MEMPHIS, Tenn. — Patients with sickle cell disease will soon have a new treatment option to reduce pain crises caused by obstructed blood flow.
The FDA has approved use of the drug crizanlizumab based on the results of a clinical trial led by Kenneth Ataga, MD, Plough Foundation Endowed Chair in Sickle Cell Disease and director of the Center for Sickle Cell Disease at the University of Tennessee Health Science Center.
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FOX13 spoke to Ericka Matthews whose 5-year-old daughter was diagnosed with sickle cell when she was just 2.
“When you see your 5-year-old on the floor balled up because she's in so much pain and there's nothing you can do, that’s heartbreaking,” Matthews said.
She says her daughter’s days are spent taking medication, going to the hospital, or being in pain.
“For the rest of her life something she will have to go through for the rest of her life sickle cell,” she said
Dr. Ataga, who is also the director of the Memphis Consortium for Sickle Cell Disease and Non-Malignant Hematology Research, was the lead principal investigator of the multicenter, Phase II SUSTAIN trial. Part of a larger program of studies, the sustain trial was designed to test the safety and efficacy of crizanlizumab in reducing the frequency of vaso-occlusive crises , or pain crises, in adult and pediatric patients ages 16 years and older with sickle cell disease.
The study participants were recruited from 60 sites in the United States, Brazil and Jamaica.
Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped which restricts the flow of blood and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage.
According to the Centers for Disease Control and Prevention, sickle cell disease affects approximately 100,000 Americans.
Considered the clinical hallmark of the disease, sickle cell pain crises are unpredictable, severe events associated with life-threatening complications. They are triggered, in part, by multicellular interactions that form clusters of cells, which can block or reduce the blood flow to organs. Sickle cell pain crises are the main reason why individuals living with sickle cell disease go to the emergency room and are admitted to the hospital.
“Having sickle cell is just like having cancer, and that’s enough to think about,” Matthews said. “She’s only five.”
Matthews hopes to learn more about the new treatment so her daughter can feel less pain.
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