Safety and Effectiveness of Lanadelumab in Long-Term Prevention of Hereditary Angiooedema
The purpose of this work is to review the results of modern studies on the effectiveness and safety of lanadelumab, a new drug for biological targeted therapy of hereditary angioedema. The article also presents the results of our own experience in clinical use of this option for long-term prevention of angioedema attacks. In recent years, new genetic mechanisms have been identified that determine the development of hereditary angioedema. C1-inhibitor deficiency (true or functional) leads to uncontrolled activation of the kallikrein-kinin system with the formation of bradykinin, which is the main mediator of hereditary angioedema. In this regard, kallikrein is one of the most significant targets for new targeted inhibitor drugs. Lanadelumab, a kallikrein inhibitor, was used for long-term prevention of exacerbations in a patient with a long history of hereditary angioedema and showed sufficient effectiveness and safety.
Shkodkina S.A., Afonina I.A. 2025. Safety and Effectiveness of Lanadelumab in Long-Term Prevention of Hereditary Angiooedema. Challenges in Modern Medicine, 48(4): 457–467 (in Russian). DOI: 10.52575/2687-0940-2025-48-4-457-467. EDN: LIPOJF
While nobody left any comments to this publication.
You can be first.
Latysheva E.A., Manto I.A., Aleshina L.V., Bobrikova E.N., Viktorova E.A., Gracheva E.M., Demina D.V., Fomina D.S., Shcherbina A.Yu., Latysheva T.V. 2023. Preliminary Results of a Non-Interventional Single-Center Study Evaluating the Efficacy of Long-Term use of Anadelumab in Routine Clinical Practice in the Russian Federation. Russian Journal of Allergy. 20(2): 164–176 (in Russian). doi: https://doi.org/10.36691/RJA5493
Anderson J., Maina N. 2022. Reviewing Clinical Considerations and Guideline Recommendations of C1 Inhibitor Prophylaxis for Hereditary Angioedema. Clin Transl Allergy. 18; 12(1): e12092. doi: 10.1002/clt2.12092
Azmy V., Brooks J.P., Hsu F.I. 2020. Clinical Presentation of Hereditary Angioedema. Allergy Asthma Proc. Nov 1; 41 (Suppl 1): 18–21. doi: 10.2500/aap.2020.41.200065
Banerji A., Bernstein J.A., Johnston D.T., Lumry W.R., Magerl M., Maurer M., Martinez-Saguer I., Zanichelli A., Hao J., Inhaber N., Yu M., Riedl M.A. 2022. HELP OLE Investigators. Long-Term Prevention of Hereditary Angioedema Attacks with Lanadelumab: The HELP OLE Study. Allergy. 77(3): 979–990. doi: 10.1111/all.15011
Banerji A., Davis K.H., Brown T.M., Hollis K., Hunter S.M., Long J., Jain G., Devercelli G. 2020. Patient-Reported Burden of Hereditary Angioedema: Findings from a Patient Survey in the United States. Ann Allergy Asthma Immunol. 124(6): 600–607. doi: 10.1016/j.anai.2020.02.018
Banerji A., Riedl M.A., Bernstein J.A., Cicardi M., Longhurst H.J., Zuraw B.L., Busse P.J., Anderson J., Magerl M., Martinez-Saguer I, Davis-Lorton M., Zanichelli A., Li H.H., Craig T., Jacobs J., Johnston D.T., Shapiro R., Yang W.H., Lumry W.R., Manning M.E., Schwartz L.B., Shennak M., Soteres D., Zaragoza-Urdaz R.H., Gierer S., Smith A.M., Tachdjian R., Wedner H.J., Hebert J., Rehman S.M., Staubach P., Schranz J., Baptista J., Nothaft W., Maurer M. 2018. HELP Investigators. Effect of Lanadelumab Compared with Placebo on Prevention of Hereditary Angioedema Attacks: A Randomized Clinical Trial. JAMA. 27; 320(20): 2108–2121. doi: 10.1001/jama.2018.16773
Bardou M.L.D., Constantino-Silva R.N., Alonso M.L.O., Teixeira A.J.R., Giavina-Bianchi P.F., Mansour E., Pesquero J.B., Valle S.O.R., Grumach A.S. 2025. Evaluating Functional C1INH with Multiple Laboratory Methods across Hereditary Angioedema Types. Front Immunol. 26; 16: 1654078. doi: 10.3389/fimmu.2025.1654078
Busse P.J., Christiansen S.C., Riedl M.A., Banerji A., Bernstein J.A., Castaldo A.J., Craig T., Davis-Lorton M., Frank M.M., Li H.H., Lumry W.R., Zuraw B.L. 2021. US HAEA Medical Advisory Board 2020 Guidelines for the Management of Hereditary Angioedema. J Allergy Clin Immunol Pract. 9(1): 132–150. e3. doi: 10.1016/j.jaip.2020.08.046
Craig T., Tachdjian R., Bernstein J.A., Anderson J., Nurse C., Watt M., Yu M., Juethner S. 2024. Long-Term Prevention of Hereditary Angioedema Attacks with Lanadelumab in Adolescents. Ann Allergy Asthma Immunol. 133(6): 712–719.e1. doi: 10.1016/j.anai.2024.08.001
Jacobs J., Neeno T. 2021. The Importance of Recognizing and Managing a Rare form of Angioedema: Hereditary Angioedema due to C1-inhibitor Deficiency. Postgrad Med. 133(6): 639–650. doi: 10.1080/00325481.2021.1905364
