In Case You Missed it: Myelofibrosis Updates

Each year, during Blood Cancer Awareness Month, HealthTree helps raise awareness about blood cancers and chronic conditions like myelofibrosis.

Myelofibrosis (MF) is a chronic condition that can cause anemia, spleen enlargement, fatigue, and other life-altering symptoms.

 In recent years, research has accelerated, offering new treatment approaches, supportive care strategies, and insights into health disparities. Below, we summarize the latest updates that could help improve outcomes and quality of life for people living with MF.

Treatment Option for Pre-Fibrotic and Low-Risk Myelofibrosis

A recent study highlights the potential of ropeginterferon alfa-2b (Besremi) as a safe and effective option for people with early-stage or low-risk MF, including pre-fibrotic MF. Among the 71 patients studied, most achieved normalized blood counts within a year, and many also saw reductions in spleen size, symptom relief, and decreases in genetic mutation levels linked to the disease. Side effects were generally manageable, with no cases of leukemia or major clotting events reported. These findings suggest ropeginterferon could fill an important gap for individuals needing blood count control at earlier stages of MF.

This development is particularly meaningful because current standard therapies are mainly used for moderate or high-risk MF, leaving those with earlier stages fewer treatment options.

Momelotinib May Reduce Transfusions and Improve Quality of Life

Momelotinib (Ojjaara, GSK), was FDA-approved in 2023 and is showing strong real-world results. Recent studies from Spain and the UK confirm that momelotinib not only shrinks enlarged spleens and relieves symptoms such as fatigue, itching, and night sweats, but also reduces the need for blood transfusions. Some patients were able to stop transfusions altogether.

Unlike many available therapies, momelotinib addresses both symptoms and anemia, making it a valuable option for newly diagnosed individuals as well as those who previously tried other drugs like ruxolitinib.

What are the Benefits of Elritercept for Myelofibrosis? Results from Phase 2 RESTORE Trial

Anemia remains one of the most challenging complications in MF care. The phase 2 RESTORE trial evaluated elritercept, a therapy designed to restore blood cell production without worsening anemia, unlike some JAK inhibitors.

Patients treated with elritercept experienced higher hemoglobin levels, reduced transfusion needs, improved platelet counts, smaller spleens, and better symptom control. The therapy was well-tolerated, and the FDA has already granted it Fast-Track designation, showing recognition of its potential to address a major unmet need.

Can Bone Marrow Fibrosis Be Reversed?

Current treatments primarily manage symptoms rather than address the underlying disease. A new therapy, PXS-5505, aims to change that by targeting enzymes that drive bone marrow scarring.

In early trials, combining PXS-5505 with ruxolitinib proved safe and well-tolerated. Early signs suggest this approach may reduce or even reverse fibrosis, raising hope that future treatments could change the natural course of MF, not just its symptoms.

Is CAR T-cell Therapy Possible for Myelofibrosis?

Researchers are also looking beyond drug therapies to explore CAR T-cell therapy for MF. This cutting-edge treatment modifies a patient’s own immune cells to fight cancer. While CAR-T is already approved for other blood cancers, MF is more complex because it lacks a clear target that distinguishes cancerous from healthy blood cells.

Laboratory studies show promise in targeting the calreticulin mutation, present in about one-third of patients. Human trials are expected within five years, marking a potential step toward harnessing the immune system in MF treatment.

Myelofibrosis Fatigue: Causes and Ways to Manage

While new therapies offer hope, daily symptom management remains a major challenge. Fatigue is one of the most common and disruptive symptoms of MF, often caused by anemia, inflammation, poor sleep, or emotional health concerns.

Practical steps such as light exercise, better sleep routines, emotional support, and treatments like ruxolitinib can help ease fatigue. This article shares helpful strategies to help people with fatigue and myelofibrosis. 

Assessing Racial and Ethnic Disparities and Outcomes in Myelofibrosis

Beyond medical advances, researchers are also uncovering important disparities in MF care. The Chicagoland study of nearly 500 patients found that non-Hispanic Black and Hispanic individuals were less likely to receive JAK inhibitors or bone marrow transplants compared to White patients.

While genetic mutation patterns were similar across groups, differences in access to therapies and higher complication rates may contribute to poorer outcomes. These findings highlight the need for equitable access to treatments as progress in MF continues.

Looking Ahead in Myelofibrosis Care

This Blood Cancer Awareness Month, we honor the resilience of patients and caregivers while also recognizing the exciting progress in research. From new drugs like ropeginterferon, momelotinib, and elritercept to emerging therapies like PXS-5505 and CAR T-cell approaches, science is expanding options for people living with MF. At the same time, addressing common symptoms and reducing disparities in care are essential to ensuring these advances benefit everyone.

The road ahead brings hope for treatments that may one day reverse fibrosis or transform MF into a manageable condition, and responsibility to ensure that all people can access these innovations.

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