Treatment of HLH

As HLH is uncommon and serious, treatment is usually coordinated by a specialist centre experienced in treating rare immune disorders.

The priority of treatment is to damp down (suppress) the immune system to reduce the over-reaction and lessen the risk of tissue damage. This will often involve courses of corticosteroids and chemotherapy medicines, usually given into a vein (intravenously) in hospital. Some of the medicines used are listed in the table below but new treatments are being developed all the time.

Treatment will be individualised to minimise side effects, which your medical team will discuss with you. If an infectious trigger is suspected, anti-infection treatment may be given, such as antibiotics or other medication. In the case of primary HLH, this treatment usually puts the condition into remission but the risk of relapse remains.

Corrective treatment of HLH

In many cases, haematopoietic stem cell transplant (HSCT, including bone marrow transplant, or BMT) offers the potential for long-term cure of primary HLH. HSCT aims to replace the faulty immune system with an immune system from a healthy donor. Stem cells, from which all the cells of the immune system develop, can be obtained from healthy bone marrow, or in some cases from umbilical cord blood or donor blood. The healthy stem cells can be given by transfusion into a vein to a child with HLH. More information on HSCT can be found at www.piduk.org/whatarepids/treatment/bonemarrowtransplantation