bloodjourГематология и трансфузиологияRussian journal of hematology and transfusiology0234-57302411-3042ООО Издательский дом «Практика»10.25837/HAT.2018.39..2..010bloodjour-101Research ArticleКЛИНИЧЕСКИЕ НАБЛЮДЕНИЯCASE REPORTSВРОЖДЕННАЯ ФОРМА ТРОМБОТИЧЕСКОЙ ТРОМБОЦИТОПЕНИЧЕСКОЙ ПУРПУРЫ. КРАТКИЙ ОБЗОР И ОПИСАНИЕ КЛИНИЧЕСКОГО СЛУЧАЯCONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA. CASE REPORT AND REVIEWhttps://orcid.org/0000-0002-8712-9813НакастоевИ. М.NakastoevI. M.slep06@gmail.comhttps://orcid.org/0000-0002-4138-1589АвдонинП. П.AvdoninP. P.https://orcid.org/0000-0002-4155-7820ГрибановаE. О.GribanovaE. O.КалининН. Н.KalininN. N.https://orcid.org/0000-0002-0977-215XЦветаеваН. В.TsvetaevaN. V.ПетроваВ. И.PetrovaV. I.КалининаИ. И.KalininaI. I.https://orcid.org/0000-0001-6840-6152КанаеваМ. Л.KanaevaM. L.https://orcid.org/0000-0003-3034-4843ЖуравлевВ. В.ZhuravlevV. V.https://orcid.org/0000-0003-1098-8406БирюковаЛ. С.BiryukovaL. S.https://orcid.org/0000-0002-4138-1589АвдонинП. В.AvdoninP. V.https://orcid.org/0000-0003-1221-3495РыжкоB. В.RyzhkoV. V.ФГБУ «Национальный медицинский исследовательский центр гематологии» Минздрава России, МоскваРоссияNational Research Center for Hematology, MoscowRussian FederationИнститут биологии развития им. Н. К. Кольцова РАН, МоскваРоссияKoltsov Institute of Developmental Biology of the Russian Academy of Sciences, MoscowRussian FederationМНОЦ МГУ им. М. В. Ломоносова, МоскваРоссияMedical Research and Educational Center of the Lomonosov Moscow State University, MoscowRussian FederationНациональный медицинский исследовательский центр детской гематологии, онкологии и иммунологии имени Дмитрия Рогачева, МоскваРоссияDmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, MoscowRussian Federation201821042019632191199Copyright © Накастоев И.М., Авдонин П.П., Грибанова E.О., Калинин Н.Н., Цветаева Н.В., Петрова В.И., Калинина И.И., Канаева М.Л., Журавлев В.В., Бирюкова Л.С., Авдонин П.В., Рыжко B.В., 20192019Накастоев И.М., Авдонин П.П., Грибанова E.О., Калинин Н.Н., Цветаева Н.В., Петрова В.И., Калинина И.И., Канаева М.Л., Журавлев В.В., Бирюкова Л.С., Авдонин П.В., Рыжко B.В.Nakastoev I.M., Avdonin P.P., Gribanova E.O., Kalinin N.N., Tsvetaeva N.V., Petrova V.I., Kalinina I.I., Kanaeva M.L., Zhuravlev V.V., Biryukova L.S., Avdonin P.V., Ryzhko V.V.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/101

Резюме. Представлен клинический случай десятилетнего наблюдения пациента с тяжелой формой врожденной тромботической тромбоцитопенической пурпуры (ТТП), которому проводилась постоянная заместительная терапия переливаниями свежезамороженной плазмы (СЗП). При первом обследовании активность ADAMTS-13 в плазме крови пациента не выявлялась, содержание мультимеров фактора фон Виллебранда было повышено. В процессе лечения течение заболевания периодически осложнялось острой почечной недостаточностью и печеночной дисфункцией. Все осложнения возникали при удлинении интервалов между процедурами плазмафереза и носили частично обратимый характер. На том основании, что при обследовании не был обнаружен ингибитор ADAMTS-13, а симптомы заболевания появились в раннем детстве (петехии на коже при инфекциях), была установлена врожденная форма ТТП. После уточнения формы заболевания процедуры плазмафереза были заменены на плановое переливание СЗП. Оценивая динамику снижения числа тромбоцитов, определяли необходимые интервалы между трансфузиями. Исследовали динамику снижения активности ADAMTS-13 после трансфузии СЗП. В результате лечения на протяжении последних 6 лет тяжелых осложнений заболевания не было, пациент ведет активный образ жизни, учится и работает. За время наблюдения больному проведено более 150 трансфузий СЗП, перелито более 100 л СЗП. Несмотря на большой объем трансфузий донорской плазмы от многих доноров, у пациента не выявляются маркеры вирусного гепатита и ВИЧ. Таким образом, индивидуально спланированная заместительная терапия переливаниями СЗП больному с врожденной формой ТТП позволила избежать развития тяжелых осложнений, приводящих пациентов к инвалидности. При этом использование карантинизированной или вирусинактивированной СЗП значительно снижает риск инфицирования.

Summary. We present a ten-year follow-up of a patient with severe congenital thrombotic thrombocytopenic purpura (TTP), who received permanent replacement therapy with freshly frozen plasma (FFP) transfusions. At presentation, the patient had no activity of plasma ADAMTS-13 and an increased concentration of von Willebrand factor multimers. During treatment, the disease was complicated by occasional acute renal failure and hepatic dysfunction. These complications developed when the intervals between plasmapheresis were extended, and were partially reversible. Given the absense of ADAMTS-13 inhibitor and the early childhood history of the signs of the disease (petechiae during infections), the patient was diagnosed with congenital TTP. After the nature of the disease was thus established, plasmapheresis was replaced with regular transfusions of FFP. The intervals between transfusions were determined by monitoring the falling platelet counts and ADAMTS-13 activity. As a result of the treatment over the past 6 years, there have been no serious complications, and the patient leads an active lifestyle, studies and works. Over the course of the treatment, the patient received over 150 transfusions of FFP (amounting to over 100 liters of FFP), but despite the numerous transfusions, the patient has not contracted viral hepatitis or HIV. Thus, a tailored replacement therapy with FFP administered to a patient with congenital TTP helped to avoid severe debilitating complications, while the use of quarantine or virus-inactivated FFP minimized the risk of transfusionrelated infections.

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