Emapalumab treatment in rheumatologic disease-associated hemophagocytic lymphohistiocytosis: a plain language summary

What is this summary about? Hemophagocytic lymphohistiocytosis (HLH) is a rare disease caused by overactivation of the body’s natural defense mechanism (the immune system) against infection. In untreated HLH, uncontrolled inflammation (hyperinflammation) can cause significant damage to the body’s organs and death. HLH can occur due to genetic defects (primary HLH) or as a complication or worsening of an underlying disease such as rheumatologic disease, cancer, or infection (secondary HLH).Interferon-gamma (IFNγ) is a type of protein that is overproduced in HLH and causes hyperinflammation. Emapalumab is a medicine that blocks IFNγ activity. The REAL-HLH study described the use of emapalumab in routine clinical practice and evaluated its effects on the health of patients with HLH in the United States. This summary presents results from the REAL-HLH study for a subgroup of patients with rheumatologic disease-associated HLH, which is a particular type of secondary HLH. What were the results? Data from medical charts of patients treated for rheumatologic disease-associated HLH who received at least one dose of emapalumab were analyzed.The results from this study showed that emapalumab was used to treat patients with rheumatologic disease-associated HLH in whom HLH either worsened, recurred, or did not respond to conventional HLH treatment after initial improvement. Importantly, most patients receiving emapalumab-containing treatment regimens achieved normal or maintained stable laboratory values for ferritin, blood cell counts, liver enzymes etc., which are important HLH markers (naturally occurring molecules that serve as a sign of a normal or abnormal condition or process). In addition, physicians were able to lower the daily dose of steroids, a standard HLH medicine with long-term side effects, in most patients treated with emapalumab-containing treatment regimens (a treatment regimen is a structured and personalized plan that generally includes medications, therapies, lifestyle changes, and other interventions for managing and treating a specific medical condition or disease). Most patients (87%) were alive at the end of the study. What were the main conclusions of the REAL-HLH study reported by the researchers?Emapalumab-containing treatment regimens controlled hyperinflammation, reduced the average daily steroid dose in most patients, and were associated with a high chance of survival.

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Journal
Immunotherapy
Published
2025-05-03
DOI
https://doi.org/10.1080/1750743x.2025.2509478
Primary Topic
Autoimmune and Inflammatory Disorders Research
Type
article
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article

Emapalumab treatment in rheumatologic disease-associated hemophagocytic lymphohistiocytosis: a plain language summary

Michelle L. Hermiston, Susmita N. Sarangi, Carl E. Allen, Mona Riskalla et al.
Immunotherapy
Autoimmune and Inflammatory Disorders Research
article

Emapalumab treatment in rheumatologic disease-associated hemophagocytic lymphohistiocytosis: a plain language summary

Michelle L. Hermiston, Susmita N. Sarangi, Carl E. Allen, Mona Riskalla, Abiola Oladapo, Renee Modica, John Carter, Anand Tandra, Lauren Draper, Adi Zoref‐Lorenz, May Chien, Michael S. Isakoff, Deepika Bhatla, Shanmuganathan Chandrakasan, Sachit Patel, J. Allyson Hays, Kelly Walkovich, Michael B. Jordan, Prakash Satwani, Rabi Hanna, Priti Pednekar, Taizo A. Nakano, Edward M. Behrens, Patricia M. Hobday, Olive S. Eckstein, Jennifer W. Leiding, John Yee, Ashley P. Hinson, Robert B. Cooper
article en

Abstract

What is this summary about? Hemophagocytic lymphohistiocytosis (HLH) is a rare disease caused by overactivation of the body’s natural defense mechanism (the immune system) against infection. In untreated HLH, uncontrolled inflammation (hyperinflammation) can cause significant damage to the body’s organs and death. HLH can occur due to genetic defects (primary HLH) or as a complication or worsening of an underlying disease such as rheumatologic disease, cancer, or infection (secondary HLH).Interferon-gamma (IFNγ) is a type of protein that is overproduced in HLH and causes hyperinflammation. Emapalumab is a medicine that blocks IFNγ activity. The REAL-HLH study described the use of emapalumab in routine clinical practice and evaluated its effects on the health of patients with HLH in the United States. This summary presents results from the REAL-HLH study for a subgroup of patients with rheumatologic disease-associated HLH, which is a particular type of secondary HLH. What were the results? Data from medical charts of patients treated for rheumatologic disease-associated HLH who received at least one dose of emapalumab were analyzed.The results from this study showed that emapalumab was used to treat patients with rheumatologic disease-associated HLH in whom HLH either worsened, recurred, or did not respond to conventional HLH treatment after initial improvement. Importantly, most patients receiving emapalumab-containing treatment regimens achieved normal or maintained stable laboratory values for ferritin, blood cell counts, liver enzymes etc., which are important HLH markers (naturally occurring molecules that serve as a sign of a normal or abnormal condition or process). In addition, physicians were able to lower the daily dose of steroids, a standard HLH medicine with long-term side effects, in most patients treated with emapalumab-containing treatment regimens (a treatment regimen is a structured and personalized plan that generally includes medications, therapies, lifestyle changes, and other interventions for managing and treating a specific medical condition or disease). Most patients (87%) were alive at the end of the study. What were the main conclusions of the REAL-HLH study reported by the researchers?Emapalumab-containing treatment regimens controlled hyperinflammation, reduced the average daily steroid dose in most patients, and were associated with a high chance of survival.

ImmunotherapyVol. 17(7)
Cincinnati Children's Hospital Medical Center (US), University of Minnesota (US), Nebraska Medical Center (US), Cleveland Clinic (US), Children's Mercy Hospital (US), Lucile Packard Children's Hospital (US), Children's Hospital of Philadelphia (US), NewYork–Presbyterian Hospital (US), Johns Hopkins University (US), Emory University (US), Tel Aviv University (IL), Oregon Health & Science University (US), University of California, San Francisco (US), Baylor College of Medicine (US), Georgetown University (US), University of Michigan (US), Columbia University Irving Medical Center (US), University of Florida Health (US), New York Hospital Queens (US), Meir Medical Center (IL), MedStar Georgetown University Hospital (US), Johns Hopkins All Children's Hospital (US), Franciscan Health Indianapolis (US), Connecticut Children's Medical Center (US), Aflac (United States) (US), Levine Children's Hospital (US), Children's Healthcare of Atlanta (US), Saint Louis University (US), University of Nebraska Medical Center (US), University of Florida Health Science Center (US), University of Cincinnati Medical Center (US)
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Autoimmune and Inflammatory Disorders Research
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