Hematopoietic stem cell transplantation with alpha/beta T-lymphocyte depletion and short course of eculizumab in adolescents and young adults with paroxysmal nocturnal hemoglobinuria
- 作者: SHASHELEVA D1, MASCHAN A1, SHELIKHOVA L1, PETROVA U1, KURNIKOVA E1, ILLARIONOVA O1, BOYAKOVA E1, NOVICHKOVA G1, MASCHAN M1
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隶属关系:
- Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Department of optimization treatment and prevention of complications of hematopoietic stem cell transplantation
- 期: 卷 90, 编号 7 (2018)
- 页面: 57-64
- 栏目: Editorial
- URL: https://journals.rcsi.science/0040-3660/article/view/32779
- DOI: https://doi.org/10.26442/terarkh201890757-64
- ID: 32779
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作者简介
D SHASHELEVA
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Department of optimization treatment and prevention of complications of hematopoietic stem cell transplantation
Email: shasheleva@mail.ru
врач-гематолог отд-ния ТГСК №1, ORCID 0000-0002-5641-8567; тел.: +7(926)576-89-12 Moscow, Russia
A MASCHAN
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Department of optimization treatment and prevention of complications of hematopoietic stem cell transplantationзам. генерального директора по научной работе, ORCID 0000-0002-0016-6698 Moscow, Russia
L SHELIKHOVA
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Department of optimization treatment and prevention of complications of hematopoietic stem cell transplantationзав. отд-нием ТГСК №1, ORCID 0000-0003-0520-5630 Moscow, Russia
U PETROVA
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Department of optimization treatment and prevention of complications of hematopoietic stem cell transplantationврач-гематолог отд-ния детской гематологии и онкологии, ORCID0000-0002-1258-8281 Moscow, Russia
E KURNIKOVA
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Department of optimization treatment and prevention of complications of hematopoietic stem cell transplantationврач-трансфузиолог отд-ния трансфузиологии, заготовки и процессинга ГСК, ORCID 0000-0003-4767-5382 Moscow, Russia
O ILLARIONOVA
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Department of optimization treatment and prevention of complications of hematopoietic stem cell transplantationврач-лаборант лаб. клеточной иммунологии и иммуногенеза, 0000-0003-2685-674X Moscow, Russia
E BOYAKOVA
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Department of optimization treatment and prevention of complications of hematopoietic stem cell transplantationзав. лаб. трансплантационной иммунологии и иммунотерапии гемобластозов Moscow, Russia
G NOVICHKOVA
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Department of optimization treatment and prevention of complications of hematopoietic stem cell transplantationмедицинский директор, зам. генерального директора по научно-клинической работе, ORCID 0000-0003-4911-0553 Moscow, Russia
M MASCHAN
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology, Department of optimization treatment and prevention of complications of hematopoietic stem cell transplantationам. генерального директора, директор Высшей школы молекулярной и экспериментальной медицины, ORCID 0000-0003-1735-0093 Moscow, Russia
参考
- Новичкова Г.А., Петрова У.Н., Калинина И.И., Масчан А.А. Пароксизмальная ночная гемоглобинурия у детей (обзор литературы). Доктор Ру Гематология. 2016; 122(5):15-20.
