Лечение врожденной тромботической тромбоцитопенической пурпуры (синдрома Апшоу–Шульмана)

1. Scully M, Cataland S, Coppo P, et al. Consensus on the standardization of terminology in thrombotic thrombocytopenic purpura and related thrombotic microangiopathies. J Thromb Haemost. 2017;15(2):312-22. DOI:10.1111/jth.13571
2. Roose E, Tersteeg C, Demeersseman R, et al. Anti-ADAMTS13 Antibodies and a Novel Heterozygous p.R1177Q Mutation in a Case of Pregnancy-Onset Immune-Mediated Thrombotic Thrombocytopenic Purpura. TH Open. 2018;2(1):e8-15. DOI:10.1055/s-0037-1615252
3. Knöbl P. Thrombotic thrombocytopenic purpura. Memo. 2018;11(3):220‑6. DOI:10.1007/s12254-018-0429-6
4. Zheng X. Structure-function and regulation of ADAMTS13. J Thromb Haemost. 2013;11(Suppl. 1):11-23. DOI:10.1111/jth.12221
5. Галстян Г.М., Гапонова Т.В., Шерстнев Ф.С., и др. Клиническое использование криосупернатантной плазмы. Гематология и трансфузиология. 2020;65(3):351-9 [Galstyan GM, Gaponova TV, Sherstnev FS, et al. Clinical guidelines for cryosupernatant transfusions. Russian journal of hematology and transfusiology. 2020;65(3):351-9 (in Russian)]. DOI:10.35754/0234-5730-2020-65-3-351-359
6. Hori Y, Hayakawa M, Isonishi A, et al. ADAMTS13 unbound to larger von Willebrand factor multimers in cryosupernatant: Implications for selection of plasma preparations for thrombotic thrombocytopenic purpura treatment. Transfusion. 2013;53(12):3192-202. DOI:10.1111/trf.12182
7. Raife TJ, Friedman KD, Dwyre DM. The pathogenicity of von Willebrand factor in thrombotic thrombocytopenic purpura: reconsideration of treatment with cryopoor plasma Thomas. Transfusion. 2006;46:74-9. DOI:10.1111/j.1537-2995.2006.00674.x
8. Freedman M, Rock G. Analysis of the products of cryoprecipitation: RiCoF is deficient in cryosupernatant plasma. Transfus Apher Sci. 2010;43(2):179-82. DOI:10.1016/j.transci.2010.07.004
9. Von Baeyer H. Plasmapheresis in thrombotic microangiopathy-associated syndromes: review of outcome data derived from clinical trials and open studies. Ther Apher. 2002;6(4):320-8. DOI:10.1046/j.1526-0968.2002.00390.x
10. Altuntas F, Aydogdu I, Kabukcu S, et al. Therapeutic plasma exchange for the treatment of thrombotic thrombocytopenic purpura: A retrospective multicenter study. Transf Apher Sci. 2007;36(1):57-67. DOI:10.1016/j.transci.2006.05.014
11. Rock G, Anderson D, Clark W, et al. Does cryosupernatant plasma improve outcome in thrombotic thrombocytopenic purpura? No answer yet. Br J Haematol. 2005;129(1):79-86. DOI:10.1111/j.1365-2141.2005.05418.x
12. Peyvandi F, Mannucci PM, Valsecchi C, et al. ADAMTS13 content in plasma-derived factor VIII/von Willebrand factor concentrates. Am J Hematol. 2013;88(10):895-8. DOI:10.1002/ajh.23527
13. Scully M, Gattens M, Khair K, et al. The use of intermediate purity factor VIII concentrate BPL 8Y as prophylaxis and treatment in congenital thrombotic thrombocytopenic purpura. Br J Haematol. 2006;135(1):101‑4. DOI:10.1111/j.1365-2141.2006.06264.x
14. Naik S, Mahoney DH. Successful treatment of congenital ttp with a novel approach using plasma-derived factor VIII. J Pediat Hematol/Oncol. 2013;35(7):551-3. DOI:10.1097/MPH.0b013e3182755c38
15. Aledort LM, Singleton TC, Ulsh PJ. Treatment of Congenital Thrombotic Thrombocytopenia Purpura: A New Paradigm. J Pediatr Hematol Oncol. 2017;39(7):524-7. DOI:10.1097/MPH.0000000000000917
