Intravenous Bevacizumab for Refractory Hereditary Hemorrhagic Telangiectasia-Related Epistaxis and Gastrointestinal Bleeding

Mayo Clin Proc. 2018 Feb;93(2):155-166. doi: 10.1016/j.mayocp.2017.11.013. Epub 2018 Jan 24.

Abstract

Objective: To present a multiyear clinical experience with intravenous bevacizumab for the management of severe gastrointestinal bleeding and/or epistaxis in patients with hereditary hemorrhagic telangiectasia (HHT).

Patients and methods: All patients treated with intravenous bevacizumab for severe hereditary hemorrhagic telangiectasia-related bleeding from June 1, 2013, through January 31, 2017, were included in this report. Severity of epistaxis (determined using the Epistaxis Severity Score questionnaire); hemoglobin, iron, and ferritin levels; and quality of life data were collected serially in all patients.

Results: Intravenous bevacizumab was administered to 34 patients using a standardized treatment protocol. Anemia was primarily related to severe epistaxis (n=15, 44%), severe gastrointestinal bleeding (n=4, 12%), or both (n=15, 44%), with a median baseline hemoglobin level of 9.1 g/dL (range, 8.3-10.5 gm/dL; to convert to mmol/L, multiply by 0.62). Red blood cell (RBC) transfusions had been administered to 28 patients (82%). Of these, 16 patients (47%) were RBC transfusion dependent and had received a median of 75 RBC transfusions (range, 4->500 RBC units) before bevacizumab initiation. The median length of follow-up was 17.6 months from the beginning of bevacizumab treatment (range, 3-42.5 months). There was a significant reduction in epistaxis severity scores (P<.001) and RBC transfusion requirements (P=.007) after completion of the initial bevacizumab treatment cycle. New-onset or worsened hypertension was noted in 4 patients, with 1 patient experiencing hypertensive urgency with a temporary decline in renal function.

Conclusion: Intravenous bevacizumab is an effective treatment option for patients with severe anemia related to epistaxis and/or gastrointestinal bleeding. Further studies are needed to establish a dose-response relationship as well as clinical, genetic, and biomarker predictors of response.

MeSH terms

  • Administration, Intravenous
  • Aged
  • Anemia, Refractory* / diagnosis
  • Anemia, Refractory* / etiology
  • Anemia, Refractory* / therapy
  • Angiogenesis Inhibitors / administration & dosage
  • Bevacizumab / administration & dosage*
  • Epistaxis* / diagnosis
  • Epistaxis* / etiology
  • Epistaxis* / therapy
  • Female
  • Ferritins / blood
  • Gastrointestinal Hemorrhage* / diagnosis
  • Gastrointestinal Hemorrhage* / etiology
  • Gastrointestinal Hemorrhage* / therapy
  • Humans
  • Male
  • Middle Aged
  • Minnesota
  • Quality of Life*
  • Retrospective Studies
  • Severity of Illness Index
  • Telangiectasia, Hereditary Hemorrhagic* / blood
  • Telangiectasia, Hereditary Hemorrhagic* / complications
  • Telangiectasia, Hereditary Hemorrhagic* / diagnosis
  • Telangiectasia, Hereditary Hemorrhagic* / psychology
  • Treatment Outcome

Substances

  • Angiogenesis Inhibitors
  • Bevacizumab
  • Ferritins