New Instrument for the Evaluation of Prodromes and Attacks of Hereditary Angioedema (HAE-EPA).


Journal

Clinical reviews in allergy & immunology
ISSN: 1559-0267
Titre abrégé: Clin Rev Allergy Immunol
Pays: United States
ID NLM: 9504368

Informations de publication

Date de publication:
Aug 2021
Historique:
accepted: 13 01 2021
pubmed: 5 2 2021
medline: 15 12 2021
entrez: 4 2 2021
Statut: ppublish

Résumé

A disease-specific, patient-reported outcome instrument suitable for evaluation of prodromes and attacks of hereditary angioedema (HAE) is a clinical unmet need. We constructed such instrument and examined its validity, acceptability, and discriminative ability. Sixty-six patients participated in a survey addressing their demographics, social, and medical status. Discriminant content validity involved: (1) construct definition by in-depth cognitive debriefing interviews, (2) item selection identifying relevant categories, and (3) judgment of the format whereby questionnaires were tested on experienced patients and its content/reliability was validated. Prodromes and attacks affecting certain body systems (domains) were organized in "clusters". Internal consistency, content, and convergent validities were analyzed. Analyses of variance and regression models were used to evaluate the discriminative ability of the instrument to differentiate between attacks and prodromes. The study demonstrates very high internal consistency (Cronbach's α: attacks 0.88-0.98, prodromes 0.78-0.98). Analysis of variance confirmed significant differences between all dimensions and in pre-defined clusters (F (4, 61) = 45.74, p < 0.001, Eta

Identifiants

pubmed: 33538950
doi: 10.1007/s12016-021-08843-8
pii: 10.1007/s12016-021-08843-8
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

29-39

Subventions

Organisme : Tel Aviv University
ID : Stanley Styer foundation

Informations de copyright

© 2021. The Author(s), under exclusive licence to Springer Science+Business Media, LLC part of Springer Nature.

