Individual approach to long-term therapy in patients with hereditary angioedema (HAE-C1-INH): A case series

Andarawewa, Shimalee; Aygören‐Pürsün, Emel

doi:10.3389/falgy.2022.949387

Cited by 2 publications

(2 citation statements)

References 9 publications

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“…Previous case series that have examined the transition between LTP have included pdC1-INH and lanadelumab [ 16 , 24 ]. Some LTP transitions have been looked at in the context of a clinical trial.…”

Section: Introductionmentioning

confidence: 99%

Androgen transition and management of hereditary angioedema long-term prophylaxis in real life: a single-center case series

Hoarau,

Maleki,

Bouillet

et al. 2024

Orphanet J Rare Dis

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Background Hereditary angioedema (HAE) is a rare and potentially life-threatening disease that manifests clinically as recurrent episodes of swelling affecting multiple anatomical locations. Long-term prophylaxis (LTP) aims to control the disease by preventing HAE attacks. Previously, treatments such as attenuated androgens have been used for LTP, but they have an unfavorable adverse effect profile. Today, these limitations may be overcome by patients transitioning to newer, targeted therapies including oral berotralstat and subcutaneous lanadelumab. This case series reports the transition process between different prophylactic therapies in a family with HAE in a real-world setting. Results Four adult patient cases from the same family who underwent transitions in HAE prophylaxis are presented. Three were female and one male. Two patients who transitioned to berotralstat were initially prescribed attenuated androgens. Two patients were not taking LTP at the time of initiating targeted treatment but had previously been prescribed tranexamic acid. The length of transition varied between the patients, with the longest time taken to stabilize on new therapy being 26 months. All patients received regular follow-up in person or by telephone and all four required an adjustment from their initial treatment plan. Conclusions Transitioning between LTP in HAE may help improve control of attacks, avoid unwanted adverse effects, or better cater to individual patient preferences. Newer targeted therapies have been shown to be effective and should be discussed with patients. Shared decision-making is a tool that can aid these discussions. The transition journey between LTP therapies in HAE may not be straightforward and is specific to each patient. Physicians should consider complicating factors such as patient anxieties around changing treatment, adverse effects, preferred routes of administration, and speed of transition. Following patients closely during the transition period helps identify any issues, including difficulties with treatment adherence, and may allow the transition plan to be adapted when necessary.

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Section: Introductionmentioning

confidence: 99%

Androgen transition and management of hereditary angioedema long-term prophylaxis in real life: a single-center case series

Hoarau,

Maleki,

Bouillet

et al. 2024

Orphanet J Rare Dis

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“…6,8 The AECT has been developed and validated to assess disease control in patients with recurrent angioedema. 17,18 Previous studies have shown that it is well suited for routine practice, 19,20 clinical research, [21][22][23][24][25] and therapeutic trials 26 given its retrospective approach, brevity, and simple scoring. The AECT has excellent internal consistency and test-retest reliability, as well as high convergent validity and good known-groups validity.…”

Section: Introductionmentioning

confidence: 99%

Sensitivity to change and minimal clinically important difference of the angioedema control test

Fijen,

Vera,

Buttgereit

et al. 2023

Clinical & Translational All

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BackgroundThe Angioedema Control Test (AECT) is a patient‐reported outcome measure developed and validated for the assessment of disease control in patients with recurrent angioedema. Its sensitivity to change and minimal clinically important difference (MCID) have hitherto not been established.MethodsPatients with recurrent angioedema due to chronic spontaneous urticaria, hereditary angioedema, or acquired C1‐inhibitor deficiency were repeatedly asked to complete the AECT along with the Angioedema Quality of Life Questionnaire (AE‐QoL), Dermatology Life Quality Index (DLQI), and anchors for disease control and whether treatment was sufficient during routine care visits. The sensitivity to the change of the AECT was determined by correlating changes in its scores over time with changes in the applied anchors. The MCID was determined using anchor‐based and distributional criterion‐based approaches.ResultsEighty‐six cases were used for this analysis. Changes in AECT scores correlated well with AE‐QoL changes (but less with changes in the DLQI) as well as other applied anchors, demonstrating its sensitivity to change. The MCID was found to be three points for improvement of angioedema control. The available number of cases with meaningful deterioration in our dataset was too low to reach a definite conclusion on the MCID for deterioration of angioedema control.ConclusionThe AECT is a valuable tool to assess changes in disease control in patients with recurrent angioedema over time. The lowest AECT score change that reflects a meaningful improvement of disease control to patients (MCID) is three points.

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Individual approach to long-term therapy in patients with hereditary angioedema (HAE-C1-INH): A case series

Cited by 2 publications

References 9 publications

Androgen transition and management of hereditary angioedema long-term prophylaxis in real life: a single-center case series

Androgen transition and management of hereditary angioedema long-term prophylaxis in real life: a single-center case series

Sensitivity to change and minimal clinically important difference of the angioedema control test

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