Pediatric Hematology/Oncology and ImmunopathologyPediatric Hematology/Oncology and ImmunopathologyВопросы гематологии/онкологии и иммунопатологии в педиатрии1726-17082414-9314Fund Doctors, Innovations, Science for Children44510.24287/1726-1708-2020-19-4suppl-39-49MATERIALS OF THE 3rd WORKING MEETING OF ALLERGISTS-IMMUNOLOGISTSМАТЕРИАЛЫ III РАБОЧЕГО СОВЕЩАНИЯ АЛЛЕРГОЛОГОВ-ИММУНОЛОГОВThe efficacy and safety of romiplostim in the treatment of thrombocytopenia in pediatric patients with Wiskott–Aldrich syndrome: the results of a retrospective studyБезопасность и эффективность ромиплостима в лечении тромбоцитопении у детей с синдромом Вискотта–Олдрича: результаты ретроспективного исследованияhttps://orcid.org/0000-0002-8697-4206KhorevaA. L.ХореваА. Л.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0003-2335-0735AbramovaI. N.АбрамоваИ. Н.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0002-9083-4783DeripapaE. V.ДерипапаЕ. В.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0001-9857-4456RodinaYu. A.РодинаЮ. А.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0001-5132-1267RoppeltA. A.РоппельтА. А.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0003-1267-9957BurlakovV. I.БурлаковВ. И.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0002-6148-7209PershinD. E.ПершинД. Е.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0002-2128-0078LarinS. S.ЛаринС. С.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0002-7634-2053RaykinaE. V.РайкинаЕ. В.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

VarlamovaT. V.ВарламоваТ. В.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0003-2467-2840KievaA. M.КиеваА. М.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0001-7578-9657VoroninK. A.ВоронинК. А.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0002-0016-6698MaschanA. A.МасчанА. А.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0002-2322-5734NovichkovaG. A.НовичковаГ. А.
Russian Federation

1 Samory Mashela St., Moscow 117997, Russia

117997, Москва, ул. Саморы Машела, 1

https://orcid.org/0000-0002-3113-4939ShcherbinaA. Yu.ЩербинаА. Ю.
Russian Federation

MD, PhD, Professor, Deputy Director, Institute of Hematology, Immunology and Cellular Technologies; Chief of Immunology Department

1 Samory Mashela St., Moscow 117997, Russia

д-р мед. наук, профессор, заместитель директора Института гематологии, иммунологии и клеточных технологий, заведующая отделением иммунологии117997, Москва, ул. Саморы Машела, 1

shcher26@hotmail.comDmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian FederationФГБУ «Национальный медицинский исследовательский центр детской гематологии, онкологии и иммунологии им. Дмитрия Рогачева» Минздрава России11012021194supplementприложение39490801202108012021Copyright ©; 2021, «D. Rogachev NMRCPHOI»Copyright ©; 2021, ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава России2021«D. Rogachev NMRCPHOI»ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава Россииhttps://creativecommons.org/licenses/by/4.0https://hemoncim.com/jour/article/view/445

Reduction of haemorrhage and improving the quality of life for patients with Wiskott–Aldrich syndrome (WAS) before hematopoietic stem cell transplantation (HSCT) are the most important goals. We aimed to investigate the efficacy and safety of thrombopoietin receptor agonist (TPO-RA) in children with WAS. This study is supported by the Independent Ethics Committee and approved by the Academic Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. 67 patients with genetically confirmed WAS treated with subcutaneous romiplostim at the dose of 9 µg/kg weekly between 2012 and 2019 in Dmitry Rogachev National Center of Pediatric Hematology, Oncology, and Immunology were enrolled. The median treatment duration was 8 months (range, 1‒12). Overall, 22 patients (33%) achieved complete response and 18 (27%) partial. There were no apparent severe adverse effects associated with romiplostim administration. The only notable and possibly not related to the treatment was arterial thrombosis in one patient who had ongoing systemic vasculitis and a history of aortic aneurysm formation. The results of our study demonstrated that use of romiplostim was safe and effective in managing of thrombocytopenia and bleeding in most WAS patients.