Kenniston J.A., Faucette R.R., Martik D., Comeau S.R., Lindberg A.P., Kopacz K.J., Conley G.P., Chen J., Viswanathan M., Kastrapeli N., Cosic J., Mason S., DiLeo M., Abendroth J., Kuzmic P., Ladner R.C., Edwards T.E., TenHoor C., Adelman B.A., Nixon A.E., Sexton D.J. 2014. Inhibition of Plasma Kallikrein by a Highly Specific Active Site Blocking Antibody. J Biol Chem. 22; 289(34): 23596–608. doi: 10.1074/jbc.M114.569061
Lumry W.R., Settipane R.A. 2020. Hereditary Angioedema: Epidemiology and Burden of Disease. Allergy Asthma Proc. 1; 41(Suppl 1): 08–13. doi: 10.2500/aap.2020.41.200050
Mak H.W.F., Wong J.C.Y., So S.W.M., Lam D.L.Y., Weller K., Maurer M., Li P.H. Validation and Correlations of the Angioedema Activity Score (AAS), Angioedema Quality of Life (AE-QoL) Questionnaire, and Angioedema Control Test (AECT) in Chinese Patients with Angioedema. 2024. J Allergy Clin Immunol Glob. 2; 3(4): 100295. doi: 10.1016/j.jacig.2024.100295
Maurer M., Aygören-Pürsün E., Banerji A., Bernstein J.A., Balle Boysen H., Busse P.J., Bygum A., Caballero T., Castaldo A.J., Christiansen S.C., Craig T., Farkas H., Grumach A.S., Hide M., Katelaris C.H., Li H.H., Longhurst H., Lumry W.R., Magerl M., Martinez-Saguer I., Riedl M.A., Zhi Y., Zuraw B. 2021. Consensus on Treatment Goals in Hereditary Angioedema: A Global Delphi Initiative. J Allergy Clin Immunol. 148(6): 1526–1532. doi: 10.1016/j.jaci.2021.05.016
Maurer M., Magerl M., Ansotegui I., Aygören-Pürsün E., Betschel S., Bork K., Bowen T., Balle Boysen H., Farkas H., Grumach A.S., Hide M., Katelaris C., Lockey R., Longhurst H., Lumry W.R., Martinez-Saguer I., Moldovan D., Nast A., Pawankar R., Potter P., Riedl M., Ritchie B., Rosenwasser L., Sánchez-Borges M., Zhi Y., Zuraw B., Craig T. 2018. The International WAO/EAACI Guideline for the Management of Hereditary Angioedema – The 2017 Revision and Update. Allergy. 73(8): 1575–1596. doi: 10.1111/all.13384
Maurer M., Magerl M., Betschel S. 2022. The International WAO/ EAACI Guideline for the Management of Hereditary Angioedema: The 2021 Revision and Update // Allergy. 744(7): 1961–1990. doi: 10.1111/all.15214
Morioke S., Takahagi S., Kawano R., Fukunaga A., Harada S., Ohsawa I., Masuda K., Irifuku R., Yokobayashi H., Kameyoshi Y., Tanaka A., Tamari S., Hide M. 2021. A Validation Study of the Japanese Version of the Angioedema Activity Score (AAS) and the Angioedema Quality of Life Questionnaire (AE-QoL). Allergol Int. 70(4): 471–479. doi: 10.1016/j.alit.2021.04.006.
Riedl M.A., Bernstein J.A., Craig T., Banerji A., Magerl M., Cicardi M., Longhurst H.J., Shennak M.M., Yang W.H., Schranz J., Baptista J., Busse P.J. 2017. An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Lanadelumab for Prevention of Attacks in Hereditary Angioedema: Design of the HELP Study Extension. Clin Transl Allergy. 6; 7: 36. doi: 10.1186/s13601-017-0172-9
Riedl M.A., Maurer M., Bernstein J.A., Banerji A., Longhurst H.J., Li H.H., Lu P., Hao J., Juethner S., Lumry W.R. 2020. HELP Investigators. Lanadelumab Demonstrates Rapid and Sustained Prevention of Hereditary Angioedema Attacks. Allergy. 75(11): 2879–2887. doi: 10.1111/all.14416
Riedl M.A., Staubach P., Farkas H., Zanichelli A., Ren H., Nurse C., Andresen I., Juethner S., Yu M., Zhang J. 2025. Lanadelumab for Prevention of Attacks of Non-Histaminergic Normal C1 Inhibitor Angioedema: Results from the Randomized, Double-Blind CASPIAN Study and CASPIAN Open-Label Extension. Front Immunol. 21; 16: 1502325. doi: 10.3389/fimmu.2025.1502325
Santacroce R., D'Andrea G., Maffione A.B., Margaglione M., d'Apolito M. 2021. The Genetics of Hereditary Angioedema: A Review. J Clin Med. 9; 10(9): 2023. doi: 10.3390/jcm10092023
Tachdjian R., Banerji A., Busse P.J., Agmon-Levin N., Anderson J., Cancian M., Spadaro G., Enciu C., Estepan D.N., Khutoryansky N., Jain S., Recke A. 2025. Effective Long-Term Prophylaxis with Lanadelumab in Adolescents with Hereditary Angioedema: EMPOWER/ENABLE. Pediatr Allergy Immunol. 36(4): e70072. doi: 10.1111/pai.70072
Weller K., Donoso T., Magerl M., Aygören-Pürsün E., Staubach P., Martinez-Saguer I., Hawro T., Altrichter S., Krause K., Siebenhaar F., Metz M., Zuberbier T., Freier D., Maurer M. 2020. Development of the Angioedema Control Test-A Patient-Reported Outcome Measure that Assesses Disease Control in Patients with Recurrent Angioedema. Allergy. 75(5): 1165–1177. doi: 10.1111/all.14144
Informed consent. Informed consent was obtained from the patient for the publication of the case report. All patient data were de-identified.