Targeted Therapy for Myelofibrosis
Targeted therapy for myelofibrosis is not currently considered disease modifying. Rather, targeted therapy is considered supportive care which helps with the myelofibrosis symptoms and blood counts. In myelofibrosis, targeted therapy drugs called JAK inhibitors block abnormal signals in the JAK-STAT pathway. This pathway is often overactive in myelofibrosis and can lead to problems such as enlarged spleen, fatigue, anemia, and other symptoms.
These are the targeted therapy drugs that are used most often in supportive care for myelofibrosis. New drugs become available all the time, so this may not be a complete list.
Please note: The side effects listed here do not represent a comprehensive list. Each targeted therapy drug has its own set of side effects and ways of working. It's important to discuss all potential side effects of a drug with your healthcare team. Be sure to tell your healthcare team about any side effects you do have.
All Available Treatments
Treatment Type
JAK inhibitors
Drug Names
Potential Side Effects
Low blood counts
Nausea/vomiting
Fatigue/weakness
Diarrhea
Blood sugar changes
Important Things to Know
- Helps reduce spleen size and improve symptoms such as itching, night sweats, and bone or muscle pain.
- Can work in patients with or without a JAK2 mutation.
- Regular blood tests are needed to monitor blood counts and liver function.
- Tell your care team about any side effects or new symptoms.