{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,10]],"date-time":"2026-10-10T05:38:56Z","timestamp":1791610736865,"version":"4.3.4"},"reference-count":39,"publisher":"Becaris Publishing Limited","issue":"12","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"published-print":{"date-parts":[[2025,12]]},"abstract":"<jats:p>\n                    <jats:bold>Aim:<\/jats:bold>\n                    To describe real-world adherence, treatment utilization, vaso-occlusive crises (VOC) and economic outcomes in patients with sickle cell disease (SCD) with recurrent VOC treated with L-glutamine, voxelotor or crizanlizumab in the US.\n                    <jats:bold>Materials &amp; methods:<\/jats:bold>\n                    In this retrospective study, patients with SCD with recurrent VOC who received L-glutamine, voxelotor, or crizanlizumab were identified from the Merative\u2122 MarketScan\n                    <jats:sup>\u00ae<\/jats:sup>\n                    Research Databases between 1 January 2015 and 30 September 2022. Eligible patients had \u226512\u00a0months continuous enrollment before and after the first chronic therapy claim (i.e., index date). Number of VOC, treatment utilization, healthcare resource utilization and healthcare costs were summarized for 12\u00a0months before (baseline) and after (follow-up) the index date. The proportion of days covered (PDC; i.e., proxy for adherence) for the index chronic therapy was measured during the 12-month follow-up period.\n                    <jats:bold>Results:<\/jats:bold>\n                    Overall, 440 patients initiated a recently approved chronic therapy (L-glutamine, n\u00a0=\u00a0254; voxelotor, n\u00a0=\u00a0110; crizanlizumab, n\u00a0=\u00a076) and met inclusion criteria. Mean (standard deviation [SD]) number of VOC during baseline and follow-up were similar for patients treated with any index therapy (n\u00a0=\u00a0440; 7.21 [8.82] vs 7.27 [9.85]); this was similar across patients treated with L-glutamine, crizanlizumab, and voxelotor, respectively. Mean (SD) PDC for patients with any index therapy was 0.37 (0.29); results were similar across patients treated with L-glutamine, crizanlizumab, and voxelotor. Healthcare resource utilization during the 12-month baseline and follow-up periods were comparable. Mean (SD) total costs for patients initiating a recently approved chronic therapy increased by \u223c50% or $38,111 during follow-up (follow-up, $118,235 [$177,125]; baseline, $80,125 [$120,950]; p\u00a0&lt;\u00a00.001); most of the increased costs ($27,108 [71.1%]) were a direct result of recently approved chronic therapies.\n                    <jats:bold>Conclusion:<\/jats:bold>\n                    Patients initiated on L-glutamine, voxelotor or crizanlizumab had low adherence (based on PDC), continued to experience frequent VOC, and incurred higher healthcare costs mostly due to the costs of these therapies. This highlights the need for additional treatment options for patients with SCD with recurrent VOC.\n                  <\/jats:p>","DOI":"10.57264\/cer-2024-0232","type":"journal-article","created":{"date-parts":[[2025,11,24]],"date-time":"2025-11-24T12:51:00Z","timestamp":1763988660000},"source":"Crossref","is-referenced-by-count":4,"title":["Adherence, treatment utilization, clinical and economic outcomes of patients with sickle cell disease with recurrent vaso-occlusive crises treated with recently approved chronic therapies in the US"],"prefix":"10.57264","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0009-0002-7840-6804","authenticated-orcid":false,"given":"Chuka","family":"Udeze","sequence":"first","affiliation":[{"name":"Vertex Pharmaceuticals Incorporated, Boston, MA 02210, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1664-8090","authenticated-orcid":false,"given":"Michelle","family":"Jerry","sequence":"additional","affiliation":[{"name":"Merative, Ann Arbor, MI 48108, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0987-1745","authenticated-orcid":false,"given":"Kristin","family":"Evans","sequence":"additional","affiliation":[{"name":"Merative, Ann Arbor, MI 48108, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-4439-0176","authenticated-orcid":false,"given":"Nanxin","family":"Li","sequence":"additional","affiliation":[{"name":"Vertex Pharmaceuticals Incorporated, Boston, MA 02210, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0001-1579-4275","authenticated-orcid":false,"given":"Siddharth","family":"Jain","sequence":"additional","affiliation":[{"name":"Vertex Pharmaceuticals