ICERA Consulting Ltd., 17 Redbridge Close, Swindon, Wiltshire, UK.
Shionogi Inc., Florham Park, NJ, USA.
Adv Ther. 2022 Sep;39(9):4169-4188. doi: 10.1007/s12325-022-02235-w. Epub 2022 Jul 14.
Lusutrombopag is an oral thrombopoietin receptor agonist (TPO-RA). Clinical trials have shown lusutrombopag's efficacy in reducing need for preoperative platelet transfusion in patients with chronic liver disease (CLD) and severe thrombocytopenia. This analysis assessed efficacy and safety of lusutrombopag in patients with severe thrombocytopenia and CLD undergoing planned invasive procedures.
An electronic database search (through 1 December 2020) identified three randomised, placebo-controlled, double-blind clinical trials comparing lusutrombopag with placebo in patients with CLD and platelet count below 50 × 10/L scheduled to undergo a procedure with a perioperative bleeding risk. A random-effects meta-analysis examined treatment effect, with Cochrane Collaboration's tool assessing risk of bias.
The meta-analysis included 343 (lusutrombopag 3 mg, n = 173; placebo, n = 170) patients. More patients met the criteria for treatment response (platelet count at least 50 × 10/L and increase of at least 20 × 10/L from baseline anytime during the study) with lusutrombopag versus placebo (risk ratio [RR] 6.39; 95% confidence interval [CI] 3.69, 11.07; p < 0.0001). The primary efficacy outcome, proportion of patients requiring no platelet transfusion and no rescue therapy for bleeding for at least 7 days post procedure, was achieved by more patients treated with lusutrombopag versus placebo (RR 3.42; 95% CI 1.86, 6.26; p = 0.0001). The risk of any bleeding event was significantly lower with lusutrombopag compared to placebo (RR 0.55; 95% CI 0.32, 0.95; p = 0.03); conversely, thrombosis event rates were similar between lusutrombopag and placebo (RR 0.79; 95% CI 0.19, 3.24; p = 0.74).
This meta-analysis showed that treatment of severe thrombocytopenia with lusutrombopag in patients with CLD prior to a planned invasive procedure was efficacious and safe in increasing platelet counts, avoiding the need for platelet transfusions, and reducing risk of bleeding, thereby enhancing the certainty of evidence supporting the efficacy and safety of lusutrombopag.
芦曲泊帕是一种口服血小板生成素受体激动剂（TPO-RA）。临床试验表明，芦曲泊帕可减少慢性肝病（CLD）和严重血小板减少症患者术前血小板输注的需求。本分析评估了芦曲泊帕在计划行有创操作的严重血小板减少症和 CLD 患者中的疗效和安全性。
电子数据库检索（截至 2020 年 12 月 1 日）确定了三项随机、安慰剂对照、双盲临床试验，比较了芦曲泊帕与安慰剂在血小板计数低于 50×10/L 且计划行围手术期出血风险的有创操作的 CLD 患者中的疗效。采用随机效应荟萃分析检查治疗效果，并用 Cochrane 协作网工具评估偏倚风险。
荟萃分析纳入了 343 名（芦曲泊帕 3mg，n=173；安慰剂，n=170）患者。与安慰剂相比，更多的患者达到了治疗反应标准（研究期间任何时候血小板计数至少为 50×10/L，且比基线至少增加 20×10/L）（风险比 [RR]6.39；95%置信区间 [CI]3.69，11.07；p<0.0001）。主要疗效结局为至少 7 天内无需血小板输注和无出血补救治疗的患者比例，接受芦曲泊帕治疗的患者多于安慰剂（RR 3.42；95%CI 1.86，6.26；p=0.0001）。与安慰剂相比，芦曲泊帕治疗组任何出血事件的风险显著降低（RR 0.55；95%CI 0.32，0.95；p=0.03）；相反，芦曲泊帕和安慰剂组血栓事件发生率相似（RR 0.79；95%CI 0.19，3.24；p=0.74）。
本荟萃分析表明，在计划行有创操作前，芦曲泊帕治疗 CLD 合并严重血小板减少症可有效且安全地增加血小板计数，避免血小板输注，并降低出血风险，从而提高支持芦曲泊帕疗效和安全性的证据确定性。
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