Medicines for Myelofibrosis

What it is

Myelofibrosis is a rare, chronic blood condition (one of a group called myeloproliferative neoplasms) in which scar tissue (fibrosis) gradually builds up in the bone marrow — the tissue that produces blood cells. As the marrow becomes scarred, it becomes less able to make normal blood cells, which can lead to a shortage of red blood cells (anaemia), and abnormal numbers of other blood cells. The body may try to make blood cells in other organs, particularly the spleen, which often becomes enlarged. Symptoms develop gradually and can include tiredness and weakness (from anaemia), breathlessness, a feeling of fullness or discomfort in the tummy (from the enlarged spleen), weight loss, night sweats, fever, and easy bruising or bleeding or increased infections (from abnormal blood counts). Some people have no symptoms at first and are found to have it after a routine blood test. Myelofibrosis mainly affects older adults. It varies greatly in how it behaves — some people remain stable for a long time, while in others it progresses — and it is managed by haematology specialists.

How it is treated

Myelofibrosis is diagnosed and managed by a haematologist, usually involving blood tests, examination, and often a bone marrow test and specific genetic tests, which also help judge how the condition is likely to behave. Treatment is individualised, based on the symptoms, blood counts, risk assessment, age and general health, and its aims are to control symptoms, manage the blood counts and enlarged spleen, and, in selected people, to consider treatments that can modify the disease. For people with few symptoms and lower-risk disease, careful monitoring ("watch and wait") may be appropriate. For others, treatments may include medicines to improve symptoms and reduce the size of the spleen, treatments to manage anaemia (such as supportive measures or blood transfusions), and targeted medicines. For selected, usually younger and fitter, people with higher-risk disease, a stem cell (bone marrow) transplant is the only treatment that can potentially cure it, though it carries significant risks and is not suitable for everyone. Supportive care for symptoms and complications is important throughout. The reassuring message is that myelofibrosis is a condition that specialists manage in a tailored way — some people remain stable for a long time, treatments can control symptoms and improve quality of life, and, for selected people, a transplant offers the possibility of cure.