Management of Refractory Malignancy-Associated Hemophagocytic
Lymphohistiocytosis in Pediatric Patients: A Case Series of Novel
Therapeutics and Treatment Challenges
Abstract
Hemophagocytic lymphohistiocytosis (HLH) is a potentially fatal syndrome
of immune dysregulation that has primary and secondary forms. HLH is
classified based on underlying triggers of malignancy, infections,
and/or autoimmune processes. Primary-HLH has a genetic etiology related
to defects in cytotoxic T and NK cells. The established standard of care
is dexamethasone and etoposide but no guidelines exist for refractory
HLH or cases triggered by malignancy. [1] We describe three
pediatric patients with malignancy-associated HLH (m-HLH) to discuss
complexities in initial diagnostic considerations, balance of
therapeutic regimens/toxicities, and novel uses of emapalumab and
ruxolitinib in refractory disease patients.