Trial reportEuropean journal of haematology2025
Real-World Evidence of Crizanlizumab Showing Reductions in Vaso-Occlusive Crises and Opioid Usage in Sickle Cell Disease.
Trial report in European journal of haematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03720626 (Managed Access Programs for SEG101, Crizanlizumab), which is not on this map. Cited by 1 paper.
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.
Managed Access Programs for SEG101, Crizanlizumab
Who cites it
1 citing paper in PubMed.
- Drug development for sickle cell disease: repeated setbacks, yet there remains an optimistic outlook for future breakthroughs.Hematology, transfusion and cell therapyArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
Abstract
objectiveAccess to crizanlizumab, a disease-modifying therapy for sickle cell disease (SCD), was provided through a managed access program (MAP, NCT03720626). The present analysis evaluated the impact of 12 months of crizanlizumab treatment on vaso-occlusive crises (VOCs), and on the use of opioids for VOC-related pain relief, in patients with SCD from the MAP.
methodsFrom June 2018 to January 2023, 112 patients with a history of recurrent VOCs completed 12 months of crizanlizumab (5 mg/kg) treatment and were monitored for adverse events (AEs).
resultsCrizanlizumab led to reductions of 18.0% and 36.2% in the proportions of patients having ≥ 1 home- and ≥ 1 healthcare-managed VOCs. Median absolute changes (interquartile range) from baseline in the rates of home- and healthcare-managed VOCs were -3.0 (-6.0, -1.0) and -2.0 (-4.0, 0), respectively. Data stratified by genotype and prior hydroxyurea use showed similar reductions in VOC rates. A 35.5% reduction in the proportion of patients requiring opioids was noted. AEs were consistent with earlier reports, and no new safety concerns were identified.
conclusionsCrizanlizumab demonstrated potential benefits in attenuating VOC episodes, irrespective of SCD genotype and prior hydroxyurea use, and in lowering opioid usage. The safety of crizanlizumab was consistent with earlier reports.
trial registrationThe MAP has been registered at ClinicalTrials.gov with the ID, NCT03720626.
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Registered trials
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