Available Treatment Options for Myelofibrosis


An overview of the currently available treatment options in myelofibrosis.

Case: A 67-Year-Old Man With Myelofibrosis

Initial presentation

RF is a 67 y/o man who visits his primary care physician for his yearly checkup. He reports having fatigue and bone pain for the last few months. More recently, he’s been complaining of abdominal discomfort which he thinks is probably related to the big Thanksgiving dinner party, he and his wife hosted.

He enjoys spending time working on antique cars in his garage, but his wife says he becomes tired after just an hour.

RF has not noticed any changes in his eating habits, but his wife mentions he doesn’t eat as much as he used too. Upon examination, RF has lost close to 13 lbs. since this last yearly exam.


Primary myelofibrosis diagnosed about 1 year ago

Diabetes (controlled with diet and exercise)


SMH: Smoked 3 packs/day but quit 5 years ago; he drinks occasionally

PE: abdominal exam reveals spleen palpable 5cm below left coastal margin, visible bruising

Initial Labs/Workup:

Platelet count: 184 x 109/L

Hgb: 10g/dL

WBC: 34 x 109/L

Bone Marrow Biopsy

Shows an increase in megakaryocytes

Bone Marrow Fibrosis

Megakaryocyte Atypia

Molecular testing

JAK-V617F mutation: positive

Initial treatment:

Patient was started on ruxolitinib 10mg BID when initially diagnosed

Current Presentation/Labs:

One year later, RF reports increasing fatigue and abdominal pain

Current Labs:

Platelet count: 57 x 109/L

Hgb: 8g/dL

WBC count: 29 x 109/L

Raajit Rampal, MD, PhD: What are our available treatment options for this patient at this point? There are a couple of things to think about. With ruxolitinib, many patients eventually do have treatment failure, which can be disease progression or intolerance of the medication. That can happen over a number of years, but it does happen to a substantial percentage of patients. The scenario that is being put forth in this vignette is not atypical. What are the options? Well, it's important to remember that historical data has shown that once patients come off of ruxolitinib, historically their outcomes are poor, with a median survival of about 14 months. Oftentimes this does coincide with the clonal evolution of the disease. Now, there are data to support other lines of therapy, fortunately. These are relatively recent developments. One option could be fedratinib, which was studied in the JAKARTA2 trial. In JAKARTA2, patients had previously been on ruxolitinib and went on to fedratinib. In that study, approximately 30% of patients went on to have a 35% spleen volume reduction or to have a symptom burden reduction by 50% or more. That study was limited to patients with a platelet count of 50,000 or higher. However, more recently the drug pacritinib was approved for treating myelofibrosis. Importantly, this is a drug that can be considered for patients with cytopenic myelofibrosis, including patients with profound thrombocytopenia.

Transcript edited for clarity.

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