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Voxelotor in sickle cell disease: There’s HOPE in the HOPE trial

Following receipt of an email from The NEJM, about the notable articles of 2019, one paper stood out for me – this involved the clinical phase 3 trial of voxelotor in sickle cell disease via the HOPE trial by Vichinsky et al. Perusing through the paper, I must tell you that there’s hope in the HOPE trial. Here are the highlights of this potential practice-changing clinical trial.

If you’re not aware yet, we’ve covered quite a lot of information about sickle cell disease here in an attempt to garner awareness among both the health care providers and the general public about one of the most common genetic disorders in the African population, and around the globe.
Sickle haemoglobin changes form with ease in the deoxygenated state. It is the root cause of all the complications of sickle cell disease. To prevent this process is to forget about all the sickle cell disease complications. They include hemolysis, anaemia, vaso-occlusive crisis and the devastating stroke.

Related article: Hydroxyurea in sickle cell disease: What You Need To Know: Part 1

However, the results exhibited by Voxelotor in this phase 3 multicenter, randomized trial can change the way we tackle sickle cell anaemia. Voxelotor is an HbS polymerization inhibitor, reversibly anchoring onto the haemoglobin molecules to prevent polymerization. It implies that the abnormal haemoglobin can deliver oxygen safely to the tissues without changing form after entering into a phase of deoxygenation.

During the HOPE trial, Voxelotor increased the levels of haemoglobin. It reduced indirect bilirubin, absolute reticulocyte count, percentage of reticulocytes and lactate dehydrogenase. They are all markers of hemolysis. The trial had a total of 274 participants who were randomized in a 1:1:1 for Voxelotor 1500 mg, 900 mg or placebo with an intention-to-treat analysis.

The increase in the haemoglobin levels and reduction in the markers of hemolysis were in line with the effects of inhibiting HbS polymerization. It is, therefore, indicated the potential to modify the disease by targeting the protein and forgetting about the gene.
However, the excitement should be put on hold until more trials are available – the drug gets approval from the relevant bodies around the globe like FDA. Nonetheless, the future is bright.


MBChB (MUK), Graduate Fellow, Department of Physiology, Makerere University Founder and Content Creator Peer reviewer, Associate Editor

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