Introduction Hereditary angioedema (HAE) is a rare disorder characterized by recurrent, unpredictable episodes of swelling. Social determinants of health (SDOH) may impact healthcare outcomes/experiences among those with HAE. Methods Using Inovalon's closed claims, patients had ≥2 medical claims for HAE (D84.1), or angioedema (T783XXX) and ≥1 prescription for an HAE medication during the case-finding period ((CFP), 1/1/2017 - 9/30/2021). Patients were continuously enrolled in the health-plan for 12- and 24-months pre/post index respectively and excluded if they had history of HAE during the pre-period. Index was the first claim for HAE/angioedema or HAE pharmacological treatment during the CFP. Race/ethnicity, income and rurality were investigated as factors impacting outcomes including the percentage of patients with: a claim for long-term prophylaxis (LTP), a claim for fresh frozen plasma (FFP), and Emergency Department (ED) visit or hospitalization. Results African American individuals had the lowest percentage of LTP use (12.8%) and highest FFP use (72.2%). LTP and FFP was used in 19.2% and 63.4% of patients, respectively. In multivariable models adjusting for age and gender, African American individuals (odds ratio (OR) 4.92, 95%CI, (2.71,8.93)) and Hispanic individuals (OR 2.67, 95% CI, (1.29,5.53)) had higher odds of having an ED visit compared with white individuals. Individuals with higher incomes ≥$50K (OR 0.35, 95%CI, 0.24,0.52) had lower odds of an ED visit compared with lower incomes (<$50K). Conclusion Traditionally underserved communities with HAE have more ED visits and a lower LTP use suggesting differences in healthcare experience. Strategies are needed to reduce health disparities among individuals with HAE.

IntroductionUS patient perspectives on medical care for hereditary angioedema (HAE) are limited for some underrepresented ethnic and racial groups.MethodsA web-based survey of patients with HAE from underrepresented ethnic and/or racial groups was conducted. Eligible participants (excluding patients self-identifying as ‘White only’ ethnicity) had a self-reported diagnosis of HAE Type I/II, were US residents aged ≥18 years able to complete the survey in English or Spanish, and provided informed consent. Descriptive statistics were reported.ResultsThis analysis included 139 eligible patients, all from underrepresented ethnic and racial groups (Table). Mean duration between first attack and HAE diagnosis was 8.4 years, most (82.3% [107/130]) had healthcare experiences before diagnosis that negatively affected their lives. The most common factor leading to diagnosis was referral to an HAE specialist (44.1% [60/136]). Once diagnosed, 90.3% (121/134) reported being satisfied/extremely satisfied with their current care, and 94.8% (128/135) were extremely/moderately aware of available treatment options. Following diagnosis, 91.8% (123/134) reported being involved in decisions concerning their current HAE treatment, and 75.4% (101/134) responded that their background/lifestyle/perspectives (e.g., culture/language/religion) were always/very often considered. The most important sources of information about HAE were healthcare providers 35.1% (47/134) and advocacy groups 35.1%; 15.7% (21/134) obtained information from their own research. Additionally, 36.6% (49/134) reported being involved in patient advocacy groups at least every few months.ConclusionFindings suggest that, following diagnosis, patients with HAE within underrepresented racial and ethnic groups are largely satisfied with their care and are involved in decisions regarding their treatment.

IntroductionHereditary angioedema (HAE) is characterized by unpredictable swelling attacks, that affect patient quality of life (QoL). Real-world data on QoL among patients receiving long-term prophylaxis (LTP) are limited.MethodsData were obtained from a cross-sectional, real-world survey of physicians and patients with HAE in the US. Physician-reported QoL, HAE attack rate, and disease severity were described in patients receiving lanadelumab or other LTP.ResultsThis analysis included 104 patients (38 receiving lanadelumab; 66 other LTP). Mean±SD time on current treatment was 39.0±22.9 months for lanadelumab and 26.1±20.3 months for other LTP. Prior to starting current LTP, a higher proportion of patients who initiated lanadelumab vs other LTP had 'very severe' chronic pain, fatigue, and 'very poor' QoL (Table). Also, a higher proportion of patients receiving lanadelumab had >3 attacks/month at diagnosis (16.7% vs 5.7%). Yet, at time of survey, a greater proportion of patients receiving lanadelumab vs other LTP had no chronic fatigue or pain, excellent QoL (Table), and experienced 0 attacks/month (88.9% vs 77.1%). Prior to starting current LTP, 18.5% of patients receiving lanadelumab and 4.2% receiving other prophylaxis had 'very severe' disease, which decreased to 0% in both groups at the time of the survey. The proportion of patients with 'mild disease' increased from 40.7% prior to starting current LTP to 51.9% after lanadelumab treatment, while those receiving other LTP remained stable at 35.4%.ConclusionFindings indicate numerically greater improvements in QoL and disease severity in patients treated with lanadelumab than other LTP in real-world settings.

