{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T19:20:49Z","timestamp":1790796049833,"version":"4.1.0"},"reference-count":30,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2020,7,16]],"date-time":"2020-07-16T00:00:00Z","timestamp":1594857600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Ann Pharmacother"],"published-print":{"date-parts":[[2021,2]]},"abstract":"<jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>To review the pharmacological characteristics, clinical evidence, and place in therapy of voxelotor for the treatment of sickle cell disease (SCD).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Data Sources:<\/jats:title>\n                    <jats:p>A comprehensive literature search of PubMed (1966 to April 2020) was conducted. Key search terms included GBT440, sickle cell, and voxelotor. Other sources were derived from bibliographies of articles, product labeling, manufacturer\u2019s website, and news releases. ClinicalTrials.gov was searched for additional studies.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Study Selection and Data Extraction:<\/jats:title>\n                    <jats:p>All English-language articles identified from the data sources were reviewed and evaluated. Case reports\/series and phase 1 through 3 clinical trials were included.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Data Synthesis:<\/jats:title>\n                    <jats:p>SCD is an inherited disorder associated with significant morbidity and early mortality. Three medications approved for SCD reduce SCD-associated complications but do not selectively ameliorate the underlying disease. Voxelotor is a novel agent that targets the pathophysiology of SCD. A phase 3 trial reported an increase in mean Hb level from baseline for voxelotor compared with placebo (1.1 vs \u22120.1 g\/dL; P &lt; 0.001). Voxelotor is generally well tolerated, with common adverse effects including headache, diarrhea, nausea, and arthralgia.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Relevance to Patient Care and Clinical Practice:<\/jats:title>\n                    <jats:p>Voxelotor may be considered for patients with SCD who have continued anemia and hemolysis despite being on maximum tolerated dose of hydroxyurea or in those who are hydroxyurea intolerant. Voxelotor is costly; therefore, both cost and benefit should be weighed before prescribing.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>Voxelotor appears to be safe and effective as monotherapy or in combination with hydroxyurea for patients with SCD who are 12 years of age and older.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/1060028020943059","type":"journal-article","created":{"date-parts":[[2020,7,17]],"date-time":"2020-07-17T03:57:01Z","timestamp":1594958221000},"page":"240-245","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":31,"title":["Voxelotor: A Novel Treatment for Sickle Cell Disease"],"prefix":"10.1177","volume":"55","author":[{"given":"Leah B.","family":"Herity","sequence":"first","affiliation":[{"name":"Virginia Commonwealth University Health System, Richmond, VA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"DaleMarie M.","family":"Vaughan","sequence":"additional","affiliation":[{"name":"Virginia Commonwealth University Health System, Richmond, VA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lindsey Ritenour","family":"Rodriguez","sequence":"additional","affiliation":[{"name":"Virginia Commonwealth University Health System, Richmond, VA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2781-9991","authenticated-orcid":false,"given":"Denise Kozella","family":"Lowe","sequence":"additional","affiliation":[{"name":"Virginia Commonwealth University Health System, Richmond, VA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2020,7,16]]},"reference":[{"key":"bibr1-1060028020943059","unstructured":"World Health Organization. Sickle-cell anemia: report by the Secretariat. Accessed March 31, 2020. http:\/\/apps.who.int\/iris\/handle\/10665\/20890"},{"key":"bibr2-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pmed.1001484"},{"key":"bibr3-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1016\/j.amepre.2009.12.022"},{"key":"bibr4-1060028020943059","unstructured":"Centers for Disease Control and Prevention. Data & statistics on sickle cell disease. Accessed April 27, 2020. https:\/\/www.cdc.gov\/ncbddd\/sicklecell\/data.html"},{"key":"bibr5-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1001\/jamanetworkopen.2019.15374"},{"key":"bibr6-1060028020943059","first-page":"1","volume":"68","author":"Arias E.","year":"2019","journal-title":"Natl Vital Stat Rep"},{"key":"bibr7-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)30193-9"},{"key":"bibr8-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0002095"},{"key":"bibr9-1060028020943059","author":"Droxia","year":"2019","journal-title":"Bristol-Myers Squibb Company;"},{"key":"bibr10-1060028020943059","author":"Endari","year":"2017","journal-title":"Emmaus Medical, Inc;"},{"key":"bibr11-1060028020943059","author":"Adakveo","year":"2019","journal-title":"Novartis Pharmaceuticals;"},{"key":"bibr12-1060028020943059","unstructured":"US Food & Drug Administration. FDA approves novel treatment to target abnormality in sickle cell disease. Published November 25, 2019. Accessed March 8, 2020. https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-novel-treatment-target-abnormality-sickle-cell-disease"},{"key":"bibr13-1060028020943059","unstructured":"Oxbyrta. Package insert. Global Blood Therapeutics, Inc; 2019."},{"key":"bibr14-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.14214"},{"key":"bibr15-1060028020943059","doi-asserted-by":"publisher","DOI":"10.3233\/CH-170340"},{"key":"bibr16-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-08-868893"},{"key":"bibr17-1060028020943059","volume-title":"Poster presented at: EHA23","author":"Washington CB"},{"key":"bibr18-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.13896"},{"key":"bibr19-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1903212"},{"key":"bibr20-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2011.08916.x"},{"key":"bibr21-1060028020943059","unstructured":"ClincalTrials.gov. Voxelotor. Accessed February 22, 2020. https:\/\/clinicaltrials.gov\/ct2\/results?cond=&term=voxelotor&cntry=&state=&city=&dist="},{"key":"bibr22-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2019-130802"},{"key":"bibr23-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25389"},{"key":"bibr24-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25139"},{"key":"bibr25-1060028020943059","doi-asserted-by":"publisher","DOI":"10.4081\/hr.2018.7643"},{"key":"bibr26-1060028020943059","doi-asserted-by":"publisher","DOI":"10.1016\/j.cca.2018.03.032"},{"key":"bibr27-1060028020943059","unstructured":"Medi-Span\u00ae Price Rx\u00ae Online, UpToDate Online. Oxbryta (voxelotor). Wolters Kluwer Clinical Drug Information, Inc; 2020."},{"key":"bibr28-1060028020943059","unstructured":"Bradt P, Spackman E, Synnott P Crizanlizumab, voxelotor, and l-glutamine for sickle cell disease: effectiveness and value. Institute for Clilnical and Economic Review, January 23, 2020. Accessed July 20, 2020. https:\/\/icer-review.org\/wp-content\/uploads\/2019\/08\/ICER_SCD_Evidence-Report_031220-FOR-PUBLICATION.pdf"},{"key":"bibr29-1060028020943059","unstructured":"Global Blood Therapeutics, Inc. Oxbryta (voxelotor). 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