Снижение риска развития жизнеугрожающих кровотечений и улучшение качества жизни пациентов с синдромом Вискотта–Олдрича (СВО) до проведения трансплантации гемопоэтических стволовых клеток является одной из главных задач ведения этих больных. Основной целью данной работы явилась оценка эффективности и безопасности терапии пациентов с СВО с использованием агониста рецептора тромбопоэтина ромиплостима, направленной на достижение нормального уровня тромбоцитов и снижение риска развития тяжелых кровотечений. Данное исследование одобрено независимым этическим комитетом и утверждено решением ученого совета ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава России. В исследование были включены 67 пациентов с генетически верифицированным диагнозом СВО, получавших терапию ромиплостимом в дозе 9 мкг/кг еженедельно в НМИЦ ДГОИ им. Дмитрия Рогачева в период с 2012 по 2019 г. Медиана длительности лечения составила 8 (1–12) мес. Полный ответ достигнут у 22 (33%) пациентов и частичный ответ – у 18 (27%). Тяжелых нежелательных явлений на фоне терапии ромиплостимом не отмечено. Единственным нежелательным явлением, возникшим на фоне проводимого лечения и, возможно, не связанным с ним, было развитие артериального тромбоза у пациента с системным васкулитом и аневризмой аорты в анамнезе. Применение ромиплостима у детей с СВО показало безопасность и высокую эффективность в коррекции тромбоцитопении и кровотечений у большинства пациентов.

