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

• Published in: PloS one Vol. 15; no. 2; p. e0228486
• Main Authors: Vázquez, Carolina, Gonzalez, María Laura, Ferraris, Augusto, Bandi, Juan Carlos, Serra, Marcelo Martín
• Format: Journal Article
• Language: English
• Published: United States Public Library of Science 07.02.2020; Public Library of Science (PLoS)
• Subjects: Adult; Aged; Analysis; Anemia; Anemia, Refractory - drug therapy; Anemia, Refractory - etiology; Anemia, Refractory - pathology; Angiogenesis inhibitors; Anticoagulants; Antineoplastic agents; Argentina; Bevacizumab; Bevacizumab - therapeutic use; Biology and Life Sciences; Bleeding; Blood; Bridge failure; Bridges (Structures); Cardiac output; Cardiac patients; Care and treatment; Congestive heart failure; Diuretics; Endoscopy; Female; Glycosylated hemoglobin; Heart failure; Hemodynamic responses; Hemodynamics; Hemoglobin; Hemoglobins; Hemorrhage; Hereditary hemorrhagic telangiectasia; Hospitals; Humans; Hypertension; Immunotherapy; Internal medicine; Iron; Iron deficiency; Iron deficiency anemia; Liver; Liver Diseases - drug therapy; Liver Diseases - etiology; Liver Diseases - pathology; Liver transplantation; Male; Medicine and Health Sciences; Middle Aged; Monoclonal antibodies; Mortality; Nutrient deficiency; Organ transplantation; Patients; Rankings; Refractory anemia; Retrospective Studies; Schedules; Severity of Illness Index; Surgery; Telangiectasia, Hereditary Hemorrhagic - complications; Telangiectasia, Hereditary Hemorrhagic - drug therapy; Telangiectasis; Transfusion; Transplantation; Treatment Outcome; Variables; Vascular endothelial growth factor; Argentina
• ISSN: 1932-6203; 1932-6203
• DOI: 10.1371/journal.pone.0228486

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.