What type of study is this?

This is a Human Clinical Trial study.

What question did this study set out to answer?

To evaluate the real-world effectiveness and safety of voxelotor in patients with sickle cell disease before its withdrawal.

December 8, 2025Blood

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To evaluate the real-world effectiveness and safety of voxelotor in patients with sickle cell disease before its withdrawal.
Utilized data from the ASH Research Collaborative Data Hub aggregating EHR from 14 SCD centers.
Included patients with PI-attested diagnoses from 2015-2024 who were on voxelotor for ≥90 days/year.
Conducted longitudinal analysis comparing clinical and lab data pre-treatment and on-treatment.
Employed random effects repeated measures models for analyses.
Among 388 patients treated with voxelotor, median Hb increased by 0.15 g/dL without significant changes in hospitalizations or complications.
No clear signal for harm was identified in 614 patient-years of voxelotor exposure.
Increase in Hb was more notable among patients with HbSS genotype.

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Real-world analysis shows voxelotor safety and marginal Hb increase in sickle cell disease, indicating limited clinical benefits.

Quinn et al. (2025) studied this question.

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