{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,15]],"date-time":"2026-04-15T19:24:52Z","timestamp":1776281092270,"version":"3.50.1"},"reference-count":38,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2020,11,1]],"date-time":"2020-11-01T00:00:00Z","timestamp":1604188800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2020,11,1]],"date-time":"2020-11-01T00:00:00Z","timestamp":1604188800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2020,10,8]],"date-time":"2020-10-08T00:00:00Z","timestamp":1602115200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"name":"FDA"},{"name":"GBT"}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Contemporary Clinical Trials"],"published-print":{"date-parts":[[2020,11]]},"DOI":"10.1016\/j.cct.2020.106161","type":"journal-article","created":{"date-parts":[[2020,9,30]],"date-time":"2020-09-30T10:23:52Z","timestamp":1601461432000},"page":"106161","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":8,"special_numbering":"C","title":["Accelerated approval of Oxbryta\u00ae (voxelotor): A case study on novel endpoint selection in sickle cell disease"],"prefix":"10.1016","volume":"98","author":[{"given":"Joshua","family":"Lehrer-Graiwer","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Linda","family":"Yokoshima","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Barbara","family":"Tong","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ted W.","family":"Love","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.cct.2020.106161_bb0005","doi-asserted-by":"crossref","first-page":"18010","DOI":"10.1038\/nrdp.2018.10","article-title":"Sickle cell disease","volume":"4","author":"Kato","year":"2018","journal-title":"Nat. Rev. Dis. Primers"},{"issue":"4 Suppl","key":"10.1016\/j.cct.2020.106161_bb0010","doi-asserted-by":"crossref","first-page":"S512","DOI":"10.1016\/j.amepre.2009.12.022","article-title":"Population estimates of sickle cell disease in the U.S.","volume":"38","author":"Hassell","year":"2010","journal-title":"Am. J. Prev. Med."},{"issue":"1","key":"10.1016\/j.cct.2020.106161_bb0015","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1182\/asheducation-2017.1.435","article-title":"Chronic organ failure in adult sickle cell disease","volume":"2017","author":"Vichinsky","year":"2017","journal-title":"Hematology Am. Soc. Hematol. Educ. Program"},{"issue":"9","key":"10.1016\/j.cct.2020.106161_bb0020","doi-asserted-by":"crossref","first-page":"1482","DOI":"10.1002\/pbc.24557","article-title":"Sickle cell disease related mortality in the United States (1999\u20132009)","volume":"60","author":"Hamideh","year":"2013","journal-title":"Pediatr. Blood Cancer"},{"issue":"11","key":"10.1016\/j.cct.2020.106161_bb0025","doi-asserted-by":"crossref","first-page":"e1915374","DOI":"10.1001\/jamanetworkopen.2019.15374","article-title":"Estimated life expectancy and income of patients with sickle cell disease compared with those without sickle cell disease","volume":"2","author":"Lubeck","year":"2019","journal-title":"JAMA Netw. Open"},{"issue":"20","key":"10.1016\/j.cct.2020.106161_bb0030","doi-asserted-by":"crossref","first-page":"2719","DOI":"10.1182\/blood-2017-02-765891","article-title":"Treating sickle cell disease by targeting HbS polymerization","volume":"129","author":"Eaton","year":"2017","journal-title":"Blood"},{"issue":"2","key":"10.1016\/j.cct.2020.106161_bb0035","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1016\/j.blre.2015.08.005","article-title":"The role of blood rheology in sickle cell disease","volume":"30","author":"Connes","year":"2016","journal-title":"Blood Rev."},{"issue":"3","key":"10.1016\/j.cct.2020.106161_bb0040","doi-asserted-by":"crossref","first-page":"750","DOI":"10.1172\/JCI89741","article-title":"Intravascular hemolysis and the pathophysiology of sickle cell disease","volume":"127","author":"Kato","year":"2017","journal-title":"J. Clin. Invest."},{"issue":"1","key":"10.1016\/j.cct.2020.106161_bb0045","doi-asserted-by":"crossref","DOI":"10.4084\/mjhid.2020.010","article-title":"The evolving pharmacotherapeutic landscape for the treatment of sickle cell disease","volume":"12","author":"Ballas","year":"2020","journal-title":"Mediterr. J. Hematol. Infect. Dis."