- Takeda J, Miyata T, Kawagoe K, Iida Y, Endo Y, Fujita T, Takahashi M, Kitani T, Kinoshita T. Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria. Cell. 1993; 73(4):703-711. https://doi.org/10.1016/0092-8674(93) 90250-t
- Parker C, Omine M, Richards S, Nishimura J.-i, Bessler M, Ware R, Hillmen P, Luzzatto L, Young N, Kinoshita T, Rosse W, Socié G. For the International P. N. H. I. G. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005; 106(12):3699-3709. https:// doi.org/10.1182/blood-2005-04-1717
- Narita A, Muramatsu H, Okuno Y, Sekiya Y, Suzuki K, Hamada M, Kataoka S, Ichikawa D, Taniguchi R, Murakami N, Kojima D, Nishikawa E, Kawashima N, Nishio N, Hama A, Takahashi Y, Kojima S. Development of clinical paroxysmal nocturnal haemoglobinuria in children with aplastic anaemia. British Journal of Haematology. 2017; 178(6):954-958. https://doi.org/10.1111/bjh.14790
- Timeus F, Crescenzio N, Lorenzati A, Doria A, Foglia L, Pagliano S, Quarello P, Ramenghi U, Saracco P. Paroxysmal nocturnal haemoglobinuria clones in children with acquired aplastic anaemia: a prospective single centre study. British Journal of Haematology. 2010; 150(4):483-485. https://doi.org/10.1111/j.1365-2141.2010.08219.x
- Tutelman P.R, Aubert G, Milner R.A, Dalal B.I, Schultz K.R, Deyell R.J. Paroxysmal nocturnal haemoglobinuria phenotype cells and leucocyte subset telomere length in childhood acquired aplastic anaemia. British Journal of Haematology. 2014; 164(5):717-721. https://doi.org/ 10.1111/bjh.12656
- Kulagin A, Lisukov I, Ivanova M, Golubovskaya I, Kruchkova I, Bondarenko S, Vavilov V, Stancheva N, Babenko E, Sipol A, Pronkina N, Kozlov V, Afanasyev B. Prognostic value of paroxysmal nocturnal haemoglobinuria clone presence in aplastic anaemia patients treated with combined immunosuppression: results of two - centre prospective study. British Journal of Haematology. 2014; 164(4):546-554. https://doi.org/10.1111/bjh.12661
- Hillmen P, Lewis S.M, Bessler M, Luzzatto L, Dacie J.V. Natural History of Paroxysmal Nocturnal Hemoglobinuria. New England Journal of Medicine. 1995; 333(19):1253-1259. https://doi.org/10.1056/nejm 199511093331904
- Socié G, Mary J.-Y, de Gramont A, Rio B, Leporrier M, Rose C, Heudier P, Rochant H, Cahn J.-Y, Gluckman E. Paroxysmal nocturnal haemoglobinuria: long - term follow - up and prognostic factors. Lancet. 1996; 348 (9027):573-577. https://doi.org/10.1016/s0140-6736(95)12360-1
- Santarone S, Bacigalupo A, Risitano A.M, Tagliaferri E, Di Bartolomeo E, Iori A.P, Rambaldi A, Angelucci E, Spagnoli A, Papineschi F, Tamiazzo S, Di Nicola M, Di Bartolomeo P. Hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria: long - term results of a retrospective study on behalf of the Gruppo ItalianoTrapiantoMidollo Osseo (GITMO). Haematologica. 2010; 95(6):983-988. https://doi.org/10.3324/haematol.2009.017269
- de Latour R.P, Schrezenmeier H, Bacigalupo A, Blaise D, de Souza C.A, Vigouroux S, Willemze R, Terriou L, Tichelli A, Mohty M, de Guibert S, Marsh J.C, Passweg J, Yves Mary J, Socié G. Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria. Haematologica. 2012; 97(11):1666-1673. https://doi.org/10.3324/haematol. 2012.062828
- Попов А. М., Кашпор С.А., Вержбицкая Т.Ю., Борисов В. И., Фечина Л. Г., Плясунова С. А., Масчан А. А., Новичкова Г. А. Определение FLAER-негативных гранулоцитов в периферической крови - простой скрининг - тест для диагностики пароксизмальной ночной гемоглобинурии. Вопросы гематологии/онкологии и иммунопатологии в педиатрии. 2016; 15(3):12-18. https://doi.org/10.20953/1726-1708-2016-3-12-18
- Наумова Е.В., Почтарь М.Е., Кисиличина Д.Г., Плеханова О.С., Сипол А.А., Бабенко Е.В. и др. Стандартизация диагностики пароксизмальной ночной гемоглобинурии с помощью проточной цитометрии. Клиническая лабораторная диагностика. 2013; 61(7):54-58.
