ADHERENCE AND PERSISTENCE AMONG HEREDITARY ANGIOEDEMA PATIENTS TREATED WITH BEROTRALSTAT, LANADELUMAB, AND SUBCUTANEOUS PLASMA-DERIVED C1-INHIBITOR

Abstract

Introduction

Berotralstat is a novel, targeted oral long-term prophylactic (LTP) treatment for hereditary angioedema (HAE). Real-world evidence comparing WAO/EAACI recommended first-line HAE LTP therapies is limited. We investigated adherence and persistence following initiation of berotralstat, lanadelumab, and subcutaneous plasma-derived C1-inhibitor (SC-pdC1-INH).

Methods

Veradigm Health Network EHR linked to claims data was utilized to select mutually exclusive cohorts of patients ≥12 years initiating one of the three LTPs between 06/22/2017-09/12/2023. Inclusion criteria also included ≥12 months continuous enrollment before and following date of initiation of LTP. Analysis: demographics, clinical characteristics, LTP adherence and persistence. Adherence was defined as the mean proportion of days covered (PDC). Persistence was defined as having no gap in treatment ≥ 45 days after the index LTP. Sensitivity analyses were conducted requiring at least 2 claims for the index LTP.

Results

Included subjects: 90 berotralstat, 189 lanadelumab, and 78 SC-pdC1-INH. Berotralstat patients were older (mean age 44) than lanadelumab (39) and SC-pdC1-INH (36) cohorts (p-value <0.025). >90% had an HAE diagnosis in the baseline period and 46-51% were LTP treatment experienced. Mean PDC was similar between treatments at 0.73, 0.78, and 0.74 for berotralstat, lanadelumab, and SC-pdC1-INH, respectively (p-value=NS). Proportion of patients persistent on index LTP at 12-months follow-up was also similar across LTPs: 61% for berotralstat compared to 58% for lanadelumab (58%) and 53% for SC-pdC1-INH (p-value=NS).

Conclusions

HAE patient adherence and persistence rates for all three LTP treatments are uniformly high. Berotralstat adherence and persistence were comparable to those observed following lanadelumab or SC-pdC1-INH initiation.