Resolution of the Expert council “Unsolved problems of targeted therapy for paroxysmal nocturnal hemoglobinuria in Russia”
https://doi.org/10.17650/1818-8346-2023-18-3-140-144
Abstract
On June 24, 2023, an Expert Council was held in St. Petersburg, during which leading experts in the field of hematology discussed current achievements and answered a number of unresolved issues of targeted therapy of paroxysmal nocturnal hemoglobinuria (APG) in order to further improve treatment results in Russia. During the Expert Council, the following aspects of targeted APG therapy were considered: • criteria for the suboptimal response of patients with APG to therapy with inhibitors of the 5th component of complement (C5); • efficacy and safety of the use of pegcetacoplan in APG in patients with insufficient efficacy of inhibitors of the C5 component of complement; • vaccination issues before starting therapy with complement inhibitors and the possibility of conducting treatment with pegcetacoplan at home.
About the Authors
A. D. KulaginRussian Federation
Alexander Dmitrievich Kulagin, MD, Director, Head of the Department
Department of Hematology, Transfusiology and Transplantology with the course of Pediatric Oncology of the FPO named after prof. B. V. Afanasyev
Saint-Petersburg
E. A. Lukina
Russian Federation
Elena Alekseevna Lukina, MD, Professor, Head of the Department
department of orphan diseases
Moscow
V. V. Ptushkin
Russian Federation
Vadim Vadimovich Ptushkin, MD, Professor, Chief Freelance Specialist Hematologist, Deputy Chief Physician for Hematology
Moscow
M. P. Kostinov
Russian Federation
Mikhail Petrovich Kostinov, Corresponding Member of the Russian Academy of Sciences, MD, Professor, Head of the Laboratory
laboratory of vaccination and immunotherapy of allergic diseases
Moscow
E. G. Arshanskaya
Russian Federation
Evgenia Grigorievna Arshanskaya, PhD, hematologist
Moscow
T. N. Babaeva
Russian Federation
Tatiana Nikolaevna Babaeva, PhD, hematologist, assistant
Faculty of advanced training and professional retraining of doctors
Department of Therapy, Hematology and Transfusiology
Novosibirsk
T. I. Ksenzova
Russian Federation
Tatiana Ilyinichna Ksenzova, PhD, Head of the Department
Department of Hematology and Chemotherapy
Tyumen
Z. T. Fidarova
Russian Federation
Zalina Taimurazovna Fidarova, PhD, Hematologist, Head of the department
hemoblastosis and hematopoietic depression chemotherapy department with bone marrow and hematopoietic stem cell transplantation unit
Moscow
M. V. Marchenko
Russian Federation
Maria Viktorovna Marchenko, hematologist
Saint-Petersburg
References
1. Kulagin A.D. Paroxysmal nocturnal hemoglobinuria: current view on a rare disease. Klinicheskaya onkogematologiya. Fundamental’nye issledovaniya i klinicheskaya praktika = Clinical Oncohematology. Basic Research and Clinical Practice 2019;12(1):4–20. (In Russ.).
2. Savchenko V.G., Lukina E.A., Mikhaylova E.A. et al. Clinical guidelines for the management of patients with paroxysmal nocturnal hemoglobinuria. Gematologiya i transfuziologiya = Russian Journal of Hematology and Transfusiology 2022;67(3): 426–39. (In Russ.). DOI: 10.35754/0234-5730-2022-67-3-426-439
3. Lisukov I.A., Kulagin A.D., Afanasyev B.V. Treatment of paroxysmal nocturnal hemoglobinuria. Onkogematologiya = Oncohematology 2012;(3):49–54. (In Russ.). URL: https://cyberleninka.ru/article/n/lechenie-paroksizmalnoy-nochnoy-gemoglobinurii?ysclid=lmhkknq4lm335167245
4. Gavriilaki E., Peffault de Latour R., Risitano A.M. Advancing therapeutic complement inhibition in hematologic diseases: PNH and beyond. Blood 2022;139(25):3571–82. DOI: 10.1182/blood.2021012860
5. Wong R.S.M. Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria. Ther Adv Hematol 2022;13:20406207221114670. DOI: 10.1177/20406207221114673
6. Hillmen P., Szer J., Weitz I. et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2021;384(11):1028–37. DOI: 10.1056/NEJMoa2029073
7. Kulagin A., Klimova O., Rudakova T. et al. Benefits and limitations of long-term eculizumab treatment for paroxysmal nocturnal hemoglobinuria (PNH): real-world data from large cohort study in Russia. Blood 2018;132(suppl 1):2589. DOI: 10.1182/blood-2018-99-120139
8. Instructions for use of eculizumab and ravulizumab. State Register of Medicinal Products. Available at: rosminzdrav.ru. (In Russ.).
9. Instructions for use of pegcetacoplan. State Register of Medicinal Products. Available at: rosminzdrav.ru. (In Russ.).
10. Mbaeyi S.A., Bozio C.H., Duffy J. et al. Meningococcal vaccination: recommendations of the advisory committee on immunization practices, United States, 2020. MMWR Recomm Rep 2020;69(9):1–41. DOI: 10.15585/mmwr.rr6909a1
11. www.gov.uk [Internet]. Public Health England. Immunisation of individuals with underlying medical conditions: the green book, chapter 7 [updated Jan 2020]. Available at: The Green book of immunisation – chapter 7 – Immunisation of immunocompromised individuals (https://pulsedive.com/ioc/publishing.service.gov.uk).
12. Meningococcal vaccines: WHO position paper, November 2011. Wkly Epidemiol Rec 2011;86(47):521–40.
13. Briko N.I., Namazova-Baranova L.S., Koroleva I.S. et al. Resolution of the Interdisciplinary Expert Council for prevention of severe infections in patients with genetic disorders of complement system regulation receiving eculizumab therapy. Epidemiologiya i vaktsinoprofilaktika = Epidemiology and Vaccinal Prevention 2017;16(1):51–4. (In Russ.).
Review
For citations:
Kulagin A.D., Lukina E.A., Ptushkin V.V., Kostinov M.P., Arshanskaya E.G., Babaeva T.N., Ksenzova T.I., Fidarova Z.T., Marchenko M.V. Resolution of the Expert council “Unsolved problems of targeted therapy for paroxysmal nocturnal hemoglobinuria in Russia”. Oncohematology. 2023;18(3):140-144. (In Russ.) https://doi.org/10.17650/1818-8346-2023-18-3-140-144