Sally Abou Melhem , Office of Communications, firstname.lastname@example.org
An article on the randomized trial of Voxelotor in sickle cell disease (SCD) was recently published in The New England Journal of Medicine, co-authored by Dr. Miguel R. Abboud, professor and chairman at the Department of Pediatrics and Adolescent Medicine at AUBMC. The study concluded that Voxelotor, which is a therapeutic agent developed by Global Blood Therapeutics, is capable of significantly improving anemia in people with SCD, with no toxicity or negative side effects. Voxelotor is the first agent to target the sickling process by altering the affinity of hemoglobin for oxygen.
Sickle Cell Disease is an inherited and lifelong disease that manifests in early childhood as painful episodes, infections, strokes, and chronic anemia. It continues to cause serious complications throughout the person's life, having an impact on their entire body and damaging multiple organ systems.
This disease, which is common in Lebanon and the region, affects hemoglobin, the red protein that transports oxygen in red blood cells. People with this disorder have abnormal hemoglobin molecules that cause red blood cells to change from their normal disc shape to a crescent, or sickle, shape. These red cells have a short life span and can occlude blood vessels.
Children and adults with sickle cell disease require close follow-up and sufficient resources, as the disease puts them at risk of having unpredictable pain crises that require hospital admission and treatment with ﬂuids and morphine; strokes and severe problems with different organs such as the lungs and eyes; as well as an increased risk of death due to infections.
Early diagnosis, supportive care, and prevention and management of infections have improved the survival rates of patients with SCD. However, so far, hydroxyurea is the only drug approved for treating this disease and proven effective in decreasing pain crises and prolonging survival. In addition, methods have been developed to predict and prevent strokes in children with SCD.
The HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization) study, funded by Global Blood Therapeutics, was a phase 3 randomized, multicenter, international, placebo-controlled trial of Voxelotor, involving children and adults with sickle cell disease.
Findings show that the drug was effective in increasing patients' hemoglobin levels. This was achieved by inhibiting Hemoglobin S polymerization, which is the process that leads to the deformation of the structure of red blood cells. The treatment increases hemoglobin's affinity for oxygen, which in turn reduces polymerization, red blood cell sickling, rupture and destruction, as well as anemia.
“Voxelotor significantly increased hemoglobin levels and reduced markers of hemolysis. These findings are consistent with the inhibition of HbS polymerization and indicate a disease-modifying potential," the study states.
“We are now looking further to see whether this treatment will improve other parameters of SCD, and preliminary findings suggest that it will," explains Dr. Abboud.
The Sickle Cell Program at AUBMC
Comprehensive care in a specialized center for people with SCD, regardless of their financial situation, is important. It provides equal access to healthcare, prevents complications, treats existing problems caused by the disease, as well as educate families. This has been shown to prolong the survival of patients. The Sickle Cell Program and fund at AUB's medical center provides such care, in addition to the latest advances in SCD treatment, to around 350 patients.
Established in 2012, and with the help of volunteers and donors, doctors and healthcare providers, the program has been able to offer state of the art care for financially disadvantaged patients. It focuses on early detection and prevention, and provides regular follow-up for physical assessment, counseling, blood tests, imaging, and adequate treatment.
“Our SCD program has been recognized nationally and internationally for its achievements. Fundraising for our SCD fund is critical for continued excellence and patient care," Dr. Abboud said.