Promising role of voxelotor in managing sickle cell disease in children: a narrative review
Sickle cell disease (SCD) is characterized by chronic hemolytic anemia and intermittent vasoocclusive crises. To date, 4 disease-modifying drugs have been approved for the treatment of SCD: hydroxyurea (an S-phase inhibitor), L-glutamine (an amino acid), crizanlizumab (a P-selectin inhibitor), and v...
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| Format: | Article |
| Language: | English |
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The Korean Pediatric Society
2025-02-01
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| Series: | Clinical and Experimental Pediatrics |
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| Online Access: | http://www.e-cep.org/upload/pdf/cep-2024-00500.pdf |
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| _version_ | 1825206623260377088 |
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| author | Amit Agrawal Gaurav Jadon Japna Singh Dalwinder Janjua |
| author_facet | Amit Agrawal Gaurav Jadon Japna Singh Dalwinder Janjua |
| author_sort | Amit Agrawal |
| collection | DOAJ |
| description | Sickle cell disease (SCD) is characterized by chronic hemolytic anemia and intermittent vasoocclusive crises. To date, 4 disease-modifying drugs have been approved for the treatment of SCD: hydroxyurea (an S-phase inhibitor), L-glutamine (an amino acid), crizanlizumab (a P-selectin inhibitor), and voxelotor (a hemoglobin S polymerization inhibitor). Preclinical studies suggested that voxelotor effectively treats SCD and sickle cell anemia (SCA). In a phase III trial, voxelotor-treated patients showed significantly elevated hemoglobin levels (>1 g/dL from baseline) compared to placebo-treated patients. The group that received voxelotor also showed a greater decrease in hemolytic markers but a comparable incidence of side effects. Six ongoing clinical trials also sought to ascertain the effectiveness and safety of high-dose voxelotor when administered to children younger than 12 years. Studies assessing their long-term efficacy and safety are needed to fully understand the role of voxelotor in treating SCD/SCA. In this review, we discuss the mechanisms, trials to date, and future treatment directions of voxelotor. |
| format | Article |
| id | doaj-art-56bda60634c34898b65844bdd5cffd0a |
| institution | Kabale University |
| issn | 2713-4148 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | The Korean Pediatric Society |
| record_format | Article |
| series | Clinical and Experimental Pediatrics |
| spelling | doaj-art-56bda60634c34898b65844bdd5cffd0a2025-02-07T07:38:39ZengThe Korean Pediatric SocietyClinical and Experimental Pediatrics2713-41482025-02-0168210611410.3345/cep.2024.0050020125555751Promising role of voxelotor in managing sickle cell disease in children: a narrative reviewAmit Agrawal0Gaurav Jadon1Japna Singh2Dalwinder Janjua3 Gandhi Medical College, Bhopal, India NMC Specialty Hospital, Dubai, UAE International Modern Hospital, Dubai, UAE Al Jalila Children's Specialty Hospital, Dubai, UAESickle cell disease (SCD) is characterized by chronic hemolytic anemia and intermittent vasoocclusive crises. To date, 4 disease-modifying drugs have been approved for the treatment of SCD: hydroxyurea (an S-phase inhibitor), L-glutamine (an amino acid), crizanlizumab (a P-selectin inhibitor), and voxelotor (a hemoglobin S polymerization inhibitor). Preclinical studies suggested that voxelotor effectively treats SCD and sickle cell anemia (SCA). In a phase III trial, voxelotor-treated patients showed significantly elevated hemoglobin levels (>1 g/dL from baseline) compared to placebo-treated patients. The group that received voxelotor also showed a greater decrease in hemolytic markers but a comparable incidence of side effects. Six ongoing clinical trials also sought to ascertain the effectiveness and safety of high-dose voxelotor when administered to children younger than 12 years. Studies assessing their long-term efficacy and safety are needed to fully understand the role of voxelotor in treating SCD/SCA. In this review, we discuss the mechanisms, trials to date, and future treatment directions of voxelotor.http://www.e-cep.org/upload/pdf/cep-2024-00500.pdfsickle cell diseasesickle cell anemiavoxelotorpharmacokineticspharmacodynamicssafety |
| spellingShingle | Amit Agrawal Gaurav Jadon Japna Singh Dalwinder Janjua Promising role of voxelotor in managing sickle cell disease in children: a narrative review Clinical and Experimental Pediatrics sickle cell disease sickle cell anemia voxelotor pharmacokinetics pharmacodynamics safety |
| title | Promising role of voxelotor in managing sickle cell disease in children: a narrative review |
| title_full | Promising role of voxelotor in managing sickle cell disease in children: a narrative review |
| title_fullStr | Promising role of voxelotor in managing sickle cell disease in children: a narrative review |
| title_full_unstemmed | Promising role of voxelotor in managing sickle cell disease in children: a narrative review |
| title_short | Promising role of voxelotor in managing sickle cell disease in children: a narrative review |
| title_sort | promising role of voxelotor in managing sickle cell disease in children a narrative review |
| topic | sickle cell disease sickle cell anemia voxelotor pharmacokinetics pharmacodynamics safety |
| url | http://www.e-cep.org/upload/pdf/cep-2024-00500.pdf |
| work_keys_str_mv | AT amitagrawal promisingroleofvoxelotorinmanagingsicklecelldiseaseinchildrenanarrativereview AT gauravjadon promisingroleofvoxelotorinmanagingsicklecelldiseaseinchildrenanarrativereview AT japnasingh promisingroleofvoxelotorinmanagingsicklecelldiseaseinchildrenanarrativereview AT dalwinderjanjua promisingroleofvoxelotorinmanagingsicklecelldiseaseinchildrenanarrativereview |