Consensus on treatment goals in hereditary angioedema: A global Delphi initiative

Maurer, Marcus; Aygören‐Pürsün, Emel; Banerji, Aleena; Bernstein, Jonathan A.; Boysen, Henrik Balle; Busse, Paula J.; Bygum, Anette; Caballero, Teresa; Castaldo, Anthony J.; Christiansen, Sandra C.; Craig, Timothy J.; Farkas, Henriette; Grumach, Anete Sevciovic; Hide, Michihiro; Katelaris, Constance H.; Li, H. Henry; Longhurst, Hilary; Lumry, William R.; Magerl, Markus; Martinez‐Saguer, Inmaculada; Riedl, Marc A.; Zhi, Yuxiang; Zuraw, Bruce L.

doi:10.1016/j.jaci.2021.05.016

Cited by 34 publications

(58 citation statements)

References 42 publications

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“…Long-term prophylaxis (LTP) aims to reduce the heavy physical and psychological burden of this disease; the quest to achieve prolonged attack-free periods or attain complete control and normalize patients' daily lives is a continuing goal. 1,5,9,10 Enhanced understanding of the underlying pathophysiology of HAE, including the critical role plasma kallikrein plays in its pathogenesis, 11 has substantially broadened the treatment armamentarium of targeted agents for this disease. Lanadelumab is a subcutaneously administered fully human monoclonal antibody that acts as a potent and specific inhibitor of active plasma kallikrein.…”

Section: Introductionmentioning

confidence: 99%

“…Importantly, the frequency of attacks does not correlate with their severity 5 (even the first laryngeal edema attack can be fatal), 8 and the historical anatomical location of attacks does not predict future attack sites. Long‐term prophylaxis (LTP) aims to reduce the heavy physical and psychological burden of this disease; the quest to achieve prolonged attack‐free periods or attain complete control and normalize patients’ daily lives is a continuing goal 1,5,9,10 …”

Section: Introductionmentioning

confidence: 99%

“…Long‐term prophylaxis (LTP) aims to reduce the heavy physical and psychological burden of this disease; the quest to achieve prolonged attack‐free periods or attain complete control and normalize patients’ daily lives is a continuing goal. 1 , 5 , 9 , 10 …”

Section: Introductionmentioning

confidence: 99%

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Long‐term prevention of hereditary angioedema attacks with lanadelumab: The HELP OLE Study

Banerji

Bernstein

Johnston

et al. 2021

Allergy

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Background The aim was to evaluate long‐term effectiveness and safety of lanadelumab in patients ≥12 y old with hereditary angioedema (HAE) 1/2 (NCT02741596). Methods Rollover patients completing the HELP Study and continuing into HELP OLE received one lanadelumab 300 mg dose until first attack (dose‐and‐wait period), then 300 mg q2wks (regular dosing stage). Nonrollovers (newly enrolled) received lanadelumab 300 mg q2wks from day 0. Baseline attack rate for rollovers: ≥1 attack/4 weeks (based on run‐in period attack rate during HELP Study); for nonrollovers: historical attack rate ≥1 attack/12 weeks. The planned treatment period was 33 months. Results 212 patients participated (109 rollovers, 103 nonrollovers); 81.6% completed ≥30 months on study (mean [SD], 29.6 [8.2] months). Lanadelumab markedly reduced mean HAE attack rate (reduction vs baseline: 87.4% overall). Patients were attack free for a mean of 97.7% of days during treatment; 81.8% and 68.9% of patients were attack free for ≥6 and ≥12 months, respectively. Angioedema Quality‐of‐Life total and domain scores improved from day 0 to end of study. Treatment‐emergent adverse events (TEAEs) (excluding HAE attacks) were reported by 97.2% of patients; most commonly injection site pain (47.2%) and viral upper respiratory tract infection (42.0%). Treatment‐related TEAEs were reported by 54.7% of patients. Most injection site reactions resolved within 1 hour (70.2%) or 1 day (92.6%). Six (2.8%) patients discontinued due to TEAEs. No treatment‐related serious TEAEs or deaths were reported. Eleven treatment‐related TEAEs of special interest were reported by seven (3.3%) patients. Conclusion Lanadelumab demonstrated sustained efficacy and acceptable tolerability with long‐term use in HAE patients.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Long‐term prevention of hereditary angioedema attacks with lanadelumab: The HELP OLE Study

Banerji

Bernstein

Johnston

et al. 2021

Allergy

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“…The goals of treatment, in HAE, are to achieve complete control of the disease and to normalize patients’ lives (Recommendation 13). 178 This can currently only be achieved by long-term prophylactic treatment, also referred to as long term prophylaxis (LTP), ie, the regular use of medication that reduces the burden of the disease by preventing attacks.…”

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

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120

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“…Hereditary angioedema (HAE) is a rare disease with autosomal dominant inheritance, characterized by recurrent episodes of subcutaneous and sub-mucosal edema attacks. HAE can be classified in HAE with C1 inhibitor deficiency (HAE-C1-INH) or HAE with normal C1 inhibitor (HAE-nC1-INH) ( 1 ). In both cases, angioedema occurs due to excessive activation of the plasma contact system, leading to increased levels of bradykinin and, consequently, an increase in vascular permeability and extravasation of fluids to the extravascular environment ( 2 ).…”

Section: Introductionmentioning

confidence: 99%

Pregnancy in Patients With Hereditary Angioedema and Normal C1 Inhibitor

et al. 2022

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BackgroundHAE with normal C1 inhibitor (HAE-nC1-INH) has been identified as a bradykinin mediated angioedema. Estrogens are one of the main trigger factors. Pregnancy in HAE with C1 inhibitor deficiency showed variable course, however, few reports are available for HAE-nC1-INH. We evaluated the course of pregnancies in women diagnosed with HAE-nC1-INH.MethodsWomen with diagnosis of HAE-nC1-INH according to the following criteria: clinical manifestations similar to HAE-C1-INH, normal biochemical evaluation and family history were included. A questionnaire about pregnancies was applied after consent. Genetic evaluation for known mutations was performed in all patients.ResultsA total of 45 pregnancies occurring in 26 HAE-nC1-INH patients were evaluated (7/26 patients with F12 variant). Spontaneous abortion was reported in 8/45 (17.8%) pregnancies. Onset of attacks started before the pregnancy in 18/26 patients; during the pregnancy in 2/26; and after the pregnancy in 6/26. HAE attacks occurred in 24/37 pregnancies (64,7%): during the 1st trimester in 41.7%; 2nd trimester in 12.5%; 3rd trimester in 20.8%; 1st and 3rd trimesters in 4.2% and during the whole pregnancy in 20.8%. Among 15/18 patients who had attacks before pregnancy, symptoms persisted with worsening in 9/15; improvement in 4/15; no change in 1/15, and no response in 1/15.ConclusionsThe occurrence of abortion in HAE-nC1-INH was similar to the expected for not affected women. The 1st trimester of the pregnancy was more symptomatic for HAE-nC1-INH women. Considering the strong relevance of estrogens in HAE-nC1-INH, pregnancy could worsen the course of disease.

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Consensus on treatment goals in hereditary angioedema: A global Delphi initiative

Cited by 34 publications

References 42 publications

Long‐term prevention of hereditary angioedema attacks with lanadelumab: The HELP OLE Study

Long‐term prevention of hereditary angioedema attacks with lanadelumab: The HELP OLE Study

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

Pregnancy in Patients With Hereditary Angioedema and Normal C1 Inhibitor

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