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美国复发性血管阻塞性危象镰状细胞病的经济和临床负担。

Economic and Clinical Burden of Managing Sickle Cell Disease with Recurrent Vaso-Occlusive Crises in the United States.

机构信息

Vertex Pharmaceuticals Incorporated, Boston, MA, USA.

Merative, Cambridge, MA, USA.

出版信息

Adv Ther. 2023 Aug;40(8):3543-3558. doi: 10.1007/s12325-023-02545-7. Epub 2023 Jun 18.

Abstract

INTRODUCTION

The aim of this study was to describe the clinical complications, treatment use, healthcare resource utilization (HCRU), and costs among patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) in the US.

METHODS

Merative MarketScan Databases were used to identify patients with SCD with recurrent VOCs from March 1, 2010, to March 1, 2019. Inclusion criteria were ≥ 1 inpatient or  ≥ 2 outpatient claims for SCD and ≥ 2 VOCs per year in any 2 consecutive years after the first qualifying SCD diagnosis. Individuals without SCD in these databases were used as matched controls. Patients were followed for ≥ 12 months, from their second VOC in the 2nd year (index date) to the earliest of inpatient death, end of continuous enrollment in medical/pharmacy benefits, or March 1, 2020. Outcomes were assessed during follow-up.

RESULTS

In total, 3420 patients with SCD with recurrent VOCs and 16,722 matched controls were identified. Patients with SCD with recurrent VOCs had a mean of 5.0 VOCs (standard deviation [SD] = 6.0), 2.7 inpatient admissions (SD 2.9), and 5.0 emergency department visits (SD 8.0) per patient per year during follow-up. Compared to matched controls, patients with SCD with recurrent VOCs incurred higher annual ($67,282 vs. $4134) and lifetime ($3.8 million vs. $229,000 over 50 years) healthcare costs.

CONCLUSION

Patients with SCD with recurrent VOCs experience substantial clinical and economic burden driven by inpatient costs and frequent VOCs. There is a major unmet need for treatments that alleviate or eliminate clinical complications, including VOCs, and reduce healthcare costs in this patient population.

摘要

简介

本研究旨在描述美国镰状细胞病(SCD)患者复发性血管阻塞性危象(VOC)的临床并发症、治疗方法、医疗保健资源利用(HCRU)和成本。

方法

使用 Merative MarketScan 数据库从 2010 年 3 月 1 日至 2019 年 3 月 1 日期间确定复发性 VOC 的 SCD 患者。纳入标准为:在首次确诊 SCD 后的任何 2 年内,每年至少有 1 次住院或 2 次以上门诊 SCD 就诊,且每年至少有 2 次 VOC。数据库中无 SCD 的个体作为匹配对照。从第 2 年的第 2 次 VOC(索引日期)开始,患者随访至少 12 个月,直至住院死亡、医疗/药房福利连续参保结束或 2020 年 3 月 1 日。在随访期间评估结果。

结果

共确定了 3420 例复发性 VOC 的 SCD 患者和 16722 例匹配对照。复发性 VOC 的 SCD 患者每年每例患者平均发生 5.0 次 VOC(标准差[SD]6.0)、2.7 次住院和 5.0 次急诊就诊(SD 8.0)。与匹配对照相比,复发性 VOC 的 SCD 患者每年的医疗费用($67282 与 $4134)和终生(50 年内$380 万与$229000)均较高。

结论

复发性 VOC 的 SCD 患者经历了严重的临床和经济负担,这是由住院费用和频繁的 VOC 导致的。在该患者人群中,需要治疗方法来减轻或消除临床并发症,包括 VOC,并降低医疗保健成本。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4149/10329958/a7a57392a41d/12325_2023_2545_Fig1_HTML.jpg

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