EurekAlert!: New targeted therapy, ulixertinib, shows promise for histiocytosis, a rare blood cancer New targeted therapy, ulixertinib, shows promise for histiocytosis, a rare blood cancer Histiocytosis is a rare group of conditions that occur when your body makes abnormal (mutant) white blood cells called histiocytes. Normal histiocytes help tune up your body’s defenses against multiple pathogens (like viruses, bacteria, fungi and parasites). In histiocytosis, the histiocytes move into tissues where they are not normally found and cause damage to those tissues.

Understanding the Context

These proliferating immune cells may form tumors, which can affect various parts of the body. These clinical practice guidelines provide the latest evidence and expert-consensus for diagnosing and treating the three most common forms of histiocytosis in adults: Langerhans cell histiocytosis (LCH), Erdheim-Chester Disease (ECD,) and Rosai Dorfman Disease (RDD). The historic term ‘histiocytosis’ meaning ‘tissue cell’ is used as a unifying concept for diseases characterized by pathogenetic myeloid cells that share histologic features with macrophages or dendritic cells. These cells may arise from the embryonic yolk sac, fetal liver, or post-natal bone marrow.

Key Insights

Learn about Histiocytosis, its early signs, risk factors, diagnosis, and treatment options to help manage the condition effectively. Understand histiocytosis: the complex mechanism of immune cell proliferation, diverse classifications, genetic causes, diagnosis, and treatment options. The clinical manifestations of histiocytosis depend on the organs and systems involved, as well as their level of involvement. Langerhans cell histiocytosis (LCH) can be localized and manifest... In medicine, histiocytosis is an excessive number of histiocytes [1] (tissue macrophages), and the term is also often used to refer to a group of rare diseases which share this sign as a characteristic.

Final Thoughts

Adult histiocyte disorders are rare diseases. The most common of these is Langerhans cell histiocytosis (LCH), which affects only a few hundred patients every year in the United States. LCH results from the body's overproduction of immature histiocytes, which are a type of white blood immune cell. Langerhans cell histiocytosis is the most common histiocytic disorder. There are other rare histiocytic disorders such as Erdheim-Chester disease and juvenile xanthogranuloma, some of which are now considered variants of Langerhans cell histiocytosis (2).