[The role and place of high-dose immunosuppressive therapy and autologous transplantation of hematopoietic stem cells for autoimmune diseases].

Abstract:

AIM:To determine the possible boundaries of high-dose immunosuppressive therapy and autologous hematopoietic stem cell transplantation (HDIT-autoHSCT) for autoimmune diseases (AUDs), such as systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and multiple sclerosis (MS). SUBJECTS AND METHODS:A long-term trial was conducted at one center to evaluate the efficiency and safety of HDIT-autoHSCT in patients with AUDs. The previous standard therapy was noted to be resistant or lowly effective. The age of 10 patients with systemic connective tissue diseases was 27.6±2.8 years; the pre-HDIT-autoHSCT disease duration was 5.9±1.3 years; the median posttransplantation follow-up was 39.3 months. The age of 49 patients with MS reached 34.9±1.33 years; the pretransplantation disease duration was 8.4±0.69 years; the median post-HDIT-autoHSCT follow-up was 42 months. The efficiency of transplantation was evaluated on the basis of clinical findings, by using scales, laboratory tests, and magnetic resonance imaging. Pretransplantation conditioning was carried out according to the protocols: a) BEAM + antilymphocyte globulin (ALG); b) fludarabine + melphalan + ALG. No fatal outcomes due to a transplant procedure were observed. RESULTS:Overall 5-year survival after transplantation was 80% for systemic connective tissue diseases and 95% for MS; 5-year progression-free survival rates were 30% in the RA and SLE groups and 45% in the MS group. HDIT-autoHSCT turned out safe and reduced the activity of the process and further disease progression for a long period of time, as confirmed by regression of clinical symptoms and/or status stabilization in 9 patients with SLE or RA and in all patients with MS. CONCLUSION:The favorable factors associated with the results of transplantation are age younger than 35 years in collagenoses with their short-term duration and moderate signs; age younger than 40 years in MS with a disease duration of less than 10 years and expanded disability status scale scores of not more than 6.5. Of importance are functional system scores, duration of first remission, and an index of disease progression in different types of MS. :Цель исследования. Определение возможных границ применения высокодозной иммуносупрессивной терапии с аутотрансплантацией стволовых кроветворных клеток (ВИСТ-аутоТСКК) при аутоиммунных заболеваниях (АИЗ): системной красной волчанке (СКВ), ревматоидном артрите (РА), рассеянном склерозе (РС). Материалы и методы. Проведено многолетнее одноцентровое исследование по оценке эффективности и безопасности применения ВИСТ-аутоТСКК у больных с АИЗ. Отмечена резистентность или низкая эффективность предшествующей стандартной терапии. Возраст 10 больных с системными заболеваниями соединительной ткани составил 27,6±2,8 года, длительность заболевания до ВИСТ-аутоТСКК - 5,9±1,3 года, медиана наблюдения после трансплантации - 39,3 мес. Возраст 49 больных РС достигал 34,9±1,33 года, длительность заболевания до трансплантации - 8,4±0,69 года, медиана наблюдения после ВИСТ-аутоТСКК 42 мес. Оценку эффективности трансплантации осуществляли на основании клинических данных с применением шкал, лабораторных методов, магнитно-резонансной томографии. Предтрансплантационное кондиционирование проводили по протоколам: а) ВЕАМ + антилимфоцитарный глобулин (АЛГ); б) флударабин + мелфалан + АЛГ. Летальных исходов, связанных с процедурой трансплантации, не отмечено. Результаты. Общая 5-летняя выживаемость от даты трансплантации при системных заболеваниях соединительной ткани составила в среднем 80%, при РС - 95%; 5-летняя выживаемость без прогрессирования в группе РА и СКВ - 30%, в группе РС - 45%. ВИСТ-аутоТСКК безопасна, снижала активность процесса и дальнейшее прогрессирование заболевания на длительный срок, что подтверждено регрессом клинических симптомов и/или стабилизацией состояния у 9 пациентов с СКВ, РА и у всех больных РС. Заключение. Благоприятными факторами, связанными с результатами трансплантации, являются при коллагенозах возраст моложе 35 лет с небольшой длительностью заболевания и умеренными признаками активности болезни; при РС возраст моложе 40 лет с длительностью заболевания менее 10 лет при показателе по расширенной шкале инвалидизации (EDSS) не более 6,5 балла. Имеют значение данные шкалы функциональных систем (FS), длительность первой ремиссии, индекс прогрессирования болезни при различных типах течения РС.

journal_name

Ter Arkh

journal_title

Terapevticheskii arkhiv

authors

Makarov SV,Rossiev VA,Mishchenko OV,Kozlov VA,Semagina OV,Alexandrova IY,Grishina GV,Minaev YL

doi

10.17116/terarkh201688153-59

subject

Has Abstract

pub_date

2016-01-01 00:00:00

pages

53-59

issue

1

eissn

0040-3660

issn

2309-5342

journal_volume

88

pub_type

杂志文章
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  • [The comparative efficacy of splenectomy and antilymphocyte globulin therapy in patients with aplastic anemia].

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  • [Immunosupressive therapy of aplastic anemia patients: successes and failures (single center experiment 2007-2016)].

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    authors: Solov'ev GM,Mikheev AA,Petrovskiĭ PF,Klembovskiĭ AA,Simonov VI,Travin NO,Alishin II,Pisareva NS

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