Characterizing Self-Reported Daily Diary Epistaxis Measures in Hereditary Hemorrhagic Telangiectasia

Area of Science:

• Medical Research
• Clinical Trials
• Patient-Reported Outcomes

Background:

• Hereditary Hemorrhagic Telangiectasia (HHT) is a genetic disorder characterized by frequent nosebleeds (epistaxis).
• Accurate measurement of bleeding severity is crucial for evaluating treatment efficacy in clinical trials.
• Existing methods for assessing epistaxis severity may lack precision or patient-centeredness.

Purpose of the Study:

• To validate a novel clinical trial outcome measure, patient-reported outcome of cumulative bleeding (PRO-CB), derived from daily nosebleed diaries.
• To assess the cumulative weekly gushing bleeding duration (PRO-CGB) as a secondary outcome.
• To evaluate the accuracy of patient recall for Epistaxis Severity Score (ESS) items compared to daily diary entries.

Main Methods:

• Prospective observational study involving 23 HHT patients over three-month run-in periods.
• Participants maintained daily diaries recording nosebleed episodes, duration, and intensity (gushing vs. non-gushing).
• Data analyzed using linear mixed-effects models, correlation tests, and partial autocorrelation analyses.

Main Results:

• High diary compliance (98.8%) was observed.
• Mean weekly PRO-CB was 71.2 minutes; gushing episodes were significantly longer than non-gushing episodes.
• PRO-CB and ESS total scores showed no significant correlation, but ESS items 1 and 2 correlated strongly with diary data.

Conclusions:

• Daily nosebleed diaries are a feasible and accurate method for tracking epistaxis severity in HHT.
• PRO-CB demonstrates potential as a meaningful outcome measure for epistaxis clinical trials, acknowledging inter-patient variability.
• Patient recall for specific ESS items shows varying degrees of accuracy compared to daily diary data.