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What Is Ruxolitinib Used For?

Ruxolitinib is prescribed to treat intermediate-risk or high-risk myelofibrosis in adults. This drug works by blocking the action of certain enzymes that cause abnormal blood cells to form in areas other than the bone marrow. Ruxolitinib is used for relieving myelofibrosis symptoms and reducing the size of an enlarged spleen. There are no off-label (unapproved) uses for this medicine at this time.

An Overview of Uses for Ruxolitinib

Ruxolitinib (Jakafi™) is a prescription medication approved to treat people with intermediate-risk or high-risk myelofibrosis, a rare disease of the bone marrow. It is the first medication approved to treat this condition.
Myelofibrosis is a disorder of the bone marrow, the soft, spongy tissue found in the center of some bones. Normally, the bone marrow produces new blood cells, including:
  • Red blood cells, which carry oxygen throughout the body
  • White blood cells, which fight infections
  • Platelets, which help the blood to clot.
However, in people with myelofibrosis, this normal production of blood cells is disrupted. In addition, fibers inside the bone marrow thicken, resulting in scarring of the bone marrow, which further interferes with the ability of the bone marrow to make blood cells.
The liver and spleen begin to produce blood cells to compensate for the lack of blood cells produced by the bone marrow. This causes the liver and spleen to enlarge.
Myelofibrosis is normally a progressive disorder, which means people usually get worse over time. Some people may progress to a serious form of leukemia. In the early stages of the disease, the main symptom is extreme fatigue, although not everyone will experience fatigue in the early stages of the disease. As the condition progresses, symptoms increase and may include:
  • Tiredness and shortness of breath from severe anemia. due to low red blood cells
  • Easy bruising and bleeding, due to low platelets
  • Increased risk for infections, due to low white blood cells
  • Pain in the left upper abdomen (stomach), due to an enlarged spleen
  • An enlarged liver
  • Fever
  • Night sweats
  • Weight loss.
Oftentimes, the cause of myelofibrosis is not known. In these cases, the condition is known as primary myelofibrosis. Myelofibrosis can also occur as a complication of other conditions, including:
  • Polycythemia vera, which is a bone marrow disorder characterized by an overproduction of red blood cells
  • Essential thrombocythemia, which is a bone marrow disorder characterized by an overproduction of platelets.
Healthcare providers may categorize people with myelofibrosis as low-, intermediate-, or high-risk, based on a variety of factors that affect survival. Some of these factors include:
  • Age
  • Hemoglobin levels (a protein found in red blood cells that carries oxygen)
  • Blood cell counts
  • The presence of symptoms.
The risk categories roughly estimate expected survival, and can be used to guide therapy decisions.
Ruxolitinib is approved to treat people with intermediate-risk or high-risk myelofibrosis, including people with:
  • Primary myelofibrosis
  • Post-polycythemia vera myelofibrosis
  • Post-essential thrombocythemia myelofibrosis.
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Ruxolitinib Drug Information

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