ppharmПедиатрическая фармакологияPediatric pharmacology1727-57762500-3089Издательство «ПедиатрЪ»10.15690/pf.v11i4.1058ppharm-67Research ArticleЛЕКЦИЯLECTUREПАТОЛОГИЯ СЕРДЦА И СОСУДОВ У ДЕТЕЙ С МУКОПОЛИСАХАРИДОЗОМ I ТИПАCARDIOVASCULAR PATHOLOGY IN CHILDREN WITH TYPE I MUCOPOLYSACCHARIDOSISВашакмадзеН. Д.VashakmadzeN. D.nato-nato@yandex.ruНамазова-БарановаЛ. С.Namazova-BaranovaL. S.namazova@nczd.ruГеворкянА. К.GevorkyanA. K.nato-nato@yandex.ruКузенковаЛ. М.KuzenkovaL. M.nato-nato@yandex.ruБабайкинаМ. А.BabaikinaM. A.nato-nato@yandex.ruБерезневаН. А.BereznevaN. A.nato-nato@yandex.ruПодклетноваТ. В.PodkletnovaT. V.nato-nato@yandex.ruНаучный центр здоровья детей, Москва, Российская ФедерацияРоссияScientific Center of Children’s Health, Moscow, Russian FederationRussian FederationНаучный центр здоровья детей, Москва, Российская Федерация Первый Московский государственный медицинский университет им. И.М. Сеченова, Москва, Российская Федерация Российский национальный исследовательский медицинский университет им. Н.И. Пирогова, Москва, Российская ФедерацияРоссияScientific Center of Children’s Health, Moscow, Russian Federation Sechenov First Moscow State Medical University, Moscow, Russian Federation Pirogov Russian National Medical Research University, Moscow, Russian FederationRussian FederationНаучный центр здоровья детей, Москва, Российская Федерация Первый Московский государственный медицинский университет им. И.М. Сеченова, Москва, Российская ФедерацияРоссияScientific Center of Children’s Health, Moscow, Russian Federation Sechenov First Moscow State Medical University, Moscow, Russian FederationRussian Federation201408082014114Педиатрическая фармакология1622Copyright © Вашакмадзе Н.Д., Намазова-Баранова Л.С., Геворкян А.К., Кузенкова Л.М., Бабайкина М.А., Березнева Н.А., Подклетнова Т.В., 20142014Вашакмадзе Н.Д., Намазова-Баранова Л.С., Геворкян А.К., Кузенкова Л.М., Бабайкина М.А., Березнева Н.А., Подклетнова Т.В.Vashakmadze N.D., Namazova-Baranova L.S., Gevorkyan A.K., Kuzenkova L.M., Babaikina M.A., Berezneva N.A., Podkletnova T.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.pedpharma.ru/jour/article/view/67

В статье представлены современные литературные данные о частоте встречаемости, патогенезе, клинических проявлениях, диагностике и лечении жизнеугрожающих поражений сердечно-сосудистой системы при мукополисахаридозе (МПС) I типа. Мукополисахаридоз ― редкое генетически детерминированное заболевание, имеющее хроническое прогрессирующее течение с широким спектром клинических проявлений. Особая актуальность болезни обусловлена ее тяжелым инвалидизирующим течением, развитием большого числа осложнений со стороны разных органов и систем (вплоть до их недостаточности), трудностями диагностики, а также высоким риском летального исхода еще в детском возрасте. Распространенность и тяжесть сердечно-сосудистых нарушений у таких пациентов очень высока. В настоящее время существуют два метода лечения пациентов с МПС, основанных на патофизиологии данного заболевания: трансплантация гемопоэтических стволовых клеток, применяемая только у определенных пациентов, и ферментозаместительная терапия. Ранняя диагностика и своевременно начатое лечение позволяют улучшить состояние больных, облегчить течение заболевания, в том числе приостановить прогрессирование кардиологических нарушений.

 

The article present current published data on the incidence rate, pathogenesis, clinical manifestations, diagnosis and treatment oflife-threatening conditions of the cardiovascular system at type I mucopolysaccharidosis (MPS). Mucopolysaccharidosis is a raregenetically determined disease with chronic progressive course and wide range of clinical manifestations. Specific relevance of thedisease is justified by its severe incapacitating potential, development of a large number of complications of various organs and systems (up to failure), difficulty diagnosing and high risk of fatal outcomes as early as in childhood. Prevalence and severity of cardiovascular disorders in such patients is very high. Currently, there are two methods of treating MPS patients based on pathophysiology of this disease: transplantation of hemopoietic stem cells, which is used only in certain patients, and enzyme-replacement therapy. Early diagnosis and timely launched treatment help to improve condition of patients, attenuate the disease course, including termination of progression of cardiac disorders.

 

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