bloodjourГематология и трансфузиологияRussian journal of hematology and transfusiology0234-57302411-3042ООО Издательский дом «Практика»10.35754/0234-5730-2022-68-4-443-455bloodjour-486Research ArticleОРИГИНАЛЬНЫЕ СТАТЬИORIGINAL ARTICLESВрожденная тромботическая тромбоцитопеническая пурпура у детейCongenital thrombotic thrombocytopenic purpura in childrenhttps://orcid.org/0000-0003-1814-6772ШутоваА. Д.ShutovaA. D.

Шутова Александра Дмитриевна, гематолог отделения трансплантации гемопоэтических клеток № 2

117998

Москва

Alexandra D. Shutova, Hematologist, Department of Haematopoietic stemcell transplantation No. 2

117198

Moscow

alexandra.shutova@fccho-moscow.ruhttps://orcid.org/0000-0002-0813-5626КалининаИ. И.KalininaI. I.

Калинина Ирина Игоревна, кандидат медицинских наук, гематолог отделения детской гематологии/онкологии

117998

Москва

Irina I. Kalinina, Cand. Sci. (Med.), Hematologist, Department of Pediatric Hematology/Oncology

117198

Moscow

burbir@mail.ruhttps://orcid.org/0000-0001-8404-1800СунцоваЕ. В.SuntsovaE. V.

Сунцова Елена Викторовна, научный сотрудник отдела депрессий кроветворения, миелоидных лейкозов, редких и наследственных болезней

117998

Москва

Elena V. Suntsova, Researcher, Department of hematopoiesis depressions, myeloidleukemias, rare and inherited diseases

117198

Moscow

elena.suncova@fccho-moscow.ruhttps://orcid.org/0000-0002-7634-2053РайкинаЕ. В.RaikinaE. V.

Райкина Елена Владиславовна, кандидат медицинских наук, заведующая лабораторией молекулярной биологии

117998

Москва

Elena V. Raikina, Cand. Sci. (Med.), Head of the Molecular Biology Laboratory

117198

Moscow

Elena.Raykina@fccho-moscow.ruhttps://orcid.org/0000-0001-8818-8949ГалстянГ. М.GalstyanG. M.

Галстян Геннадий Мартинович, доктор медицинских наук, заведующий отделением реанимации и интенсивной терапии

125167

Москва

Gennady M. Galstyan, Dr. Sci. (Med.), Head of the Resuscitation and Intensive Care Unit

125167

Moscow

gengalst@gmail.comhttps://orcid.org/0000-0002-8805-1499СметанинаН. С.SmetaninaN. S.

Сметанина Наталья Сергеевна, доктор медицинских наук, профессор, заместитель директора Института гематологии, иммунологии и клеточныхтехнологий

117998

Москва

Nataliya S. Smetanina, Dr. Sci. (Med.), Professor, Deputy Director of the Institute of Hematology, Immunology and Cell Technologies

117198

Moscow

nataliya.smetanina@fnkc.ruhttps://orcid.org/0000-0002-0016-6698МасчанА. А.MaschanA. A.

Масчан Алексей Александрович, доктор медицинских наук, профессор, член-корр. РАН, заместитель генерального директора по научно-клинической работе

117998

Москва

Alexei A. Maschan, Dr. Sci. (Med.), Professor, corresponding member of the RAS, Deputy Director

117198

Moscow

Aleksey.Maschan@fccho-moscow.ruФГБУ «Национальный медицинский исследовательский центр детской гематологии, онкологии и иммунологии им. Дмитрия Рогачева» Министерства здравоохранения Российской ФедерацииРоссияDmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and ImmunologyRussian FederationФГБУ «Национальный медицинский исследовательский центр гематологии» Министерства здравоохранения Российской ФедерацииРоссияNational Medical Research Center for HematologyRussian Federation202328122023684443455Copyright © Шутова А.Д., Калинина И.И., Сунцова Е.В., Райкина Е.В., Галстян Г.М., Сметанина Н.С., Масчан А.А., 20232023Шутова А.Д., Калинина И.И., Сунцова Е.В., Райкина Е.В., Галстян Г.М., Сметанина Н.С., Масчан А.А.Shutova A.D., Kalinina I.I., Suntsova E.V., Raikina E.V., Galstyan G.M., Smetanina N.S., Maschan A.A.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.htjournal.ru/jour/article/view/486

Введение. Врожденная тромботическая тромбоцитопеническая пурпура (вТТП) — это редкое жизнеугрожающее генетически обусловленное заболевание, характеризующееся микроангиопатической гемолитической анемией, тромбоцитопенией и ишемическими поражениями внутренних органов вследствие дефицита или снижения активности фермента ADAMTS13. Диагностика заболевания сложна ввиду широкого круга дифференциальных диагнозов, схожих с ТТП.Цель: анализ клинических проявлений и результатов лечения выявленных случаев вТТП. Материалы и методы. Приведен анализ клинических наблюдений 11 больных вТТП, подтвержденной генетически или характерной клиническо-лабораторной картиной.Результаты. Клинические и лабораторные признаки ТТП были вариабельны и неспецифичны. Триггерным фактором выступала перенесенная острая респираторная вирусная инфекция, а стабилизация заболевания наступала после трансфузий компонентов крови. Основным подтверждающим диагноз методом являлось молекулярно-генетическое исследование, которое было проведено у 8 больных. Всем больным с установленным диагнозам вТТП проводили терапию плазмой с эффектом.Заключение. Ранняя диагностика вТТП затруднительна вследствие отсутствия специфических клинических проявлений и легкодоступных лабораторных тестов

Introduction. Congenital thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening genetic disorder characterized by recurrent episodes of microangiopathic hemolytic anemia, thrombocytopenia, and organ dysfunction due to the defi ciency or dysfunction of the ADAMTS13 enzyme. Despite the availability of clinical and laboratory diagnostic criteria the diagnosis remains challenging due to a wide range of diseases with similar appearance.Aim: to analyze the clinical manifestations and treatment results of identifi ed cases of congenital TTP at the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology.Methods. This article reviews 11 clinical cases with congenital TTP genetically confi rmed or with typical clinical and laboratory signs.Results. Clinical and laboratory signs of TTP are highly variable and nonspecifi c, which was observed in the described cohort of patients. One of the trigger factors is often an acute respiratory viral infection, and stabilization of the disease can occur after transfusions of blood components. A delay of diagnosis can often lead not only to a delay of correct therapy, but also to the prescription of wrong therapy. The main diagnosis confi rmatory method was a molecular genetic test, which was carried out in 8 cases. All patients diagnosed with congenital TTP were successfully treated with FFP therapy.Conclusion. Early diagnosis of TTP is diffi cult due to the lack of specifi c clinical manifestations and easily accessible laboratory tests

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The authors declare that there are no conflicts of interest present.