Real-world US Experience with Voxelotor for Sickle Cell Disease: A Plain Language Summary of the RETRO and PROSPECT...

What is this summary about? This summary describes two studies in people with sickle cell disease, or SCD. Both studies looked at a medicine called voxelotor, also known as Oxbryta^®, which was previously available for treating people with SCD. This is a summary of an article published in Blood Advances. What were the results of the studies? In the RETRO and PROSPECT studies, researchers looked at people with SCD taking voxelotor in everyday, real-world medical practice. In other words, they took voxelotor as part of their usual care when it was available for prescription in the United States. In RETRO, 216 participants were enrolled and the length of voxelotor treatment was about 1 year, on average. In PROSPECT, 265 participants were enrolled, and the length of voxelotor treatment was almost 3 years, on average. After taking voxelotor, participants had increases in hemoglobin, the protein in red blood cells that carries oxygen. Their red blood cells also broke apart less quickly when taking voxelotor. The most common symptom related to SCD was acute pain crisis, but participants did not experience it more often after starting voxelotor. What do the results mean? All medicines are tested in clinical trials before they are approved as treatments. However, it is always important to study a medicine in real-world medical practice as well. In RETRO and PROSPECT, the effects of voxelotor were like the effects seen in the clinical trials, and researchers did not see any unexpected symptoms related to SCD or unexpected side effects related to the study drug.