Observational studyBlood advances2026
Safety and effectiveness of voxelotor in individuals with sickle cell disease in the RETRO and PROSPECT US registries.
Observational study in Blood advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Not yet cited in PubMed.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Retrospective Data Collection and Analysis Study of Patients With Sickle Cell Disease (SCD) Who Have Been Treated With Oxbryta® (Voxelotor)
An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)
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0 citing papers in PubMed.
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
abstractTwo registry studies, RETRO and PROSPECT, were conducted to evaluate real-world safety and effectiveness outcomes in US participants with sickle cell disease (SCD) who received voxelotor as standard of care. RETRO collected retrospective data of participants aged ≥12 years at 9 US sites for ∼1 year after voxelotor initiation. PROSPECT collected retrospective and prospective data of participants aged ≥4 years at 24 US sites, with a 5-year follow-up planned. Both studies collected data for 1 year before voxelotor initiation. RETRO enrolled 216 participants. PROSPECT enrolled 265 participants before voxelotor was withdrawn from global markets in September 2024; of these, 260 participants received voxelotor. The mean ± standard deviation (SD) voxelotor treatment duration was 50.52 ± 25.1 and 143.2 ± 65.6 weeks in RETRO and PROSPECT, respectively. Safety analyses included all participants who received voxelotor. The mean ± SD observed change in hemoglobin from baseline was from 0.6 ± 1.6 to 0.8 ± 1.5 g/dL in RETRO (∼1 year) and from 0.1 ± 1.7 to 0.7 ± 1.5 g/dL in PROSPECT (54 months). After starting voxelotor, decreases were observed from baseline in hemolysis markers (absolute/percentage reticulocyte counts, total/indirect bilirubin) across all RETRO time points and most PROSPECT time points. Acute pain crisis (APC) was the most common SCD complication (annualized incidence rates before and after voxelotor, respectively: RETRO: 1.33 and 1.54; PROSPECT 4.78 and 3.15). No new safety findings were identified. Despite inherent limitations of registry studies, voxelotor treatment increased hemoglobin and decreased hemolysis markers in US clinical practice, with no evidence of increased frequency of APC. These studies were registered at www.ClinicalTrials.gov as NCT04930328 and NCT04930445.
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Registered trials
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