Language
English
Publication Date
5-14-2024
Journal
Blood Advances
DOI
10.1182/bloodadvances.2023012217
PMID
38429096
PMCID
PMC11117018
PubMedCentral® Posted Date
3-5-2024
PubMedCentral® Full Text Version
Post-print
Abstract
Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening, hyperinflammatory syndrome. Emapalumab, a fully human monoclonal antibody that neutralizes the proinflammatory cytokine interferon gamma, is approved in the United States to treat primary HLH (pHLH) in patients with refractory, recurrent, or progressive disease, or intolerance with conventional HLH treatments. REAL-HLH, a retrospective study, conducted across 33 US hospitals, evaluated real-world treatment patterns and outcomes in patients treated with ≥1 dose of emapalumab between 20 November 2018 and 31 October 2021. In total, 46 patients met the pHLH classification criteria. Median age at diagnosis was 1.0 year (range, 0.3-21.0). Emapalumab was initiated for treating refractory (19/46), recurrent (14/46), or progressive (7/46) pHLH. At initiation, 15 of 46 patients were in the intensive care unit, and 35 of 46 had received prior HLH-related therapies. Emapalumab treatment resulted in normalization of key laboratory parameters, including chemokine ligand 9 (24/33, 72.7%), ferritin (20/45, 44.4%), fibrinogen (37/38, 97.4%), platelets (39/46, 84.8%), and absolute neutrophil count (40/45, 88.9%). Forty-two (91.3%) patients were considered eligible for transplant. Pretransplant survival was 38 of 42 (90.5%). Thirty-one (73.8%) transplant-eligible patients proceeded to transplant, and 23 of 31 (74.2%) of those who received transplant were alive at the end of the follow-up period. Twelve-month survival probability from emapalumab initiation for the entire cohort (N = 46) was 73.1%. There were no discontinuations because of adverse events. In conclusion, results from the REAL-HLH study, which describes treatment patterns, effectiveness, and outcomes in patients with pHLH treated with emapalumab in real-world settings, are consistent with the emapalumab pivotal phase 2/3 pHLH trial.
Keywords
Humans, Lymphohistiocytosis, Hemophagocytic, Female, Male, Treatment Outcome, Adolescent, Child, Retrospective Studies, Child, Preschool, Infant, Young Adult, Antibodies, Monoclonal, Adult
Published Open-Access
yes
Recommended Citation
Chandrakasan, Shanmuganathan; Jordan, Michael B; Baker, Ashley; et al., "Real-World Treatment Patterns and Outcomes in Patients With Primary Hemophagocytic Lymphohistiocytosis Treated With Emapalumab" (2024). Faculty, Staff and Students Publications. 6414.
https://digitalcommons.library.tmc.edu/baylor_docs/6414
Graphical Abstract