Is neutralization of IFN‐γ sufficient to control inflammation in HLH?

EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity

“…More targeted novel therapies including alemtuzumab may reduce this toxicity and improve the patients' condition prior to HSCT [81]. The efficacy of anti IFN gamma antibodies [82,83] and JAK-Inhibitors need yet to be evaluated in prospective studies. VOD is common in this group of patients, mainly due to treatment related toxicity and disease, and therefore we suggest the use of prophylactic defibrotide in infants under 18 months of age or those over 18 months with clear hepatic involvement.…”

Clinical features, diagnosis and therapy of familial haemophagocytic lymphohistiocytosis

“…Up to one‐third of patients needed additional HLH‐directed therapies. A commentary from several leading HLH experts critically analysed the results of the study, suggesting that until there is more convincing evidence, more broadly acting agents such as etoposide and dexamethasone should remain the standard of care for frontline treatment of HLH 33 . Whereas the Food and Drug Administration in the USA has granted marketing authorisation for emapalumab in 2020, the European Medicines Agency confirmed the refusal of marketing authorisation after reexamination.…”

Combined emapalumab and ruxolitinib in patients with haemophagocytic Lymphohistiocytosis

“…Concerns about the combination therapy, for ruxolitinib, are mainly reported by Jordon et al [ 7 ], who believe that the addition of ruxolitinib worsens myelosuppression. For emapalumab, the main concern lies in the heightened GM-CSF-producing capacity by T cells in the absence of IFN-γ signaling [ 12 , 14 ]. In our study, the low-dosage agents’ combination proved to have a beneficial effect.…”