bloodjourГематология и трансфузиологияRussian journal of hematology and transfusiology0234-57302411-3042ООО Издательский дом «Практика»10.35754/0234-5730-2019-64-3-342-352bloodjour-152Research ArticleОБЗОРЫ ЛИТЕРАТУРЫREVIEWS OF LITERATUREНАЛИЧИЕ КЛОНА ПАРОКСИЗМАЛЬНОЙ НОЧНОЙ ГЕМОГЛОБИНУРИИ И ДРУГИЕ ФАКТОРЫ, ВЛИЯЮЩИЕ НА ЭФФЕКТИВНОСТЬ ИММУНОСУПРЕССИВНОЙ ТЕРАПИИ У БОЛЬНЫХ ИДИОПАТИЧЕСКОЙ АПЛАСТИЧЕСКОЙ АНЕМИЕЙCLONE OF PAROXYSMAL NOCTURNAL HAEMOGLOBINURIA AND OTHER PREDICTORS OF THE RESPONSE TO IMMUNOSUPPRESSIVE THERAPY IN PATIENTS WITH IDIOPATHIC APLASTIC ANAEMIAhttps://orcid.org/0000-0003-0934-6094ФидароваЗ. Т.FidarovaZ. T.

кандидат медицинских наук, заведующая отделением химиотерапии гемобластозов и депрессий кроветворения с дневным стационаром,

125167, Москва

Cand. Sci. (Med.), Head of the Department of Chemotherapy for Hemoblastoses and Hematopoiesis Depressions with a Day In-patient Facility,

125167, Moscow

zalinafidarova@mail.ruhttps://orcid.org/0000-0002-8113-6115АбрамоваА. В.AbramovaA. V.

врач отделения высокодозной интенсивной химиотерапии гемобластозов и депрессий кроветворения с круглосуточным стационаром,

125167, Москва

MD, Department of High-Dose Intensive Chemotherapy for Hemoblastoses and Haematopoiesis Depressions with a 24-hour In-patient facility, 

125167, Moscow

anastasia.abramova@list.ruhttps://orcid.org/0000-0002-4400-4711ЛучкинА. В.LuchkinA. V.

врач отделения высокодозной интенсивной химиотерапии гемобластозов и депрессий кроветворения с круглосуточным стационаром,

125167, Москва

MD, Department of High-Dose Intensive Chemotherapy for Hemoblastoses and Haematopoiesis Depressions with a 24-hour In-patient facility, 

125167, Moscow

a_luchkin@rambler.ruФГБУ «Национальный медицинский исследовательский центр гематологии» Министерства здравоохранения Российской ФедерацииРоссияNational Research Center for HematologyRussian Federation201909112019643342352Copyright © Фидарова З.Т., Абрамова А.В., Лучкин А.В., 20192019Фидарова З.Т., Абрамова А.В., Лучкин А.В.Fidarova Z.T., Abramova A.V., Luchkin A.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/152Введение

Введение. В основе патогенеза приобретенной апластической анемии (АА) лежит иммуноопосредованное развитие костномозговой недостаточности. Отсутствие однозначных причин развития иммунной агрессии делает актуальными исследования, направленные на изучение генетических нарушений в оставшемся пуле гемопоэтических стволовых клеток, в кроветворной нише, а также механизмов срыва иммунологической толерантности.

Цель настоящего обзора литературы — описание наиболее актуальных маркеров, позволяющих охарактеризовать больных АА в зависимости от возможного ответа на ИСТ и сформировать группы риска развития рефрактерности и клональной эволюции.

Основные сведения

Основные сведения. Вероятность общей выживаемости больных АА, которым проведена программная иммуносупрессивная терапия (ИСТ), сопоставима с результатами трансплантации аллогенных гемопоэтических стволовых клеток крови (алло-ТГСК) от родственного донора в первой линии терапии. Согласно современным отечественным и международным рекомендациям, выбор тактики лечения больных АА определяется возрастом больного и наличием HLA-идентичного сиблинга. Методом выбора лечения больных младше 40 лет является алло-ТГСК от родственного HLA-идентичного донора, но возможность проведения алло-ТГСК ограничена наличием донора. Несмотря на то что вероятность бессобытийной выживаемости при проведении ИСТ уступает результатам алло-ТГСК, для большинства больных АА ИСТ остается основным методом лечения. С целью минимизации неблагоприятных исходов необходимо учитывать наличие предикторов эффективности лечения и вероятность развития поздней клональной эволюции уже на этапе диагностики АА. Оценка и формирование групп риска больных позволит на этапе планирования выбрать оптимальный подход, включающий добавление к ИСТ агонистов тромбопоэтиновых рецепторов, или поиск неродственного HLA-совместимого донора и переход к алло-ТГСК в более ранние сроки. 

Introduction

Introduction. The pathogenesis of acquired aplastic anaemia (AA) is based on immune-mediated development of bone marrow failure. The absence of clear reasons for the development of immune aggression determines the relevance of investigations aimed at studying genetic disorders in the remaining pool of hematopoietic stem cells, in the hematopoietic niche, as well as mechanisms underlying the failure of immunological tolerance.

Aim

Aim. The present literature review describes the most relevant markers used for characterising AA patients on the basis of their possible response to immunosuppressive therapy (IT) and for forming groups being at risk of developing refractoriness and clonal evolution.

General findings

General findings. The overall survival probability in patients with AA following program IT is comparable to the results of transplanting allogeneic hematopoietic blood stem cells (allo-HSCT) from a related donor in the first line of therapy. According to current Russian and international recommendations, the tactics for treating AA patients is determined by the patient’s age and the presence of an HLA-identical sibling. Allo-HSCT from a related HLA-identical donor is a method used for treating patients younger than 40 years; however, the possibility of performing allo-HSCT is limited by donor availability. Although the event-free survival probability during IT is inferior to the results of allo-HSCT, IT remains the main treatment method for most patients with AA. In order to minimise adverse outcomes, it is necessary to consider predictors of treatment efficacy along with the likelihood of developing late clonal evolution as early as at the AA diagnosis stage. Patient evaluation and formation of risk groups will facilitate selection of the most optimal treatment approach at the therapy planning stage, which includes either IT combination with thrombopoietin receptor agonists, or a search for an unrelated HLA-compatible donor and timely allo-HSCT. 

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