Bevacizumab for treating Hereditary Hemorrhagic Telangiectasia patients with severe hepatic involvement or refractory anemia

Abstract

ObjectiveTo report our clinical experience with bevacizumab in a cohort of Hereditary Hemorrhagic Telangiectasia (HHT) patients with severe hepatic involvement and/or refractory anemia. MethodsObservational, 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. ResultsTwenty 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][3][4][5][6]. In heart failure group, pretreatment median cardiac index was 4.5 L/min/m 2 [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.

Hereditary Hemorrhagic Telangiectasia Induced Portosystemic Encephalopathy: A Case Report and Literature Review

“…In patients who are unresponsive to these medications, liver transplantation has been proposed as a curative option ( 3 ). In addition, systemic treatment, such as with Bevacizumab, has been given to HHT patients with hepatic AVMs ( 19 , 20 ).…”

Hereditary Hemorrhagic Telangiectasia and Refractory Ascites

“… 8 There are reports of the antiangiogenic agent bevacizumab improving cardiac hemodynamics in patients with HHT, and in some cases, resulting in optimization of HOCF before OLT. 9 …”

Hereditary hemorrhagic telangiectasia (Osler‑Weber‑Rendu syndrome) - Part II. Pharmacological therapy and international guidelines for the therapy 2020

“…Játra k transplantaci však nejsou ihned k dispozici, a proto je třeba interval do transplantace jater překlenout vhodnou léčbou. Literatura uvádí, že pravidelné aplikace bevacizumabu, který bude podrobněji zmíněn v dalším odstavci, snížily průtok jaterními cévními malformacemi, snížily plicní hypertenzi a umožnily tak pacientovi dožít se transplantace jater (29,30).…”