The efficacy of voxelotor, 900 mg in patients with sickle cell anaemia: A meta-analysis of the randomised controlled trials

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dc.authoridErkurt, Mehmet Ali/0000-0002-3285-417X
dc.authoridParlakpınar, Hakan/0000-0001-9497-3468
dc.authoridTanriverdi, Lokman Hekim/0000-0003-4263-5234
dc.authoridParlakpinar, Hakan/0000-0001-9497-3468
dc.authoridSARICI, Ahmet/0000-0002-5916-0119
dc.authorwosidErkurt, Mehmet Ali/ABI-7232-2020
dc.authorwosidParlakpınar, Hakan/T-6517-2018
dc.authorwosidTanriverdi, Lokman Hekim/M-5701-2015
dc.authorwosidParlakpinar, Hakan/V-6637-2019
dc.authorwosidSARICI, Ahmet/ABI-7512-2020
dc.contributor.authorTanriverdi, Lokman Hekim
dc.contributor.authorSarici, Ahmet
dc.contributor.authorErkurt, Mehmet Ali
dc.contributor.authorParlakpinar, Hakan
dc.date.accessioned2024-08-04T20:49:14Z
dc.date.available2024-08-04T20:49:14Z
dc.date.issued2021
dc.departmentİnönü Üniversitesien_US
dc.description.abstractObjective Sickle cell anaemia (SCA), an inherited chronic hematological disease affecting hundreds of thousand people worldwide, causes significant morbidity and reduced life expectancy about two or three decades. This study aimed to conduct a meta-analysis of the efficacy of voxelotor, 900 mg in patients with SCA. Methods The research protocol was registered at the International Register of Prospective Systematic Reviews (PROSPERO), under the registration number: CRD42020147796. ClinicalTrials.gov, Cochrane Central Register of Controlled Trials, Conference s, Google Scholar, Ovid Medline, Scopus, Web of Science, and Wiley Online Library from 2015 through July 25, 2019, and bibliographies of review articles and eligible studies. Eleven eligible studies that evaluated the effectiveness of voxelotor, 900 mg in SCA. Based on pre-specified inclusion and exclusion criteria, 2 randomized, placebo-controlled studies were included in the meta-analysis. Results The primary outcome measured was hemoglobin elevation, assessed in a highly similar fashion in both trials. There was a significant difference between voxelotor and placebo in haemoglobin change from baseline (mean difference [MD]: 0.87, 95% confidence interval [CI]: 0.67-1.06). Voxelotor also reduced markers of haemolysis, MD: -36.79, 95% CI: (-75.05) to 1.48 for unconjugated bilirubin that changes from baseline; MD: -19.09, 95% CI: (-44.06) to 5.88 for the percentage of reticulocytes that change from baseline and MD: -23.29, 95% CI: (-65.14) to 18.55 for LDH that change from baseline) but the difference was not statistically significant. Conclusions As a conclusion, voxelotor, 900 mg use significantly increased hemoglobin levels which of 1 g/dL elevation predicts a reduced risk of stroke (41%), albuminuria (53%), pulmonary arterial hypertension (57%), and mortality (64%) in recent studies. Voxelotor also reduced markers of hemolysis but failed to reach statistically significance in current evidence. Multicenter, randomized, placebo-controlled studies are on the way and will provide more evidence to see the potential of disease-modifying effects of voxelotor.en_US
dc.identifier.doi10.1111/ijcp.13967
dc.identifier.issn1368-5031
dc.identifier.issn1742-1241
dc.identifier.issue6en_US
dc.identifier.pmid33369007en_US
dc.identifier.scopus2-s2.0-85099425247en_US
dc.identifier.scopusqualityQ2en_US
dc.identifier.urihttps://doi.org/10.1111/ijcp.13967
dc.identifier.urihttps://hdl.handle.net/11616/99718
dc.identifier.volume75en_US
dc.identifier.wosWOS:000607802300001en_US
dc.identifier.wosqualityQ2en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherWiley-Hindawien_US
dc.relation.ispartofInternational Journal of Clinical Practiceen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subject[No Keywords]en_US
dc.titleThe efficacy of voxelotor, 900 mg in patients with sickle cell anaemia: A meta-analysis of the randomised controlled trialsen_US
dc.typeArticleen_US

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