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.
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| Titel: | 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. |
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| Autoren: | Udeze C; Vertex Pharmaceuticals Incorporated, Boston, MA 02210, USA., Jerry M; Merative, Ann Arbor, MI 48108, USA., Evans K; Merative, Ann Arbor, MI 48108, USA., Li N; Vertex Pharmaceuticals Incorporated, Boston, MA 02210, USA., Jain S; Vertex Pharmaceuticals Incorporated, Boston, MA 02210, USA., Andemariam B; University of Connecticut Health, Farmington, CT 06030, USA. |
| Quelle: | Journal of comparative effectiveness research [J Comp Eff Res] 2025 Dec; Vol. 14 (12), pp. e240232. Date of Electronic Publication: 2025 Nov 24. |
| Publikationsart: | Journal Article |
| Sprache: | English |
| Info zur Zeitschrift: | Publisher: Becaris Publishing Country of Publication: England NLM ID: 101577308 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2042-6313 (Electronic) Linking ISSN: 20426305 NLM ISO Abbreviation: J Comp Eff Res Subsets: MEDLINE |
| Imprint Name(s): | Publication: 2023- : Royston, UK : Becaris Publishing Original Publication: London : Future Medicine |
| MeSH-Schlagworte: | Anemia, Sickle Cell*/drug therapy , Anemia, Sickle Cell*/complications , Anemia, Sickle Cell*/economics , Patient Acceptance of Health Care*/statistics & numerical data , Antisickling Agents*/therapeutic use , Antisickling Agents*/economics, Humans ; Female ; Male ; Retrospective Studies ; Adult ; Antibodies, Monoclonal, Humanized/therapeutic use ; Antibodies, Monoclonal, Humanized/economics ; United States ; Young Adult ; Adolescent ; Middle Aged ; Recurrence ; Health Care Costs/statistics & numerical data ; Medication Adherence ; Vaso-Occlusive Crises |
| Abstract: | Aim: 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. Materials & methods: In this retrospective study, patients with SCD with recurrent VOC who received L-glutamine, voxelotor, or crizanlizumab were identified from the Merative™ MarketScan ® Research Databases between 1 January 2015 and 30 September 2022. Eligible patients had ≥12 months 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 months 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. Results: Overall, 440 patients initiated a recently approved chronic therapy (L-glutamine, n = 254; voxelotor, n = 110; crizanlizumab, n = 76) 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 = 440; 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 ∼50% or $38,111 during follow-up (follow-up, $118,235 [$177,125]; baseline, $80,125 [$120,950]; p < 0.001); most of the increased costs ($27,108 [71.1%]) were a direct result of recently approved chronic therapies. Conclusion: 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. |
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| Contributed Indexing: | Keywords: L-glutamine; adherence; costs; crizanlizumab; healthcare resource utilization; real-world; sickle cell disease; treatment utilization; vaso-occlusive crises; voxelotor |
| Substance Nomenclature: | 0 (Antibodies, Monoclonal, Humanized) L7451S9126 (crizanlizumab) 0 (Antisickling Agents) |
| Entry Date(s): | Date Created: 20251124 Date Completed: 20251203 Latest Revision: 20251207 |
| Update Code: | 20251207 |
| PubMed Central ID: | PMC12679662 |
| DOI: | 10.57264/cer-2024-0232 |
| PMID: | 41277817 |
| Datenbank: | MEDLINE |
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Nájsť tento článok vo Web of Science | Abstract: | Aim: 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. Materials & methods: In this retrospective study, patients with SCD with recurrent VOC who received L-glutamine, voxelotor, or crizanlizumab were identified from the Merative™ MarketScan <sup>®</sup> Research Databases between 1 January 2015 and 30 September 2022. Eligible patients had ≥12 months 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 months 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. Results: Overall, 440 patients initiated a recently approved chronic therapy (L-glutamine, n = 254; voxelotor, n = 110; crizanlizumab, n = 76) 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 = 440; 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 ∼50% or $38,111 during follow-up (follow-up, $118,235 [$177,125]; baseline, $80,125 [$120,950]; p < 0.001); most of the increased costs ($27,108 [71.1%]) were a direct result of recently approved chronic therapies. Conclusion: 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. |
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| ISSN: | 2042-6313 |
| DOI: | 10.57264/cer-2024-0232 |