Watch-and-Wait in Myelofibrosis: Why Do We Do It?

If you’ve been diagnosed with myelofibrosis, your doctor might have mentioned the watch-and-wait approach for managing the cancer. 

If you're curious about when early treatment for myelofibrosis (also referred to as early intervention) is recommended, click here to learn more.

Myelofibrosis specialist Prithviraj Bose, MD, from MD Anderson Cancer Center, published a report explaining this strategy and when it may be appropriate. Read a summary of his insights below. 

What is Myelofibrosis, and How is Risk Assessed?

Myelofibrosis is often a slow-growing cancer where scar tissue forms in the bone marrow, disrupting blood cell production. This can lead to symptoms such as fatigue, night sweats, abdominal pain, and an enlarged spleen. The severity of symptoms and risks associated with the cancer can vary widely from person to person. 

Doctors classify myelofibrosis into four risk levels: 

• Low
• Intermediate-1
• Intermediate-2
• High

These levels are determined by factors such as age, genetic mutations, and symptom severity, and they play a critical role in guiding treatment decisions.

What is Watch-and-Wait for Myelofibrosis? What are its Benefits? 

The watch-and-wait approach involves closely monitoring myelofibrosis without starting treatment immediately. It’s typically recommended for people with low or intermediate-1 risk levels who have mild symptoms. This strategy doesn’t mean ignoring the cancer; instead, it’s about balancing the potential benefits and risks of treatment.

Myelofibrosis often progresses slowly and may remain stable for years. If the cancer symptoms are mild, watch-and-wait allows you to avoid unnecessary treatment-related side effects and maintain your quality of life. 

For example, treatments for myelofibrosis like the JAK inhibitor ruxolitinib (Jakafi by Incyte) can improve symptoms and slow the cancer’s progression, but they also come with potential side effects like anemia (low red blood cell counts) and thrombocytopenia (low platelet counts). 

If myelofibrosis is not yet interfering with your quality of life, delaying treatment minimizes exposure to side effects for as long as possible. 

When Should Myelofibrosis Treatment Begin?

While watch-and-wait is common for those with low and intermediate-1 risk myelofibrosis, it’s important to stay vigilant. Regular check-ups, blood tests, and symptom tracking allow your myelofibrosis specialist to determine the right time to start therapy.

For individuals with low-risk or intermediate-1 myelofibrosis who experience moderate to severe symptoms, Dr. Bose stated these patients would benefit from active treatment. 

For people with intermediate-2 or high-risk myelofibrosis, Dr. Bose suggests an allogeneic stem cell transplant when possible, preceded by treatment with a JAK inhibitor. For patients who cannot receive or do not want a transplant, early treatment with ruxolitinib is recommended to manage symptoms and improve survival, as demonstrated in the COMFORT-I study. 

To learn more about early treatment for myelofibrosis, click here.

Stay Informed and Proactive

Every person’s experience with myelofibrosis is unique. The watch-and-wait approach isn’t about inaction—it’s a thoughtful, personalized strategy. Work closely with your healthcare team, ask questions, and keep them informed of any changes in your symptoms. Together, you can create the best plan for managing myelofibrosis. 

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Sources: 

• Management of Patients with Early Myelofibrosis: A Discussion of Best Practices
• Long-term treatment with ruxolitinib for patients with myelofibrosis: 5-year update from the randomized, double-blind, placebo-controlled, phase 3 COMFORT-I trial
• Why Does Early Intervention in Myelofibrosis Matter?