Pediatric Hematology/Oncology and ImmunopathologyPediatric Hematology/Oncology and ImmunopathologyВопросы гематологии/онкологии и иммунопатологии в педиатрии1726-17082414-9314Fund Doctors, Innovations, Science for Children24210.24287/1726-1708-2019-18-2-30-42ORIGINAL ARTICLESОРИГИНАЛЬНЫЕ СТАТЬИThe experience of hematopoietic stem cell transplantation in primary immunodeficiencies in the Russian Children's Clinical HospitalОпыт трансплантации гемопоэтических стволовых клеток при первичных иммунодефицитах в Российской детской клинической больницеhttps://orcid.org/0000-0003-2395-4045MachnevaE. B.МачневаЕ. Б.
Russian Federation

Elena B. Machneva, MD, hematologist at Department of Bone marrow transplantation.

117997, Moscow, Leninsky p-kt, 117.

Мачнева Елена Борисовна, канд. мед. наук, врач-гематолог отделения трансплантации костного мозга.

117997, Москва, Ленинский пр-кт, 117.

lena.machneva@yandex.ruhttps://orcid.org/0000-0003-4431-1444SkorobogatovaE. V.СкоробогатоваЕ. В.
Russian Federation
https://orcid.org/0000-0002-4569-657XPristanskovaE. A.ПристансковаE. А.
Russian Federation
https://orcid.org/0000-0002-2652-8642KonstantinovaV. V.КонстантиноваВ. В.
Russian Federation
https://orcid.org/0000-0003-4170-7152BuryaA. E.БуряА. Е.
Russian Federation
https://orcid.org/0000-0002-7531-6443OlkhovaL. V.ОльховаЛ. В.
Russian Federation
https://orcid.org/0000-0003-3797-5808SidorovaN. V.СидороваН. В.
Russian Federation
https://orcid.org/0000-0003-3211-6158MezentsevaA. V.МезенцеваА. В.
Russian Federation
https://orcid.org/0000-0002-2945-284XKirgizovK. I.КиргизовК. И.
Russian Federation
https://orcid.org/0000-0002-1587-3256BlagonravovaO. L.БлагонравоваО. Л.
Russian Federation
https://orcid.org/0000-0001-7034-1730NikolaevaYu. A.НиколаеваЮ. А.
Russian Federation
https://orcid.org/0000-0002-2136-3287FilinaO. A.ФилинаО. А.
Russian Federation
https://orcid.org/0000-0003-0575-2028VakhlyarskayaS. C.ВахлярскаяС. С.
Russian Federation
https://orcid.org/0000-0002-0566-053XSkvortsovaYu. V.СкворцоваЮ. В.
Russian Federation
https://orcid.org/0000-0003-4834-4075KondratenkoI. V.КондратенкоИ. В.
Russian Federation
https://orcid.org/0000-0002-0265-5778BologovA. A.БологовА. А.
Russian Federation
https://orcid.org/0000-0002-0016-6698MaschanA. A.МасчанА. А.
Russian Federation
Russian Children's Clinical Hospital of the Pirogov Russian National Research Medical University of the Ministry of Healthcare of the Russian FederationРоссийская детская клиническая больница ФГБОУ ВО «Российский национальный исследовательский медицинский университет им. Н.И. Пирогова» Минздрава РоссииDmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian FederationФГБУ «Национальный медицинский исследовательский центр детской гематологии, онкологии и иммунологии им. Дмитрия Рогачева» Минздрава России2906201918230422906201929062019Copyright ©; 2019, «D. Rogachev NMRCPHOI»Copyright ©; 2019, ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава России2019«D. Rogachev NMRCPHOI»ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава Россииhttps://creativecommons.org/licenses/by/4.0https://hemoncim.com/jour/article/view/242