Incorporated, Boston, MA 02210, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2540-6037","authenticated-orcid":false,"given":"Biree","family":"Andemariam","sequence":"additional","affiliation":[{"name":"University of Connecticut Health, Farmington, CT 06030, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"36897","reference":[{"issue":"8","key":"e_1_3_10_3_2","doi-asserted-by":"crossref","first-page":"3543","DOI":"10.1007\/s12325-023-02545-7","article-title":"Economic and clinical burden of managing sickle cell disease with recurrent vaso-occlusive crises in the United States","volume":"40","author":"Udeze C","year":"2023","unstructured":"Udeze C, Evans KA, Yang Y Economic and clinical burden of managing sickle cell disease with recurrent vaso-occlusive crises in the United States. Adv. Ther. 40(8), 3543\u20133558 (2023).","journal-title":"Adv. Ther."},{"key":"e_1_3_10_4_2","volume-title":"StatPearls [Internet]","author":"Mangla A","year":"2025","unstructured":"Mangla A, Agarwal N, Maruvada S. Sickle cell anemia [updated 2023 Sep 4]. In: StatPearls [Internet]. StatPearls Publishing, FL, USA (2025)."},{"key":"e_1_3_10_5_2","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1146\/annurev-pathmechdis-012418-012838","article-title":"Pathophysiology of sickle cell disease","volume":"14","author":"Sundd P","year":"2019","unstructured":"Sundd P, Gladwin MT, Novelli EM. Pathophysiology of sickle cell disease. Annu. Rev. Pathol. 14, 263\u2013292 (2019).","journal-title":"Annu. Rev. Pathol."},{"issue":"2","key":"e_1_3_10_6_2","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1080\/17474086.2022.2031967","article-title":"Real-world effectiveness of voxelotor for treating sickle cell disease in the US: a large claims data analysis","volume":"15","author":"Shah N","year":"2022","unstructured":"Shah N, Lipato T, Alvarez O Real-world effectiveness of voxelotor for treating sickle cell disease in the US: a large claims data analysis. Expert Rev. Hematol. 15(2), 167\u2013173 (2022).","journal-title":"Expert Rev. Hematol."},{"key":"e_1_3_10_7_2","unstructured":"Emmaus Medical Inc. ENDARI\u00ae(L-glutamine oral powder) (2020). https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2020\/208587s003lbl.pdf"},{"key":"e_1_3_10_8_2","unstructured":"Global Blood Therapeutics Inc. OXBRYTA\u00ae(voxelotor) tablets for oral use and oral suspension (2023). https:\/\/labeling.pfizer.com\/ShowLabeling.aspx?id=19593&Section=PPI"},{"key":"e_1_3_10_9_2","unstructured":"Novartis Pharmaceuticals Corporation. ADAKVEO\u00ae(crizanlizumab-tmca) injection for intravenous use (2024). https:\/\/www.novartis.com\/us-en\/sites\/novartis_us\/files\/adakveo.pdf"},{"key":"e_1_3_10_10_2","unstructured":"Pfizer. Pfizer voluntarily withdraws all lots of sickle cell disease treatment OXBRYTA\u00ae (voxelotor) from worldwide markets (2024). https:\/\/www.pfizer.com\/news\/press-release\/press-release-detail\/pfizer-voluntarily-withdraws-all-lots-sickle-cell-disease"},{"key":"e_1_3_10_11_2","unstructured":"Agency EM. Adakveo - referral. (2023). https:\/\/www.ema.europa.eu\/en\/medicines\/human\/referrals\/adakveo#:\u223c:text=Commission%20final%20decision- Overview older%20with%20sickle%20cell%20disease"},{"issue":"6","key":"e_1_3_10_12_2","doi-asserted-by":"crossref","first-page":"352.e1","DOI":"10.1016\/j.jtct.2025.02.025","article-title":"Best practices in gene therapy for sickle cell disease and transfusion-dependent \u03b2-thalassemia","volume":"31","author":"Frangoul H","year":"2025","unstructured":"Frangoul H, Stults A, Bruce K Best practices in gene therapy for sickle cell disease and transfusion-dependent \u03b2-thalassemia. Transplant. Cell Ther. 31(6), 352.e1\u2013352.e10 (2025).","journal-title":"Transplant. 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Manag. Care Spec. Pharm. 21(12), 1195\u20131202 (2015).","journal-title":"J. Manag. Care Spec. Pharm."},{"issue":"12","key":"e_1_3_10_29_2","doi-asserted-by":"crossref","first-page":"e2247787","DOI":"10.1001\/jamanetworkopen.2022.47787","article-title":"Association of baseline adherence to antihypertensive medications with adherence after shelter-in-place guidance for COVID-19 among US adults","volume":"5","author":"Egan BM","year":"2022","unstructured":"Egan BM, Sutherland SE, Macri CI Association of baseline adherence to antihypertensive medications with adherence after shelter-in-place guidance for COVID-19 among US adults. JAMA Netw. Open 5(12), e2247787 (2022).","journal-title":"JAMA Netw. 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