ReviewTherapeutic advances in hematology2021
Voxelotor: alteration of sickle cell disease pathophysiology by a first-in-class polymerization inhibitor.
Review in Therapeutic advances in hematology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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.
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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.
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Who cites it
16 citing papers in PubMed, 34 citations in OpenAlex.
- The evolving landscape of drug targets.Nature reviews. Drug discovery · 2026Review
- Two birds with one stone: An antibiotic hit blockingiScience · 2026Article
- The role of hemoglobin F in sickle cell disease complications: a descriptive secondary analysis.Blood vessels, thrombosis & hemostasis · 2026Article
- The influence of early intravenous immunoglobulin administration on the prognosis of severe hemolytic disorders in neonates.BMC pediatrics · 2026Article
- Updated Review of Current Therapeutic Approaches for the Management of Sickle Cell Disease.Cardiovascular & hematological disorders drug targets · 2026Review
- Article
- Promising role of voxelotor in managing sickle cell disease in children: a narrative review.Clinical and experimental pediatrics · 2025Article
- A review on disease modifying pharmacologic therapies for sickle cell disease.Annals of hematology · 2025Review
- CRISPR/Cas9 in the treatment of sickle cell disease (SCD) and its comparison with traditional treatment approaches: a review.Annals of medicine and surgery (2012) · 2024Review
- Article
- Hydroxyurea in the sickle cell disease modern era.Expert review of clinical pharmacology · 2024Review
- Sickle cell disease: combination new therapies vs. CRISPR-Cas9 potential and challenges - review article.Annals of hematology · 2024Review
- OcclusionChip: A functional microcapillary occlusion assay complementary to ektacytometry for detection of small-fraction red blood cells with abnormal deformability.Frontiers in physiology · 2022Article
- Practical Guidance for the Use of Voxelotor in the Management of Sickle Cell Disease.Journal of blood medicine · 2022Review
- Article
- Genome-based therapeutic interventions for β-type hemoglobinopathies.Human genomics · 2021Review
Corrections and comments
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Authors and funding
4 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sickle cell disease, despite its recognition as a severely debilitating genetic condition affecting hundreds of thousands of neonates throughout the world each year, was not a target of pharmaceutical research focus for most of its 100-year existence in the medical consciousness. This has changed in recent years as many novel therapeutics are currently under investigation, with three new disease-modifying drugs achieving FDA approval in the last 4 years. One of these drugs, voxelotor, is especially encouraging as an inhibitor of sickling for its ability to safely improve the chronic hemolytic anemia of sickle cell disease. This was demonstrated during all clinical phases of investigation by an average improvement in hemoglobin of greater than 1 g/dL, as well as statistically significant improvements in established markers of hemolysis. While anemia itself represents a potential cause of morbidity, it is more importantly a marker of the hemolysis known to cause the long-term vascular and organ damage that makes sickle cell disease so debilitating and frequently fatal early in life. Given the recency of the approval, there has not been sufficient long-term follow-up to demonstrate improvement in the chronic sequelae of sickle cell disease as a result of voxelotor-induced improvements in hemolytic anemia. There is hope, however, based on the experience with hydroxyurea improving morbidity and mortality
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.