REVIEW
Ethical aspects of clinical trials of blood clotting factors in children with hemophilia
1 I.M. Sechenov First Moscow State Medical University (Sechenov University), Russia, Moscow
2 NRC Institute of Immunology FMBA of Russia
3 AO “GENERIUM”, Russia, Vladimir region, Volginsky
4 Morozovskaya Children’s City Clinical Hospital, Russia, Moscow
5 Odintsovo Regional Hospital of the Moscow region, Russia, Odintsovo
6 Yaroslavl State Medical University, Yaroslavl, Russia
7 Samara State Medical University, Samara, Russia
Correspondence should be addressed: Anton Yu. Borozinets
ul. Testovskaya, d.10, pod. 2, Moscow, 123112, Russia; ur.muireneg@stenizorob.a
Authors contribution: Kudlay DA — article design development, analysis of scientific material, review of publications on the topic of the article, article editing; Vdovin VV — literary editing of the article, scientific editing of the article; Schiller EE — analysis of scientific material, review of publications on the topic of the article, editing of the article; Khokhlov AL — literary editing; scientific editing of the article; Davydkin IL — literary editing, scientific editing of the article, summary writing; Borozinets AYu — development of article design, analysis of scientific material, review of publications on the topic of the article, compilation of a summary, writing the text of the article, preparation of a list of references.
- Vol’skaya E. Novye perspektivy dlya detskikh lekarstv. Remedium. 2007; 8: 6–11. Russian.
- Zavidova SS, Namazova- Baranova LS, Topolyanskaya SV. Klinicheskie issledovaniya lekarstvennykh preparatov v pediatrii: problemy i dostizheniya. Pediatricheskaya farmakologiya. 2010; 7 (1): 6–14. Russian.
- Mosikyan AA, Tomasheva AO, Galankin TL, Kolbin AS. Klinicheskie issledovaniya v pediatrii i neonatologii: uspekhi i prichiny neudach. Pediatricheskaya farmakologiya. 2016; 13 (3): 232–238. DOI: 10.15690/ pf.v13i3.1572. Russian.
- Vdovin VV, Shiller EE, Svirin PV, Petrov VYu, Lavrent’eva IN, Perina FG, Kudlay DA, Shuster AM, Borozinets AYu. Osobennosti klinicheskikh issledovaniy rekombinantnykh faktorov svertyvaniya krovi IX u detey s gemofiliey B. Pediatriya im. GN Speranskogo. 2020; 99(3): 168–175. Russian.
- Belousov YuB, Strachunskiy LS, Belousov DYu, Zubkov VV, Malaya IP, Gratsianskaya AN, Dorozhko OV, Popov AO. Voprosy etiki biomeditsinskikh issledovaniy v pediatrii. Farmateka. 2001; 5: 10–14. Russian.
- Federal’nyy zakon ot 12.04.2010 № 61-FZ (red. ot 02.06.2021) “Ob obrashchenii lekarstvennykh sredstv”. Rezhim dostupa: https://base.garant.ru/12174909/ Russian.
- Topolyanskaya SV. Obshchie metodologicheskie problemy provedeniya klinicheskikh issledovaniy u detey. Pediatricheskaya farmakologiya. 2010; 7 (5): 6–10.
- Khel’sinkskaya deklaratsiya Vsemirnoy Meditsinskoy Assotsiatsii. Eticheskie printsipy pri provedenii meditsinskikh issledovaniy s privlecheniem cheloveka. Meditsinskaya etika. 2014; 2(1): 3–6. Russian.
- Federal’nyy zakon ot 21.11.2011 № 323-FZ (red. ot 02.07.2021) “Ob obrashchenii lekarstvennykh sredstv”. Rezhim dostupa: https://base.garant.ru/12191967/ Russian.
- Melikhov OG. Klinicheskie issledovaniya. 3-e izd., dop. M.: Izdatel’stvo «Atmosfera», 2013; 200 s. Russian.
- Ling G, Luo P. Inherited bleeding disorders. Medicine. 2021; 49(4): 225–228. https://doi.org/10.1016/j.mpmed.2021.01.009
- Kuznik BI, Sturov VG, Levshin NYu, Maksimova OG, Kudlay DA. Gemorragicheskie i tromboticheskie zabolevaniya i sindromy u detey i podrostkov: Patogenez, klinika, diagnostika, terapiya i profilaktika. Novosibirsk: Nauka, 2018; 524 s. Russian.
- Rumyantsev AG, Maschan AA, Vdovin VV, Svirin PV. Federal’nye klinicheskie rekomendatsii po diagnostike i lecheniyu detey s gemofiliey A, gemofiliey V i bolezn’yu Villebranda u detey. M., 2015; 76 s. Russian.
- Ay C, Perschy L, Rejtö J, Kaider A, Pabinger I. Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a real-world setting. Annals of Hematology. 2020; 99: 2763–2771. https://doi.org/10.1007/s00277–020–04250–9
- Report on the Annual Global Survey 2019. World Federation of Hemophilia, 2020: 85. WWW document. URL: https://www1.wfh. org/publications/files/pdf-2045.pdf
- Guideline on the clinical investigation of recombinant and human plasma- derived factor VIII products // EMA/CHMP/ BPWP/144533/2009 rev.1. 2016: 22.
