Myelofibrosis (MF)

MF is an uncommon type of blood cancer that interrupts the body’s normal production of blood cells. This condition can cause widespread scarring in the bone marrow, leading to severe anaemia that can cause weakness and fatigue. Bone marrow scarring can also lead to a reduced number of platelets which are important blood-clotting cells.

What causes MF?

Primary MF is a chronic disorder that occurs over time where the bone marrow stem cells change or mutate. Stem cells have the ability to replicate and divide into the components that make up the blood – red and white blood cells, and platelets. It is not well-defined or understood what causes the bone marrow stem cell mutations, however the disease can be classified based on certain gene mutations identified by DNA testing.

Secondary MF develops from another blood disorder, such as thrombocythemia or polycythemia vera. Thrombocythemia is a condition where you have a high platelet count, which can cause blood clots. Polycythemia vera is a blood cancer where the bone marrow produces too many red blood cells, causing the blood to thicken and also develop blood clots.

Compared with other similar chronic blood cancers, MF carries the poorest prognosis.

Symptoms

Most myelofibrosis develops slowly and in the early stages of the disease people might not experience any signs or symptoms. Early symptoms include:

Tired or weak,
Shortness of breath,
Pain or fullness below the ribs on the left side – resulting from an enlarged spleen,
Bleeds and bruises easily,
Night sweats,
Fever,
Bone pain.

Treatment

The only known cure for Myelofibrosis is stem cell transplant. Therefore, early diagnosis is critical for long-term management of the condition. PBI currently has three clinical trials focusing on Myelofibrosis. The aim of each trial is to assess the safety, tolerability and/or efficacy of different drugs and treatment options. One of our trials is looking at the drug BMS-986158 in combination with one of two specific drugs - Ruxolitinib or Fedratinib. In July 2023, The Lancet published an article referencing these treatments saying that 'In 2011, the approval by the US Food and Drug Administration for Ruxolitinib has advanced the treatment for myelofibrosis and ushered further advances in the development of Fedratinib in 2019. These agents have greatly improved the lives of patients with myelofibrosis through reducing symptom burden and potentially providing survival benefit'.

To read more about these trials please visit our website PBI clinical trials.

References