The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviews

Jean-Baptiste, Milenka; Itzler, Robbin; Prusty, Subhransu; Supina, Dylan; Martin, Mona L

doi:10.1186/s13023-022-02360-3

Cited by 3 publications

(2 citation statements)

References 24 publications

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“…One of the potential limitations of current tools for QoL assessment is that the convenience or side effects of treatments are not considered, even though they may have an important impact on patient QoL. There is a need for larger studies that help to differentiate between disease symptoms and treatment-associated adverse events [65,66].…”

Section: Adverse Eventsmentioning

confidence: 99%

Expert Review and Consensus on the Treat-to-Target Management of Hereditary Angioedema: From Scientific Evidence to Clinical Practice

Caballero¹,

Lleonart-Bellfill²,

Pedrosa³

et al. 2023

J Investig Allergol Clin

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Background: Hereditary angioedema with C1 inhibitor deficiency (HAE-C1INH) is a rare disease characterized by swelling episodes. It affects quality of life (QoL) and can be lethal, when involving upper airways. Treatment is individualized, with therapeutic options including on-demand treatment (ODT), and short- and long-term prophylaxis (STP, LTP). However, available guidelines are not always clear about treatment selection, its goals or assessment of goal achievement. Objective: To review the available evidence for the management of HAE-C1INH and build a Spanish expert consensus to steer HAE-C1INH management towards a treat-to-target (T2T) approach, while addressing some of the unclear aspects of the Spanish guidelines. Methods: We reviewed the literature on the management of HAE-C1INH with a T2T approach, focusing on: 1) treatment selection and goals; and 2) available tools to assess goal achievement. We discussed the literature based on clinical experience and drew up 45 statements on undefined management aspects. A panel of 53 HAE experts validated the statements through a two-round Delphi process. Results: The goals for ODT and STP are minimizing the morbidity and mortality of attacks, and preventing attacks caused by known triggers, respectively, while the main goal of LTP is to decrease the rate, severity and duration of attacks. Furthermore, when prescribing, clinicians should consider the reduction in adverse events, while increasing patient QoL and satisfaction. Appropriate instruments for assessing goal achievement have also been indicated. Conclusions: We provide recommendations on previously unclear aspects of HAE-C1INH management with ODT, STP and LTP, focusing particularly on clinical and patient-oriented goals.

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Section: Adverse Eventsmentioning

confidence: 99%

Expert Review and Consensus on the Treat-to-Target Management of Hereditary Angioedema: From Scientific Evidence to Clinical Practice

Caballero¹,

Lleonart-Bellfill²,

Pedrosa³

et al. 2023

J Investig Allergol Clin

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“…Here, we sought to further build on the earlier research of Castaldo et al [ 1 ] and evaluate the real-world benefits of LTP over a longer time period. Jean-Baptiste et al have recently evaluated the symptom experience of HAE patients and concluded that more research is needed on the effect of longer attack-free durations on HRQoL [ 27 ]. Using data drawn from a wide-ranging survey of members of the US HAEA and Hereditary Angioedema International (HAEi), we examined the relationship between duration of time attack free, and HRQoL, exploring the possibility that there is an association between observed improvement in HRQoL and attack-free duration.…”

Section: Introductionmentioning

confidence: 99%

An international survey assessing the effects of the duration of attack-free period on health-related quality of life for patients with hereditary angioedema

Itzler,

Lumry,

Sears

et al. 2024

Orphanet J Rare Dis

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Background Hereditary angioedema (HAE) is characterized by unpredictable and often severe cutaneous and mucosal swelling that affects the extremities, face, larynx, gastrointestinal tract, or genitourinary area. Introduction of novel long-term prophylactic treatment options (lanadelumab, berotralstat, and C1-esterase inhibitor SC [human]) into the treatment armamentarium has substantially reduced HAE attacks, allowing patients to be attack free for longer with improvements to their quality of life. Using data drawn from a wide-ranging survey of patients with HAE, we examined the relationship between duration of time attack free and health-related quality of life (HRQoL), exploring the possibility that there is an association between observed improvement in HRQoL and attack-free duration. Methods A survey among patients with HAE on long-term prophylaxis (LTP) in six countries (the US, Australia, Canada, UK, Germany, and Japan) assessed the relationship between attack-free duration and mean Angioedema Quality of Life (AE-QoL) scores, quality of life benefits, and rescue medication used. Analysis of covariance (ANCOVA) was used to assess the roles of LTP and attack-free period (< 1 month, 1– < 6 months, ≥ 6 months) on total AE-QoL scores. Results include descriptive p-values for strength of association, without control for multiplicity. Descriptive statistics were used to show the relationship between time attack free and quality of life benefits. Results Longer durations of time for which participants reported being attack free at the time of the survey correlated with better AE-QoL scores and less use of rescue medication. The mean total AE-QoL scores were 51.8, 33.2, and 19.9 for those who reported having been attack free for < 1 month, 1– < 6 months, and ≥ 6 months, respectively, with higher scores reflecting more impairment. The ANCOVA results showed a strong association between attack-free duration and AE-QoL total score. Conclusion This study shows that longer attack-free duration has an influential role for better HRQoL in patients receiving LTP. Prolonging the attack-free period is an important goal of therapy and recent advances in LTP have increased attack-free duration. However, opportunities exist for new treatments to further increase attack-free duration and improve HRQoL for all patients with HAE.

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Los migrantes menos pensados. Zamuco y chiquito-hablantes en la fundación de Vila Maria do Paraguai (Mato Grosso), siglo XVIII

Martínez

2022

revindias

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A partir del cotejo de dos documentos tardocoloniales provenientes de la Gobernación de Chiquitos y de la Capitanía de Mato Grosso se demuestra la participación mayoritaria de indígenas chiquito y zamuco-hablantes de la gobernación de Chiquitos (actual Bolivia) en la fundación de Vila Maria do Paraguai, al contrario de las versiones más extendidas que sostienen su filiación arawak y otuqui. Al analizar el escenario en el que tuvo lugar su migración a Mato Grosso, la idea del retorno al lugar de origen como principal motivación resulta enfáticamente cuestionada. En cambio, se pone en consideración la pertenencia étnica de los migrantes, sus relaciones interétnicas y el cambio sociocultural que experimentaron como consecuencia de su participación activa en los procesos de historia regional.

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The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviews

Cited by 3 publications

References 24 publications

Expert Review and Consensus on the Treat-to-Target Management of Hereditary Angioedema: From Scientific Evidence to Clinical Practice

Expert Review and Consensus on the Treat-to-Target Management of Hereditary Angioedema: From Scientific Evidence to Clinical Practice

An international survey assessing the effects of the duration of attack-free period on health-related quality of life for patients with hereditary angioedema

Los migrantes menos pensados. Zamuco y chiquito-hablantes en la fundación de Vila Maria do Paraguai (Mato Grosso), siglo XVIII

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