Racial and Ethnic Disparities in the Research and Care of Hereditary Angioedema Patients in the United States

Sylvestre, Sebastian; Craig, Timothy J.; Ajewole, Oyindamola; Craig, Sansanee; Kaur, Sukhdeep; Al‐Shaikhly, Taha

doi:10.1016/j.jaip.2021.08.019

Cited by 11 publications

(7 citation statements)

References 52 publications

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“…Limitations of this study include the fact that the psychometric analysis was retrospective and the sample size was small; the small size is a common disadvantage of studies in rare diseases. The patient population was predominantly female; while this is consistent with numerous clinical trials [ 32 – 35 , 46 ] and other studies [ 3 , 47 , 48 ] in which a higher number of female patients with HAE was enrolled or reported, the disease experience in females may differ from that in male patients [ 49 ]. Although sensitivity to change and meaningful change for the AE-QoL were established previously [ 12 ], anchor-based analyses of meaningful change thresholds were limited in this study, as there were no global assessments of symptom severity or change to use as anchors.…”

Section: Discussionsupporting

confidence: 83%

Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema

Vanya

Watt

Shahraz

et al. 2023

J Patient Rep Outcomes

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Background There is considerable burden of illness in hereditary angioedema (HAE). However, instruments to assess health-related quality of life (HRQoL) in HAE are limited. The Angioedema Quality of Life Questionnaire (AE-QoL) was developed to measure HRQoL in patients with recurrent angioedema; the validity of the AE-QoL in patients with HAE is described. Methods To identify disease-related experiences with a focus on the impact of HAE on HRQoL, interviews were conducted with a group of clinician experts and patients with HAE from Canada, France, Germany, Spain, the United Kingdom, and the United States, along with a targeted literature review. Concepts were mapped to the AE-QoL to assess item relevance, interpretation, and conceptual coverage. Cognitive interviews assessed item clarity and relevance. A psychometric validation was performed using data from a phase 3 trial. Results Interviews were conducted with seven clinicians and 40 adult patients. Patients reported 35 unique impacts of HAE on their lives, the most frequent being on work/school, social relationships, physical activities, and emotions, particularly fear/worrying and anxiety. Saturation for these impacts was reached, and all concepts covered in the AE-QoL were reported during the interviews. Patients agreed that the questionnaire items and response options were clear and relevant, and the 4-week recall period was appropriate. The psychometric validation included data from 64 patients. For AE-QoL total scores, excellent internal consistency (Cronbach’s alpha > 0.90), test-retest reliability (intraclass coefficient > 0.80), convergent validity with the Sheehan Disability Scale (r = 0.663), divergent validity with the EQ-5D-5L index (r = 0.292) and EQ-VAS (r = 0.337), and known-groups validity (p < 0.0001; ɳ2 = 0.56) were demonstrated. Conclusions Qualitative and psychometric analyses showed that the AE-QoL is a reliable and valid instrument for measuring HRQoL in adult patients with HAE from six countries.

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Section: Discussionsupporting

confidence: 83%

Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema

Vanya

Watt

Shahraz

et al. 2023

J Patient Rep Outcomes

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“…Even though "genetic HAE" is equal in sexes, symptomatic disease is greater in females, and this may account for the asymmetry between the number of men and women receiving care. Our prior data suggest care for African American patients is not disparate, however, secondary to the demographics of our area care for African Americans and other races is underrepresented [17] Our analysis suggests that several aspects of HAE care were consistently addressed in patient visits. Note elements such as recording the number of attacks and the use of rescue medications, as well as inquiring into the patient's quality of life, are basic components of any complete history that can be analogized to the treatment of other chronic diseases.…”

Section: Discussionmentioning

confidence: 76%

Evaluating Quality of Care for Hereditary Angioedema in a Health Care System

Craig

2023

JOJPH

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Background: Hereditary angioedema (HAE) is an orphan disease that inflicts significant morbidity, post-traumatic stress, anxiety, productivity loss, and potential mortality on its affected patients. For this reason, treatment must be uniquely designed to ensure comprehensive and holistic quality of care.Objective: To evaluate HAE care in a health care system (HCS) for adequacy and disparities. Methods:We reviewed the HAE Guidelines and literature and from this developed a survey to assess quality of care for HAE in a HCS. This study met Institutional Review Board exception since it was for quality improvement. RedCap (Vanderbilt, Nashville, TN, USA) was used for collection and evaluation of the data. Chi-square testing was used to compare demographic groups. Results:The entire population had high rates of their quality of life, attack frequency, rescue medication use, and short-and long-term prophylaxis being recorded. Surgical history, dental hygiene, and avoidance of triggering medications were infrequently addressed. Discrepancies across age and sex categories exist for medication choices, decisions to leave attacks untreated, and patient education. The number of African American patients with HAE in the HCS were too few to determine if disparity of care exists. Conclusion:Assessment of the care provided suggests that the use of a standardized instrument to gauge HAE control and educate patients during follow-up should be encouraged.

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“…Of note, the majority of confirmed HAE patients in the database were managed by a primary care physician; thus patients who were managed by an allergist may have been underrepresented. A recent retrospective study of data from the TriNetX Diamond Network covering more than 2000 patients with HAE from 2014 to 2021 showed an equal prevalence of HAE in White and Black populations (1.64 and 1.47 patients, respectively, per 100,000) and a lower prevalence in Hispanic patients (0.80 per 100,000) [15]. Underdiagnosis of rare genetic disorders in racial and ethnic minority groups is not uncommon [16][17][18] and may contribute to the apparent lower prevalence of HAE in these populations.…”

Section: Discussionmentioning

confidence: 99%

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

Craig

Zaragoza-Urdaz²,

Li³

et al. 2022

Allergy Asthma Clin Immunol

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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.

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Racial and Ethnic Disparities in the Research and Care of Hereditary Angioedema Patients in the United States

Cited by 11 publications

References 52 publications

Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema

Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema

Evaluating Quality of Care for Hereditary Angioedema in a Health Care System

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

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