ArticleAnnals of the rheumatic diseases2023
Efficacy and safety of emapalumab in macrophage activation syndrome.
Article in Annals of the rheumatic diseases, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 87 papers, 5 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Multicenter Study for the Long-term Follow-up of HLH Patients Who Received Treatment With NI-0501, an Anti-interferon Gamma Monoclonal Antibody
A Pilot, Open-label, Single Arm, Multicenter Study to Evaluate Safety, Tolerability, Pharmacokinetics and Efficacy of Intravenous Administrations of Emapalumab, an Anti-interferon Gamma (Anti-IFNγ) Monoclonal Antibody, in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)
Who cites it
87 citing papers in PubMed, 5 syntheses or guidelines pooled it, 128 citations in OpenAlex.
- The Asia-Pacific League of Associations for Rheumatology Consensus Recommendations on the Management of Systemic Juvenile Idiopathic Arthritis (Juvenile Still's Disease).International journal of rheumatic diseases · 2026Guideline
- Real-world use of emapalumab in hemophagocytic lymphohistiocytosis: a scoping review of published evidence.Frontiers in pharmacology · 2026Pooled it
- Current treatment in macrophage activation syndrome worldwide: a systematic literature review to inform the METAPHOR project.Rheumatology (Oxford, England) · 2025Pooled it
- Efficacy and safety of therapies for Still's disease and macrophage activation syndrome (MAS): a systematic review informing the EULAR/PReS guidelines for the management of Still's disease.Annals of the rheumatic diseases · 2024Pooled it
- EULAR/PReS recommendations for the diagnosis and management of Still's disease, comprising systemic juvenile idiopathic arthritis and adult-onset Still's disease.Annals of the rheumatic diseases · 2024Guideline
- Advancements in Diagnosis and Care for Immune Dysregulation and Inborn Errors of Immunity in Pediatric Rheumatology: Our 50-Year Journey.Arthritis care & research · 2026Review
- Challenging diagnosis of drug reaction with eosinophilia and systemic symptoms (DRESS) in a patient with adult-onset Still disease.JAAD case reports · 2026Article
- Variation in the access and use of biologic and targeted synthetic DMARDs in juvenile idiopathic arthritis: insights from a national UK survey.Pediatric rheumatology online journal · 2026Article
- Type I Interferon-Driven Monocyte Dysregulation and MAS-associated CD8bioRxiv : the preprint server for biology · 2026Article
- Hepatic Manifestations in Systemic Juvenile Idiopathic Arthritis and Macrophage Activation Syndrome.The Journal of rheumatology · 2026Article
- Potential new targets for rheumatic diseases.EULAR rheumatology open · 2026Review
- Chimeric antigen receptor T-cell therapies related to immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome: Diagnosis, high-risk factors, and management.Chinese medical journal · 2026Review
- Speaking the same language: international cross-validation of emerging biomarkers for juvenile idiopathic arthritis and Still's Disease.Pediatric rheumatology online journal · 2026Article
- Emapalumab use in malignancy-associated hemophagocytic lymphohistiocytosis in the United States: the REAL-HLH study.Blood advances · 2026Article
- CXCL9 as a novel prognostic marker to identify high-risk adults with hemophagocytic lymphohistiocytosis.Blood · 2026Article
- CellAwareGNN: Single-Cell Enhanced Knowledge Graph Foundation Model for Drug Indication Prediction.bioRxiv : the preprint server for biology · 2026Article
- Still's Disease and Autoinflammation: Positioning an Inflammatory Syndrome on the Autoinflammation-Autoimmunity Spectrum.Current rheumatology reports · 2026Review
- STAT1 drives the immune landscape of murine Toll-like receptor 9-induced liver inflammation.JHEP reports : innovation in hepatology · 2026Article
- Cytokine storm.Nature reviews. Disease primers · 2026Review
- The many faces of cytokine storm syndrome: immunopathogenic mechanisms and clinical implications for a better patient management.Clinical and experimental immunology · 2026Review
27 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
17 authors at 7 institutions in 6 countries.
Funding
Abstract
objectivesMacrophage activation syndrome (MAS) is a severe, life-threatening complication of systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD). The objective of this study was to confirm the adequacy of an emapalumab dosing regimen in relation to interferon-γ (IFNγ) activity by assessing efficacy and safety. The efficacy outcome was MAS remission by week 8, based on clinical and laboratory criteria.
methodsWe studied emapalumab, a human anti-IFNγ antibody, administered with background glucocorticoids, in a prospective single-arm trial involving patients who had MAS secondary to sJIA or AOSD and had previously failed high-dose glucocorticoids, with or without anakinra and/or ciclosporin. The study foresaw 4-week treatment that could be shortened or prolonged based on investigator's assessment of response. Patients entered a long-term (12 months) follow-up study.
resultsFourteen patients received emapalumab. All patients completed the trial, entered the long-term follow-up and were alive at the end of follow-up. The investigated dosing regimen, based on an initial loading dose followed by maintenance doses, was appropriate, as shown by rapid neutralisation of IFNγ activity, demonstrated by a prompt decrease in serum C-X-C motif chemokine ligand 9 (CXCL9) levels. By week 8, MAS remission was achieved in 13 of the 14 patients at a median time of 25 days. Viral infections and positive viral tests were observed.
conclusionsNeutralisation of IFNγ with emapalumab was efficacious in inducing remission of MAS secondary to sJIA or AOSD in patients who had failed high-dose glucocorticoids. Screening for viral infections should be performed, particularly for cytomegalovirus. TRIAL REGISTRATION NUMBER: NCT02069899 and NCT03311854.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.