},{"key":"10.1016\/j.cct.2020.106161_bb0050","series-title":"Evidence-Based Management of Sickle Cell Disease. Expert Panel Report, 2014","first-page":"1","author":"National Heart Lung and Blood Institute","year":"2014"},{"issue":"1","key":"10.1016\/j.cct.2020.106161_bb0055","doi-asserted-by":"crossref","first-page":"625","DOI":"10.1182\/asheducation-2016.1.625","article-title":"Sickle cell disease: when and how to transfuse","volume":"2016","author":"Howard","year":"2016","journal-title":"Hematology Am. Soc. Hematol. Educ. Program"},{"key":"10.1016\/j.cct.2020.106161_bb0060","author":"US Food and Drug Administration"},{"key":"10.1016\/j.cct.2020.106161_bb0065","author":"US Food and Drug Administration"},{"key":"10.1016\/j.cct.2020.106161_bb0070","author":"US Food and Drug Administration"},{"key":"10.1016\/j.cct.2020.106161_bb0075","author":"Moscicki"},{"issue":"1","key":"10.1016\/j.cct.2020.106161_bb0080","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1056\/NEJM199107043250103","article-title":"Pain in sickle cell disease: rates and risk factors","volume":"325","author":"Platt","year":"1991","journal-title":"N. Engl. J. Med."},{"issue":"6","key":"10.1016\/j.cct.2020.106161_bb0085","doi-asserted-by":"crossref","first-page":"575","DOI":"10.1177\/1740774515590811","article-title":"Systematic review of interventional sickle cell trials registered in ClinicalTrials.gov","volume":"12","author":"Lebensburger","year":"2015","journal-title":"Clinical Trials"},{"issue":"1","key":"10.1016\/j.cct.2020.106161_bb0090","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1002\/ajh.21550","article-title":"Definitions of the phenotypic manifestations of sickle cell disease","volume":"85","author":"Ballas","year":"2010","journal-title":"Am. J. Hematol."},{"key":"10.1016\/j.cct.2020.106161_bb0095","author":"National Heart Lung and Blood Institute"},{"issue":"2","key":"10.1016\/j.cct.2020.106161_bb0100","doi-asserted-by":"crossref","first-page":"94","DOI":"10.7326\/0003-4819-148-2-200801150-00004","article-title":"Daily assessment of pain in adults with sickle cell disease","volume":"148","author":"Smith","year":"2008","journal-title":"Ann. Intern. Med."},{"issue":"5","key":"10.1016\/j.cct.2020.106161_bb0105","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1080\/21678707.2018.1471983","article-title":"Advances in new drug therapies for the management of sickle cell disease","volume":"6","author":"Ataga","year":"2018","journal-title":"Expert Opin. Orphan. Drugs"},{"key":"10.1016\/j.cct.2020.106161_bb0110","author":"US Food and Drug Administration"},{"issue":"6","key":"10.1016\/j.cct.2020.106161_bb0115","doi-asserted-by":"crossref","first-page":"363","DOI":"10.1097\/01.md.0000189089.45003.52","article-title":"Outcome of sickle cell anemia: a 4-decade observational study of 1056 patients","volume":"84","author":"Powars","year":"2005","journal-title":"Medicine (Baltimore)"},{"key":"10.1016\/j.cct.2020.106161_bb0120","author":"US Food and Drug Administration"},{"issue":"1","key":"10.1016\/j.cct.2020.106161_bb0125","doi-asserted-by":"crossref","first-page":"482","DOI":"10.1182\/asheducation-2018.1.482","article-title":"Measuring success: utility of biomarkers in sickle cell disease clinical trials and care","volume":"2018","author":"Kalpatthi","year":"2018","journal-title":"Hematology Am. Soc. Hematol. Educ. Program"},{"issue":"4","key":"10.1016\/j.cct.2020.106161_bb0130","doi-asserted-by":"crossref","first-page":"433","DOI":"10.1111\/j.1365-2141.2011.08961.x","article-title":"Biomarkers in sickle cell disease","volume":"156","author":"Rees","year":"2012","journal-title":"Br. J. Haematol."},{"issue":"25","key":"10.1016\/j.cct.2020.106161_bb0135","doi-asserted-by":"crossref","first-page":"2973","DOI":"10.1002\/sim.5403","article-title":"Biomarkers and surrogate endpoints in clinical trials","volume":"31","author":"Fleming","year":"2012","journal-title":"Stat. Med."},{"issue":"8","key":"10.1016\/j.cct.2020.106161_bb0140","doi-asserted-by":"crossref","first-page":"790","DOI":"10.1001\/jama.282.8.790","article-title":"Are surrogate markers adequate to assess cardiovascular disease drugs?","volume":"282","author":"Temple","year":"1999","journal-title":"JAMA"},{"issue":"1","key":"10.1016\/j.cct.2020.106161_bb0145","doi-asserted-by":"crossref","first-page":"95","DOI":"10.3233\/CH-170340","article-title":"GBT440 improves red blood cell deformability and reduces viscosity of sickle cell blood under deoxygenated conditions","volume":"70","author":"Dufu","year":"2018","journal-title":"Clin. Hemorheol. Microcirc."