16. Zheng XL, Vesely SK, Cataland SR, et al. ISTH Guidelines for Treatment of Thrombotic Thrombocytopenic Purpura. J Thromb Haemost. 2020;18(10):2496-502. DOI:10.1111/jth.15010
17. Tersteeg C, Schiviz A, De Meyer SF, et al. Potential for Recombinant ADAMTS13 as an Effective Therapy for Acquired Thrombotic Thrombocytopenic Purpura. Arter Thromb Vasc Biol. 2015;35(11):2336‑42. DOI:10.1161/ATVBAHA.115.306014
18. Schiviz A, Wuersch K, Piskernik C, et al. A new mouse model mimicking thrombotic thrombocytopenic purpura: Correction of symptoms by recombinant human ADAMTS13. Blood. 2012;119(25):6128-35. DOI:10.1182/blood-2011-09-380535
19. Scully M, Knöbl P, Kentouche K, et al. A recombinant ADAMTS-13: First-in-human pharmacokinetics and safety in congenital thrombotic thrombocytopenic purpura. Blood. 2017;130(19):2055-63. DOI:10.1182/blood-2017-06-788026
20. FDA. FDA Fast Tracks Recombinant ADAMTS13 Enzyme Replacement Therapy. 2017.
21. ClinicalTrials.gov. A Study of Prophylactic and On-demand Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP) With BAX 930 (rADAMTS13) – Full Text View – ClinicalTrials.gov. 2017.
22. Nguyen TC, Han YY. Plasma exchange therapy for thrombotic microangiopathies. Organogenesis. 2011;7(1):28-31. DOI:10.4161/org.7.1.14027
23. Аксельрод Б.А., Балашова Е.Н., Баутин А.Е., и др. Клиническое использование эритроцитсодержащих компонентов донорской крови. Гематология и трансфузиология. 2018;63(4):372‑435 [Akselrod BA, Balashova EN, Bautin AE, et al. [Clinical guidelines for red blood cell transfusion]. Russian journal of hematology and transfusiology. 2018;63(4):372-435 (in Russian)]. DOI:10.25837/HAT.2019.62.39.006
24. Benhamou Y, Baudel JL, Wynckel A, et al. Are platelet transfusions harmful in acquired thrombotic thrombocytopenic purpura at the acute phase? Experience of the French thrombotic microangiopathies reference center. Am J Hematol. 2015;90(6):E127-9. DOI:10.1002/ajh.23997
25. Bobbio-Pallavicini E, Gugliotta L, Centurioni R, et al. Antiplatelet agents in thrombotic thrombocytopenic purpura (TTP). Results of a randomized multicenter trial by the Italian Cooperative Group for TTP. Haematologica. 1997;82(4):429-35.
26. Moatti-Cohen M, Garrec C, Wolf M, et al. Unexpected frequency of Upshaw-Schulman syndrome in pregnancy-onset thrombotic thrombocytopenic purpura. Blood. 2012;119(24):5888-97. DOI:10.1182/blood-2012-02-408914
27. Savignano C, Rinaldi C, de Angelis V. Pregnancy associated thrombotic thrombocytopenic purpura: Practical issues for patient management. Transfus Apher Sci. 2015;53(3):262-8. DOI:10.1016/j.transci.2015.11.005
28. Scully M, Thomas M, Underwood M, et al. Thrombotic thrombocytopenic purpura and pregnancy: Presentation, management, and subsequent pregnancy outcomes. Blood. 2014;124(2):211-9. DOI:10.1182/blood-2014-02-553131
29. Gangaraju R, Rodgers GM. Upshaw-Schulman syndrome and pregnancy: successful management with plasma infusions. Ann Hematol. 2016;95(10):1745-6. DOI:10.1007/s00277-016-2755-2
30. Kasht R, Borogovac A, George JN. Frequency and severity of pregnancy complications in women with hereditary thrombotic thrombocytopenic purpura. Ame J Hematol. 2020;95(11):E316-8. DOI:10.1002/ajh.25964
31. Joly BS, Stepanian A, Leblanc T, et al. Child-onset and adolescent-onset acquired thrombotic thrombocytopenic purpura with severe ADAMTS13 deficiency: a cohort study of the French national registry for thrombotic microangiopathy. Lancet Haematol. 2016. 
DOI:10.1016/S2352-3026(16)30125-9

