Effectiveness and safety of lanadelumab in ethnic and racial minority subgroups of patients with hereditary angioedema: results from phase 3 studies

Timothy J Craig; Rafael H Zaragoza-Urdaz; H Henry Li; Ming Yu; Hong Ren; Salomé Juethner; John Anderson; HELP and HELP OLE Study Investigators

doi:10.1186/s13223-022-00721-y

Effectiveness and safety of lanadelumab in ethnic and racial minority subgroups of patients with hereditary angioedema: results from phase 3 studies

Allergy Asthma Clin Immunol. 2022 Sep 24;18(1):85. doi: 10.1186/s13223-022-00721-y.

Authors

Timothy J Craig¹, Rafael H Zaragoza-Urdaz², H Henry Li³, Ming Yu⁴, Hong Ren⁵, Salomé Juethner⁴, John Anderson⁶; HELP and HELP OLE Study Investigators

Affiliations

¹ Allergy Asthma and Immunology, Departments of Medicine, Pediatrics, Biomedical Sciences, Penn State University, 500 University Drive, Hershey, PA, 17033, USA. tcraig@pennstatehealth.psu.edu.
² University of Puerto Rico School of Medicine, UPH, San Juan, Puerto Rico, USA.
³ Institute for Asthma and Allergy, Chevy Chase, MD, USA.
⁴ Takeda Pharmaceuticals, Inc., Lexington, MA, USA.
⁵ Cytel Inc., Cambridge, MA, USA.
⁶ Alabama Allergy & Asthma Center, an affiliate of AllerVie Health, Birmingham, AL, USA.

Abstract

Background: The COVID-19 pandemic has highlighted disparities in healthcare, particularly in the United States, even though disparities have existed since the organization of the modern healthcare system. Recruitment of patients from racial and ethnic minority groups is often minimal in phase 3 clinical trials, and is further exacerbated in the case of trials for rare diseases such as hereditary angioedema (HAE). This can lead to a gap in the understanding of minority patients' experiences with these diseases and their response to potential treatment options.

Methods: We reviewed data from phase 3 double-blind (HELP) and open-label extension (HELP OLE) trials of lanadelumab, a monoclonal antibody developed for long-term prophylaxis against attacks of HAE. Efficacy (attack rate reduction) and safety (adverse events) results from White patients were compared descriptively to those from Hispanic/Latino patients, Black/African Americans, and other minority Americans.

Results: Not surprisingly, few minorities were recruited across both studies: 9.5% Black, 2.4% Asian, and 7.1% Hispanic/Latino versus 88.1% White and 91.7% non-Hispanic/non-Latino received lanadelumab in HELP, and 4.7% Black, 0.9% Asian, 0.9% other, and 6.1% Hispanic/Latino versus 93.4% White and 93.4% non-Hispanic/non-Latino were enrolled in HELP OLE. Although these studies were conducted in the United States, Canada, Europe, and Jordan, all minorities were from the United States. Despite the number of minority patients being far less than expected for the population, there was no evidence that either efficacy or adverse event profiles differed between ethnic or racial groups.

Conclusions: The HELP and HELP OLE studies described herein recruited far fewer minorities than would be ideal to represent these populations. However, evidence suggests that the effectiveness and tolerance of lanadelumab are similar between the groups. Nonetheless, the disparity in recruitment into research for minorities has significant room for improvement. Trial registration NCT02586805, registered 26 October 2015, https://clinicaltrials.gov/ct2/show/record/NCT02586805 . NCT02741596, registered 18 April 2016, https://clinicaltrials.gov/ct2/show/NCT02741596 .

Keywords: Disparity; Ethnicity; Hereditary angioedema; Lanadelumab; Long-term prophylaxis; Race.

Associated data

ClinicalTrials.gov/NCT02586805
ClinicalTrials.gov/NCT02741596