanos Intzes, Kelly Walkovich, Michael B. Jordan, Blachy J. Dávila Saldaña, Priti Pednekar, Edward M. Behrens, Anish Ray, Jennifer W. Leiding, Ashley R. Hinson, Arun Panigrahi, John Yee
article en
1 citations

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.

Blood AdvancesVol. 10(5)
Providence Sacred Heart Medical Center (US), Cincinnati Children's Hospital Medical Center (US), Lurie Children's Hospital (US), Lucile Packard Children's Hospital (US), Children's Hospital of Philadelphia (US), Children's National (US), Johns Hopkins University (US), Emory University (US), Tel Aviv University (IL), Palo Alto University (US), University of California, San Francisco (US), Baylor College of Medicine (US), University of Michigan (US), Johns Hopkins Medicine (US), Meir Medical Center (IL), Cook Children's Medical Center (US), Johns Hopkins All Children's Hospital (US), Connecticut Children's Medical Center (US), Nippon Soken (Japan) (JP), Center for Cancer and Blood Disorders (US), Michigan United (US), Michigan Medicine (US), Precision for Medicine (United States) (US), Duke Medical Center (US), Levine Children's Hospital (US), NeuroMetrix (United States) (US), VinUniversity (VN), Office of Extramural Research (US), Children's Healthcare of Atlanta (US), University of California Davis Medical Center (US), Robert H. Lurie Comprehensive Cancer Center of Northwestern University, University of Nebraska Medical Center (US), University of California, Davis (US), University of Cincinnati Medical Center (US), Stanford University (US)
Swedish Orphan Biovitrum
Openalex Percentile: Top 11%
Autoimmune and Inflammatory Disorders Research
1.77
DOIOpen Access PDFOpenAlex
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