Xing Shan, Bullano Michael, Hale Sarah, Lokhandwala Tasneem, Murty Sharanya
Takeda Pharmaceuticals U.S.A., Inc., Lexington, MA, USA.
Xcenda LLC, Carrollton, TX, USA.
Am J Health Syst Pharm. 2024 Dec 5;81(24):1278-1287. doi: 10.1093/ajhp/zxae167.
Severe ADAMTS13 deficiency (activity <10%) is the diagnostic threshold for thrombotic thrombocytopenic purpura (TTP) and is associated with various clinical symptoms, abnormal laboratory results, and long-term complications.
This retrospective, noninterventional cohort study used the Premier Healthcare Database to identify patients with ADAMTS13 activity of <10% in US hospitals from January 1, 2016, through March 31, 2020. The objective was to describe patient characteristics, laboratory results, comorbidities (as measured by the Elixhauser comorbidity index), symptoms, length of stay, treatment patterns, mortality, inpatient costs, and readmission rates (summarized descriptively). Inpatient costs were calculated as total cost to the hospital.
There were 211 patients with severe ADAMTS13 deficiency; 89% of patients had a TTP-related diagnosis, of whom 62% had a primary diagnosis of thrombotic microangiopathy. Over 80% of patients with available data had a decreased platelet count and elevated lactate dehydrogenase; schistocytes were detected in 99%. The most prevalent symptoms/complications were neurological, bleeding, and pain. Most patients (86%) had 2 or more Elixhauser comorbidities. Over 80% of patients received 1 or more TTP-related treatments, mostly plasma exchange. The mean length of stay was 11.5 days; 5% of patients died during their stay. Readmission rates at 30, 60, and 90 days were 20%, 26%, and 28%, respectively. The median (interquartile range) total inpatient cost to the hospital throughout the index admission was $33,221 ($19,431-$64,901).
Patients with severe ADAMTS13 deficiency have substantial clinical burden, have high mortality and readmission rates, and generate high costs for hospitals. There is a high need for a therapy that replaces ADAMTS13, thus addressing the root cause of the symptoms and complications caused by this deficiency.
严重的ADAMTS13缺乏（活性<10%）是血栓性血小板减少性紫癜（TTP）的诊断阈值，与各种临床症状、异常实验室检查结果及长期并发症相关。
这项回顾性、非干预性队列研究利用Premier医疗数据库，识别2016年1月1日至2020年3月31日期间美国医院中ADAMTS13活性<10%的患者。目的是描述患者特征、实验室检查结果、合并症（通过Elixhauser合并症指数衡量）、症状、住院时间、治疗模式、死亡率、住院费用及再入院率（进行描述性总结）。住院费用计算为医院的总费用。
有211例严重ADAMTS13缺乏患者；89%的患者有TTP相关诊断，其中62%的患者原发性诊断为血栓性微血管病。超过80%有可用数据的患者血小板计数降低且乳酸脱氢酶升高；99%检测到裂红细胞。最常见的症状/并发症是神经症状、出血和疼痛。大多数患者（86%）有2种或更多Elixhauser合并症。超过80%的患者接受了1种或更多TTP相关治疗，主要是血浆置换。平均住院时间为11.5天；5%的患者在住院期间死亡。30天、60天和90天的再入院率分别为20%、26%和28%。整个索引住院期间医院的住院总费用中位数（四分位间距）为33,221美元（19,431 - 64,901美元）。
严重ADAMTS13缺乏患者有巨大的临床负担，死亡率和再入院率高，给医院带来高昂费用。非常需要一种能替代ADAMTS13的疗法，从而解决由这种缺乏导致的症状和并发症的根本原因。