- Guideline on clinical investigation of recombinant and human plasma- derived factor IX products // EMA/CHMP/ BPWP/144552/2009. 2011: 20. WWW document. URL: https:// www.ema.europa.eu/en/documents/scientific- guideline/guideline- clinical-investigation-r ecombinant-human- plasma-derived-f actorix-products_en.pdf.
- Prikaz Minpromtorga Rossii ot 23.10.2009 № 965 “Ob utverzhdenii Strategii razvitiya farmatsevticheskoy promyshlennosti Rossiyskoy Federatsii na period do 2020 goda”. Rezhim dostupa: [Elektronnyy resurs]. URL: http://www.consultant.ru/cons/cgi/online. Russian.
- Gosudarstvennaya programma «Razvitie farmatsevticheskoy i meditsinskoy promyshlennosti» na 2013–2020 gody. Osnovnye dannye po gosudarstvennoy programme «Razvitie farmatsevticheskoy i meditsinskoy promyshlennosti» na 2013– 2020 gody. M.: Minpromtorg Rossii, 2014. Rezhim dostupa: [Elektronnyy resurs]. URL: http://minpromtorg.gov.ru/common/ upload/files/docs/MinProm_02.06.14.pdf. Russian.
- Kudlay DA. Razrabotka i primenenie otechestvennykh rekombinantnykh preparatov faktorov svertyvaniya krovi VII, VIII, IX u detey s gemofiliey A i B. Pediatriya. 2019; 98 (1): 9–17. Russian.
- Pravila nadlezhashchey klinicheskoy praktiki Evraziyskogo ekonomicheskogo soyuza. Utverzhdeny resheniem № 79 ot 3.11.2016. 232s. Rezhim dostupa: [Elektronnyy resurs]. URL: http://docs.cntd.ru/document/456026110. Russian.
- Pravila provedeniya issledovaniy biologicheskikh lekarstvennykh sredstv Evraziyskogo ekonomicheskogo soyuza. Utverzhdeny resheniem № 89 ot 3.11.2016. 714s. Rezhim dostupa: [Elektronnyy resurs]. URL: http://docs.cntd.ru/document/456026116. Russian.
- Ivanov R, Sekareva G, Kravtsova O, Kudlay D, Luk’yanov S, Tikhonova I, Demin A, Maksumova L, Nikitina I, Obukhov A, Zaytsev D, Stepanov A, Nosyreva M, Samsonov M. Pravila provedeniya issledovaniy bioanalogovykh lekarstvennykh sredstv (bioanalogov). Farmakokinetika i farmakodinamika. 2014; 1: 21–36. Russian.
- Zorenko VYu, Mishin GV, Severova TV, Kudlay DA, Borozinets AYu. Farmakokineticheskie svoystva, bezopasnost’ i perenosimost’ preparata Oktofaktor (rezul’taty I fazy klinicheskogo issledovaniya u bol’nykh gemofiliey A). Voprosy gematologii/onkologii i immunopatologii v pediatrii. 2013; 12(2): 30–37. Russian.
- Zorenko VYu, Mishin GV, Severova T, Kudlay DA, Borozinets AS. Pharmacokinetic properties, safety and tolerability of new b-domain deleted recombinant factor VIII (Octofactor) in patients with severe and moderately severe hemophilia A. Haemophilia. 2014. V. 20. № S3; p. 24.
- Davydkin IL, Andreeva TA, Zorenko VYu, Konstantinova VN, Zalepukhina OE, Klimova NI, Kurtov IV, Noskova MS, Gusyakova OA, Mishin GV, Severova TV, Shuster AM, Kudlay DA, Luk’yanov SV, Borozinets AYu. Effektivnost’ i bezopasnost’ preparata Oktofaktor pri profilakticheskom lechenii bol’nykh s tyazheloy i srednetyazheloy formoy gemofilii A (rezul’taty 1-y chasti klinicheskogo issledovaniya II–III fazy). Voprosy gematologii/ onkologii i immunopatologii v pediatrii. 2013; 12(3): 29–37. Russian.
- Andreeva TA, Zorenko VYu, Davydkin IL, Konstantinova VN, Zalepukhina OE, Klimova NI, Kurtov IV, Avdoshkina MS, Gusyakova OA, Mishin GV, Severova TV, Shuster AM, Kudlay DA, Luk’yanov SV, Borozinets AYu. Effektivnost’ i bezopasnost’ preparata Oktofaktor v lechenii bol’nykh s tyazheloy i srednetyazheloy formoy gemofilii A (rezul’taty 2-y chasti klinicheskogo issledovaniya II i III fazy). Voprosy gematologii/onkologii i immunopatologii v pediatrii. 2013; 12(4): 31–37. Russian.
- Vdovin VV, Shiller EE, Svirin PV, Petrov VYu, Perina FG, Shuster AM, Kudlay DA, Borozinets AYu. Osobennosti klinicheskikh issledovaniy rekombinantnykh faktorov svertyvaniya krovi VIII u detey s gemofiliey A. Pediatriya. 2019; 98 (6): 103–110. Russian.
