Clinical outcomes and healthcare utilization in patients with sickle cell disease: a nationwide cohort study of Medicaid beneficiaries
Clinical outcomes and healthcare utilization in patients with sickle cell disease: a nationwide cohort study of Medicaid beneficiaries
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DOI：
10.1007/s00277-020-04233-w
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发表时间：
2020-09-01
影响因子：
3.5
通讯作者：
Schneeweiss, Sebastian
中科院分区：
文献类型：
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作者：
Desai, Rishi J.;Mahesri, Mufaddal;Schneeweiss, Sebastian
为了补充现有关于美国医疗补助计划受益者中镰状细胞病(SCD)临床结果和医疗保健使用情况的有限证据，我们使用全国医疗补助申领数据(2000-2013年)进行了一项队列研究。根据HbSS SCD诊断纳入患者，并进行跟踪，直到医疗补助取消登记、死亡、骨髓移植或数据可用终止，以评估血管闭塞危象(VOC)、急诊室(ER)就诊、住院、门诊就诊和输血。报告了年化事件发生率(95%可信区间[CI])。用多变量COX模型分析VOC对死亡风险的影响，其中VOC被建模为时变的，并每年更新。在44,033名平均年龄为15.7(13.6)岁的SCD患者中，VOC发生率(95%CI)为3.71(3.70-3.72)/人年，其中19-35岁患者的VOC发生率最高(13.20[13.15-13.26])。其他结果的事件发生率(95%CI)分别为2.97(2.97-2.98)次急诊室就诊、2.39(2.38-2.40)次住院、5.80(5.79-5.81)次门诊和0.91(0.90-0.91)次输血。前一年更高的VOC负担与更高的死亡风险相关，2-4 VOC的风险比(95%CI)为1.26(1.14-1.40)，VOC=5的风险比(1.41-1.74)为1.57(1.41-1.74)。总之，我们记录了美国医疗补助参与者的SCD负担，特别是在成年早期，并注意到持续的VOC负担与这些患者的死亡率有关。
To add to the limited existing evidence on clinical outcomes and healthcare use in sickle cell disease (SCD) among beneficiaries of the US Medicaid program, we conducted a cohort study using nationwide Medicaid claims data (2000-2013). Patients were included based on HbSS SCD diagnosis and followed until Medicaid disenrollment, death, bone marrow transplant, or end of data availability to assess vasoocclusive crises (VOC), emergency room (ER) visits, hospitalizations, outpatient visits, and blood transfusions. Annualized event rates (with 95% confidence intervals [CI]) were reported. The impact of VOCs on the risk of mortality was analyzed using a multivariable Cox model with VOC modeled as time-varying and updated annually. In a total of 44,033 SCD patients included with a mean (SD) age of 15.7 (13.6) years, the VOC rate (95% CI) was 3.71 (3.70-3.72) per person-year, with highest rate among patients 19-35 years who had >= 5 VOCs at baseline (13.20 [13.15-13.26]). Event rates (95% CI) per person per year for other outcomes were 2.97 (2.97-2.98) ER visits, 2.39 (2.38-2.40) hospitalizations, 5.80 (5.79-5.81) outpatient visits, and 0.91 (0.90-0.91) blood transfusions. A higher VOC burden in the preceding year was associated with an increased risk of mortality, with a hazard ratio (95% CI) of 1.26 (1.14-1.40) for 2-4 VOC vs. < 2 and 1.57 (1.41-1.74) for >= 5 VOC vs < 2. In conclusion, we documented a substantial burden of SCD in US Medicaid enrollees, especially during early adulthood and noted that ongoing burden of VOC is associated with mortality in these patients.