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ruxolitinib (Jakafi, Jakavi, Opzelura)
Targeted Therapy

How it is administered

Ruxolitinib is available as oral tablets for systemic use and as a topical cream for skin conditions. For blood cancers like Chronic Myelomonocytic Leukemia (CMML), the oral tablet form is used. The tablets are taken by mouth, usually twice daily, with or without food. The dose depends on your platelet count and other blood counts, and your doctor will adjust the dose as needed.

For those unable to swallow tablets, ruxolitinib tablets can be suspended in water and given through a nasogastric tube. The topical cream form (used for skin conditions) is not used for blood cancers.

How it works

Ruxolitinib is a type of medication known as a Janus kinase (JAK) inhibitor. It works by blocking the activity of certain enzymes called JAK1 and JAK2. These enzymes are involved in signaling pathways that control the production and growth of blood cells and immune system function.

In blood cancers such as myeloproliferative neoplasms (which include diseases similar to CMML), abnormal JAK signaling can lead to excessive or abnormal blood cell growth. By inhibiting JAK1 and JAK2, ruxolitinib helps to reduce the abnormal growth of blood cells, decrease inflammation, and improve symptoms such as enlarged spleen and fatigue.

Ruxolitinib also helps regulate the immune response, which can be beneficial in conditions where the immune system is overactive or attacking the body’s own tissues.

Common side effects

  • Thrombocytopenia (low platelet count)
  • Anemia (low red blood cell count)
  • Neutropenia (low white blood cell count)
  • Bruising
  • Dizziness
  • Headache
  • Diarrhea
  • Infections (including bacterial, viral, and fungal)
  • Weight gain
  • Increased cholesterol levels

If you experience unusual bleeding, signs of infection (fever, chills), or other concerning symptoms, contact your healthcare provider promptly.

Who Should take it

Ruxolitinib is approved for adults with certain blood cancers, specifically intermediate or high-risk myelofibrosis (a related myeloproliferative neoplasm), polycythemia vera (in adults who do not respond to or cannot tolerate hydroxyurea), and for steroid-refractory acute or chronic graft-versus-host disease in patients 12 years and older.

While ruxolitinib is not specifically approved for Chronic Myelomonocytic Leukemia (CMML), it may be considered in some cases where CMML overlaps with myeloproliferative features, or in clinical trials. It is important to discuss with your doctor whether ruxolitinib is suitable for your specific type of blood cancer.

Who should not take it

There are no absolute contraindications listed for ruxolitinib, but it should be used with caution in certain groups. Patients with severe low blood counts (especially platelets or neutrophils) may not be able to start ruxolitinib until these counts improve.

People with active, serious infections should not start ruxolitinib until the infection is under control. Caution is also advised in patients with a history of tuberculosis, hepatitis B, or other chronic infections.

Pregnant or breastfeeding women should not use ruxolitinib, and it should be avoided in patients with severe liver or kidney impairment unless specifically advised by a doctor.

Commonly used with

Ruxolitinib may be used alongside other supportive treatments such as blood transfusions for anemia or low platelets. In some cases, it is used with other medications to manage symptoms or complications of blood cancers. For graft-versus-host disease, it may be used with corticosteroids or other immunosuppressive drugs.

It is important to tell your doctor about all medications you are taking, as ruxolitinib can interact with drugs that affect liver enzymes (such as strong CYP3A4 inhibitors or inducers).

Commonly tested with

Ruxolitinib has been tested in combination with other therapies in clinical trials, especially for myeloproliferative neoplasms and graft-versus-host disease. It has been studied with hydroxyurea, corticosteroids, and other immunosuppressants.

If you are considering participation in a clinical trial or combination therapy, your doctor will discuss the risks and benefits with you.