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卢苏替尼在准备接受侵入性操作的低血小板计数肝硬化患者中的真实世界经验。

Real-life experience of lusutrombopag for cirrhotic patients with low platelet counts being prepared for invasive procedures.

机构信息

Department of Gastroenterology and Hepatology, Musashino Red Cross Hospital, Tokyo, Japan.

First Department of Internal Medicine, Faculty of Medicine, University of Yamanashi, Yamanashi, Japan.

出版信息

PLoS One. 2019 Feb 15;14(2):e0211122. doi: 10.1371/journal.pone.0211122. eCollection 2019.

Abstract

BACKGROUND AND AIMS

The present study aimed to report our real-life experience of the TPO receptor agonist lusutrombopag for cirrhotic patients with low platelet counts.

METHODS

We studied platelet counts in 1,760 cirrhotic patients undergoing invasive procedures at our hospital between January 2014 and December 2017. In addition, we studied 25 patients who were administered lusutrombopag before invasive procedures between June 2017 and January 2018. Effectiveness of lusutrombopag to raise platelet counts and to avoid transfusion and treatment-related adverse events were analyzed.

RESULTS

In 1,760 cirrhotic patients without lusutrombopag prior to invasive procedures, proportion of patients whose platelet counts <50,000/μL and needed platelet transfusions were 66% (n = 27/41) for radiofrequency ablation, 43% (n = 21/49) for transarterial chemoembolization, and 55% (n = 21/38) for endoscopic injection sclerotherapy / endoscopic variceal ligation, respectively. In 25 cirrhotic patients treated by lusutrombopag prior to the invasive procedures, platelet counts significantly increased compared with baseline (82,000 ± 26,000 vs. 41,000 ± 11,000/μL) (p < 0.01). Out of 25 patients, only 4 patients (16%) needed platelet transfusion before the invasive procedures. The proportion of patients with low platelet count and who needed platelet transfusions was significantly low in patients treated with lusutrombopag compared to those not treated with lusutrombopag (16% (4/25) vs. 54% (69/128), p = 0.001). Platelet counts after lusutrombopag treatment and prior to invasive procedures were lower in patients with a baseline platelet count ≤30,000/μL (n = 8) compared with those with a baseline platelet count >30,000/μL (n = 17) (50,000 ± 20,000 vs 86,000 ± 26,000/μL, p = 0.002). Patients with a baseline platelet count ≤30,000/μL with spleen index (calculated by multiplying the transverse diameter by the vertical diameter measured by ultrasonography) ≥40 cm2 (n = 3) had a lower response rate to lusutrombopag compared to those with spleen index <40 cm2 (n = 5) (0% vs. 100%, p = 0.02). Hemorrhagic complication was not observed. Recurrence of portal thrombosis was observed and thrombolysis therapy was required in one patient who had prior history of thrombosis.

CONCLUSIONS

Lusutrombopag is an effective and safe drug for thrombocytopenia in cirrhotic patients, and can reduce the frequency of platelet transfusions.

摘要

背景与目的

本研究旨在报告我们在真实世界中使用 TPO 受体激动剂芦曲泊帕治疗伴有血小板计数降低的肝硬化患者的经验。

方法

我们研究了 2014 年 1 月至 2017 年 12 月期间在我院接受侵入性操作的 1760 例肝硬化患者的血小板计数。此外,我们还研究了 2017 年 6 月至 2018 年 1 月期间 25 例在侵入性操作前接受芦曲泊帕治疗的患者。分析了芦曲泊帕升高血小板计数、避免输血和治疗相关不良事件的有效性。

结果

在 1760 例未接受芦曲泊帕治疗的肝硬化患者中,血小板计数<50,000/μL且需要血小板输注的患者比例分别为射频消融术患者 66%(27/41)、经动脉化疗栓塞术患者 43%(21/49)和内镜下硬化剂注射/内镜下静脉曲张结扎术患者 55%(21/38)。在 25 例接受芦曲泊帕治疗的肝硬化患者中,与基线相比,血小板计数显著升高(82,000±26,000 vs. 41,000±11,000/μL)(p<0.01)。25 例患者中,仅 4 例(16%)在侵入性操作前需要血小板输注。与未接受芦曲泊帕治疗的患者相比,接受芦曲泊帕治疗的患者中血小板计数低且需要血小板输注的患者比例显著降低(16%(4/25)vs. 54%(69/128),p=0.001)。在基线血小板计数≤30,000/μL(n=8)的患者中,芦曲泊帕治疗后和侵入性操作前的血小板计数低于基线血小板计数>30,000/μL(n=17)的患者(50,000±20,000 vs 86,000±26,000/μL,p=0.002)。基线血小板计数≤30,000/μL且脾指数(通过超声测量的横径乘以纵径计算)≥40 cm2(n=3)的患者对芦曲泊帕的反应率低于脾指数<40 cm2(n=5)的患者(0% vs. 100%,p=0.02)。未观察到出血并发症。1 例有血栓形成史的患者出现门静脉血栓再发,需要溶栓治疗。

结论

芦曲泊帕是治疗肝硬化患者血小板减少症的一种有效且安全的药物,可减少血小板输注的频率。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6ef3/6377090/5836cf89733c/pone.0211122.g001.jpg

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