Wiskott–Aldrich syndromeromiplostimthrombocytopeniahaemorrhagic syndromeсиндром Вискотта–Олдричаромиплостимтромбоцитопениягеморрагический синдром1. Sullivan K.E., Mullen C.A., Blaese R.M., Winkelstein J.A. A multiinstitutional survey of the Wiskott–Aldrich syndrome. J Pediatr 1994; 125: 876–885. DOI: 10.1016/s0022-3476(05)82002-5Sullivan K.E., Mullen C.A., Blaese R.M., Winkelstein J.A. A multiinstitutional survey of the Wiskott–Aldrich syndrome. J Pediatr 1994; 125: 876–885. DOI: 10.1016/s0022-3476(05)82002-52. Moratto D., Giliani S., Bonfim C., Mazzolari E., Fischer A., Ochs H.D., et al. Long-term outcome and lineage-specific chimerism in 194 patients with WiskottAldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. Blood 2011; 118: 1675–84. DOI: 10.1182/blood-2010-11-319376Moratto D., Giliani S., Bonfim C., Mazzolari E., Fischer A., Ochs H.D., et al. Long-term outcome and lineage-specific chimerism in 194 patients with WiskottAldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. Blood 2011; 118: 1675–84. DOI: 10.1182/blood-2010-11-3193763. Balashov D., Laberko A., Shcherbina A., Trakhtman P., Abramov D., Gutovskaya E., et al. Conditioning Regimen with Plerixafor Is Safe and Improves the Outcome of TCRab+ and CD19+ Cell-Depleted Stem Cell Transplantation in Patients with Wiskott–Aldrich Syndrome. Biol Blood Marrow Transplant 2018; 24 (7): 1432–40. DOI: 10.1016/j.bbmt.2018.03.006Balashov D., Laberko A., Shcherbina A., Trakhtman P., Abramov D., Gutovskaya E., et al. Conditioning Regimen with Plerixafor Is Safe and Improves the Outcome of TCRab+ and CD19+ Cell-Depleted Stem Cell Transplantation in Patients with Wiskott–Aldrich Syndrome. Biol Blood Marrow Transplant 2018; 24 (7): 1432–40. DOI: 10.1016/j.bbmt.2018.03.0064. Ferrua F., Marangoni F., Aiuti A., Roncarolo M.G. Gene therapy for WiskottAldrich syndrome: History, new vectors, future directions. J Allergy Clin Immunol 2020; 146 (2): 262–5. DOI: 10.1016/j.jaci.2020.06.018Ferrua F., Marangoni F., Aiuti A., Roncarolo M.G. Gene therapy for WiskottAldrich syndrome: History, new vectors, future directions. J Allergy Clin Immunol 2020; 146 (2): 262–5. DOI: 10.1016/j.jaci.2020.06.0185. Rivers E., Worth A., Thrasher A.J., Burns S.O. Bleeding and splenectomy in Wiskott–Aldrich syndrome: A single-centre experience. J Allergy Clin Immunol Pract 2019; 7: 1042–4. DOI: 10.1016/j.jaip.2018.07.009Rivers E., Worth A., Thrasher A.J., Burns S.O. Bleeding and splenectomy in Wiskott–Aldrich syndrome: A single-centre experience. J Allergy Clin Immunol Pract 2019; 7: 1042–4. DOI: 10.1016/j.jaip.2018.07.0096. Lengline E., Drenou B., Peterlin P., Tournilhac O., Abraham J., Berceanu A., et al. Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia. Haematologica 2018; 103 (2): 212–20. DOI: 10.3324/haematol.2017.176339Lengline E., Drenou B., Peterlin P., Tournilhac O., Abraham J., Berceanu A., et al. Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia. Haematologica 2018; 103 (2): 212–20. DOI: 10.3324/haematol.2017.1763397. Giordano P., Lassandro G., Barone A., Cesaro S., Fotzi I., Giona F., et al. Use of Eltrombopag in Children with Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP). Front Med (Lausanne) 2020; 7: 66. DOI: 10.3389/fmed.2020.00066Giordano P., Lassandro G., Barone A., Cesaro S., Fotzi I., Giona F., et al. Use of Eltrombopag in Children with Chronic Immune Thrombocytopenia (ITP): A Real Life Retrospective Multicenter Experience of the Italian Association of Pediatric Hematology and Oncology (AIEOP). Front Med (Lausanne) 2020; 7: 66. DOI: 10.3389/fmed.2020.000668. Bussel J.B., de Miguel P.G., Despotovic J.M., Grainger J.D., Sevilla J., Blanchette V.S., et al. Eltrombopag for the treatment of children with persistent and chronic immune thrombocytopenia (PETIT): a randomised, multicentre, placebo-controlled study. Lancet Haematol 2015; 2 (8): e315–25. DOI: 10.1016/S2352-3026(15)00114-3Bussel J.B., de Miguel P.G., Despotovic J.M., Grainger J.D., Sevilla J., Blanchette V.S., et al. Eltrombopag for the treatment of children with persistent and chronic immune thrombocytopenia (PETIT): a randomised, multicentre, placebo-controlled study. Lancet Haematol 2015; 2 (8): e315–25. DOI: 10.1016/S2352-3026(15)00114-39. Townsley D.M., Scheinberg P., Winkler T., Desmond R., Dumitriu B., Rios O., et al. