Momelotinib May Reduce Transfusions and Improve Quality of Life

Momelotinib (Ojjaara, GSK) was FDA approved for people with myelofibrosis in September 2023. It can help mitigate common myelofibrosis symptoms by reducing the spleen size and increasing red blood cell levels, which is difficult to achieve with other therapies.

Recently, real-world studies from Spain and the United Kingdom have confirmed that momelotinib works well outside of clinical trials. In this article, we’ll cover what you need to know about these findings.

What is Momelotinib? 

Momelotinib is a small-molecule inhibitor that targets three key pathways involved in myelofibrosis:

• JAK1 and JAK2, which are proteins involved in abnormal blood cell growth and inflammation.
• ALK2, a protein that regulates hepcidin, a hormone controlling iron metabolism. High hepcidin can worsen anemia by blocking iron availability.

By addressing these processes, momelotinib helps manage not only the growth of abnormal blood cells and inflammation but also the anemia that often leads to transfusions and a low quality of life.

A Real-World Study of Momelotinib 

This was a real-world, observational study conducted in Spain. It involved 154 patients with myelofibrosis symptoms who had an enlarged spleen and anemia. These patients received momelotinib between March 2023 and July 2024 as part of a managed-access program.

Nearly 69% of patients had mutations linked to a higher risk of cancer progression, such as ASXL1 (the most common), U2AF1, SRSF2, EZH2, and IDH1 or IDH2.

Most patients had already tried other therapies, 91% of patients had ruxolitinib in the past, and 77% of patients had also tried medications that stimulate red blood cell production.

Before starting momelotinib:

• 93.5% of patients had symptoms
• 83% had an enlarged spleen
• The average hemoglobin level (a measure of red blood cells) was 8.0 grams per deciliter
• 65% were dependent on blood transfusions, needing about three units of red blood cells per month

What Were the Study Results?

After an average of five months on momelotinib, 75% of patients reported reduced fatigue, itching, and night sweats. They also improved their appetite and stomach discomfort, and stopped losing weight. More than 50% of patients experienced spleen shrinkage.

Momelotinib also helped patients increase their hemoglobin levels after one month. In 52% of patients who were dependent on transfusions, they no longer needed them. This improvement also lasted, and by 12 months, 44% of patients remained transfusion-independent. This improvement in anemia happened in both patients who were new to treatment and those who had already tried other therapies.

What Were the Side Effects of Momelotinib?

The most common side effects were diarrhea, low platelet counts, infection, nausea, and dizziness; however, most side effects were manageable, and only six patients stopped treatment due to side effects. 

This study gives real-world reassurance that momelotinib can:

• Reduce the need for blood transfusions
• Improve quality of life by easing difficult symptoms
• Be an option even after other treatments like ruxolitinib

Understanding how a new treatment performs outside of clinical trials can help patients and caregivers advocate for the best care. If anemia and symptoms like fatigue or spleen enlargement are still a problem, asking your doctor about momelotinib could open up new possibilities, especially since it is effective in both newly diagnosed patients and those who have already tried other options.

A Study Across 16 Centers in the UK 

This real-world study included 85 patients with myelofibrosis who received momelotinib outside of a clinical trial. The results supported the findings from Spain:

• Spleen reduction was seen in 43% of patients after three months.
• Symptoms improved in 24% of patients.
• Anemia responses (either becoming transfusion independent or gaining at least a 20 g/L increase in hemoglobin) were seen in: 36.7% of patients at 6 weeks, 43.8% at 3 months.
• The average hemoglobin increase was 10.8 g/L at 6 weeks and 9.2 g/L at 3 months.

Safety remained consistent, with 75% of patients continuing treatment. About 10% of patients experienced more serious side effects, including low platelets, fatigue, and elevated liver enzymes.

Learning From Real-World Studies Can Help You Become A Better Self-Advocate!

Understanding this data is important for patients' and caregivers' self-advocacy. By learning from international onset, you can learn what has worked for others and advocate for your best treatment options.

Real-world results also help you decide if momelotinib might be an option as a second-line therapy. If you are newly diagnosed and have anemia, it may be worth asking your doctor if starting with momelotinib is right for you.

Final Takeaway

Momelotinib offers a meaningful step forward in the care of people with myelofibrosis, especially those struggling with anemia. Real-world evidence supports what clinical trials suggested: this treatment improves symptoms, reduces the need for transfusions, and offers hope to those with few options left. As always, talk with your healthcare team about which treatment is right for your unique situation.

You can keep learning from more momelotinib articles here: 

• Momelotinib: A New Treatment Option For Myelofibrosis
• New Treatments for Improving Low Blood Cell Counts in Myelofibrosis Patients

We Need Your Help! Easily Contribute to Myelofibrosis Research

If you are living with myelofibrosis, we need your help improving patient outcomes for all by taking simple, anonymous surveys that contribute to real-world research in HealthTree Cure Hub®. Click the button below to get started! 

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Sources: 

• Real-World Outcomes of Momelotinib As an Alternative Therapy to Other JAK Inhibitors in Myelofibrosis Patients with Anemia
• Real World Efficacy and Safety of Momelotinib for Myelofibrosis: Evaluation of a UK-Wide Study Confirms 40% Anaemia Response Rate in a Non-Clinical Trial Cohort\