Systemic bevacizumab for high-output cardiac failure in hereditary hemorrhagic telangiectasia: an international survey of HHT centers
Systemic bevacizumab for high-output cardiac failure in hereditary hemorrhagic telangiectasia: an international survey of HHT centers
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DOI：
10.1186/s13023-019-1239-6
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发表时间：
2019-11-14
影响因子：
3.7
通讯作者：
Iyer, Vivek N.
中科院分区：
文献类型：
--
作者：
Al-Samkari, Hanny;Albitar, Hasan A.;Iyer, Vivek N.
背景全身性贝伐单抗是一种新型的靶向抗血管生成治疗遗传性出血性毛细血管扩张症（HHT）高输出量心力衰竭（HOCF）的药物，但已发表的数据有限。这项基于调查的研究测量了医生报告的HHT-HOCF中全身性贝伐珠单抗的安全性、有效性和当前治疗实践。方法对31个国际HHT卓越中心的中心主任进行了27项调查。结果有效率为74%，各中心共报告150例患者接受了系统性贝伐单抗治疗HHT-HOCF。大约三分之二的中心治疗了≥ 5例患者。所有研究中心均采用5 mg/kg剂量进行诱导治疗，大多数研究中心每2周一次给药6次（范围4-6次），尽管维持治疗方案差异很大。中心主任报告贝伐珠单抗是有效的，55%的患者报告心脏指数和HOCF症状显着改善，在大多数患者接受贝伐珠单抗治疗，虽然正常化的心脏参数是罕见的。不良事件不常见，四分之三的中心报告不良事件发生率<10%。因不良事件或无效而停药的情况很少。贝伐珠单抗通常由血液科医生和肺病科医生（分别为50%和39%的中心）给药，启动阈值差异很大。虽然有一半的中心报告说，在保险审批过程中遇到了困难，但70%的中心最终能够为大多数或所有患者获得保险。结论贝伐珠单抗治疗HHT HOCF安全有效，是一种广泛应用的治疗方法。在HHT相关HOCF中，HHT中心在维持治疗实践和开始贝伐珠单抗治疗的疾病严重程度阈值方面似乎差异很大。
Background Systemic bevacizumab is a novel targeted anti-angiogenic therapy for high-output cardiac failure (HOCF) in hereditary hemorrhagic telangiectasia (HHT) but published data is limited. This survey-based study measured physician-reported safety, effectiveness and current treatment practices for systemic bevacizumab in HHT-HOCF. Methods A 27-item survey was sent to center directors of 31 international HHT Centers of Excellence. Results Response rate was 74% with centers reporting 150 total patients receiving systemic bevacizumab for HHT-HOCF. Approximately two-thirds of centers had treated >= 5 patients. All centers utilize a 5 mg/kg dose for induction treatment and most administer 6 doses (range, 4-6) every 2 weeks, although maintenance regimens varied considerably. Center directors reported bevacizumab to be effective, with 55% reporting significant improvement in cardiac index and HOCF symptoms in most patients treated with bevacizumab, although normalization of cardiac parameters was uncommon. Adverse events were uncommon with three-quarters of centers reporting adverse event rates < 10%. Discontinuation for adverse events or ineffectiveness was rare. Bevacizumab was typically administered by hematologists and pulmonologists (50 and 39% of centers, respectively), with highly variable thresholds for initiation. Although half the centers reported difficulty with the insurance approval process, 70% of centers were ultimately able to obtain coverage for most or all of their patients. Conclusions Systemic bevacizumab is a widely-used therapy for HHT-HOCF with reasonable safety and effectiveness. HHT centers appear to vary considerably in maintenance treatment practices and disease severity thresholds for initiation of bevacizumab in HHT-related HOCF.