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Abstract：
European real-world data indicate that front-line treatment with caplacizumab is associated with improved clinical outcomes compared with delayed caplacizumab treatment. The objective of the study was to describe the characteristics, treatment patterns, and outcomes in hospitalized patients with an immune-mediated thrombotic thrombocytopenic purpura (iTTP) episode treated with front-line versus delayed caplacizumab in the US. This retrospective cohort analysis of a US hospital database included adult patients (≥18 years) with an acute iTTP episode (a diagnosis of thrombotic microangiopathy and ≥1 therapeutic plasma exchange [TPE] procedure) from January 21, 2019, to February 28, 2021. Unadjusted baseline characteristics, treatment patterns, healthcare resource utilization, and costs were compared between patients who received front-line versus delayed (<2 vs ≥2 days after TPE initiation) caplacizumab treatment. Out of 39 patients, 16 (41.0%) received front-line and 23 (59.0%) received delayed treatment with caplacizumab. Baseline characteristics and symptoms were similar between the two groups. Patients who received front-line caplacizumab treatment had significantly fewer TPE administrations (median: 5.0 vs 12.0); and a significantly shorter hospital stay (median: 9.0 days vs 16.0 days) than patients receiving delayed caplacizumab therapy. Both of these were significantly lower in comparison of means (t-test P < .01). Median inpatient costs (inclusive of caplacizumab costs) were 54% higher in the delayed treated patients than in the front-line treated patients (median: $112 711 vs $73 318). TPE-specific cost was lower in the front-line treated cohort (median: $6 989 vs $10 917). In conclusion, front-line treatment with caplacizumab had shorter hospitalizations, lower healthcare resource utilization, and lower costs than delayed caplacizumab treatment after TPE therapy.
摘要：
欧洲现实世界的数据表明，与延迟的caplacizumab治疗相比，使用caplacizumab的一线治疗与改善的临床结果相关。这项研究的目的是描述特征，治疗模式,在美国，使用一线治疗与延迟卡普拉斯珠单抗治疗的免疫介导性血栓性血小板减少性紫癜（iTTP）发作的住院患者的结局和结局。这项对美国医院数据库的回顾性队列分析包括2019年1月21日至2021年2月28日患有急性iTTP发作（诊断为血栓性微血管病和≥1次治疗性血浆置换[TPE]手术）的成年患者（≥18岁）。未调整的基线特征，治疗模式,医疗保健资源利用，比较了接受一线治疗与延迟治疗(TPE开始后<2天vs≥2天)卡普拉斯单抗治疗的患者之间的费用.在39名患者中，16例（41.0%）接受一线治疗，23例（59.0%）接受卡普拉斯单抗延迟治疗。两组的基线特征和症状相似。接受一线caplacizumab治疗的患者的TPE给药明显减少（中位数：5.0vs12.0）；与接受延迟的caplacizumab治疗的患者相比，住院时间明显缩短（中位数：9.0天vs16.0天）。两者在平均值比较中显著较低(t检验P<.01)。延迟治疗患者的住院费用中位数（包括卡普拉斯单抗费用）比一线治疗患者高54％（中位数：$112711vs$73318）。一线治疗队列中TPE特异性费用较低（中位数：$6989vs$10917）。总之,卡普拉斯单抗一线治疗的住院时间较短，医疗资源利用率较低，与TPE治疗后延迟的caplacizumab治疗相比，费用更低。
