nodgoРоссийский журнал детской гематологии и онкологии (РЖДГиО)Russian Journal of Pediatric Hematology and Oncology2311-12672413-5496LTD “Graphica”10.17650/2311-1267-2018-5-3-23-35nodgo-403Research ArticleИЗБРАННЫЕ ЛЕКЦИИSELECTED LECTURESМиелодиспластический синдром у детейPediatric myelodysplastic syndromeАфанасьевБ. В.AfanasyevB. V.

Борис Владимирович Афанасьев.

197022, Санкт-Петербург, ул. Рентгена, 12

12 Rentgena St., Saint Petersburg, 197022

bmt-director@1spbgmu.ruЗубаровскаяЛ. С.ZubarovskayaL.

197022, Санкт-Петербург, ул. Рентгена, 12

12 Rentgena St., Saint Petersburg, 197022

Научно-исследовательский институт детской онкологии, гематологии и трансплантологии им. Р.М. Горбачевой ФГБОУ ВО «Первый Санкт-Петербургский государственный медицинский университет им. акад. И.П. Павлова» Минздрава РоссииРоссияRaisa Gorbacheva Memorial Research Institute of Children Oncology, Hematology and Transplantation, First Pavlov State Medical University of St. Petersburg, Ministry of Health of RussiaRussian Federation201814092018532335Copyright © Афанасьев Б.В., Зубаровская Л.С., 20182018Афанасьев Б.В., Зубаровская Л.С.Afanasyev B.V., Zubarovskaya L.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://journal.nodgo.org/jour/article/view/403

Миелодиспластический синдром (МДС) у детей – гетерогенная группа клональных состояний, возникающих, как правило, вследствие наследственных синдромов костномозговой недостаточности, приобретенной апластической анемии или генов предрасположенности. Среди них врожденные состояния, обусловленные мутациями RUNX1, ANKRD, GATA2, ETV6, SRP72, DDX41, являющиеся фактором развития фамильного МДС или острого миелоидного лейкоза. Ювенильный миеломоноцитарный лейкоз (ЮММЛ) также связан с наличием наследованных или соматических мутаций PTPN11, KRAS, NRAS, CBL, NF1. Патогенез этих состояний обеспечивается несколькими факторами – гиперметилированием, возникновением клонального гемопоэза/цитопении неопределенного значения, изменениями микроокружения костного мозга, длины теломер, иммунными механизмами. Аллогенная трансплантация гемопоэтических стволовых клеток является основным методом лечения МДС у детей и ЮММЛ, но необходимо учитывать особые показания при рефрактерной цитопении (частые инфекции, зависимость от переливаний крови) и ЮММЛ с мутацией CBL.

Pediatric myelodysplastic syndrome (MDS) are a heterogeneous group of clonal disorders often occur in the context of inherited bone marrow failure syndromes, acquired aplastic anemia or gene predisposition. Germ line syndromes predisposing individuals to develop familial MDS or acute myeloid leukemia have recently been identified – mutations in RUNX1, ANKRD, GATA2, ETV6, SRP72, DDX41. Juvenile myelomonocytic leukemia (JMML) occurs in context of inherited and somatic mutations PTPN11, KRAS, NRAS, CBL, NF1. In pathogenesis of these disorders there are a several factors – hypermethylation, clonal hematopoiesis/cytopenia of undetermined significance, disturbances of bone marrow microenvironment, telomeres, immune mechanisms. Allogeneic hematopoietic stem cell transplantation is the main method of MDS and JMML treatment but it is necessary to take into account special indications for refractory cytopenia (infections, dependence on blood transfusions) and be careful for JMML with CBL mutation.

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