Adding caplacizumab to standard of care in thrombotic thrombocytopenic purpura: a systematic review and meta-analysis.
Adding caplacizumab to standard of care in thrombotic thrombocytopenic purpura: a systematic review and meta-analysis.
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DOI：
10.1182/bloodadvances.2022008443
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发表时间：
2023-05-23
期刊：
影响因子：
7.5
通讯作者：
Pishko, Allyson M.
中科院分区：
文献类型：
--
作者：
Djulbegovic, Mia;Tong, Jiayi;Xu, Alice;Yang, Joanna;Chen, Yong;Cuker, Adam;Pishko, Allyson M.
免疫血栓性血小板减少紫癜（ITTP）是未经治疗的可产生的致命微血管病。随机对照试验（RCT）表明，通过在护理标准（SOC）中添加caplacizumab的响应速度更快。然而，人们对RCT选择偏见和caplacizumab的高昂成本的担忧，保证所有证据，包括现实世界的观察研究。在这项系统的综述和荟萃分析中，我们搜索了使用或不使用caplacizumab的SOC进行评估的比较研究，以治疗ITTP。我们使用Cochrane风险偏置2工具（RCT）和纽卡斯尔 - 奥塔瓦量表（观察性研究）评估了偏见的风险。主要的功效和安全性结果分别是全因死亡率和治疗生气的出血。次要结果包括加剧和复发，难治性ITTP以及响应时间。我们包括2个高质量的RCT和3项观察性研究，其中包括632个参与者的高风险。与SOC相比，Caplacizumab与RCT中死亡的相对风险[RR]无显着降低有关（RR，0.21; 95％置信区间[CI]，0.05-1.74）和观察性研究（RR，0.62; 95％CI，0.07-4.41）。与SOC相比，Caplacizumab与RCT的出血风险增加有关（RR，1.37; 95％CI，1.06-1.77）。在观察性研究中，出血风险没有显着增加（RR，7.10； 95％CI，0.90-56.14）。 caplacizumab的添加与难治性ITTP和加剧风险的显着降低和缩短的响应时间相关，但复发风险增加了。与单独使用SOC相比，毛扫珠蛋白酶的前线并不能显着降低全因死亡率，尽管它会降低难治性疾病风险，缩短反应时间并提高加重率，而牺牲了复发和出血风险。
Immune thrombotic thrombocytopenic purpura (iTTP) is an acquired, fatal microangiopathy if untreated. Randomized controlled trials (RCTs) demonstrated faster time to response with addition of caplacizumab to standard of care (SOC). However, concerns about RCT selection bias and the high cost of caplacizumab warrant examination of all evidence, including real-world observational studies. In this systematic review and meta-analysis, we searched for comparative studies evaluating SOC with or without caplacizumab for the treatment of iTTP. We assessed risk of bias using the Cochrane risk-of-bias-2 tool (RCTs) and the Newcastle-Ottawa Scale (observational studies). The primary efficacy and safety outcomes were all-cause mortality and treatment-emergent bleeding, respectively. Secondary outcomes included exacerbation and relapse, refractory iTTP, and time to response. We included 2 high-quality RCTs and 3 observational studies at high risk of bias comprising 632 total participants. Compared with SOC, caplacizumab was associated with a nonsignificant reduction in the relative risk [RR] of death in RCTs (RR, 0.21; 95% confidence interval [CI], 0.05-1.74) and observational studies (RR, 0.62; 95% CI, 0.07-4.41). Compared with SOC, caplacizumab was associated with an increased bleeding risk in RCTs (RR, 1.37; 95% CI, 1.06-1.77). In observational studies, bleeding risk was not significantly increased (RR, 7.10; 95% CI, 0.90-56.14). Addition of caplacizumab was associated with a significant reduction in refractory iTTP and exacerbation risks and shortened response time but increased relapse risk. Frontline addition of caplacizumab does not significantly reduce all-cause mortality compared with SOC alone, although it reduces refractory disease risk, shortens time to response, and improves exacerbation rates at the expense of increased relapse and bleeding risk.
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影响因子：
4
作者：
Hozo SP;Djulbegovic B;Hozo I
通讯作者：
Hozo I
影响因子：
15.8
作者：
Moher D;Liberati A;Tetzlaff J;Altman DG;PRISMA Group
通讯作者：
PRISMA Group
DOI：
10.1136/bmj.c3920
发表时间：
2010-08-18
期刊：
BMJ (Clinical research ed.)
影响因子：
--
作者：
Cordoba G;Schwartz L;Woloshin S;Bae H;Gøtzsche PC
通讯作者：
Gøtzsche PC
影响因子：
4
作者：
Lo CK;Mertz D;Loeb M
通讯作者：
Loeb M
影响因子：
7.5
作者：
Peyvandi, Flora;Cataland, Spero;Callewaert, Filip
通讯作者：
Callewaert, Filip