High importance
Jul 13, 2026
To evaluate the effectiveness of doxycycline in reducing epistaxis severity in patients with hereditary hemorrhagic telangiectasia (HHT), with a focus on genotype-stratified responses (specifically active receptor-like kinase 1, endoglin, and SMAD4).
A retrospective cohort study at the University of Florida's Hereditary Hemorrhagic Telangiectasia Center, involving 41 adult patients diagnosed with HHT. Patients were classified as responders or nonresponders based on changes in epistaxis severity scores (ESS) pre- and post-treatment. Additional assessments included hemoglobin and hematocrit values.
Among the 41 patients, there was a statistically significant reduction in ESS from a median baseline of 4.4 to 3.3 after treatment (P < .0001), with 26 patients classified as responders showing a notable decrease in ESS from 4.6 to 2.4 (P < .0001). However, changes in hemoglobin and hematocrit were not statistically significant, and genotype did not correlate with the treatment response.
This study was limited by its retrospective design, small sample size, and heterogeneity in genotype distribution, which may limit the generalizability of findings. The relationship between genotype and treatment response was not significant, suggesting further exploration is warranted.
These findings suggest that doxycycline could be a viable treatment option for patients with HHT experiencing epistaxis, potentially providing a safer and more accessible intervention. Understanding treatment responses across different genetic backgrounds can inform personalized management approaches in HHT.
Patients with hereditary hemorrhagic telangiectasia (HHT) experience recurrent epistaxis. Doxycycline has been proposed as a possible treatment, although its efficacy remains controversial. Whether the most common genotypes (activin receptor-like kinase 1, endoglin, and SMAD4) contribute to a differential response has not been investigated. In this study, we evaluate the effectiveness of doxycycline among the different HHT genotypes. A retrospective cohort study was conducted at the University of Florida's Hereditary Hemorrhagic Telangiectasia Center. Forty-one adult patients (aged ≥18 years) with HHT, diagnosed by Curacao criteria and genetic testing, were classified as responders and nonresponders based on the minimal clinically important change between pre- and posttreatment epistaxis severity scores (ESS). Hemoglobin and hematocrit values were also collected to assess treatment response. Overall, the cohort was 61% female and 90.2% White, with a mean age of 58.1 years; 26 responders had a decrease (P < .0001) in ESS from a baseline median of 4.6 (interquartile range [IQR], 3.3) to posttreatment ESS of 2.4 (IQR, 1.9). The median baseline ESS for the total cohort was 4.4 (IQR, 3.3), and after a mean follow-up of 4.1 months, it significantly (P < .0001) decreased to an ESS of 3.3 (IQR, 3.2). The mean hemoglobin and hematocrit values did not exhibit significant changes. The relationship between genotype and doxycycline response was not statistically significant. Patients with HHT treated with doxycycline showed an overall reduction in epistaxis severity, which did not appear to be associated with genotype. Doxycycline may be a safe, effective, and accessible treatment option for epistaxis in HHT.