Emapalumab Treatment in Patients With Rheumatologic Disease–Associated Hemophagocytic Lymphohistiocytosis in the United States: A Retrospective Medical Chart Review Study
Authors' conclusion
Emapalumab-containing regimens stabilized or normalized most key laboratory parameters, reduced glucocorticoid dose, and were associated with low disease-related mortality, thereby demonstrating potential benefits in patients with rheumatologic disease-associated HLH.
Study context
- Population
- Patients with hemophagocytic lymphohistiocytosis (HLH) treated with ≥1 dose of emapalumab, 33 US hospitals, November 20, 2018–October 31, 2021; subset with rheumatologic disease-associated HLH (9 sJIA, 1 AOSD); median (range) age at HLH diagnosis 5 (0.9–39) yearsof 105 REAL-HLH patients
- Participants
- 15 patients
- Outcome
- Laboratory parameters stabilized or normalized (physician-determined) with emapalumab-containing regimens
Methodological detailssubset analysis of REAL-HLH, multicentre (33 US hospitals), real-world
- Subset analysis of REAL-HLH
- Multicentre (33 US hospitals)
- Real-world
Main finding
As reported in the abstract
Absolute neutrophil count and absolute lymphocyte count stabilized or normalized
13 of 14; 92.9%
Chemokine ligand 9 stabilized or normalized
9 of 11; 81.8%
Platelets and alanine transaminase stabilized or normalized
11 of 14; 78.6%
Reduction in GC dose
80%
Observational data: associations, not effects.
Further findings
- Overall survival and 12-month survival probability from emapalumab initiation
- 86.7%
Original publication
Emapalumab Treatment in Patients With Rheumatologic Disease–Associated Hemophagocytic Lymphohistiocytosis in the United States: A Retrospective Medical Chart Review Study. Arthritis Rheumatol. Published 5 November 2024. doi:10. 1002/ art. 42985
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