Successful first-line treatment of hemophagocytic lymphohistiocytosis
with ruxolitinib in a pediatric patient with trisomy 21
Abstract
Hemophagocytic lymphohistiocytosis (HLH) is an uncommon immunologic
disorder associated with high rates of morbidity and mortality
characterized by systemic inflammation and multiorgan dysfunction. The
standard of care for primary treatment of HLH is chemotherapy (i.e.
etoposide), but consideration of alternative therapies is warranted to
support treatment goals for critically ill pediatric patients. We
present the case of a 7-year-old male with trisomy 21, acute multiorgan
failure secondary to infection, and subsequent HLH who was successfully
treated with ruxolitinib. This represents the first use of ruxolitinib
as a first-line agent for secondary HLH in a critically ill child with
trisomy 21.