Assessment and management of disease burden and quality of life in patients with hereditary angioedema: a consensus report

Allergy, Asthma & Clinical Immunology

Hilary J. Longhurst¹  Teresa Caballero²  Douglas T. Johnston³  John T. Anderson⁴  Konrad Bork⁵  Timothy Craig⁶  Cristine Radojicic⁷  H. Henry Li⁸  Marc A. Riedl⁹ 

[1] Addenbrookes Hospital, Cambridge Universities NHS Foundation Trust, Cambridge and University College Hospital London, CB2 0QQ, Cambridge, UK;Allergy Department, Hospital La Paz Institute for Health Research (IdiPaz), Biomedical Research Network on Rare Diseases (CIBERER, U754), Paseo Castellana 261, 28406, Madrid, Spain;Asthma and Allergy Specialists, 8405 Providence Road, Suite 300, 28277, Charlotte, NC, USA;Clinical Research Center of Alabama, 504 Brookwood Boulevard, Suite 250, 35209, Birmingham, AL, USA;Department of Dermatology, University Medical Center, Johannes Gutenberg University, Langenbeckstraße 1, 55131, Mainz, Germany;Department of Medicine and Pediatrics, Penn State University, 200 Campus Drive, Suite 1300, Entrance 4, Hershey, 17033, University Park, PA, USA;Division of Allergy and Clinical Immunology, Department of Medicine, Duke University, 1821 Hillandale Rd, 27705, Durham, NC, USA;Institute for Asthma and Allergy, 2 Wisconsin Circle, Suite 250, 20815, Chevy Chase, MD, USA;University of California San Diego, 8899 University Center Ln, 92122, San Diego, CA, USA;

关键词: Consensus document;    Disease burden;    Hereditary angioedema;    Management;    Quality of life;   

DOI  :  10.1186/s13223-021-00537-2

来源: Springer

【 摘 要 】

BackgroundHereditary 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.MethodsA 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.ResultsFinal 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 outcomemeasures 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.ConclusionThis consensus report builds on existing guidelines by expanding the assessment of disease burden and QoL measures for patients with HAE.

【 授权许可】

CC BY