Primary immunodeficiencies (PID) include a group of congenital diseases, many of which are associated with a high risk of developing life-threatening infectious and non-infectious complications. Many of PIDs require hematopoietic stem cell transplantation (HSCT), which can lead to a complete cure of the disease. The article presents more than 20 years of experience in conducting HSCT with PID in the Russian Children's Clinical Hospital for the period from 1997 to 2018. 88 HSCTs were performed in 80 patients (64 boys and 16 girls) with various PIDs: severe combined immune deficiency (SCID, n = 34), hemophagocytic lymphohistiocytosis (HLH, n = 12), chronic granulomatous disease (CGD, n = 11), Wiskott–Aldrich syndrome (WAS, n = 10), congenital agranulocytosis (n = 4), hyper IgM syndrome type 1 (n = 3), Nijmegen breakage syndrome (n = 2), lymphoproliferative syndrome (n = 2), Chediak–Higashi syndrome (n = 1), leukocyte adhesion deficiency (n = 1). Оverall survival (OS) and event-free survival (EFS) after HSCT with PID was 63.1% and 49.3%. OS after HSCT with SCID was 65.5%, EFS – 48.4%. The article presents the results of HSCT taking into account the type of HSCT, the source of hematopoietic stem cells (HSC) and the type of graft manipulation, conditioning regimen. Growth of positive results of HSCT in patients with PID in recent years is associated with the improvement of accompanying therapy (improving the quality of infection control, the introduction of new drugs for the prevention and treatment of hepatic veno-occlusive disease); technology application TcRα+β+/CD19+ depletion at haploidentical transplantation; optimization of conditioning regimens; successes in the prevention and treatment of the graftversus- host disease (antithymocyte globulin and rituximab administration during the period of conditioning, post-transplant administration of cyclophosphamide at haploidentical HSCT). The study was approved by the Independent Ethics Committee of Russian Children's Clinical Hospital.

Первичные иммунодефициты (ПИД) – это группа врожденных заболеваний, многие из которых сопряжены с высоким риском развития жизнеугрожающих инфекционных и неинфекционных осложнений. При многих ПИД показано проведение трансплантации гемопоэтических стволовых клеток (ТГСК), которая может привести к полному излечению заболевания. В статье представлен более чем 20-летний опыт проведения ТГСК при ПИД в РДКБ ФГБОУ ВО РНИМУ им. Н.И. Пирогова Минздрава России. За период с 1997 по 2018 год выполнены 88 ТГСК 80 пациентам (64 мальчикам и 16 девочкам) с различными ПИД: тяжелой комбинированной иммунной недостаточностью (ТКИН, n = 34), гемофагоцитарным лимфогистиоцитозом (n = 12), хронической гранулематозной болезнью (n = 11), синдромом Вискотта–Олдрича (n = 10), врожденным агранулоцитозом (n = 4), синдромом гипериммуноглобулинемии IgM 1-го типа (n = 3), синдромом Ниймеген (n = 2), лимфопролиферативным синдромом (n = 2), синдромом Чедиака–Хигаши (n = 1), дефицитом адгезии лейкоцитов (n = 1). Общая (ОВ) и бессобытийная выживаемость (БСВ) после ТГСК при ПИД составила 63,1 и 49,3% соответственно; ОВ после ТГСК при ТКИН – 65,5%; БСВ – 48,4%. В статье представлены результаты ТГСК при всех перечисленных нозологиях с учетом вида ТГСК, источника гемопоэтических стволовых клеток, вида манипуляций с трансплантатом и режима кондиционирования. Рост положительных результатов ТГСК у пациентов с ПИД за последние годы связан с совершенствованием сопроводительной терапии (повышение качества инфекционного контроля, внедрение новых препаратов для профилактики и лечения веноокклюзионной болезни печени), применение технологий TcRα+β+/CD19+ деплеции при гаплоидентичных трансплантациях, оптимизация режимов кондиционирования, успехи в профилактике и лечении реакции «трансплантат против хозяина» (введение антитимоцитарного иммуноглобулина и ритуксимаба в период кондиционирования, посттрансплантационное введение циклофосфамида при гаплоидентичных ТГСК). Данное исследование поддержано Независимым этическим комитетом Российской детской клинической больницы.

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