},{"issue":"1","key":"10.1016\/j.cct.2020.106161_bb0150","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1111\/bjh.14214","article-title":"GBT 440 increases haemoglobin oxygen affinity, reduces sickling and prolongs RBC half-life in a murine model of sickle cell disease","volume":"175","author":"Oksenberg","year":"2016","journal-title":"Br. J. Haematol."},{"issue":"17","key":"10.1016\/j.cct.2020.106161_bb0155","doi-asserted-by":"crossref","first-page":"1865","DOI":"10.1182\/blood-2018-08-868893","article-title":"A phase 1\/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease","volume":"133","author":"Howard","year":"2019","journal-title":"Blood"},{"issue":"6","key":"10.1016\/j.cct.2020.106161_bb0160","first-page":"719","article-title":"Transfusion of blood and blood products: indications and complications","volume":"83","author":"Sharma","year":"2011","journal-title":"Am. Fam. Physician"},{"issue":"1","key":"10.1016\/j.cct.2020.106161_bb0165","doi-asserted-by":"crossref","first-page":"49","DOI":"10.7326\/0003-4819-157-1-201206190-00429","article-title":"Red blood cell transfusion: a clinical practice guideline from the AABB*","volume":"157","author":"Carson","year":"2012","journal-title":"Ann. Intern. Med."},{"issue":"23","key":"10.1016\/j.cct.2020.106161_bb0170","doi-asserted-by":"crossref","first-page":"4002","DOI":"10.1182\/bloodadvances.2019000883","article-title":"End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings","volume":"3","author":"Farrell","year":"2019","journal-title":"Blood Adv."},{"issue":"4","key":"10.1016\/j.cct.2020.106161_bb0175","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0229959","article-title":"Low hemoglobin increases risk for cerebrovascular disease, kidney disease, pulmonary vasculopathy, and mortality in sickle cell disease: a systematic literature review and meta-analysis","volume":"15","author":"Ataga","year":"2020","journal-title":"PLoS One"},{"issue":"5","key":"10.1016\/j.cct.2020.106161_bb0180","doi-asserted-by":"crossref","first-page":"699","DOI":"10.1002\/ana.410420505","article-title":"Long-term stroke risk in children with sickle cell disease screened with transcranial Doppler","volume":"42","author":"Adams","year":"1997","journal-title":"Ann. Neurol."},{"issue":"14","key":"10.1016\/j.cct.2020.106161_bb0185","doi-asserted-by":"crossref","first-page":"1814","DOI":"10.1182\/blood-2015-10-675231","article-title":"Long-term treatment follow-up of children with sickle cell disease monitored with abnormal transcranial Doppler velocities","volume":"127","author":"Bernaudin","year":"2016","journal-title":"Blood"},{"issue":"23","key":"10.1016\/j.cct.2020.106161_bb0190","doi-asserted-by":"crossref","first-page":"3982","DOI":"10.1182\/bloodadvances.2019000882","article-title":"End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain","volume":"3","author":"Farrell","year":"2019","journal-title":"Blood Adv."}],"container-title":["Contemporary Clinical Trials"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1551714420302391?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1551714420302391?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,27]],"date-time":"2025-10-27T08:51:57Z","timestamp":1761555117000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1551714420302391"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,11]]},"references-count":38,"alternative-id":["S1551714420302391"],"URL":"https:\/\/doi.org\/10.1016\/j.cct.2020.106161","relation":{},"ISSN":["1551-7144"],"issn-type":[{"value":"1551-7144","type":"print"}],"subject":[],"published":{"date-parts":[[2020,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Accelerated approval of Oxbryta\u00ae (voxelotor): A case study on novel endpoint selection in sickle cell disease","name":"articletitle","label":"Article Title"},{"value":"Contemporary Clinical Trials","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.cct.2020.106161","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2020 The Authors. Published by Elsevier Inc.","name":"copyright","label":"Copyright"}],"article-number":"106161"}}