Introduction: Hereditary angioedema (HAE) is a potentially fatal disease characterized by unpredictable, recurrent, and often disabling swellings.Donidalorsen is an investigational ligand-conjugated antisense oligonucleotide designed for hepatic uptake and inhibition of prekallikrein production.We report the 2-year analysis of patients with HAE (Type 1 or 2) who received donidalorsen Q8W in a phase 2 open-label extension (OLE) study (NCT04307381).Methods: The OLE study had fixed (Weeks 1-16; donidalorsen 80 mg subcutaneously every 4 weeks [Q4W]) and flexible (Weeks 17 -105; 80 mg Q4W, 80 mg Q8W, or 100 mg Q4W) treatment periods.Patients who were HAE attack-free for ≥12 weeks after entering OLE study could switch to Q8W.Change in quality of life (QoL) was assessed versus the randomized study baseline (ISIS 721744-CS2, NCT04030598).Results: Seventeen patients enrolled in the OLE and 8 received donidalorsen 80 mg Q8W; 5 patients remained attack-free at 2 years, and 3 patients returned to Q4W.No study drug-related treatment-emergent adverse event occurring in >1 patient was reported in this group.There was a mean 83% (95% CI, À113.8 to À52.0; median, 100%) reduction in HAE attack rate across all 8 patients; mean monthly attack rate was 0.29 (range, 0.0-1.7;95% CI, À0.2 to 0.8; median, 0.0).The mean Angioedema-QoL total score improved by 27.4 points from baseline to week 105.Conclusion: Donidalorsen Q8W was well-tolerated and resulted in a mean 83% reduction in HAE attacks; 5/8 patients remained attackfree.These results suggest Q8W dosing is safe and effective, warranting further study.

Introduction Hereditary angioedema (HAE) subtypes I/II are defined by abnormal plasma levels/activity of C1 esterase inhibitor (C1-INH). Diagnosis and management of the ultra-rare subtype with normal C1-INH (NC1-INH) is challenging without routine diagnostic assessments1 and phase 3 trial data. This study describes diagnostic pathways used in real-world lanadelumab-treated patients with NC1-INH-HAE. Methods Four expert HAE clinicians were interviewed about NC1-INH-HAE diagnosis in their clinical practice, 3 then completed a case report form using data from patient records. Patients eligible for retrospective chart review had physician-diagnosed NC1-IHN-HAE, initiated lanadelumab treatment (index date) with prior medical and treatment history available and ≥ 1 follow-up assessment. Results Physician-experts discussed 6 diagnostic criteria, 4 were endorsed by all (Table). Charts from 24 patients (3 US centers) were reviewed: mean (SD) age at index 44.0 (18.0) years, 92% female, median (range) 5.0 (0-40.3) years between symptom onset and NC1-INH-HAE diagnosis. Top 3 previous misdiagnoses: ‘other GI disorder' (71%), ‘irritable bowel syndrome' (67%), and ‘anaphylaxis' (54%). Of 4 criteria endorsed by all experts at interview, 3 were well-represented in patient charts (100-92%), except family history (25%). Expert endorsement was lower for criteria 3 (50%) and 6 (75%) found in 8% and 100% of patient charts, respectively. For patients on prophylaxis prior to index date, monthly attack rates were 5.60 at baseline (n=10) and 2.41 7-12 months after lanadelumab treatment (n=7). Conclusion Real-world NC1-INH-HAE diagnosis aligned with US guidelines on diagnostic criteria, except genetic testing and family history. Broader study of diagnostic guideline implementation in clinical practice is warranted.