Recombinant human C1 esterase inhibitor (Conestat alfa) for prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema

Valerieva, Anna; Caccia, Sonia; Cicardi, Marco

doi:10.1080/1744666x.2018.1503055

Cited by 9 publications

(8 citation statements)

References 90 publications

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“…Alternative options for LTP, in the absence of all three first-line LTP treatments, include the off-label use of intravenous recombinant C1-INH. 245 Importantly, first-line LTP treatments should be initiated as approved. For lanadelumab, and to some extent for C1-INH, adapting the dose and/or treatment interval, after achieving complete response, can decrease treatment burden.…”

Section: Long-term Prophylaxis With Berotralstatmentioning

confidence: 99%

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The international WAO/EAACI guideline for the management of hereditary angioedema—The 2021 revision and update

Maurer

Magerl

Betschel

et al. 2022

Allergy

237

295

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Hereditary Angioedema (HAE) is a rare and disabling disease. Early diagnosis and appropriate therapy are essential. This update and revision of the global guideline for HAE provides up-to-date consensus recommendations for the management of HAE. In the development of this update and revision of the guideline, an international expert panel reviewed the existing evidence and developed 20 recommendations that were discussed, finalized and consented during the guideline consensus conference in June 2016 in Vienna. The final version of this update and revision of the guideline incorporates the contributions of a board of expert reviewers and the endorsing societies. The goal of this guideline update and revision is to provide clinicians and their patients with guidance that will assist them in making rational decisions in the management of HAE with deficient C1-inhibitor (type 1) and HAE with dysfunctional C1-inhibitor (type 2). The key clinical questions covered by these recommendations are: 1) How should HAE-1/2 be defined and classified?, 2) How should HAE-1/2 be diagnosed?, 3) Should HAE-1/2 patients receive prophylactic and/or on-demand treatment and what treatment options should be used?, 4) Should HAE-1/2 management be different for special HAE-1/2 patient groups such as pregnant/lactating women or children?, and 5) Should HAE-1/2 management incorporate self-administration of therapies and patient support measures? This article is co-published with permission in Allergy and the World Allergy Organization Journal.

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Section: Long-term Prophylaxis With Berotralstatmentioning

confidence: 99%

“…Where none of the three recommended first‐line LTP treatments are available, efforts should aim to change this. Alternative options for LTP, in the absence of all three first‐line LTP treatments, include the off‐label use of intravenous recombinant C1‐INH 245 …”

Section: Long‐term Prophylactic Treatment Of Haementioning

confidence: 99%

The international WAO/EAACI guideline for the management of hereditary angioedema—The 2021 revision and update

Maurer

Magerl

Betschel

et al. 2022

Allergy

237

295

View full text Add to dashboard Cite

show abstract

“…Alternative options for LTP, in the absence of all 3 first-line LTP treatments, include the off-label use of intravenous recombinant C1–INH. 245 …”

Section: Long-term Prophylactic Treatment Of Haementioning

confidence: 99%

The international WAO/EAACI guideline for the management of hereditary angioedema – The 2021 revision and update

Maurer

Magerl

Betschel

et al. 2022

World Allergy Organization Journal

120

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“…14,18 The concept that replacement therapy should restore C1-INH functional activity to the normal range is intuitively attractive, although additional research is required to validate that plasma concentrations correlate sufficiently with attack prevention. 14,20 Moreover, plasma functional C1 inhibitor levels are not informative when rhC1-INH is used, despite good observed efficacy. 21 Questions about a correlation between C1-INH plasma concentrations and clinical effectiveness have been raised, given the efficacy of rhC1-INH as prophylaxis despite its relatively short plasma half-life compared with pdC1-INH therapies.…”

Section: Long-term Prophylaxis To Address Hae Pathophysiologymentioning

confidence: 99%

A Review of Randomized Controlled Trials of Hereditary Angioedema Long-Term Prophylaxis with C1 Inhibitor Replacement Therapy: Alleviation of Disease Symptoms Is Achievable

Longhurst¹,

Valerieva²

2023

JAA

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Through its fluctuating disease activity and unpredictable attacks, hereditary angioedema (HAE) imposes a substantial patient burden. To minimize HAE burden and improve quality of life, treatment should involve individualized management strategies that address on-demand therapy and short-term/long-term prophylaxis. Goals of long-term prophylaxis include reducing the number, severity, and burden of HAE attacks. The best characterized forms of HAE arise from deficiency or dysfunction of C1-inhibitor (C1-INH; types I/II), and C1-INH replacement therapy is a first-line intervention for on-demand (acute) treatment of HAE attacks, shortterm prophylaxis before high-risk procedures, and long-term prophylaxis. Randomized, double-blind, placebo-controlled crossover trials have shown dose-dependent efficacy with plasma-derived C1-INH (pdC1-INH) 40-60 IU/kg subcutaneously, pdC1-INH 1000 U intravenously, and recombinant human C1-INH (rhC1-INH) 50 IU/kg (maximum 4200 IU) intravenously, all administered twice weekly, as long-term prophylaxis in patients with a history of 2 to ≥4 attacks/month. Overall, up to 83% (pdC1-INH 60 IU/kg) of patients experienced an HAE attack reduction threshold of ≥70%, and up to 58% (pdC1-INH 60 IU/kg) achieved an attack reduction threshold of ≥90%. Lower-dose intravenous pdC1-INH therapy (1000 U) was seemingly less effective, with 45% of 22 patients experiencing an HAE attack reduction threshold of ≥70%, and up to 23% achieving an attack reduction threshold of ≥90%. Higherdose intravenous rhC1-INH 50 IU/kg (maximum, 4200 IU) twice weekly was of intermediate benefit. Despite a baseline mean attack frequency of 17.9 (during the 3 months prior to study treatment) and a mean attack frequency during a 4-week placebo period of 7.2, 52% of 23 patients experienced ≥70% reduction in attack frequency and 26% of 23 patients experienced ≥90% reduction in attack frequency. The increasing patient percentages treated with C1-INH replacement therapy as long-term prophylaxis meeting these high thresholds reinforces hopes and expectations that "attack freedom" is achievable, including for those with moderate or severe disease.

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Recombinant human C1 esterase inhibitor (Conestat alfa) for prophylaxis to prevent attacks in adult and adolescent patients with hereditary angioedema

Cited by 9 publications

References 90 publications

The international WAO/EAACI guideline for the management of hereditary angioedema—The 2021 revision and update

The international WAO/EAACI guideline for the management of hereditary angioedema—The 2021 revision and update

The international WAO/EAACI guideline for the management of hereditary angioedema – The 2021 revision and update

A Review of Randomized Controlled Trials of Hereditary Angioedema Long-Term Prophylaxis with C1 Inhibitor Replacement Therapy: Alleviation of Disease Symptoms Is Achievable

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