Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Department of Internal Medicine, Mayo Clinic, Rochester, MN, USA.
Haemophilia. 2020 Nov;26(6):1038-1045. doi: 10.1111/hae.14034. Epub 2020 May 20.
Systemic bevacizumab is a novel targeted therapy for severe epistaxis and chronic gastrointestinal bleeding in hereditary haemorrhagic telangiectasia (HHT), but published data are very limited.
We conducted a survey-based study to characterize current treatment practices and physician-reported safety and effectiveness of systemic bevacizumab for bleeding in (HHT).
A 27-item survey was sent to physician centre directors of 31 International HHT Centers of Excellence.
Response rate was 84%. Approximately half of centres had treated >10 HHT patients with systemic bevacizumab for chronic bleeding for a total of 291 patients treated. All centres utilize a 5 mg/kg dose for induction treatment and most administer six doses (range, 4-8) every 2 weeks. However, maintenance regimens varied considerably between centres. Bevacizumab was highly effective, with 86% reporting significant (>50%) improvement in GI bleeding and/or epistaxis and haemoglobin rise in most patients treated with bevacizumab; 52% reported haemoglobin normalization in most patients. All centres reported adverse event rates <30% and two-thirds of centres reported adverse event rates <10%. Discontinuation for adverse events or inefficacy was rare. Bleeding severity thresholds for initiation of bevacizumab were highly variable, and it is typically administered by haematologists (76% of centres). Two-thirds of centres reported obtaining insurance approval for bevacizumab for most or all patients but 48% reported difficulty in obtaining coverage.
Systemic bevacizumab is widely used to treat bleeding in HHT with excellent physician-reported effectiveness and safety. There is considerable variation in maintenance treatment practices and thresholds for initiation of bevacizumab among HHT centres.
系统贝伐珠单抗是遗传性出血性毛细血管扩张症（HHT）严重鼻出血和慢性胃肠道出血的新型靶向治疗方法，但已发表的数据非常有限。
我们进行了一项基于调查的研究，以描述目前治疗实践以及医生报告的系统贝伐珠单抗治疗（HHT）出血的安全性和有效性。
向 31 个国际 HHT 卓越中心的医师中心主任发送了一份包含 27 个问题的调查问卷。
应答率为 84%。大约一半的中心治疗了> 10 名接受系统贝伐珠单抗治疗慢性出血的 HHT 患者，共治疗了 291 名患者。所有中心均使用 5mg/kg 剂量进行诱导治疗，大多数中心每 2 周给予 6 剂（范围为 4-8 剂）。然而，维持方案在各中心之间差异很大。贝伐珠单抗非常有效，86%的中心报告大多数接受贝伐珠单抗治疗的患者胃肠道出血和/或鼻出血有显著改善（> 50%），大多数患者的血红蛋白升高；52%的中心报告大多数患者的血红蛋白正常化。所有中心报告的不良事件发生率<30%，三分之二的中心报告不良事件发生率<10%。因不良事件或疗效不佳而停药的情况很少见。启动贝伐珠单抗的出血严重程度阈值差异很大，通常由血液科医生（76%的中心）进行。三分之二的中心报告为大多数或所有患者获得了贝伐珠单抗的保险批准，但 48%的中心报告说获得覆盖范围存在困难。
系统贝伐珠单抗广泛用于治疗 HHT 出血，具有出色的医生报告的有效性和安全性。在 HHT 中心，维持治疗实践和贝伐珠单抗启动阈值存在相当大的差异。
