Managing Myelofibrosis: Breakthrough Drug for Anemia


LOS ANGELES, Calif. (Ivanhoe Newswire) – Your blood accounts for about eight percent of your total body weight. It’s vital for life, but when cancer happens in the blood, it can become life-altering and even life-threatening. Ten percent of all cancers are blood cancers, one of which is myelofibrosis that affects about 25,000 people in the United States. There are few drugs approved for treating it. But now, the FDA has just approved one that helps patients with anemia – a devastating side effect of myelofibrosis.

Andrew and Esther Schorr are on a mission – spreading the word about myelofibrosis – something they know all too well about.

“I had some pain in my legs, turned out to be some blood clots, which was a shocker,” Andrew recalls.

Andrew was diagnosed with the rare blood cancer in 2011. At the time, there were no drugs for myelofibrosis. He religiously worked out, ran eight marathons, was a world traveler, but now, he struggles with exhaustion.

“When you’re anemic, you’re not getting the oxygen circulating around your body with your blood and you can be dead tired,” Andrew explains.

City of Hope hematologist oncologist, Haris Ali, MD tells Ivanhoe, “All the patients with myelofibrosis can have anemia at some point in their disease.”

Dr. Ali says patients have limited treatment options. Usually, a patient is anemic and they can’t get blood transfusions without significant risks. More than 30 percent are forced to quit MF treatment. Now, the FDA has approved the first treatment for anemia to help MF patients.

“So, OJJAARA is a JAK inhibitor. It actually targets that pathway to help with the symptoms and also with the anemia,” Dr. Ali explains.

The once-a-day pill helps to treat anemia so patients can get back on their treatment plans. Andrew is hopeful that this is the first of many for people struggling with this disease.

“I’m hoping for another 30 years out of this guy. Are you kidding?!,” expresses, Andrew’s care partner, Esther.

Ojjaara is the fourth drug to ever be approved to treat myelofibrosis, but is the only one that helps the anemia these patients suffer from. The main side effects are low platelet counts, dizziness, diarrhea and nausea.

Contributors to this news report include: Marsha Lewis, Producer; Matt Goldschmidt, Videographer; Roque Correa, Editor. 

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REPORT:        MB #5335

BACKGROUND: Myelofibrosis is a rare and chronic bone marrow disorder characterized by the abnormal production of blood cells and the development of fibrous tissue in the bone marrow. It falls under the category of myeloproliferative neoplasms, a group of conditions where the bone marrow produces too many blood cells. Bone marrow scarring also can create platelets from a low number of blood clotting cells. Myelofibrosis affects one per 100,000 people. It can occur at any age but it is most common in people aged 60 to 70. The primary cause of the rare disorder is still currently unknown. As the disruption of normal blood cells gets worse, symptoms include increased tiredness, pain or sense of fullness below the ribs, easy bruising, easy bleeding, excessive sweating, fevers, and bone pain.


DIAGNOSING: Diagnosing myelofibrosis involves a thorough process that combines clinical evaluation, laboratory tests, imaging studies, and a definitive bone marrow biopsy. Given the complexity of the disorder, a comprehensive approach is essential. The initial step involves a detailed medical history and physical examination. Routine blood tests are conducted to assess the levels and characteristics of blood cells and a bone marrow aspiration and biopsy are crucial for confirming the diagnosis of myelofibrosis. These procedures involve the extraction of a small sample of bone marrow from the hipbone or another large bone. Cytogenetic studies may be performed to evaluate the chromosomal changes in the bone marrow cells and imaging tests may be conducted to assess the size of the spleen and liver. After diagnosis, regular monitoring is crucial to assess disease progression and treatment response.


NEW TECHNOLOGY: The FDA recently approved the newest technology, the JAK inhibitor supported by phase III trials. Momelotinib is the fourth JAK inhibitor to be approved by the agency for myelofibrosis. This development is paving the way for new treatments because while JAK inhibitors were the standard treatment for myelofibrosis the only available option for patients with a hemoglobin level lower than 10 g/dL. The medication that was once-daily was approved after passing through two trials.



Zen Logsdon

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