Using an extended treatment regimen of lanadelumab in the prophylaxis of hereditary angioedema: a single-centre experience

Abuzakouk, Mohamed; Ghorab, Omar; Al-Hameli, Hamad; Salvo, Fulvio; Grandon, Deepa; Maurer, Marcus

doi:10.1016/j.waojou.2022.100664

Cited by 4 publications

(1 citation statement)

References 9 publications

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“…More recently, Abuzakouk et al compared the efficacy of standard and extended dosing regimens of lanadelumab in nine patients in a single-center retrospective study and found the effect of lanadelumab in terms of number of angioedema attacks was significant in both extended and standard group at each time point (Months 1, 2, 3 and 9). 53 Additionally, they found statistically significant improvement in the number of attacks requiring emergency care and all patients reported a significant quality of life improvement. Therefore, authors concluded that lanadelumab extended dosing regimen was equally effective compared to the standard one and could improve the rational use of the drug.…”

Section: United States Food and Drug Administration (Us Fda) Adverse ...mentioning

confidence: 92%

Safety Aspects and Rational Use of Lanadelumab Injections in the Treatment of Hereditary Angioedema (HAE): Clinical Insights

Petkova¹,

Yordanova²,

Staevska³

et al. 2022

DHPS

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Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent episodes of skin/mucosal swelling, and/ or attacks of severe abdominal pain when it affects the gastrointestinal tract. The disease might be unexpectedly fatal when the upper airways are compromised. HAE clinical presentation, disease course and prognosis are associated with significant disease burden and severely impaired quality of life. Lanadelumab is a breakthrough therapy for the prevention of attacks in HAE type 1 and 2 patients. This revolutionary approach to administer a single subcutaneous injection (once every two to four weeks) and achieve complete disease control has dramatically improved patient care resulting in significant change in the life of affected families. Current data support the drug's tolerability in adult and adolescent patients without notable safety concerns in both clinical research and real-world settings. Rational use of prophylactic treatments of HAE searches for a socio-economic balance, taking into account the life-long course of the disease, the public health funds who pay the monetary price, and the patients who might need to receive the therapy for a period longer than investigated during the development program. In this review, we address the current evidence on lanadelumab's tolerability, highlighting aspects of the drug's rationale use in clinical practice. Further studies need to investigate whether this therapy might be appropriate in other forms of angioedema, such as idiopathic primary angioedema and HAE with normal C1 inhibitor. Future efforts must focus to improve modern drugs' accessibility in more countries. Although modern prophylactic options lessen the risk of fatal laryngeal attacks, patients must be equipped with reliable on-demand therapies and be trained how to use them as such a risk cannot be fully diminished with potentially life-threatening attacks occurring even in subjects with successful and stable long-term prophylaxis. Notwithstanding, further studies are needed to identify early responders from non-responders and develop therapies for the latter.

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Section: United States Food and Drug Administration (Us Fda) Adverse ...mentioning

confidence: 92%

Safety Aspects and Rational Use of Lanadelumab Injections in the Treatment of Hereditary Angioedema (HAE): Clinical Insights

Petkova¹,

Yordanova²,

Staevska³

et al. 2022

DHPS

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Hereditary Angioedema Attacks in Patients Receiving Long-Term Prophylaxis: A Systematic Review

Longhurst,

Cancian,

Grivcheva-Panovska

et al. 2024

Clinic Rev Allerg Immunol

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Long-term prophylaxis (LTP) has been shown to reduce the frequency of hereditary angioedema (HAE) attacks; however, attacks occurring in patients receiving LTP have not been well characterized. The objective of this systematic review was to evaluate the proportion of type I/II HAE (HAE-C1INH) patients who experience attacks while receiving LTP, the characteristics of these attacks, and associated on-demand therapy use. A systematic search was conducted in PubMed to identify studies reporting LTP use with plasma-derived C1 inhibitor (pdC1INH), lanadelumab, berotralstat, androgens, or antifibrinolytics in patients with HAE-C1INH. Forty-five primary studies met the inclusion criteria. In phase 3 trials, attack-free rates were 40% for subcutaneous pdC1INH 60 IU/kg twice weekly at 16 weeks, and 44% for lanadelumab 300 mg every second week at 6 months (77% during steady-state [days 70–182]); there was no difference in attack-free rate for berotralstat 150 mg versus placebo at 24 weeks. Phase 3 studies reported a lower average attack severity with subcutaneous and intravenous pdC1INH versus placebo. With lanadelumab and berotralstat, the prophylactic treatment effect was more pronounced in peripheral attacks than in abdominal and laryngeal attacks. Laryngeal attacks accounted for 2%-7% of all attacks in observational and interventional studies, regardless of the LTP agent received. On-demand therapy was used in 49%-94% of attacks occurring in the presence of LTP. In conclusion, patients receiving LTP experienced attacks in all anatomic locations, including the larynx. Most attacks were treated with on-demand therapy, although outcomes were not reported. Access to on-demand therapy remains essential for all people with HAE-C1INH.

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Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema

Riedl,

Bordone,

Revenko

et al. 2024

The Journal of Allergy and Clinical Immunology: In Practice

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Using an extended treatment regimen of lanadelumab in the prophylaxis of hereditary angioedema: a single-centre experience

Cited by 4 publications

References 9 publications

Safety Aspects and Rational Use of Lanadelumab Injections in the Treatment of Hereditary Angioedema (HAE): Clinical Insights

Safety Aspects and Rational Use of Lanadelumab Injections in the Treatment of Hereditary Angioedema (HAE): Clinical Insights

Hereditary Angioedema Attacks in Patients Receiving Long-Term Prophylaxis: A Systematic Review

Clinical Progress in Hepatic Targeting for Novel Prophylactic Therapies in Hereditary Angioedema

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