Language
English
Publication Date
5-1-2025
Journal
Immunotherapy
DOI
10.1080/1750743X.2025.2509478
PMID
40528399
PMCID
PMC12506739
PubMedCentral® Posted Date
6-17-2025
PubMedCentral® Full Text Version
Post-print
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.
Published Open-Access
yes
Recommended Citation
Chandrakasan, Shanmuganathan; Allen, Carl E; Bhatla, Deepika; et al., "Emapalumab Treatment in Rheumatologic Disease-Associated Hemophagocytic Lymphohistiocytosis: A Plain Language Summary" (2025). Faculty, Staff and Students Publications. 6431.
https://digitalcommons.library.tmc.edu/baylor_docs/6431