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People with HLH usually develop symptoms within the first months or years of life. Symptoms may include fever, enlarged liver or spleen, cytopenia (decreased number of blood cells), and neurological abnormalities. All forms of HLH, including cases treated adequately, may have a high mortality rate. The long-term outlook (prognosis) of familial forms without treatment is poor, with a median survival of less than 2 months to 6 months after diagnosis. These disorders can be either inherited (familial or primary) or secondary to other conditions (rheumatic diseases, cancer or infections) such as Macrophage Activation Syndrome (MAS), a severe complication of rheumatic diseases. NLRC4 mutation and XIAP deficiency can be considered part of primary HLH.

Macrophage Activation Syndrome. Macrophage Activation Sysdrome (MAS) is a very severe complication of rheumatic diseases. Mortality ranges between 10-30% for patients with MAS and there is no registered therapy. MAS occurs frequently in systemic Juvenile Idiopathic Arthritis (soJIA) and in Adult onset Still’s Disease (AOSD) patients. It is characterized by severe systemic inflammation with pancytopenia, liver insufficiency, coagulopathy and neurological symptoms. The underlying cellular mechanism is the uncontrolled activation and proliferation of T lymphocytes and macrophages leading to a cytokine storm with a predominant role of IL-18.