Références

Busse PJ, Christiansen SC (2020) Hereditary angioedema. N Engl J Med 382:1136–1148. https://doi.org/10.1056/NEJMra1808012
doi: 10.1056/NEJMra1808012 pubmed: 32187470
Reshef A, Kidon M, Leibovich I (2016) The story of angioedema: from Quincke to bradykinin. Clin Rev Allergy Immunol 51(2):121–139. https://doi.org/10.1007/s12016-016-8553-8
doi: 10.1007/s12016-016-8553-8 pubmed: 27287037
Zuraw BL, Christiansen SC (2016) HAE: pathophysiology and underlying mechanisms. Clin Rev Allergy Immunol 51(2):216–229. https://doi.org/10.1007/s12016-016-8561-8
doi: 10.1007/s12016-016-8561-8 pubmed: 27459852
Kaplan AP, Joseph K (2017) Pathogenesis of hereditary angioedema: the role of the bradykinin-forming cascade. Immunol Allergy Clin N Am 37(3):513–525. https://doi.org/10.1016/j.iac.2017.04.001
doi: 10.1016/j.iac.2017.04.001
Lumry WR, Castaldo AJ, Vernon MK et al (2010) The humanistic burden of hereditary angioedema: impact on health-related quality of life, productivity, and depression. Allergy Ast Proc 31(5):407–414
doi: 10.2500/aap.2010.31.3394
Wilson D, Bork K, Shea EP et al (2010) Economic costs associated with acute attacks and long-term management of hereditary angioedema. Ann Allergy, Ast and Immunol 104(4):314–320. https://doi.org/10.1016/j.anai.2010.01.024
doi: 10.1016/j.anai.2010.01.024
Lumry WR (2018) Hereditary angioedema: the economics of treatment of an orphan disease. Front Med 5:22. https://doi.org/10.3389/fmed.2018.00022
doi: 10.3389/fmed.2018.00022
Kemp JG, Craig TJ (2009) Variability of prodromal signs and symptoms associated with hereditary angioedema attacks: a literature review. Allergy Ast Proc 30:493–499
doi: 10.2500/aap.2009.30.3278
Prematta MJ, Kemp JG, Gibbs JG, Craig TJ (2009) Frequency, timing, and type of prodromal symptoms associated with hereditary angioedema attacks. Allergy Ast Proc 30:506–511
doi: 10.2500/aap.2009.30.3279
Prematta MJ, Bewtra AK, Levy RJ et al (2012) Per-attack reporting of prodromal symptoms concurrent with C1-inhibitor treatment of hereditary angioedema attacks. Adv Ther 29(10):913–922. https://doi.org/10.1007/s12325-012-0053-5
doi: 10.1007/s12325-012-0053-5 pubmed: 23054691
Reshef A, Prematta M, Craig TJ (2013) Signs and symptoms preceding acute attacks of hereditary angioedema: results of three recent surveys. Allergy Ast Proc 34(3):261–266
doi: 10.2500/aap.2013.34.3663
Leibovich-Nassi I, Golander H, Somech R, Har-Even D, Reshef A (2019) Are HAE patients able to distinguish prodromes from attacks, and are they correlated? Allergy, Ast Clin Immunol 15(Suppl 4):P10
Santana MJ, Feeny D (2014) Framework to assess the effects of using patient-reported outcome measures in chronic care management. Qual Life Res 23:1505–1513
doi: 10.1007/s11136-013-0596-1
Nelson EC, Eftimovska E, Lind C et al (2015) Patient reported outcome measures in practice. BMJ 350:g7818. https://doi.org/10.1136/bmj.g7818
doi: 10.1136/bmj.g7818 pubmed: 25670183
Kelman L (2004) The premonitory symptoms (prodrome): a tertiary care study of 893 migraineurs. Headache 44(9):865–872
doi: 10.1111/j.1526-4610.2004.04168.x
Schoonman GG, Evers DJ, Terwindt GM, van Dijk JG, Ferrari MD (2006) The prevalence of premonitory symptoms in migraine: a questionnaire study in 461 patients. Cephalalgia 26:1209–1213
doi: 10.1111/j.1468-2982.2006.01195.x
Houtveen JH, Sorbi MJ (2013) Prodromal functioning of migraine patients relative to their interictal state—an ecological momentary assessment study. PLoS ONE 8(8):e72827. https://doi.org/10.1371/journal.pone.0072827
doi: 10.1371/journal.pone.0072827 pubmed: 23977358 pmcid: 3745475
Lidar M, Yaqubov M, Zaks N et al (2006) The prodrome: a prominent yet overlooked pre-attack manifestation of familial Mediterranean fever. J Rheumatol 33(6):1089–1092
pubmed: 16755655
Babaoglu H, Varan O, Kucuk H et al (2019) On demand use of anakinra for attacks of familial Mediterranean fever (FMF). Clin Rheumatol 38(2):577–581
doi: 10.1007/s10067-018-4230-z
Vernon MK, Rentz AM, Wyrwich KW, White MV, Grienenberger A (2009) Psychometric validation of two patient-reported outcome measures to assess symptom severity and changes in symptoms in hereditary angioedema. Qual Life Res 18:929–939. https://doi.org/10.1007/s11136-009-9509-8
doi: 10.1007/s11136-009-9509-8 pubmed: 19597964
McMillan CV, Speight J, Relan A et al (2012) Content validity of visual analog scales to assess symptom severity of acute angioedema attacks in adults with hereditary angioedema: an interview study. Patient 5(2):113–126. https://doi.org/10.2165/11597490-000000000-00000
doi: 10.2165/11597490-000000000-00000 pubmed: 22462765
Weller K, Groffik A, Magerl M et al (2013) Development, validation, and initial results of the Angioedema Activity Score. Allergy 68:1185–1192
pubmed: 23919330
Bonner N, Abetz-Webb L, Renault L et al (2015) Development and content validity testing of a patient-reported outcomes questionnaire for the assessment of hereditary angioedema in observational studies. Health Qual Life Outcomes 3:92. https://doi.org/10.1186/s12955-015-0292-7
doi: 10.1186/s12955-015-0292-7