________________________________________________

1. Scully M, Cataland S, Coppo P, et al. Consensus on the standardization of terminology in thrombotic thrombocytopenic purpura and related thrombotic microangiopathies. J Thromb Haemost. 2017;15(2):312-22. DOI:10.1111/jth.13571
2. Roose E, Tersteeg C, Demeersseman R, et al. Anti-ADAMTS13 Antibodies and a Novel Heterozygous p.R1177Q Mutation in a Case of Pregnancy-Onset Immune-Mediated Thrombotic Thrombocytopenic Purpura. TH Open. 2018;2(1):e8-15. DOI:10.1055/s-0037-1615252
3. Knöbl P. Thrombotic thrombocytopenic purpura. Memo. 2018;11(3):220‑6. DOI:10.1007/s12254-018-0429-6
4. Zheng X. Structure-function and regulation of ADAMTS13. J Thromb Haemost. 2013;11(Suppl. 1):11-23. DOI:10.1111/jth.12221
5. Galstyan GM, Gaponova TV, Sherstnev FS, et al. Clinical guidelines for cryosupernatant transfusions. Russian journal of hematology and transfusiology. 2020;65(3):351-9 (in Russian) DOI:10.35754/0234-5730-2020-65-3-351-359
6. Hori Y, Hayakawa M, Isonishi A, et al. ADAMTS13 unbound to larger von Willebrand factor multimers in cryosupernatant: Implications for selection of plasma preparations for thrombotic thrombocytopenic purpura treatment. Transfusion. 2013;53(12):3192-202. DOI:10.1111/trf.12182
7. Raife TJ, Friedman KD, Dwyre DM. The pathogenicity of von Willebrand factor in thrombotic thrombocytopenic purpura: reconsideration of treatment with cryopoor plasma Thomas. Transfusion. 2006;46:74-9. DOI:10.1111/j.1537-2995.2006.00674.x
8. Freedman M, Rock G. Analysis of the products of cryoprecipitation: RiCoF is deficient in cryosupernatant plasma. Transfus Apher Sci. 2010;43(2):179-82. DOI:10.1016/j.transci.2010.07.004
9. Von Baeyer H. Plasmapheresis in thrombotic microangiopathy-associated syndromes: review of outcome data derived from clinical trials and open studies. Ther Apher. 2002;6(4):320-8. DOI:10.1046/j.1526-0968.2002.00390.x
10. Altuntas F, Aydogdu I, Kabukcu S, et al. Therapeutic plasma exchange for the treatment of thrombotic thrombocytopenic purpura: A retrospective multicenter study. Transf Apher Sci. 2007;36(1):57-67. DOI:10.1016/j.transci.2006.05.014
11. Rock G, Anderson D, Clark W, et al. Does cryosupernatant plasma improve outcome in thrombotic thrombocytopenic purpura? No answer yet. Br J Haematol. 2005;129(1):79-86. DOI:10.1111/j.1365-2141.2005.05418.x
12. Peyvandi F, Mannucci PM, Valsecchi C, et al. ADAMTS13 content in plasma-derived factor VIII/von Willebrand factor concentrates. Am J Hematol. 2013;88(10):895-8. DOI:10.1002/ajh.23527
13. Scully M, Gattens M, Khair K, et al. The use of intermediate purity factor VIII concentrate BPL 8Y as prophylaxis and treatment in congenital thrombotic thrombocytopenic purpura. Br J Haematol. 2006;135(1):101‑4. DOI:10.1111/j.1365-2141.2006.06264.x
14. Naik S, Mahoney DH. Successful treatment of congenital ttp with a novel approach using plasma-derived factor VIII. J Pediat Hematol/Oncol. 2013;35(7):551-3. DOI:10.1097/MPH.0b013e3182755c38
15. Aledort LM, Singleton TC, Ulsh PJ. Treatment of Congenital Thrombotic Thrombocytopenia Purpura: A New Paradigm. J Pediatr Hematol Oncol. 2017;39(7):524-7. DOI:10.1097/MPH.0000000000000917
16. Zheng XL, Vesely SK, Cataland SR, et al. ISTH Guidelines for Treatment of Thrombotic Thrombocytopenic Purpura. J Thromb Haemost. 2020;18(10):2496-502. DOI:10.1111/jth.15010
17. Tersteeg C, Schiviz A, De Meyer SF, et al. Potential for Recombinant ADAMTS13 as an Effective Therapy for Acquired Thrombotic Thrombocytopenic Purpura. Arter Thromb Vasc Biol. 2015;35(11):2336‑42. DOI:10.1161/ATVBAHA.115.306014
