GREENVILLE, N.C. (WNCT) – Sickle cell disease affects millions of people worldwide.
It’s an inherited red blood cell disorder that causes unpredictable pain crises.
Majara Mooring has been living with sickle cell disease for more than thirty years.
“Sickle cell is very unpredictable it has affected my work, spending time with my loved ones…I even describe my sickle cell pain as a large dump truck being placed on top of my body,” she says.
The disease mainly affects African Americans here in the United States.
Dr. Darla Liles is a Professor of Medicine and Division Chief at the Brody School of Medicine at East Carolina University.
Dr. Liles explains the name behind the disease, “the red blood cells become sickle-shaped but all of the blood cells become stickier and they stick inside of the blood vessel wall and this causes many complications.”
Dr. Liles was involved in a clinical trial to help patients with the disease receive medical attention.
Patients living with sickle cell disease have to overcome barriers from day today.
“I’ve missed holidays, I’ve missed birthdays I have been in and out of the hospital I think pretty much all my adolescent years…I find myself balled up in a fetal position at times, crying, screaming, yelling, just looking for help at that moment,” says Majara.
Now a new medical treatment approved by the FDA is helping patients like Majara, live with this disease.
“It’s called ADAKVEO and it’s the first therapy that is targeted to that stickiness of the blood cells, to prevent that and so unlike old therapies that were pain medications to mask the pain this actually attacks that,” explains Dr. Liles.
Patients from the clinical trial experienced fewer pain crises by almost 50% and they were given the medicine as an IV infusion once a month for a whole year.
Some side effects were fairly mild including things like nausea, back pain, and fever.
Majara emphasizes the importance of a doctor-patient relationship.
“I would say build a relationship with your physician. Get to know your disease, I say your disease because it’s something that we’re living with day today. Ask plenty of questions, if you don’t know, there’s nothing wrong with asking,” she explains.
Dr. Liles encourages anyone living with sickle cell disease to talk with their physician to determine if this new treatment is the right therapy for you.
You can access more information on the medication ADAKVEO here.