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New MF Medication Effective but Dangerous

Brian Hendricks1 year ago

According to Medscape, a new pharmaceutical drug called fedratinib has recently been tested on people that suffer from Myelofibrosis (MF). The researchers found that the drug was effective on MF symptoms, but sometimes resulted in serious side effects.

Background Information About Myelofibrosis

According to the Mayo Clinic, Myelofibrosis is a serious bone marrow disorder. MF disrupts the body’s ability to produce blood cells. As a result, the bone marrow becomes scarred and weak.

MF is classified as a rare type of chronic leukemia that develops when blood cells experience genetic mutation. The mutation will eventually result in the loss of red blood cells and an abundance of white blood cells. This serious complication is called anemia.

Some common symptoms of Myelofibrosis include lethargy, an enlarged spleen, chronic pain, pale skin, night sweats, fever, and frequent bruising or bleeding.

About the Fedratinib Study

Fedratinib is a Janus Kinase (JAK2)-selective inhibitor, which means it prevents the activity of Janus Kinase enzymes in the body. When the JAK2 inhibitor reduces enzyme function, many Myelofibrosis patients experience relief from their symptoms.

Researchers from the Mayo Clinic in Rochester, Minnesota conducted a randomized controlled study to examine the potential benefits and risks of fedratinib. The study consisted of 289 people diagnosed with high-risk MF.

The study found that 36% of patients experienced relief after 24 weeks on 400 milligrams of fedratinib. In addition, 40% of patients responded well to 500 milligrams during the same time frame.

Serious Side Effects Associated With Fedratinib

Despite the proven efficacy of the drug, the researchers pointed out some rare but serious side effects. There were four cases of Wernicke encephalopathy during the study. According to medical professionals, Wernicke encephalopathy is a neurological disorder characterized by mental confusion, amnesia, and psychosis. Wernicke encephalopathy often develops as a result of a thiamine deficiency in the gut.

The risk of encephalopathy promptly put an end to the production of fedratinib. As a result, there remains only one FDA-approved treatment for MF. This drug is called ruxolitinib, and it works similar to fedratinib in that it is a selective JAK2 inhibtor.


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