Bygum A, Busse P, Caballero T et al (2017) Disease severity, activity, impact and control and how to assess them in patients with hereditary angioedema. Front Med 4:212
doi: 10.3389/fmed.2017.00212
Weller K, Donoso T, Magerl M et al (2020a) Development of the Angioedema Control Test—a patient-reported outcome measure that assesses disease control in patients with recurrent angioedema. Allergy 75:1165–1177
doi: 10.1111/all.14144
Weller K, Donoso T, Magerl M et al (2020b) Validation of the Angioedema Control Test (AECT)—a patient reported outcome instrument for assessing angioedema control. J Allergy Clin Immunol Pract 8(6):2050–2057
doi: 10.1016/j.jaip.2020.02.038
João Forjaz M, Ayala A, Caminoa M et al (2020) HAE-AS, a specific disease activity scale for hereditary angioedema with C1-inhibitor deficiency. J Investig Allergol Clin Immunol 10. Online ahead of print. https://doi.org/10.18176/jiaci.0479
Weller K, Groffik A, Magerl M et al (2012) Development and construct validation of the angioedema quality of life questionnaire. Allergy 67:1289–1298
doi: 10.1111/all.12007
Prior N, Remor E, Gomez-Traseira C et al (2012) Development of a disease-specific quality of life questionnaire for adult patients with hereditary angioedema due to C1 inhibitor deficiency (HAE-QoL): Spanish multi-centre research project. Health Qual Life Outcomes 10:82
doi: 10.1186/1477-7525-10-82
Weller K, Magerl M, Peveling-Oberhag A et al (2016) The Angioedema Quality of Life Questionnaire (AE-QoL)—assessment of sensitivity to change and minimal clinically important difference. Allergy 71:1203–1209
doi: 10.1111/all.12900
Prior N, Remor E, Pérez-Fernández E et al (2016) Psychometric field study of Hereditary Angioedema Quality of Life Questionnaire for adults: HAE-QoL. J Allergy Clin Immunol Pract 4:464–473
doi: 10.1016/j.jaip.2015.12.010
Strauss AL, Glaser BG (1975) Chronic illness and the quality of life (2
Leibovich-Nassi I, Golander H, Somech R, Har-Even D, Reshef A (2019) A new instrument for the evaluation of premonitory signs and symptoms (prodromes) of Hereditary Angioedema. Allergy 74: S106 (P. 215)
Farrar JT, Portenoy RK, Berlin JA, Kinman JL, Strom BL (2000) Defining the clinically important difference in pain outcome measures. Pain 88:287–294. https://doi.org/10.1016/s0304-3959(00)00339-0
doi: 10.1016/s0304-3959(00)00339-0 pubmed: 11068116
Cronbach L (1951) Coefficient alpha and the internal structure of tests. Psychometrika 16:297–334
doi: 10.1007/BF02310555
Baiardini I, Bousquet PJ, Brzoza Z et al (2010) Recommendations for assessing patient reported outcomes and health-related quality of life in clinical trials on allergy: a GA(2)LEN taskforce position paper. Allergy 65(3):290–295. https://doi.org/10.1111/j.1398-9995.2009.02263.x
doi: 10.1111/j.1398-9995.2009.02263.x pubmed: 19930232
Baiardini I, Braido F, Bindslev-Jensen C et al (2011) Recommendations for assessing patient-reported outcomes and health-related quality of life in patients with urticaria: a GA(2) LEN taskforce position paper. Allergy 66(7):840–844. https://doi.org/10.1111/j.1398-9995.2011.02580.x
doi: 10.1111/j.1398-9995.2011.02580.x pubmed: 21385184
Leibovich-Nassi I, Reshef A, Somech R, Golander H (2017) A survey of hereditary angioedema in Israel. Allergy, Ast Clin Immunol 13(Suppl 2):34
Barlow JH, Cullen A, Rowe IF (1999) Comparison of knowledge and psychological well-being between patients with a short disease duration and patients with more established rheumatoid arthritis. Pat Edu Counsel 38:195–203
doi: 10.1016/S0738-3991(98)00144-X
Scully JL (2004) What is a disease? Disease, disability and their definitions. Europ Moll Biology Organ Rep 5(7):650–653
Folkman S, Lazarus R (1980) An analysis of coping in a middle-aged community sample. J Health Soc Behav 21:219–239
doi: 10.2307/2136617
Lazarus R, Folkman S (1984) Stress. Appraisal and Coping, New York, Springer
Maurer M, Aberer W, Bouillet L et al (2013) Hereditary angioedema attacks resolve faster and are shorter after early icatibant treatment. PLoS ONE 8(2):e53773. https://doi.org/10.1371/journal.pone.0053773
doi: 10.1371/journal.pone.0053773 pubmed: 23390491 pmcid: 3563637
Betschel S, Badiou J, Binkley K et al (2019) The International/Canadian Hereditary Angioedema Guideline. Allergy Ast Clin Immunol 15:72. https://doi.org/10.1186/s13223-019-0376-8
doi: 10.1186/s13223-019-0376-8
Kreiberg KB, Bygum A (2019) Reporting through smartphone application results in detailed data on acquired and hereditary angioedema attacks. Allergy 74:1800–1802
doi: 10.1111/all.13792

Auteurs

Iris Leibovich-Nassi (I)

Angioedema Research Center, Barzilai University Medical Center, 2 Ha'histadrout St, 7830604, Ashkelon, Israel.
Tel Aviv University, Tel Aviv, Israel.

Hava Golander (H)

Tel Aviv University, Tel Aviv, Israel.

Raz Somech (R)

Safra Pediatric Medical Center, Ramat Gan, Israel.

Dov Har-Even (D)

Bar Ilan University, Ramat Gan, Israel.

Avner Reshef (A)

Angioedema Research Center, Barzilai University Medical Center, 2 Ha'histadrout St, 7830604, Ashkelon, Israel. aresh@netvision.net.il.

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