TY - JOUR
T1 - Assessment and management of disease burden and quality of life in patients with hereditary angioedema
T2 - a consensus report
AU - Bork, Konrad
AU - Anderson, John T.
AU - Caballero, Teresa
AU - Craig, Timothy
AU - Johnston, Douglas T.
AU - Li, H. Henry
AU - Longhurst, Hilary J.
AU - Radojicic, Cristine
AU - Riedl, Marc A.
N1 - Funding Information:
KB has received grant research support and/or speaker fees from CSL Behring and Shire (a Takeda company). JA is on the speakers’ bureau for Pharming, Shire (a Takeda company), and CSL Behring. He has served as a consultant and on advisory boards and conducted clinical research for Pharming, Shire (a Takeda company), BioCryst, and CSL Behring. TC has received grant research support and/or speaker/consultancy fees from BioCryst, CSL Behring, Merck, Novartis, Octapharma, Pharming, and Shire (a Takeda company); has received funding to attend conferences/educational events from CSL Behring, Novartis, and Shire; is/has been a clinical trial/registry investigator for BioCryst, CSL Behring, Novartis, Pharming, and Shire; and is a researcher from the IdiPAZ program for promoting research activities. TC reports past research, consultancy, and speaker fees from CSL Behring and Takeda; speaker and consultancy fees from Pharming; research and consultancy fees from BioCryst Pharmaceuticals; and is a member of the US HAEA Medical Advisory Board. DJ reports personal fees from BioCryst Pharmaceuticals, CSL Behring, Pharming, Regenxbio, and Takeda outside the submitted work. HHL reports grants from BioCryst Pharmaceuticals during the conduct of the study and grants and personal fees from CSL Behring, Pharming, and Shire/Takeda outside the submitted work. HL reports grants and personal fees from BioCryst, CSL Behring, and Pharming; personal fees from Octapharma, Pfizer, and Pharvaris; and personal fees and nonfinancial support from Takeda/Shire outside the submitted work. CR has served on scientific advisory boards for CSL Behring and BioCryst. MR is a research investigator for BioCryst, CSL Behring, Ionis, Pharming, Takeda; consultant for Attune, BioCryst, CSL Behring, Kalvista, Pfizer, Pharming, Pharvaris, Takeda; and speaker for CSL Behring, Pharming, and Takeda.
Publisher Copyright:
© 2021, The Author(s).
PY - 2021/12
Y1 - 2021/12
N2 - Background: Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, potentially life-threatening attacks, resulting in significant physical and emotional burdens for patients and families. To optimize care for patients with HAE, an individualized management plan should be considered in partnership with the physician, requiring comprehensive assessment of the patient’s frequency and severity of attacks, disease burden, and therapeutic control. Although several guidelines and consensus papers have been published concerning the diagnosis and treatment of HAE, there has been limited specific clinical guidance on the assessment of disease burden and quality of life (QoL) in this patient population. Practical guidance is critical in supporting effective long-term clinical management of HAE and improving patient outcomes. The objective of this review is to provide evidence-based guidelines for an individualized assessment of disease burden and QoL in patients with HAE. Methods: A consensus meeting was held on February 29, 2020, consisting of 9 HAE experts from the United States and Europe with extensive clinical experience in the treatment of HAE. Consensus statements were developed based on a preliminary literature review and discussions from the consensus meeting. Results: Final statements reflect the consensus of the expert panel and include the assessment of attack severity, evaluation of disease burden, and long-term clinical management of HAE caused by C1-esterase inhibitor deficiency. Patient-reported outcome measures for assessing HAE attack severity and frequency are available and valuable tools; however, attack frequency and severity are insufficient markers of disease severity unless they are evaluated in the broader context of the effect on an individual patient’s QoL. QoL assessments should be individualized for each patient and minimally, they should address the interference of HAE with work, school, social, family, and physical activity, along with access to and burden of HAE treatment. Advances in HAE therapies offer the opportunity for comprehensive, individualized treatment plans, allowing patients to achieve minimal attack burden with reduced disease and treatment burden. Conclusion: This consensus report builds on existing guidelines by expanding the assessment of disease burden and QoL measures for patients with HAE.
AB - Background: Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, potentially life-threatening attacks, resulting in significant physical and emotional burdens for patients and families. To optimize care for patients with HAE, an individualized management plan should be considered in partnership with the physician, requiring comprehensive assessment of the patient’s frequency and severity of attacks, disease burden, and therapeutic control. Although several guidelines and consensus papers have been published concerning the diagnosis and treatment of HAE, there has been limited specific clinical guidance on the assessment of disease burden and quality of life (QoL) in this patient population. Practical guidance is critical in supporting effective long-term clinical management of HAE and improving patient outcomes. The objective of this review is to provide evidence-based guidelines for an individualized assessment of disease burden and QoL in patients with HAE. Methods: A consensus meeting was held on February 29, 2020, consisting of 9 HAE experts from the United States and Europe with extensive clinical experience in the treatment of HAE. Consensus statements were developed based on a preliminary literature review and discussions from the consensus meeting. Results: Final statements reflect the consensus of the expert panel and include the assessment of attack severity, evaluation of disease burden, and long-term clinical management of HAE caused by C1-esterase inhibitor deficiency. Patient-reported outcome measures for assessing HAE attack severity and frequency are available and valuable tools; however, attack frequency and severity are insufficient markers of disease severity unless they are evaluated in the broader context of the effect on an individual patient’s QoL. QoL assessments should be individualized for each patient and minimally, they should address the interference of HAE with work, school, social, family, and physical activity, along with access to and burden of HAE treatment. Advances in HAE therapies offer the opportunity for comprehensive, individualized treatment plans, allowing patients to achieve minimal attack burden with reduced disease and treatment burden. Conclusion: This consensus report builds on existing guidelines by expanding the assessment of disease burden and QoL measures for patients with HAE.
KW - Consensus document
KW - Disease burden
KW - Hereditary angioedema
KW - Management
KW - Quality of life
UR - http://www.scopus.com/inward/record.url?scp=85104556620&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85104556620&partnerID=8YFLogxK
U2 - 10.1186/s13223-021-00537-2
DO - 10.1186/s13223-021-00537-2
M3 - Review article
C2 - 33875020
AN - SCOPUS:85104556620
VL - 17
JO - Allergy, Asthma and Clinical Immunology
JF - Allergy, Asthma and Clinical Immunology
SN - 1710-1484
IS - 1
M1 - 40
ER -