Hemophagocytic lymphohistiocytosis (HLH) requires prompt recognition and treatment. Even when not all diagnostic criteria for HLH are fully present, initiating therapy is crucial if there is a strong suspicion of the disorder. Patients benefit most from care provided by specialists, typically pediatric hematologists in centers with expertise in managing HLH.
Several factors guide the treatment strategy, including family history of HLH, concurrent infections, and identified immune system deficiencies. Treatment for HLH commonly involves cytokine inhibitors, immune therapies, and chemotherapy regimens incorporating etoposide and dexamethasone. Combinations of these approaches are often utilized, and hematopoietic stem cell transplantation may be considered in certain cases.
Early use of anakinra, an IL-1 antagonist, has shown promise in treating secondary HLH, potentially reducing the need for intensive chemotherapy, though further research is needed. Additionally, ruxolitinib, targeting the JAK-STAT pathway, is under investigation as a primary treatment for HLH through the North American Consortium for Histiocytosis (NACHO). Emapalumab, an interferon gamma-blocking monoclonal antibody, offers another therapeutic option for patients with HLH that is refractory, recurrent, progressive, or unresponsive to standard treatments.
These treatments underscore the importance of recognizing HLH promptly and initiating therapy based on clinical suspicion, even while fully meeting Hlh Diagnosis Criteria may still be in progress.
