Intermediate results of a prospective multicenter observational study of long-term therapy with eculizumab biosimilar in patients with atypical hemolytic uremic syndrome (ECU-aHUS-N03)
Background. Atypical hemolytic uremic syndrome (aHUS) is a rare systemic disease of hereditary or acquired origin, included in the group of thrombotic microangiopathies, the progressive course of which leads to life-threatening damage to organs and systems, most often accompanied by acute kidney injury. The disease is based on uncontrolled activation of the alternative complement pathway. The main drug for the pathogenetic therapy of aHUS is eculizumab, a recombinant humanized monoclonal IgG antibody to the C5 component of complement. Objective. Prospective multicenter observational study was conducted to collect and analyze data on the efficacy and safety of the eculizumab bioanalogue Elizaria® (JSC GENERIUM) in long-term pathogenetic therapy in patients with aHUS in Moscow. Material and methods. The intermediate results of the study included data from 30 patients who received Elizaria® in routine clinical practice. The patients were divided into two groups: dialysis-dependent and with conservative stage of renal failure, stratified by GFR and proteinuria/albuminuria level. The follow-up period of patients was at least 32 weeks and included 3 visits. Results. Lactate dehydrogenase activity remained stable throughout the analyzed follow-up period and was 195.3±59.49 U/L at Visit 3 in all patients, 200±65.44 and 176.8±29.81 U/L in the groups of patients without hemodialysis (HD) and on HD, respectively. At Visit 3, the platelet count in patients on HD remained stable within normal limits. In the group of patients without HD, a slight decrease in the platelet count within normal values was recorded. TMA-related events were absent in 22 (96%) patients not receiving hemodialysis at Visit 3. In 5 (17%) patients with acute disease, the absence of TMA-related events by Visit 3 was recorded in 40% of cases. Hemoglobin concentration remained stable throughout the follow-up period. At Visit 3, a statistically significant decrease in the reticulocyte count was recorded in the overall group (p=0.021) and in the group without HD (p<0.001). A statistically significant decrease in the schistocyte count was noted in the overall group (p=0.015). Quality of life according to the FACIT-F scale remained stable throughout the follow-up period. During the safety assessment, 13 episodes of mild adverse reactions were recorded in 2 patients on HD. Conclusion. Long-term pathogenetic therapy with Elizaria® in patients with aHUS was characterized by a stable effect and a favorable safety profile..Kotenko O.N., Vinogradov V.E., Ivanova E.S., Shutov E.V., Dudko M.Yu., Mayorov V.V., Kudlay D.A., Markova O.A., Kolobov E.P., Borozinets A.Yu.
Keywords
atypical hemolytic uremic syndrome
thrombotic microangiopathy
estimated glomerular filtration rate
hemodialysis
eculizumab
About the Authors
Oleg N. Kotenko – Dr.Sci. (Med.), Chief External Expert in Nephrology of the Moscow City, Head of the Moscow City Scientific and Practical Center for Nephrology and Transplanted Kidney Pathology, City Clinical Hospital No. 52. Address: 3 Pekhotnaya St., Moscow, 123182; Professor at the Department of General Therapy, Faculty of Continuous Professional Education, Pirogov Russian National Research Medical University. Address: 1 Ostrovityanova St., Moscow,117513. ORCID: https://orcid.org/0000-0001-8264-7374.Vladimir E.Vinogradov – Head of the Consultative and Diagnostic Nephrology Department, City Clinical Hospital No. 52. Address: 3 Pekhotnaya St., Moscow, 123182
Ekaterina S. Ivanova – Cand.Sci. (Med.), Nephrologist, Nephrology Department No. 1, City Clinical Hospital No. 52. Address: 3 Pekhotnaya St., Moscow, 123182.
ORCID: https://orcid.org/0000-0001-7407-5695.
Evgeny V.Shutov – Dr.Sci. (Med.), Professor, Senior Researcher, Head of the Interdistrict Nephrology Center, Moscow Botkin Multidisciplinary Scientific and Clinical Center. Address: 5 2nd Botkinsky proezd, Moscow,125284
Marina Yu. Dudko – Cand.Sci. (Med.), Head of the Consultative and Diagnostic Nephrology Department, Moscow Botkin Multidisciplinary Scientific and Clinical Center. Botkina DZM. Address: 5 2nd Botkinsky proezd, Moscow,125284
Vasily V. Mayorov – Head of the Nephrology Department, City Clinical Hospital n.a. S.S. Yudin,. Address: 4 Kolomensky proezd, Moscow, 115446
Dmitry A. Kudlay – Dr.Sci. (Med.), Corresponding Member of the Russian Academy of Sciences, Professor, Department of Pharmacology, Institute of Pharmacy, Sechenov First Moscow State Medical University (Sechenov University). Address: Bldg. 2, 8 Trubetskaya st., Moscow, 119991; tel.: +7 (499) 248-05-53; Vice President for the Implementation of New Medical Technologies, JSC GENERIUM. Address: Entrance 2, 10 Testovskaya st., Moscow, 123112; NRC Institute of Immunology FMBA of Russia.
Address: 24, Kashirskoye Shosse, Moscow, 115522. tel.: 8 (495) 988-47-94. ORCID: https://orcid.org/0000-0003-1878-4467.
Oksana A. Markova – Head of the Scientific Expertise and Pharmacovigilance Department, JSC GENERIUM Address: Entrance 2, 10 Testovskaya st., Moscow, 123112;
tel.: 8 (495) 988-47-94; e-mail: oamarkova@generium.ru. ORCID: http://orcid.org/0000-0002-1179-3881.
Evgeny P. Kolobov – Director of the Marketing and Promotion Department of Onco-Hematology Drugs, JSC GENERIUM. Address: Entrance 2, 10 Testovskaya st., Moscow, 123112; Tel.: 8 (495) 988-47-94. ORCID: https://orcid.org/0009-0000-9798-5640.
Anton Yu. Borozinets – Cand.Sci. (Med.), Senior Researcher, Department of Marketing and Promotion of Onco-Hematological Drugs, JSC GENERIUM.
Address: Entrance 2, 10 Testovskaya st., Moscow, 123112; Tel.: 8 (495) 988-47-94; E-mail: a.borozinets@generium.ru. ORCID: https://orcid.org/0000-0002-4863-8471
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