- Vdovin VV, Andreeva TA, Davydkin IL, Svirin PV, Shiller EE, Petrov VYu, Lavrichenko IA, Klimova NI, Kim AV, Kurtov IV, Shamina MS, Gusyakova OA, Kalinina EV, Shuster AM, Kudlay DA. Effektivnost’ i bezopasnost’ preparata Oktofaktor pri profilakticheskom lechenii podrostkov s tyazheloy gemofiliey A. Rossiyskiy zhurnal detskoy gematologii i onkologii. 2017; 4 (1): 61–70. Russian.
- Shiller E, Vdovin V, Petrov V, Svirin P, Andreeva T, Lavrichenko I, Bullikh A, Koltunov I, Davydkin I, Kurtov I, Shuster A, Kudlay DA, Lukyanov S, Borozinets A, Nikitin E, Klykova E. Safety and efficacy of new moroctocog alfa drug (Octofactor) in prophylactic treatment in adolescent patients with severe and moderate hemophilia A. Blood. 2015; 126(23): 4703.
- Andreeva TA, Zorenko VYu, Davydkin IL, Konstantinova VN, Zalepukhina OE, Klimova NI, Kim AV, Mishin GV, Krechetova AV, Kurtov IV, Shamina MS, Gusyakova OA, Kurapova MV, Shuster AM, Kudlay DA. Effektivnost’i bezopasnost’ preparata Oktofaktor pri profilakticheskom lechenii u patsientov s tyazheloy gemofiliey A. Rossiyskiy zhurnal detskoy gematologii i onkologii. 2018; 5 (3): 60–73. Russian.
- Timofeeva MA, Andreeva TA, Vdovin VV, Mamaev AN, Davydkin IL, Lebedev VV, Zozulya NI, Chernov VM, Kopylov KG, Basalaeva LG, Lavrichenko IA, Krasheninnikova OA, Svirin PV, Petrov VYu, Lavrent’eva IN, Rumyantsev AA, Kurtov IV, Asekretova TV, Shkryabunova VV, Shiller EE, Kudlay DA, Gapchenko EV, Markova OA, Borozinets AYu. Rezul’taty otkrytogo mnogotsentrovogo prospektivnogo klinicheskogo issledovaniya effektivnosti, bezopasnosti i farmakokinetiki moroktokoga al’fa u detey v vozraste ot 6 do 12 let s gemofiliey A. Pediatriya im. GN Speranskogo. 2021; 100 (2): 236–245. Russian.
- Timofeeva MA, Lebedev VV, Plaksina OI, Zozulya NI, Chernov VM, Kopylov KG, Ivashkina EP. Asekretova TV, Farafonova SN, Shiller EE, Kudlay DA, Gapchenko EV, Markova OA, Borozinets AYu. Effektivnost’ i bezopasnost’ moroktokoga al’fa v otkrytom mnogotsentrovom prospektivnom klinicheskom issledovanii u detey ot 2 do 6 let s tyazheloy formoy gemofilii A. Pediatriya im. GN Speranskogo. 2021; 100 (6): 154–161. Russian.
- Zozulya NI, Yastrubenetskaya OI, Belyaeva SS, Potapkova VM, Davydkin IL, Kurtov IV, Shelekhova TV, Evzerova TV, Gladilina OG, Shcherbinina ON, Volkova SA, Maksimova AS, Arkhipova NV, Esef’eva NB, Smelyanskaya MG, Molostvova VZ, Kazankova TA, Kirillova EG, Chelnov VG, Pospelova TI, Babaeva TN, Mamaev AN, Momot AP, Kosinova MV, Shcherbakova NV, Leshina OA, Smirnova AS, Korolenko TS, Kuchin AA, Novoselov KP, Bulieva NB, Morozov SV, Perina FG, Kosacheva NB, Safuanova GSh, Galina GA, Yanturina NKh, Saitova AR, Botvinovskiy VE, Timofeeva MA, Noskova EV, Konyashina NI, Shiller EE, Shuster AM, Kudlay DA, Borozinets AYu. Rezul’taty nablyudatel’nogo issledovaniya effektivnosti i bezopasnosti preparata Oktofaktor. Rossiyskiy zhurnal detskoy gematologii i onkologii. 2019; 6 (2): 30–47. Russian.
- Vdovin VV, Kosinova MV, Kalinina EV, Timofeeva MA, Mustafina GN, Rogov AV, Aslanyan KS, Duryagina SN, Lemazina EN, Svirin PV, Petrov VYu, Lavrent’eva IN, Shcherbakova NV, Shiller EE, Kudlay DA, Gapchenko EV, Markova OA, Borozinets AYu. Spetsifika molekuly moroktokoga al’fa: rezul’taty prospektivnogo mnogotsentrovogo nablyudatel’nogo issledovaniya effektivnosti i bezopasnosti u podrostkov s gemofiliey A. Pediatriya im. GN Speranskogo. 2021; 100 (5): 159–169. Russian.