- Pantin J, Tian X, Geller N, Ramos C, Cook L, Cho E, Scheinberg P, Vasu S, Khuu H, Stroncek D, Barrett J, Young N.S, Donohue T, Childs R.W. Long - term outcome of fludarabine - based reduced intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria. Biology of blood and marrow transplantation: journal of the American Society for Blood and Marrow Transplantation. 2014; 20(9):1435-1439. https://doi.org/ 10.1016/ j.bbmt.2014.05.012
- Vallet N, de Fontbrune F.S, Loschi M, Desmier D, Villate A, Barraco F, Chevallier P, Terriou L, Yakoub-Agha I, Ruggeri A, Mohty M, Maillard N, Rohrlich P-S, Ceballos P, Nguyen S, Poiré X, Guillerm G, Tabrizi R, Farhi J, Devillier R, Rubio M-T, Socié G, de Latour R.P. Hematopoietic stem cell transplantation for patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a retrospective study of 21 patients from SFGM-TC centers. Haematologica. 2018; 103(3):e103-e105. https://doi.org/10.3324/haematol.2017.182360
- Murphy W.J, Bennett M, Kumar V, Longo D.L. Donor - type activated natural killer cells promote marrow engraftment and B cell development during allogeneic bone marrow transplantation. The Journal of Immunology. 1992; 148(9):2953-2960.
- Alvarez M, Sun K, Murphy W.J. Mouse host unlicensed NK cells promote donor allogeneic bone marrow engraftment. Blood. 2016; 127(9):1202-1205. https://doi.org/10.1182/blood-2015-08-665570
- Maschan M, Shelikhova L, Ilushina M, Kurnikova E, Boyakova E, Balashov D, Persiantseva M, Skvortsova Y, Laberko A, Muzalevskii Y, Kazachenok A, Glushkova S, Bobrynina V, Kalinina V, Olshanskaya Y, Baidildina D, Novichkova G, Maschan A. TCR-alpha/beta and CD19 depletion and treosulfan - based conditioning regimen in unrelated and haploidentical transplantation in children with acute myeloid leukemia. Bone Marrow Transplantation. 2016; 51:668. https://doi.org/10.1038/bmt.2015.343
- Sanders J, Hawley J, Levy W, Gooley T, Buckner C, Deeg H, Doney K, Storb R, Sullivan K, Witherspoon R, Appelbaum F. Pregnancies following high - dose cyclophosphamide with or without high - dose busulfan or total - body irradiation and bone marrow transplantation. Blood. 1996; 87(7):3045-3052. https://doi.org/10.1016/0360-3016(92)90909-2
- Скворцова Ю.В. Руднева А.Е., Делягин В.М., Лерхендорф Ю.А., Терещенко Г.В., Балашов Д.Н., Масчан М.А., Масчан А.А. Поздние неинфекционные поражения легких у детей после аллогенной трансплантации гемопоэтических стволовых клеток. Онкогематология. 2015;10(1):29-36. https://doi.org/10.17650/1818-8346-2015-1-29-36
- Скворцова Ю.В. Балашов Д.Н., Скоробогатова Е.В., Шеховцова Ж.Б., Воронин К.А., Масчан А.А. Нарушение минерализации костей после аллогенной трансплантации гемопоэтических стволовых клеток у детей: одноцентровое когортное исследование. Педиатрическая фармакология. 2017; 14(6):459-468. https://doi.org/10.15690/pf.v14i6.1829
- Скворцова Ю.В., Балашов Д.Н., Масчан А.А. Остеопения и остеопороз после аллогенной трансплантации гемопоэтических стволовых клеток, особенности нарушений костного минерального обмена у детей. Вопросы гематологии/онкологии и иммунопатологии в педиатрии. 2017; 16(4):98-106. https://doi.org/10. 24287/1726-1708-2017-16-4-98-106