Beohar Nirat, Davidson Charles J, Kip Kevin E, Goodreau Lynne, Vlachos Helen Aslanidou, Meyers Sheridan N, Benzuly Keith H, Flaherty James D, Ricciardi Mark J, Bennett Charles L, Williams David O
Northwestern University Feinberg School of Medicine, Chicago, Ill, USA.
JAMA. 2007 May 9;297(18):1992-2000. doi: 10.1001/jama.297.18.1992.
Limited data exist regarding use of drug-eluting stents outside of approved indications in real-world settings.
To determine the frequency, safety, and effectiveness of drug-eluting stents for off-label (restenosis, bypass graft lesion, long lesions, vessel size outside of information for use recommendation) and untested (left main, ostial, bifurcation, or total occlusion lesions) indications in percutaneous coronary intervention (PCI).
DESIGN, SETTING, AND PATIENTS: Observational, prospective, multicenter registry to evaluate in-hospital, 30-day, and 1-year outcomes among patients undergoing PCI between January and June 2005 in 140 US academic and community medical centers. Of 7752 PCI-treated patients, 6993 (90%) received drug-eluting stents; of these, 5851 (84%) received no other devices. Standard, off-label, and untested use was determined in 5541 (95%) of these 5851 patients, constituting the study cohort.
Frequency of off-label and untested use, 1-year repeat target vessel revascularization, and composite of death, myocardial infarction (MI), or stent thrombosis at in-hospital follow-up and during 1 year of follow-up.
Of 5541 patients receiving drug-eluting stents, 2588 (47%) received stents for off-label or untested indications. Adjusted in-hospital risk of death, MI, or stent thrombosis was not statistically different with off-label or untested vs standard use. At 30 days, the risk of this composite end point was significantly higher with off-label use (adjusted hazard ratio [HR], 2.08; 95% confidence interval [CI], 1.24-3.48; P = .005) but not untested use (adjusted HR, 1.45; 95% CI, 0.79-2.67; P = .23). Excluding early events, this end point was not different at 1 year with off-label use (adjusted HR, 1.10; 95% CI, 0.79-1.54; P = .57) or untested use (adjusted HR, 0.91; 95% CI, 0.60-1.38; P = .66). At 1 year, compared with standard use, significantly higher rates of target vessel revascularization were associated with off-label use (adjusted HR, 1.49; 95% CI, 1.13-1.98; P = .005) and untested use (adjusted HR, 1.49; 95% CI, 1.10-2.02; P = .01), although absolute rates were low (standard, 4.4% [n = 113]; off-label, 7.6% [n = 95]; untested, 6.7% [n = 72]).
In contemporary US practice, off-label and untested use of drug-eluting stents is common. Compared with standard use, relative early safety is lower with off-label use, and the long-term effectiveness is lower with both off-label and untested use. However, the absolute event rates remain low.
关于在现实环境中超出批准适应症使用药物洗脱支架的数据有限。
确定药物洗脱支架用于经皮冠状动脉介入治疗（PCI）中标签外（再狭窄、旁路移植病变、长病变、血管尺寸超出使用建议信息范围）和未经测试（左主干、开口、分叉或完全闭塞病变）适应症的频率、安全性和有效性。
设计、设置和患者：一项观察性、前瞻性、多中心注册研究，以评估2005年1月至6月期间在美国140家学术和社区医疗中心接受PCI治疗的患者的住院、30天和1年结局。在7752例接受PCI治疗的患者中，6993例（90%）接受了药物洗脱支架；其中，5851例（84%）未接受其他器械。在这5851例患者中的5541例（95%）中确定了标准、标签外和未经测试的使用情况，构成研究队列。
标签外和未经测试使用的频率、1年再次靶血管血运重建以及住院随访期间和1年随访期间死亡、心肌梗死（MI）或支架血栓形成的复合终点。
在5541例接受药物洗脱支架的患者中，2588例（47%）接受了用于标签外或未经测试适应症的支架。标签外或未经测试使用与标准使用相比，住院调整后死亡、MI或支架血栓形成的风险无统计学差异。在30天时，标签外使用时该复合终点的风险显著更高（调整后风险比[HR]，2.08；95%置信区间[CI]，1.24 - 3.48；P = 0.005），但未经测试使用时无差异（调整后HR，1.45；95% CI，0.79 - 2.67；P = 0.23）。排除早期事件后，1年时标签外使用（调整后HR，1.10；95% CI，0.79 - 1.54；P = 0.57）或未经测试使用（调整后HR，0.91；95% CI，0.60 - 1.38；P = 0.66）时该终点无差异。在1年时，与标准使用相比，标签外使用（调整后HR，1.49；95% CI，1.13 - 1.98；P = 0.005）和未经测试使用（调整后HR，1.49；95% CI，1.10 - 2.02；P = 0.01）的靶血管血运重建率显著更高，尽管绝对发生率较低（标准，4.4%[n = 113]；标签外，7.6%[n = 95]；未经测试，6.7%[n = 72]）。
在当代美国的实践中，药物洗脱支架的标签外和未经测试使用很常见。与标准使用相比，标签外使用相对早期安全性较低，标签外和未经测试使用的长期有效性均较低。然而，绝对事件发生率仍然较低。
