Preliminary results of a non-interventional single-center study evaluating the efficacy of long-term use of lanadelumab in routine clinical practice in the Russian Federation
- Authors: Latysheva E.A.1,2, Manto I.A.1, Aleshina L.V.3, Bobrikova E.N.4, Viktorova E.A.5, Gracheva E.M.6, Demina D.V.7, Fomina D.S.4,8, Shcherbina A.Y.5, Latysheva T.V.1,9
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Affiliations:
- National Research Center — Institute of Immunology
- The Russian National Research Medical University named after N.I. Pirogov
- Saratov State Medical University named after V.I. Razumovsky
- City Clinical Hospital No. 52
- Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology
- Vologda region clinical hospital
- Research Institute of Fundamental and Clinical Immunology
- The First Sechenov Moscow State Medical University
- Moscow State University of Medicine and Dentistry named after A.I. Evdokimov
- Issue: Vol 20, No 2 (2023)
- Pages: 164-176
- Section: Original studies
- URL: https://journals.rcsi.science/raj/article/view/253519
- DOI: https://doi.org/10.36691/RJA5493
- ID: 253519
Cite item
Abstract
BACKGROUND: Hereditary angioedema with C1 inhibitor deficiency is a rare disease caused by a deficiency and/or a decrease in the functional activity of the C1 inhibitor. The primary symptom of this condition is recurrent angioedema of various localizations. According to the modern concept of treatment, the therapy aims to stop emerging angioedema and prevent death as well as achieve complete control of the disease and a high quality of life. Lanadelumab is a modern medicine developed and used to prevent attacks in patients with hereditary angioedema aged ≥12 years.
AIM: A retrospective study (IISR-2021-200085) was conducted to evaluate the efficiency and safety of lanadelumab in real-life practice in Russia.
MATERIALS AND METHODS: In all, 16 patients with hereditary angioedema and C1 inhibitor deficiency were enrolled at the initiation of lanadelumab treatment. The patients were predominantly female (81%; 13/16). The average age of patients was 29.9 years; 19% (3/16) of the patients were adolescents. The effectiveness was evaluated by comparing the patient-reported attack rates. The following PROs for the adults only were assessed initially and during the treatment: angioedema activity score, angioedema control test (AECT), angioedema quality of life questionnaire (AE-QoL), and hereditary angioedema activity score. The incidences of adverse events were evaluated.
RESULTS: Before lanadelumab, 69% (11/16) of the patients received alternative long-term prophylaxis, which was canceled after the start of lanadelumab treatment. The average number of attacks per month and treated attacks per month prelanadelumab were 10 and 4.7 per patient, respectively. After 6 months of treatment, these values were 0.26 and 0.09, respectively (10 patients were symptom free at 6 months after the initiation of the treatment). After 3 months of treatment, the mean AECT values improved from 5.6 to 14.2 (p <0.001), and all patients showed adequate disease control. After 6 months of treatment, AE-QoL decreased from 58 to 19 (p <0.001). No serious adverse events related to lanadelumab were observed.
CONCLUSION: Our study demonstrated that the composite effect of lanadelumab minimizes the attack rate and improves the quality of life in patients with hereditary angioedema. A good safety profile of lanadelumab is shown.
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##article.viewOnOriginalSite##About the authors
Elena A. Latysheva
National Research Center — Institute of Immunology; The Russian National Research Medical University named after N.I. Pirogov
Email: ealat@mail.ru
ORCID iD: 0000-0002-1606-205X
SPIN-code: 2063-7973
MD, Dr. Sci. (Med.), Senior Research Associate
Russian Federation, 24 Kashirskoye shosse, 115522 Moscow; MoscowIrina A. Manto
National Research Center — Institute of Immunology
Author for correspondence.
Email: irina.manto@yandex.ru
ORCID iD: 0000-0001-6432-394X
SPIN-code: 7944-5159
MD, Research Associate
Russian Federation, 24 Kashirskoye shosse, 115522 MoscowLiubov V. Aleshina
Saratov State Medical University named after V.I. Razumovsky
Email: Lubov-sk@mail.ru
ORCID iD: 0000-0002-3281-7379
SPIN-code: 7650-6647
MD, Cand. Sci. (Med.)
Russian Federation, SaratovElena N. Bobrikova
City Clinical Hospital No. 52
Email: elena.bobrikova.69@mail.ru
ORCID iD: 0000-0002-6534-5902
SPIN-code: 5806-7260
MD
Russian Federation, MoscowEkaterina A. Viktorova
Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology
Email: vikat.10@mail.ru
ORCID iD: 0000-0002-2427-1417
MD, Cand. Sci. (Med.)
Russian Federation, MoscowElena M. Gracheva
Vologda region clinical hospital
Email: grachevaem11@gmail.com
ORCID iD: 0000-0002-0746-4794
Russian Federation, Vologda
Darya V. Demina
Research Institute of Fundamental and Clinical Immunology
Email: immunology@mail.ru
ORCID iD: 0000-0002-0342-5368
SPIN-code: 4793-0879
MD, Cand. Sci. (Med.)
Russian Federation, NovosibirskDaria S. Fomina
City Clinical Hospital No. 52; The First Sechenov Moscow State Medical University
Email: daria.s.fomina@gmail.com
ORCID iD: 0000-0002-5083-6637
SPIN-code: 3023-4538
MD, Cand. Sci. (Med.)
Russian Federation, Moscow; MoscowAnna Yu. Shcherbina
Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology
Email: shcher26@hotmail.com
ORCID iD: 0000-0002-3113-4939
SPIN-code: 6759-0031
MD, Dr. Sci. (Med.), Professor
Russian Federation, MoscowTatiana V. Latysheva
National Research Center — Institute of Immunology; Moscow State University of Medicine and Dentistry named after A.I. Evdokimov
Email: tvlat@mail.ru
ORCID iD: 0000-0003-1508-0640
SPIN-code: 8929-7644
MD, Dr. Sci. (Med.), Professor
Russian Federation, 24 Kashirskoye shosse, 115522 Moscow; MoscowReferences
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