Long-term Impact of Lanadelumab on Patients with Hereditary Angioedema (HAE) Type 1/2: Patient Reported Outcome (PRO) Findings from the HELP Open-label Extension Study (OLE)

Abstract

Improving HAE patients’ health-related quality of life (QoL) is a continuing goal. The objective was to investigate the long-term benefit of lanadelumab from the patient perspective. Patients ≥12 years old with HAE type 1/2 who continued from HELP (rollovers) received 300mg lanadelumab on day 0, then 300mg every 2 weeks (q2wks) after their first attack (regular dosing stage). Newly enrolled patients (nonrollovers) received 300mg q2wks from day 0. The planned treatment period was 132 weeks. QoL was assessed with the Angioedema QoL Questionnaire (AE-QoL [higher scores=poorer outcome]) at baseline and every 4-8 weeks, and the Treatment Satisfaction Questionnaire for Medication (TSQM-9; treatment effectiveness, convenience, and global-satisfaction domains [higher scores=greater satisfaction]) at days 364, 574, and end of study (EOS). All endpoints were summarized using descriptive statistics. 212 patients received treatment (n=109 rollovers; n=103 nonrollovers). Rollovers experienced improvement in AE-QoL total and domain scores from day 0 to EOS (respective mean change [SD]: in total score -10.2 [17.9], functioning -11.1 [24.3], fatigue/mood -7.4 [23.8], fears/shame -12.9 [19.2], nutrition -7.2 [26.1]), with highest improvement observed in the fears/shame domain. Nonrollovers experienced numerically greater improvement in all AE-QoL scores (respective mean change [SD]: -19.5 [21.3], -26.2 [27.7], -11.6 [25.8], -22.2 [24.3], -18.3 [24.4]). Mean TSQM-9 scores showed rollovers and nonrollovers were very satisfied with treatment effectiveness (>90), perceived treatment was convenient (>81), and were very satisfied with lanadelumab (>87). The results confirm the sustained long-term improvements in QoL and positive impact of lanadelumab on patients’ experience as observed in the HELP trial.