Fast Five Quiz: Hemophagocytic Lymphohistiocytosis

Sarah K. Tasian, MD


June 19, 2020

Current initial therapy in adult patients with HLH consists of chemoimmunotherapy with etoposide and dexamethasone for 8 weeks with the goal of achieving disease remission. Intrathecal chemotherapy with methotrexate is also used in patients with evidence of central nervous system involvement with HLH. In the HLH-2004 trial, cyclosporine was added to the initial treatment regimen (etoposide and dexamethasone), but this third agent was not shown to improve outcomes and has subsequently been discontinued from modern HLH regimens.

HSCT is recommended for all patients with severe familial HLH. HSCT is also increasingly used in patients with HLH that is difficult to control, as well as in those with disease relapse following remission or who respond slowly to traditional therapy. Some studies support the use of reduced-intensity conditioning, such as with fludarabine and busulfan, as opposed to fully myeloablative conditioning in patients with HLH. The anti-CD52 monoclonal antibody alemtuzumab is also often used as a preparatory agent for T-cell depletion.

Emapalumab, a monoclonal antibody that binds to and neutralizes interferon-gamma, was approved by the US Food and Drug Administration in 2018 as a treatment for patients with refractory, recurrent, or progressive HLH or those who are intolerant of conventional HLH therapy.

Learn more about the treatment of patients with HLH.


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