March 2020
Ibrutinib for chronic Graft versus Host Disease
Ibrutinib belongs to class of drugs called Bruton’s Tyrosine Kinase (BTK) inhibitors that work against defective B lymphocytes, which are a type of white blood cells affected by these diseases. Earlier studies have demonstrated that ibrutinib works by blocking the BTK signalling pathway, resulting in reduced production of the defective blood cells in GvHD and some other types of blood cancers. Ibrutinib is available as tablets and taken orally. If licensed, ibrutinib will offer an additional first line treatment option for cGvHD which may improve patients’ quality of life by helping reduce the dose of steroids used and reduce the severity of GvHD symptoms.
February 2020
Avapritinib for advanced systemic mastocytosis
Avapritinib is in clinical development for the treatment of advanced systemic mastocytosis (SM) in adults. SM is a condition where mast cells grow uncontrollably and accumulate in body organs/tissues such as the skin, internal organs, lymph nodes and bones. Mast cells are immune cells that release inflammatory mediators that are important in the body’s allergic responses. When mast cells are present in large numbers there is a high release of these mediators leading to symptoms such as itching, fever, abdominal pain, nausea and vomiting. In advanced SM, mast cells collect in such high quantities that they lead to organ damage and dysfunction, bone fractures and anaemia.
January 2020
Mepolizumab for hypereosinophilic syndrome – add-on therapy
Mepolizumab is a medicinal product currently in development as an add-on for the treatment of hypereosinophilic syndrome (HES). HES is a rare group of inflammatory disorders characterised by an overproduction of eosinophils (a type of disease-fighting white blood cell). When eosinophils infiltrate certain tissues, they can cause inflammation and organ damage which, over time, can impact patients’ day-to-day ability to function. Although any organ system can be involved in HES, the heart, central nervous system, skin, and respiratory tract are the most commonly affected.
November 2019
Ruxolitinib for chronic graft versus host disease (cGvHD)
Ruxolitinib is in clinical development for chronic graft versus host disease (cGvHD). After a donor stem cell transplant, the donor’s stem cells (the graft) may sometimes react against the host’s own cells. This is called GVHD. cGvHD may happen more than three months after transplant. It can develop from acute GVHD or happen on its …