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. N Engl J Med 2017; 376 (16): 1540–50. DOI: 10.1056/NEJMoa1613878Townsley D.M., Scheinberg P., Winkler T., Desmond R., Dumitriu B., Rios O., et al. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. N Engl J Med 2017; 376 (16): 1540–50. DOI: 10.1056/NEJMoa161387810. Erickson-Miller C.L., Delorme E., Tian S.S., Hopson C.B., Landis A.J., Valoret E.I., et al. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells 2009; 27 (2): 424–30. DOI: 10.1634/stemcells.2008-0366Erickson-Miller C.L., Delorme E., Tian S.S., Hopson C.B., Landis A.J., Valoret E.I., et al. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells 2009; 27 (2): 424–30. DOI: 10.1634/stemcells.2008-036611. Wang B., Nichol J.L., Sullivan J.T. Pharmacodynamics and pharmacokinetics of AMG 531, a novel thrombopoietin receptor ligand. Clin Pharmacol Ther 2004; 76 (6): 628–38. DOI: 10.1016/j.clpt.2004.08.010Wang B., Nichol J.L., Sullivan J.T. Pharmacodynamics and pharmacokinetics of AMG 531, a novel thrombopoietin receptor ligand. Clin Pharmacol Ther 2004; 76 (6): 628–38. DOI: 10.1016/j.clpt.2004.08.01012. Al-Samkari H., Kuter D.J. Optimal use of thrombopoietin receptor agonists in immune thrombocytopenia. Ther Adv Hematol 2019; 10: 2040620719841735. DOI: 10.1177/2040620719841735Al-Samkari H., Kuter D.J. Optimal use of thrombopoietin receptor agonists in immune thrombocytopenia. Ther Adv Hematol 2019; 10: 2040620719841735. DOI: 10.1177/204062071984173513. Gerrits A.J., Leven E.A., Frelinger A.L. III, Brigstocke S.L., Berny-Lang M.A., Mitchell W.B., et al. Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia. Blood. 2015; 126 (10): 1367–78. DOI: 10.1182/blood-2014-09-602573Gerrits A.J., Leven E.A., Frelinger A.L. III, Brigstocke S.L., Berny-Lang M.A., Mitchell W.B., et al. Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia. Blood. 2015; 126 (10): 1367–78. DOI: 10.1182/blood-2014-09-60257314. Zaninetti C., Gresele P., Bertomoro A., Klersy C., De Candia E., Veneri D., et al. Eltrombopag for the treatment of inherited thrombocytopenias: a phase II clinical trial. Haematologica 2020; 105: 820–8. DOI: 10.3324/haematol.2019.223966Zaninetti C., Gresele P., Bertomoro A., Klersy C., De Candia E., Veneri D., et al. Eltrombopag for the treatment of inherited thrombocytopenias: a phase II clinical trial. Haematologica 2020; 105: 820–8. DOI: 10.3324/haematol.2019.22396615. Першин Д.Е., Лодоева О.Б., Фадеева М.С., Мерсиянова И.В., Хорева А.Л., Владимиров И.С. и др. Разработка метода диагностики синдрома Вискотта–Олдрича путем оценки экспрессии белка WASP с использованием проточной цитофлуориметрии. Вопросы гематологии/онкологии иПершин Д.Е., Лодоева О.Б., Фадеева М.С., Мерсиянова И.В., Хорева А.Л., Владимиров И.С. и др. Разработка метода диагностики синдрома Вискотта–Олдрича путем оценки экспрессии белка WASP с использованием проточной цитофлуориметрии. Вопросы гематологии/онкологии ииммунопатологии в педиатрии 2020; 19 (2): 141-151. DOI: 10.24287/1726-1708-2020-19-2-141-15116. Zhu Q., Watanabe C., Liu T., Hollenbaugh D., Blaese R.M., Kanner S.B., et al. Wiskott–Aldrich syndrome/X-linked thrombocytopenia: WASP gene mutations, protein expression, and phenotype. Blood 1997; 90: 2680–9.Zhu Q., Watanabe C., Liu T., Hollenbaugh D., Blaese R.M., Kanner S.B., et al. Wiskott–Aldrich syndrome/X-linked thrombocytopenia: WASP gene mutations, protein expression, and phenotype. Blood 1997; 90: 2680–9.17. Kaufman R.M., Djulbegovic B., Gernsheimer T., Kleinman S., Tinmouth A.T., Capocelli K.E., et al. Platelet transfusion: a clinical practice guideline from the AABB. Ann Intern Med 2015; 162 (3): 205–13. DOI: 10.7326/M14-1589Kaufman R.M., Djulbegovic B., Gernsheimer T., Kleinman S., Tinmouth A.T., Capocelli K.E., et al. Platelet transfusion: a clinical practice guideline from the AABB. Ann Intern Med 2015; 162 (3): 205–13. DOI: 10.7326/M14-158918. Moiseeva A., Mersiyanova I., Hachatrian L., Karelin A., Kovrigin S., Pershin D., et al. Wiskott–Aldrich syndrome in a girl with skewed X-chromosome inactivation. ESID 2018 Meeting abstracts.Moiseeva A., Mersiyanova I., Hachatrian L., Karelin A., Kovrigin S., Pershin D., et al. Wiskott–Aldrich syndrome in a girl with skewed X-chromosome inactivation. ESID 2018 Meeting abstracts.19. Saleh M.N., Bussel J.B., Cheng G., Meyer O., Bailey C.K., Arning M., et al. Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open- label EXTEND study. Blood 2013; 121: 537–45. DOI: 10.1182/blood-2012-04-425512Saleh M.N., Bussel J.B., Cheng G., Meyer O., Bailey C.K., Arning M., et al. Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open- label EXTEND study. Blood 2013; 121: 537–45. DOI: 10.1182/blood-2012-04-42551220. Cheng G., Saleh M.N., Marcher C., Vasey S., Mayer B., Aivado M., et al. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study. Lancet 2011; 377 (9763): 393–402. DOI: 10.1016/S0140-6736(10)60959-2Cheng G., Saleh M.N., Marcher C., Vasey S., Mayer B., Aivado M., et al. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study. Lancet 2011; 377 (9763): 393–402. DOI: 10.1016/S0140-6736(10)60959-221. Grace R.F., Shimano K.A., Bhat R., Neunert C., Bussel J.B., Klaassen R.J., et al. Second-line treatments in children with immune thrombocytopenia: effect on platelet count and patient-centered outcomes. Am J Hematol 2019; 94: 741–50. DOI: 10.1002/ajh.25479Grace R.F., Shimano K.A., Bhat R., Neunert C., Bussel J.B., Klaassen R.J., et al. Second-line treatments in children with immune thrombocytopenia: effect on platelet count and patient-centered outcomes. Am J Hematol 2019; 94: 741–50. DOI: 10.1002/ajh.2547922. Kajiwara M., Nonoyama S., Eguchi M., Morio T., Imai K., Okawa H., et al. WASP is involved in proliferation and differentiation of human haemopoietic progenitors in vitro. Br J Haematol 1999; 107 (2): 254–62. DOI: 10.1046/j.1365-2141.1999.01694.xKajiwara M., Nonoyama S., Eguchi M., Morio T., Imai K., Okawa H., et al. WASP is involved in proliferation and differentiation of human haemopoietic progenitors in vitro. Br J Haematol 1999; 107 (2): 254–62. DOI: 10.1046/j.1365-2141.1999.01694.x23. Sabri S., Foudi A., Boukour S., Franc B., Charrier S., Jandrot-Perrus M., et al. Deficiency in the Wiskott–Aldrich protein induces premature proplatelet formation and platelet production in the bone marrow compartment. Blood 2006; 108: 134–40. DOI: 10.1182/blood-2005-03-1219Sabri S., Foudi A., Boukour S., Franc B., Charrier S., Jandrot-Perrus M., et al. Deficiency in the Wiskott–Aldrich protein induces premature proplatelet formation and platelet production in the bone marrow compartment. Blood 2006; 108: 134–40. DOI: 10.1182/blood-2005-03-121924. Ingrungruanglert P., Amarinthnukrowh P., Rungsiwiwut R., Maneesri-le Grand S., Sosothikul D., Suphapeetiporn K., et al. Wiskott–Aldrich syndrome iPS cells produce megakaryocytes with defects in cytoskeletal rearrangement and proplatelet formation. Thromb Haemost 2015; 113: 792–805. DOI: 10.1160/TH14-06-0503Ingrungruanglert P., Amarinthnukrowh P., Rungsiwiwut R., Maneesri-le Grand S., Sosothikul D., Suphapeetiporn K., et al. Wiskott–Aldrich syndrome iPS cells produce megakaryocytes with defects in cytoskeletal rearrangement and proplatelet formation. Thromb Haemost 2015; 113: 792–805. DOI: 10.1160/TH14-06-050325. Gröttum K.A., Hovig T., Holmsen H., Abrahamsen A.F., Jeremic M., Seip M. Wiskott–Aldrich syndrome: qualitative platelet defects and short platelet survival. Br J Haematol 1969; 17: 373–88. DOI: 10.1111/j.1365-2141.1969.tb01383.xGröttum K.A., Hovig T., Holmsen H., Abrahamsen A.F., Jeremic M., Seip M. Wiskott–Aldrich syndrome: qualitative platelet defects and short platelet survival. Br J Haematol 1969; 17: 373–88. DOI: 10.1111/j.1365-2141.1969.tb01383.x26. Shcherbina A., Rosen F.S., Remold-O'Donnell E. Pathological events in platelets of Wiskott–Aldrich syndrome patients. BrShcherbina A., Rosen F.S., Remold-O'Donnell E. Pathological events in platelets of Wiskott–Aldrich syndrome patients. BrJ Haematol 1999; 106: 875–883. DOI: 10.1046/j.1365-2141.1999.01637.x27. Shcherbina A., Miki H., Kenney D.M., Rosen F.S., Takenawa T., Remold-O’Donnell E. WASP and N-WASP in human platelets differ in sensitivity to protease calpain. Blood 2001; 98: 2988–91. DOI: 10.1182/blood.v98.10.2988Shcherbina A., Miki H., Kenney D.M., Rosen F.S., Takenawa T., Remold-O’Donnell E. WASP and N-WASP in human platelets differ in sensitivity to protease calpain. Blood 2001; 98: 2988–91. DOI: 10.1182/blood.v98.10.298828. Prislovsky A., Marathe B., Hosni A., Bolen A.L., Nimmerjahn F., Jackson C.W., et al. Rapid platelet turnover in WASP(–) mice correlates