Pediatric rheumatology

Emapalumab Treatment in Patients With Rheumatologic Disease–Associated Hemophagocytic Lymphohistiocytosis in the United States: A Retrospective Medical Chart Review Study

Arthritis RheumatolPublished 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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