18. Schiviz A, Wuersch K, Piskernik C, et al. A new mouse model mimicking thrombotic thrombocytopenic purpura: Correction of symptoms by recombinant human ADAMTS13. Blood. 2012;119(25):6128-35. DOI:10.1182/blood-2011-09-380535
19. Scully M, Knöbl P, Kentouche K, et al. A recombinant ADAMTS-13: First-in-human pharmacokinetics and safety in congenital thrombotic thrombocytopenic purpura. Blood. 2017;130(19):2055-63. DOI:10.1182/blood-2017-06-788026
20. FDA. FDA Fast Tracks Recombinant ADAMTS13 Enzyme Replacement Therapy. 2017.
21. ClinicalTrials.gov. A Study of Prophylactic and On-demand Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP) With BAX 930 (rADAMTS13) – Full Text View – ClinicalTrials.gov. 2017.
22. Nguyen TC, Han YY. Plasma exchange therapy for thrombotic microangiopathies. Organogenesis. 2011;7(1):28-31. DOI:10.4161/org.7.1.14027
23. Clinical guidelines for red blood cell transfusion]. Russian journal of hematology and transfusiology. 2018;63(4):372-435 (in Russian) DOI:10.25837/HAT.2019.62.39.006
24. Benhamou Y, Baudel JL, Wynckel A, et al. Are platelet transfusions harmful in acquired thrombotic thrombocytopenic purpura at the acute phase? Experience of the French thrombotic microangiopathies reference center. Am J Hematol. 2015;90(6):E127-9. DOI:10.1002/ajh.23997
25. Bobbio-Pallavicini E, Gugliotta L, Centurioni R, et al. Antiplatelet agents in thrombotic thrombocytopenic purpura (TTP). Results of a randomized multicenter trial by the Italian Cooperative Group for TTP. Haematologica. 1997;82(4):429-35.
26. Moatti-Cohen M, Garrec C, Wolf M, et al. Unexpected frequency of Upshaw-Schulman syndrome in pregnancy-onset thrombotic thrombocytopenic purpura. Blood. 2012;119(24):5888-97. DOI:10.1182/blood-2012-02-408914
27. Savignano C, Rinaldi C, de Angelis V. Pregnancy associated thrombotic thrombocytopenic purpura: Practical issues for patient management. Transfus Apher Sci. 2015;53(3):262-8. DOI:10.1016/j.transci.2015.11.005
28. Scully M, Thomas M, Underwood M, et al. Thrombotic thrombocytopenic purpura and pregnancy: Presentation, management, and subsequent pregnancy outcomes. Blood. 2014;124(2):211-9. DOI:10.1182/blood-2014-02-553131
29. Gangaraju R, Rodgers GM. Upshaw-Schulman syndrome and pregnancy: successful management with plasma infusions. Ann Hematol. 2016;95(10):1745-6. DOI:10.1007/s00277-016-2755-2
30. Kasht R, Borogovac A, George JN. Frequency and severity of pregnancy complications in women with hereditary thrombotic thrombocytopenic purpura. Ame J Hematol. 2020;95(11):E316-8. DOI:10.1002/ajh.25964
31. Joly BS, Stepanian A, Leblanc T, et al. Child-onset and adolescent-onset acquired thrombotic thrombocytopenic purpura with severe ADAMTS13 deficiency: a cohort study of the French national registry for thrombotic microangiopathy. Lancet Haematol. 2016. 
DOI:10.1016/S2352-3026(16)30125-9

Г.М. Галстян*1, А.А. Масчан2, Е.Е. Клебанова1, И.И. Калинина2

1 ФГБУ «Национальный медицинский исследовательский центр гематологии» Минздрава России, Москва, Россия;
2 ФГБУ «Национальный медицинский исследовательский центр детской гематологии, онкологии и иммунологии им. Дмитрия Рогачева» Минздрава России, Москва, Россия
*gengalst@gmail.com

________________________________________________

Gennadii M. Galstyan*1, Alexei A. Maschan2, Elizaveta E. Klebanova1, Irina I. Kalinina2

1 National Research Center for Hematology, Moscow, Russia;
2 Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia
*gengalst@gmail.com