Treatment should be started immediately once a diagnosis of HLH is established. Treatment includes dexamethasone and etoposide. Alternative “salvage” therapies that target T cells include anti-thymocyte globulin and alemtuzumab.
While patients are undergoing treatment, efforts should be made to determine if there is a genetic defect present. Screening tests can provide early data that helps guide therapy while confirmative genetic testing is still pending. If a genetic defect is observed, curative treatment with allogeneic hematopoietic cell transplantation should be considered. In the absence of a genetic diagnosis, transplant should also be considered for young patients, those with a family history of bad inflammation / death, recurrent clinical phenotype, or functional laboratory data suggesting an inherited defect in lymphocyte cytotoxicity.
Clinical Trial for HLH
New biological agents have been developed that block the pro-inflammatory proteins, such as interferon gamma, which are significantly elevated in HLH:
These strategies and novel agents may offer alternative pathways to cure or provide adequate disease control while the patient awaits hematopoietic (blood or marrow) stem cell transplantation. Ongoing research at our center is helping provide clarity as to their exact role in HLH management.