HR-QoL prespecified exploratory endpoint

The burden of HAE goes beyond the attack

The unpredictable nature of HAE can affect various facets of a patient’s life.¹¹

In the HELP study, quality of life (QoL) measures were evaluated using the AE-QoL and EQ-5D-5L questionnaires¹²

Limitations: These results should be interpreted with caution as they are based on patient recall and are observational/descriptive in nature. These data were also from an exploratory objective and had less evidentiary value than the primary and secondary objectives. The AE-QoL was administered to 10 adolescent patients in the study, an age group for which the instrument was not validated.¹²

For the EQ-5D-5L questionnaire, an instrument used to measure health status on a given day, no differences were observed. The nondisease-specific EQ-5D-5L questionnaire was administered on days 0, 98, and 182.¹²

Significantly more patients taking TAKHZYRO vs placebo experienced improvements in AE-QoL in the 6.5-month HELP study¹²:

• 81% of patients receiving TAKHZYRO 300 mg Q2W (95% CI, 61-93; n=26) experienced improvement in AE-QoL total score vs 37% of patients taking placebo (P<0.05; 95% CI, 22-54; n=38)¹²
• Patients receiving TAKHZYRO 300 mg Q2W were 7.2 times more likely to achieve improvement in AE-QoL total score vs patients taking placebo (P<0.01)¹²

Definitions: The AE-QoL is a validated, angioedema-specific questionnaire in adults that was administered monthly, consisting of 4 domains (functioning, fatigue/mood, fears/shame, nutrition) and total scores. The minimal clinically important difference (MCID) is the minimum change in score that is meaningful to patients. For the AE-QoL total score, the predefined MCID is a reduction of 6 points.^12-14

AE-QoL=Angioedema Quality of Life Questionnaire; EQ-5D-5L=5-level EuroQol 5-dimensional; HR-QoL=health-related quality of life; Q2W=every 2 weeks.