Supportive data on the utilization of the Patient Global Impression of Change (PGI-C) scale as a clinical endpoint in patients with hereditary angioedema (HAE) were presented at the 2022 HAE International (HAEi) Global Leadership Workshop held in Frankfurt, Germany. The PGI-C is recommended as a means of assessing the efficacy of on-demand treatments for HAE attacks.
Qualitative interviews with individuals with HAE conducted by the investigators demonstrated a clear preference for the PGI-C (ie, >70%), compared to other scales, as a measurement tool for the evaluation of patient outcomes.
KalVista Pharmaceuticals which presented the data, has been focused on the discovery, development, and commercialization of oral small molecule protease inhibitors for patients with HAE.
The key impact of HAE attacks on the mental health, daily activities, and quality of life of individuals living with the rare disorder was a major focus of the meeting. According to KalVista chief executive officer Andrew Crockett, “the goal of this and other research we conduct is to better understand the consequences of HAE and to learn how [to] improve the treatment experience for these patients with our pipeline of oral therapies.”
Learn more about experimental therapies for HAE
KalVista presented 2 posters at the workshop: (1) Patient Perspectives on an Optimal Measure to Assess Efficacy in the Acute Treatment of Hereditary Angioedema Attacks, presented by Marc A. Riedl, MD, University of California San Diego, La Jolla, California, and (2) The Global and Regional Impact of Hereditary Angioedema (HAE) Attacks on Mental Health, Activities of Daily Living, and Quality of Life, presented by Paula J. Busse, MD, Mount Sinai Hospital, New York, New York.
KalVista chose PGI-C as the primary outcome measure for the ongoing phase 3 KONFIDENT trial because of its clinical meaningfulness and patient preference, the company said in a press release. The trial is investigating the use of sebetralstat as an orally administered on-demand treatment for HAE attacks.
KalVista Pharmaceuticals presents new patient-centric data at 2022 HAEi Global Leadership Workshop. News release. KalVista Pharmaceuticals, Inc; October 7, 2022.