Abstract：
CONCLUSIONS: In an effort to expedite the publication of articles, AJHP is posting manuscripts online as soon as possible after acceptance. Accepted manuscripts have been peer-reviewed and copyedited, but are posted online before technical formatting and author proofing. These manuscripts are not the final version of record and will be replaced with the final article (formatted per AJHP style and proofed by the authors) at a later time.
OBJECTIVE: 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.
METHODS: 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.
RESULTS: 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).
CONCLUSIONS: 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.
OBJECTIVE: 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.
METHODS: 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.
RESULTS: 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).
CONCLUSIONS: 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.
摘要：
结论：为了加快文章的发表，AJHP在接受后尽快在线发布手稿。接受的手稿经过同行评审和复制编辑，但在技术格式化和作者打样之前在线发布。这些手稿不是记录的最终版本，将在以后替换为最终文章（按照AJHP样式格式化并由作者证明）。
目的：严重ADAMTS13缺乏症（活度<10%）是血栓性血小板减少性紫癜（TTP）的诊断阈值，并与各种临床症状相关，异常的实验室结果，和长期并发症。
方法：本回顾性研究，非干预性队列研究使用PremierHealthcare数据库确定了2016年1月1日至2020年3月31日美国医院ADAMTS13活动<10%的患者.目的是描述病人的特征，实验室结果,合并症（通过Elixhauser合并症指数测量），症状,逗留时间,治疗模式,死亡率,住院费用,和再入院率(描述性总结)。住院费用计算为医院的总费用。
结果：有211例ADAMTS13严重缺乏；89%的患者有TTP相关诊断，其中62%的人主要诊断为血栓性微血管病。有可用数据的患者中有80％以上的血小板计数降低和乳酸脱氢酶升高；99％的人检测到分裂细胞。最常见的症状/并发症是神经系统，出血,和痛苦。大多数患者（86％）有2种或更多的Elixhauser合并症。超过80％的患者接受了1种或多种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、60和90天再入院率为20%，26%,28%，分别。在整个指数入院期间，医院的住院总费用中位数（四分位数范围）为$33,221（$19,431-$64,901）。
结论：严重ADAMTS13缺乏的患者具有巨大的临床负担，有很高的死亡率和再入院率，并为医院带来高昂的成本。对于替代ADAMTS13的疗法存在高度需求,从而解决由这种缺陷引起的症状和并发症的根本原因。
目的：严重ADAMTS13缺乏症（活度<10%）是血栓性血小板减少性紫癜（TTP）的诊断阈值，并与各种临床症状相关，异常的实验室结果，和长期并发症。
方法：本回顾性研究，非干预性队列研究使用PremierHealthcare数据库确定了2016年1月1日至2020年3月31日美国医院ADAMTS13活动<10%的患者.目的是描述病人的特征，实验室结果,合并症（通过Elixhauser合并症指数测量），症状,逗留时间,治疗模式,死亡率,住院费用,和再入院率(描述性总结)。住院费用计算为医院的总费用。
结果：有211例ADAMTS13严重缺乏；89%的患者有TTP相关诊断，其中62%的人主要诊断为血栓性微血管病。有可用数据的患者中有80％以上的血小板计数降低和乳酸脱氢酶升高；99％的人检测到分裂细胞。最常见的症状/并发症是神经系统，出血,和痛苦。大多数患者（86％）有2种或更多的Elixhauser合并症。超过80％的患者接受了1种或多种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、60和90天再入院率为20%，26%,28%，分别。在整个指数入院期间，医院的住院总费用中位数（四分位数范围）为$33,221（$19,431-$64,901）。
结论：严重ADAMTS13缺乏的患者具有巨大的临床负担，有很高的死亡率和再入院率，并为医院带来高昂的成本。对于替代ADAMTS13的疗法存在高度需求,从而解决由这种缺陷引起的症状和并发症的根本原因。
