A.R.G Argentine Rendu Study Group, Buenos Aires, Argentina.
Internal Medicine Department, Hospital Italiano, Buenos Aires, Argentina.
PLoS One. 2020 Feb 7;15(2):e0228486. doi: 10.1371/journal.pone.0228486. eCollection 2020.
To report our clinical experience with bevacizumab in a cohort of Hereditary Hemorrhagic Telangiectasia (HHT) patients with severe hepatic involvement and/or refractory anemia.
Observational, ambispective study of the Institutional Registry of HHT at Hospital Italiano de Buenos Aires. Patients were treated with bevacizumab due to iron deficiency refractory anemia secondary to nasal/gastrointestinal bleeding and/or high output cardiac failure. We describe basal clinical data, bevacizumab schedules, efficacy outcomes and adverse events. Wilcoxon signed ranks test and longitudinal analysis were conducted.
Twenty adult patients were included from July 2013 to June 2019. Clinical indications were: 13 for anemia, 4 for heart failure and 3 for both. In the anemia group, median pretreatment hemoglobin was 8.1 g/dl [IQR: 7.2-8.4] and median transfusion requirement was 4 units [2-6]. In heart failure group, pretreatment median cardiac index was 4.5 L/min/m2 [4.1-5.6] and cardiac output was 8.3 L/min [7.5-9.2]. Bevacizumab 5 mg/kg/dose every 2 weeks for 6 applications was scheduled. By the end of induction, median hemoglobin at 3 months was 10.9 g/dl [9.5-12.8] (p = 0.01) and median transfusion requirement 0 units [0-1] (p<0.01), and this effect was more or less sustained during a year. Regarding heart failure group, two patients had complete hemodynamic response and achieved liver transplantation and two had partial response. No serious adverse events were registered.
Bevacizumab is a promising line of treatment for HHT patients with refractory anemia. For patients with high output cardiac failure, bevacizumab may be useful as bridge therapy awaiting for liver transplantation.
报告我们在一组有严重肝脏受累和/或难治性贫血的遗传性出血性毛细血管扩张症（HHT）患者中使用贝伐珠单抗的临床经验。
对布宜诺斯艾利斯意大利医院 HHT 机构注册处进行观察性、前瞻性研究。由于鼻腔/胃肠道出血和/或高输出性心力衰竭导致缺铁性难治性贫血，患者接受贝伐珠单抗治疗。我们描述了基础临床数据、贝伐珠单抗方案、疗效结果和不良事件。采用 Wilcoxon 符号秩检验和纵向分析。
2013 年 7 月至 2019 年 6 月共纳入 20 例成年患者。临床适应证为：贫血 13 例，心力衰竭 4 例，两者均有 3 例。在贫血组中，预处理血红蛋白中位数为 8.1 g/dl [IQR：7.2-8.4]，中位输血需求为 4 单位 [2-6]。在心力衰竭组中，预处理时的心脏指数中位数为 4.5 L/min/m2 [4.1-5.6]，心输出量为 8.3 L/min [7.5-9.2]。贝伐珠单抗 5mg/kg/剂量，每 2 周 6 次。诱导结束时，3 个月时的血红蛋白中位数为 10.9 g/dl [9.5-12.8]（p=0.01），输血需求中位数为 0 单位 [0-1]（p<0.01），并且在一年期间这种效果或多或少地保持稳定。对于心力衰竭组，两名患者有完全的血液动力学反应，成功进行了肝移植，两名患者有部分反应。未出现严重不良事件。
贝伐珠单抗是治疗难治性贫血的 HHT 患者的一种有前途的治疗方法。对于高输出性心力衰竭患者，贝伐珠单抗可能作为桥接治疗，等待肝移植。
