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With World Sickle Cell Day approaching, Dr. Titi Fasipe reflects on the developments within the field, such as the continued conversation of disparities and the increased comfort level with new therapies.
Titilope Fasipe, MD, PhD, Assistant Professor, Department of Pediatrics, Texas Children's Cancer & Hematology Centers, Baylor College of Medicine, joined HCPLive for an interview to talk about World Sickle Cell Day, Sunday June 19th, and reflected on the developments within the field that took place over the last year.
"There continues to be more progress–the drive for legislative advocacy, for instance–as well as ongoing clinical trials to help understand the disease better," Fasipe said.
An exciting topic has been the progress around gene therapy and the clinical trials taking place, according to Fasipe. A topic that she elaborated on later in the interview.
"The other exciting things have been various organizations have continued to champion and promote sickle cell disease," she said. "And so, we have a lot to talk about in that regard, too. There's a new podcast that's out on sickle cell disease, there have been new reports that continue to shine a light on the disparities of sickle cell disease."
The conversation about the disparities of this rare inherited blood disorder is not a novel one. However, the continuation of the conversation warrants progress.
"When I think back to this time last year, I always feel like I still have learned something new or met a group of new people," she said, "and it's just a growing body of individuals who are invested in working in sickle cell disease. So, it's exciting to see. I'm thrilled."
While there were no new treatments to emerge this year, treatment options for the disease have drastically evolved within the last 5 years, with 3 of the 4 sickle cell drugs becoming available to patients.
In 1988 hydroxyurea was the first approved treatment for sickle cell disease.
"Now we have 4 disease modifying drugs for sickle cell disease," she said, "and so because the other ones are relatively new, we are gradually learning how they help the body and how they help sickle cell disease."
"After their approval, there's, of course, a post-marketing phase–they call it– where we observe how the drug works in everyday life with more people than what was seen in the clinical trial. Some of these studies are ongoing, and we're awaiting their publications anxiously."
Fasipe emphasized that sickle cell is a very complicated disease and that the new therapies don't serve as replacements for hyrdoxyurea, which does "wonders for sickle cell in most individuals." However, that treatment alone isn't enough to address all the elements of the disease.
"When I think of the new therapies, I actually think of more than one approach, or what they call the multimodal approach to therapy," she explained. "So, you're targeting sickle cell from many angles and the goal is that you're going to help a person become healthier and live longer."
She said that the upcoming years will bring physicians an even higher comfort level and understanding while utilizing the treatment in their practice.
In the next segment of the interview, Fasipes discussed the patient side, and how along with a provider's confidence, a patient's adherence to the treatment plays a major role in treatment success.