CONFERENCE UPDATE: AAAAI 2024

Interim analysis of the real-world CHOPIN study shows reduction in HAE attacks and improvement in PROs with lanadelumab

08 May 2024

STUDY DESIGN

Hereditary angioedema (HAE) is a rare genetic disease caused by a deficiency of the C1-inhibitor protein which causes uncontrolled plasma kallikrein activity.1 HAE is characterized by recurrent swelling episodes that affect the abdomen, larynx or extremities.1 It is also associated with laryngeal edema attacks which constrict the respiratory tract and may become a life-threatening emergency.1 Based on global guidelines for HAE management, lanadelumab is recommended as first-line long-term prophylactic HAE management.1 It is a fully monoclonal antibody that inhibits active plasma kallikrein, and has been shown to reduce the frequency of HAE attacks and improve patient-reported health-related quality of life (QoL) in the phase 3 HELP trial.1 However, data on the efficacy of lanadelumab in HAE patients in the real world remain limited.1

CHOPIN is a noninterventional, prospective, multicenter real-world study conducted to assess the effectiveness of lanadelumab in HAE patients naïve to prophylaxis.1 The study included patients with HAE aged ≥12 years who qualified for treatment in the Polish National Drug Program (NDP) and consented to participate in the study.1 Data from the 6-month interim analysis of 16 patients were represented.1

The primary endpoints were the change in the total number of HAE attacks from baseline to 3 months after lanadelumab initiation, the proportion of HAE attacks requiring rescue treatment and the characteristics of HAE attacks.1 Secondary endpoints included the change in the Work Productivity and Activity Impairment Questionnaire: General Health (WPAI:GH) and Angioedema Quality of Life (AE-QoL) scores from baseline to 6 months after lanadelumab initiation.1 Other exploratory endpoints were the change in the Angioedema Control Test (AECT) score from baseline to 6 months and the incidence, type, severity, seriousness, and treatment-relatedness of adverse events (AEs).1

FINDINGS

Primary endpoints: 
  • The primary endpoints were the change in the total number of HAE attacks, the proportion of attacks requiring rescue treatment and the characteristics of HAE attacks1
  • The number of HAE attacks decreased from 172 at baseline (mild: 9; moderate: 37; severe: 126) to 5 (mild: 2; moderate: 1; severe: 2)1 at 6 months
  • The number of HAE attacks requiring rescue drug decreased from 145 at baseline to 2 at six months after lanadelumab initiation1
  • Similarly, attacks requiring a medical visit or an emergency department visit decreased from 12 and 27 times respectively at baseline to 0 times at 6 months1
Secondary endpoints:
  • Other endpoints were PROs which included the change in the WPAI:GH, AE-QoL and AECT scores from baseline at 6 months after lanadelumab initiation, and a description of the AEs1
  • Improvement was observed in the WPAI:GH scores across all domains of absenteeism, presenteeism, activity impairment, and work productivity loss, which was reduced from 53.3% to <1% within the first month of lanadelumab treatment1
  • Similarly, lanadelumab was associated with a significant improvement in AE-QoL, with a baseline score of 59.9 denoting significant impairment to 16.4 after 1 month of treatment and was sustained throughout the 6 months of treatment1
  • Mean AECT scores were also improved from 4.4 (poorly-controlled HAE) at baseline to 15.3 (well-controlled HAE) at 6 months with lanadelumab administration1
Safety:
  • 4 lanadelumab-related AEs were reported including administration site conditions and a respiratory tract infection, all of which were non-serious1

 

“These findings are consistent with those from pivotal and real-world studies supporting the use of lanadelumab as first-line prophylactic treatment in patients with HAE"

Dr. Aleksandra Kucharczyk
Department of Internal Diseases,
Pneumonology, Allergology, Clinical Immunology,
Military Institute of Medicine, Warsaw, Poland

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