Maureen Achebe, MD, MPH, Shares Extended Durable Data on Voxelotor for SCD

The latest results from HOPE trial open label extension showed that after 144 weeks, the majority of patients with sickle cell disease (SCD) taking voxelotor (Oxbryta) continued to see the positive results previously revealed in the trial, without any unexpected adverse events.

Maureen Achebe, MD, MPH, Assistant Professor of Medicine, Hematology, Brigham and Women's Hospital, presented the interim analysis of the open label extension of the HOPE trial at the 2021 American Society of Hematology (ASH) Annual Meeting & Exposition.

According to Achebe, there were no severe adverse events but there were some.

"The adverse events were pain crisis, which we expect in patients who have sickle cell disease, arthralgias, some abdominal upset, and minor adverse events that were reversible," she said.

The open label extension followed the HOPE trial. HOPE was a randomized control trial that examined the use of voxelotor, a molecule that increases hemoglobin's affinity for oxygen, currently indicated for patients with sickle cell disease and anemia.

"The HOPE trial was, I think, a remarkable study and that we were able to enroll patients onto the study in record time with positive results," Achebe said. "And the open label extension shows us that the improvements in hemoglobin that were achieved in HOPE are durable, and that the side effect profile, even over a longer period of time, has not expanded to include side effects that we didn't see on trial."

Watch the first segment of Dr. Achebe's interview with HCPLive®.