What type of study is this?

This is a Human Clinical Trial study.

December 8, 2025BloodOpen Access

Real-world experience of voxelotor treatment in patients with sickle cell disease

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Authors

CHCaroline Horne ASAhmed Salem MRMyra Robinson

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Implication

Observational analysis reveals voxelotor improves hemoglobin and reduces hemolysis in sickle cell disease patients, indicating notable toxicity patterns.

Key Points

Hemoglobin increased significantly by a median of 2.0 g/dL in sickle cell disease patients treated with voxelotor, and most reported adherence despite adverse events.
The median time on voxelotor was 20.1 months, with notable GI toxicity reported in about a third of patients, showing a trend in adverse event occurrences.
Retrospective and prospective observational analysis revealed improvements in lab parameters, including total bilirubin and lactate dehydrogenase levels.
Findings underscore the efficacy and safety of voxelotor in managing sickle cell disease, reinforcing the need for future assessments post-market withdrawal.

Cite This Study

Horne et al. (2025) studied this question.

synapsesocial.com/papers/69362f514fa91c937236d990 https://doi.org/10.1182/blood-2025-2948

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Implication

Observational analysis reveals voxelotor improves hemoglobin and reduces hemolysis in sickle cell disease patients, indicating notable toxicity patterns.

Real-world experience of voxelotor treatment in patients with sickle cell disease

Discussion

Member takes

Implication

Key Points

Cite This Study

Real-world experience of voxelotor treatment in patients with sickle cell disease

Discussion

Member takes

Implication

Key Points

Cite This Study

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