CHAPEL HILL, NC – Kenneth Ataga, MD, professor of medicine at UNC School of Medicine, was recently featured in a “Conversations with Innovators” video segment produced by the American Society of Hematology (ASH).
Ataga’s lab has been studying crizanlizumab as a therapy for patients with sickle cell disease, which affects an estimated 80,000 people in the United States. The video coincides with a column in The Hematologist about Ataga’s crizanlizumab research, penned by Vanderbilt Medical Center’s Michael DeBaun, MD, MPH, a professor of pediatrics and medicine, and director of the Vanderbilt-Meharry Center for Excellence in Sickle Cell Disease.
In the video, Ataga shares his lab’s novel strategies in dealing with acute pain events in patients with sickle cell disease, one of the world’s most common genetic diseases, which affects millions of people worldwide. Sickle cell disease is an inherited blood disorder that causes anemia and complications due to sickle-shaped red cells and white blood cells adhering to small vessels, which block blood flow to downstream organs. This results in painful crisis events, which leads to repeated hospitalizations, and can lead to multi-organ dysfunction and death.
“Painful crises in patients with sickle cell disease presents the primary reason why patients with sickle cell disease would typically seek healthcare providers,” Ataga, the director of UNC’s Comprehensive Sickle Cell Program, said. “There are two basic approaches to treating painful crisis. One of them is an intervention-type approach, in which physicians try to shorten the duration of a painful crisis once it’s ongoing.”
Currently, there aren’t any drugs that have been approved by the Federal Drug Administration (FDA) to intervene during a painful crisis, though there are several trials underway, Ataga said.
“The treatment is mainly supportive,” Ataga said. “We give patients pain medications, IV fluids, oxygen perhaps. Depending on what complications patients might have, we might need to give them blood transfusions.”
The second method of treatment involves a preventive approach, using a drug intended to prevent the painful crisis from occurring. While it has shown to be efficacious – patients require fewer hospitalizations and fewer blood transfusions – patients continue to get sick and patients still have a lower life expectancy than the general population, Ataga said.
“It’s clear that we need more treatments,” Ataga said.
In Ataga’s yearlong clinical trial studying crizanlizumab, patients were divided into three groups: those who received a high dose of crizanlizumab, those who received a low dose and those who received a placebo.
“We evaluated a variety of efficacy endpoints,” Ataga said. “What we found was that when we compared the median rates of painful crises per year, there was a 45 percent reduction in in the median rates of painful crises in the patients who got the high-dose treatment compared to patients who got placebo.
“This was not only clinically meaningful, but it is also statistically significant, so we were quite pleased with that.”