Intranasal bevacizumab injections improve quality of life in HHT patients

Abstract

Background: Epistaxis is the most common symptom in patients with hereditary hemorrhagic telangiectasia (HHT), with the greatest negative impact on quality of life (QoL). Repeated intranasal submucosal bevacizumab injections (RISBI) is a relatively new treatment option for moderate or severe grades of epistaxis in HHT. However, the effect of RISBI on QoL is not fully evaluated.Study Design: Prospective, non-comparative study. Materials and Methods: Patients treated by RISBI for HHT-associated epistaxis between June 2011 and August 2013 were prospectively invited to the present study. The end of follow-up was October 2013. The patients were requested to answer QoL questionnaires before the first treatment, and 6-8 weeks after the last treatment. Three levels of QoL were assessed: Overall QoL using Cantril's Self-Anchoring Ladder; Health-related QoL using Short Form 36 (SF-36), and Disease-specific QoL. Psychological distress was measured with the Hospital Anxiety and Depression scale (HADS).Results: Thirty-three patients were treated with RISBI during the period referred to above. Twenty-three patients completed the QoL questionnaires. The average number of treatments per patient was 2.15 AE 1.3 (Range: 1-5). The mean overall QoL improved from 6.47 AE 1.9 to 7.26 AE 1.6 (P < .05). Several dimensions measured by SF-36 were significantly improved with a medium to strong effect size. HADS demonstrated a significant decrease in psychological distress after the last treatment.Conclusion: HHT patients treated by RISBI improved in several aspects of quality of life, and psychological distress decreased. RISBI was an effective treatment option for moderate and severe grades of HHT-associated epistaxis.

Health-Related Quality of Life Outcome Measures in Individuals With Hereditary Hemorrhagic Telangiectasia: A Scoping Review

“…Physical health was assessed in 56 studies (88.9%). 12,14–17,19,22–32,34–44,46–51,53–55,57,59–75,100 Twenty pre-existing instruments were used, and 15 studies used custom instruments. 22,24,32,34,35,38,39,42,50,53,54,57,59,65,…”

“…Social health was assessed in 39 studies (61.9%). 12,16,17,19,22,23,25,27–29,31,34,38,41,44,45,47–50,52,53,55–57,60–63,66–68,70,74,75,100 Among them, 12 pre-existing instruments were used, and 7 studies used custom instruments. The SF-36 (22; 57.9%) was the most used tool, followed by the GBI (4; 6.4%), the NOSE-HHT (2; 5.1%), and the SNOT-22 (2; 5.1 %).…”

A comparative study of two grading systems for epistaxis in hereditary haemorrhagic telangiectasia

“…This database includes HHT patients, diagnosed clinically according to Curaçao criteria 3-4, and/or by additional genetic testing in the case of Curaçao 1-2. The patients included in this study were previously included in other studies concerning HHT-associated epistaxis (12)(13)(14)(15) and we collected IFT and ESS scores simultaneously. In these previous studies, the IFT and ESS scoring was performed either by the patients themselves or by the responsible doctor.…”

An in vitro study on the effect of bevacizumab on endothelial cell proliferation and VEGF concentration level in patients with hereditary hemorrhagic telangiectasia

“…Genetically, HHT is caused by mutations encoding the ENG , ACVRL1 and Smad4 proteins. These proteins are part of the TGF-ß1/BMP signaling pathway ( 7 , 8 ), which regulates cell differentiation and angiogenesis in endothelial cells ( 9 ).…”