Resolution of the Expert council “Unsolved problems of targeted therapy for paroxysmal nocturnal hemoglobinuria in Russia”

Author:

Kulagin A. D.1,Lukina E. A.2,Ptushkin V. V.3,Kostinov M. P.4,Arshanskaya E. G.5,Babaeva T. N.6,Ksenzova T. I.7,Fidarova Z. T.2,Marchenko M. V.1

Affiliation:

1. R. M. Gorbacheva Research Institute of Pediatric Oncology, Hematology and Transplantology of the First St. Petersburg State Medical University named after Academician I. P. Pavlova" Ministry of Health of Russia

2. Federal State Budgetary Institution "National Medical Research Center of Hematology" of the Ministry of Health of the Russian Federation

3. Department of Health of Moscow; GBUZ of Moscow "City Clinical Hospital named after S. P. Botkin of the Department of Health of Moscow"

4. I. I. Mechnikov Scientific Research Institute of Vaccines and Serums

5. Moscow City Hematology Center of the State Medical Institution of Moscow "S. P. Botkin City Clinical Hospital of the Moscow Department of Health"

6. Novosibirsk State Medical University of the Ministry of Health of the Russian Federation

7. GBUZ TO "Regional Clinical Hospital No. 1"

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.

Publisher

Publishing House ABV Press

Subject

Oncology,Hematology

Reference13 articles.

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

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