with increased ex vivo phagocytosis of opsonized WASP(–) platelets. Exp Hematol 2008; 36: 609–23. DOI: 10.1016/j.exphem.2007.12.019Prislovsky A., Marathe B., Hosni A., Bolen A.L., Nimmerjahn F., Jackson C.W., et al. Rapid platelet turnover in WASP(–) mice correlates with increased ex vivo phagocytosis of opsonized WASP(–) platelets. Exp Hematol 2008; 36: 609–23. DOI: 10.1016/j.exphem.2007.12.01929. Prislovsky A., Zeng X., Sokolic R.A., Garabedian E.N., Anur P., Candotti F., et al. Platelets from WAS patients show an increased susceptibility to ex vivo phagocytosis. Platelets 2013; 24: 288–96. DOI: 10.3109/09537104.2012.693991Prislovsky A., Zeng X., Sokolic R.A., Garabedian E.N., Anur P., Candotti F., et al. Platelets from WAS patients show an increased susceptibility to ex vivo phagocytosis. Platelets 2013; 24: 288–96. DOI: 10.3109/09537104.2012.69399130. Prislovsky A., Strom T.S. Increased uptake by splenic red pulp macrophages contributes to rapid platelet turnover in WASP– mice. Exp Hematol 2013; 41: 789–98. DOI: 10.1016/j.exphem.2013.05.003Prislovsky A., Strom T.S. Increased uptake by splenic red pulp macrophages contributes to rapid platelet turnover in WASP– mice. Exp Hematol 2013; 41: 789–98. DOI: 10.1016/j.exphem.2013.05.00331. Obydennyi S., Artemenko E., Sveshnikova A., Ignatova A.A., Varlamova T.V., Gambaryan S. et al. Mechanisms of increased mitochondria-dependent necrosis In Wiskott–Aldrich syndrome platelets. Haematologica 2020; 105: 1095–106. DOI: 10.3324/haematol.2018.214460Obydennyi S., Artemenko E., Sveshnikova A., Ignatova A.A., Varlamova T.V., Gambaryan S. et al. Mechanisms of increased mitochondria-dependent necrosis In Wiskott–Aldrich syndrome platelets. Haematologica 2020; 105: 1095–106. DOI: 10.3324/haematol.2018.21446032. Bussel J.B., Kuter D.J., Pullarkat V., Lyons R.M., Guo M., Nichol J.L. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood 2009; 113: 2161–71. DOI: 10.1182/blood-2008-04-150078Bussel J.B., Kuter D.J., Pullarkat V., Lyons R.M., Guo M., Nichol J.L. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood 2009; 113: 2161–71. DOI: 10.1182/blood-2008-04-15007833. Al-Samkari H., Kuter D.J. Immune Thrombocytopenia in Adults: Modern Approaches to Diagnosis and Treatment. Semin Thromb Hemost 2020; 46 (3): 275–88. DOI: 10.1055/s-0039-1700512Al-Samkari H., Kuter D.J. Immune Thrombocytopenia in Adults: Modern Approaches to Diagnosis and Treatment. Semin Thromb Hemost 2020; 46 (3): 275–88. DOI: 10.1055/s-0039-170051234. Kuter D.J., Rummel M., Boccia R., Macik B.G., Pabinger I., Selleslag D., et al. Romiplostim or standard of care in patients with immune thrombocytopenia. N Engl J Med 2010; 363 (20): 1889–99. DOI: 10.1056/NEJMoa1002625Kuter D.J., Rummel M., Boccia R., Macik B.G., Pabinger I., Selleslag D., et al. Romiplostim or standard of care in patients with immune thrombocytopenia. N Engl J Med 2010; 363 (20): 1889–99. DOI: 10.1056/NEJMoa100262535. Bussel J.B., Cheng G., Saleh M.N., Psaila B., Kovaleva L., Meddeb B., et al. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med 2007; 357 (22): 2237–47. DOI: 10.1056/NEJMoa073275Bussel J.B., Cheng G., Saleh M.N., Psaila B., Kovaleva L., Meddeb B., et al. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med 2007; 357 (22): 2237–47. DOI: 10.1056/NEJMoa07327536. Gernsheimer T.B., George J.N., Aledort L.M., Tarantino M.D., Sunkara U., Matthew Guo D., et al. Evaluation of bleeding and thrombotic events during long-term use of romiplostim in patients with chronic immune thrombocytopenia (ITP). J Thromb Haemost 2010; 8 (6): 1372–82. DOI: 10.1111/j.1538-7836.2010.03830.xGernsheimer T.B., George J.N., Aledort L.M., Tarantino M.D., Sunkara U., Matthew Guo D., et al. Evaluation of bleeding and thrombotic events during long-term use of romiplostim in patients with chronic immune thrombocytopenia (ITP). J Thromb Haemost 2010; 8 (6): 1372–82. DOI: 10.1111/j.1538-7836.2010.03830.x37. Al-Samkari H., Kuter D.J. Thrombopoietin level predicts response to treatment with eltrombopag and romiplostim in immune thrombocytopenia. Am J Hematol 2018; 93 (12): 1501–8. DOI: 10.1002/ajh.25275Al-Samkari H., Kuter D.J. Thrombopoietin level predicts response to treatment with eltrombopag and romiplostim in immune thrombocytopenia. Am J Hematol 2018; 93 (12): 1501–8. DOI: 10.1002/ajh.25275