TY - JOUR
T1 - Letting the patients speak
T2 - an in-depth, qualitative research-based investigation of factors relevant to health-related quality of life in real-world patients with hereditary angioedema using subcutaneous C1 inhibitor replacement therapy
AU - Anderson, John
AU - Levy, Donald S.
AU - Lumry, William
AU - Koochaki, Patricia
AU - Lanar, Sally
AU - Henry Li, H.
N1 - Funding Information:
This study was funded by CSL Behring.
Funding Information:
J Anderson is a PI, consultant, and speaker for CSL Behring, Shire/Takeda, and Pharming and a PI for BioCryst. W Lumry is a consultant for Adverum, Attune, BioCryst, CSL Behring, Kalvista, Pharming, and Takeda; a speaker for CSL Behring, Pharming, and Takeda; and has received research grants from BioCryst, CSL Behring, Pharming, and Takeda. DS Levy has served as a consultant, speaker, and has received research grants from CSL Behring; he has served as a consultant for BioCryst; he has served as a speaker for Takeda. P Koochaki and S Lanar are employees of ICON plc, which received funding through Churchill Communications to conduct this study on behalf of the funding sponsor, CSL Behring. HH Li has conducted research with and has served a consultant/received speakers’ fees from Shire/Takeda, BioCryst, and CSL Behring; he has served as a consultant/received speakers’ fees from Pharming.
Funding Information:
The authors acknowledge the writing assistance of Sandra Westra, PharmD of Churchill Communications (Maplewood, NJ), funded by CSL Behring. The authors acknowledge the assistance of Selam Shah, of ICON plc (Boston, MA) and of Ashlee Watts, formerly of ICON plc, (Gaithersburg, MD) for their help in performing some of the patient interviews.
Publisher Copyright:
© 2021, The Author(s).
PY - 2021/12
Y1 - 2021/12
N2 - Background: While many studies of effective hereditary angioedema (HAE) therapy have demonstrated improved health-related quality of life (HRQoL) using validated instruments, specific reasons behind the improved scores have never been investigated using qualitative methods. A non-interventional, qualitative research study was designed to investigate the reasons for improvements in HRQoL while using effective prophylaxis, in this case subcutaneous C1INH (C1INH[SC]) replacement therapy. Methods: Adult patients with HAE-C1INH type 1 or 2 who had been using C1INH(SC) for ≥ 3 consecutive months were recruited through four HAE specialty practices in the US to participate in a 60-min phone interview performed by a trained qualitative research specialist (ICON plc) using a semi-structured interview guide with open-ended questions developed with the Angioedema Quality of Life (AE-QoL) items in mind. Interview transcripts were analyzed using thematic analysis methods to identify concepts (specific symptoms/impacts) and themes (higher-level categories grouping related concepts). A cross-mapping exercise was performed between interview-identified concepts and items included in the AE-QoL. Results: Fourteen patients were interviewed and included in the analysis (age range, 28–82 years [mean 47.5 years]; 64% female; 93% white). In 10 interviews, patients mentioned having no or nearly no HAE attacks, no longer feeling limited by HAE, less HAE-related anxiety/worry and depression, an improved ability to travel, fewer emergency room/hospital visits, and ease of administration of C1INH(SC), including not requiring assistance from others. Other commonly expressed concepts included: increased feelings of confidence, independence, optimism, and normalcy; less absence from work/school; better productivity; improved sleep and energy; healthier family relationships; and improved cognition. While all AE-QoL items emerged from patient interviews, a number of identified concepts were not addressed by the AE-QoL, including sensitivity to various potential attack-triggers (e.g., stress/anxiety, sports), attack frequency, not having to cancel social plans, improvements in ability to perform day-to-day tasks, and a lower burden from medical visits. Conclusions: From these interviews, a large number of common themes and concepts emerged: a greater sense of freedom and normalcy, increased productivity, and improved interpersonal relationships while using convenient and effective prophylaxis. These findings provide insights into real-world experiences and the many facets of HRQoL that are important to patients with HAE-C1INH.
AB - Background: While many studies of effective hereditary angioedema (HAE) therapy have demonstrated improved health-related quality of life (HRQoL) using validated instruments, specific reasons behind the improved scores have never been investigated using qualitative methods. A non-interventional, qualitative research study was designed to investigate the reasons for improvements in HRQoL while using effective prophylaxis, in this case subcutaneous C1INH (C1INH[SC]) replacement therapy. Methods: Adult patients with HAE-C1INH type 1 or 2 who had been using C1INH(SC) for ≥ 3 consecutive months were recruited through four HAE specialty practices in the US to participate in a 60-min phone interview performed by a trained qualitative research specialist (ICON plc) using a semi-structured interview guide with open-ended questions developed with the Angioedema Quality of Life (AE-QoL) items in mind. Interview transcripts were analyzed using thematic analysis methods to identify concepts (specific symptoms/impacts) and themes (higher-level categories grouping related concepts). A cross-mapping exercise was performed between interview-identified concepts and items included in the AE-QoL. Results: Fourteen patients were interviewed and included in the analysis (age range, 28–82 years [mean 47.5 years]; 64% female; 93% white). In 10 interviews, patients mentioned having no or nearly no HAE attacks, no longer feeling limited by HAE, less HAE-related anxiety/worry and depression, an improved ability to travel, fewer emergency room/hospital visits, and ease of administration of C1INH(SC), including not requiring assistance from others. Other commonly expressed concepts included: increased feelings of confidence, independence, optimism, and normalcy; less absence from work/school; better productivity; improved sleep and energy; healthier family relationships; and improved cognition. While all AE-QoL items emerged from patient interviews, a number of identified concepts were not addressed by the AE-QoL, including sensitivity to various potential attack-triggers (e.g., stress/anxiety, sports), attack frequency, not having to cancel social plans, improvements in ability to perform day-to-day tasks, and a lower burden from medical visits. Conclusions: From these interviews, a large number of common themes and concepts emerged: a greater sense of freedom and normalcy, increased productivity, and improved interpersonal relationships while using convenient and effective prophylaxis. These findings provide insights into real-world experiences and the many facets of HRQoL that are important to patients with HAE-C1INH.
KW - HAEGARDA
KW - Health-related quality of life
KW - Hereditary angioedema
KW - Productivity
KW - Qualitative research
KW - Subcutaneous C1-inhibitor
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UR - http://www.scopus.com/inward/citedby.url?scp=85121283772&partnerID=8YFLogxK
U2 - 10.1186/s13223-021-00550-5
DO - 10.1186/s13223-021-00550-5
M3 - Article
C2 - 34176500
AN - SCOPUS:85121283772
SN - 1710-1484
VL - 17
JO - Allergy, Asthma and Clinical Immunology
JF - Allergy, Asthma and Clinical Immunology
IS - 1
M1 - 60
ER -