The use of allogeneic bone marrow transplantation and immunosuppressive therapy in the treatment of patients with acquired aplastic anemia

Aim. To evaluate the efficiency of related and unrelated allogeneic bone marrow transplantation (allo-BMT) versus immunosuppressive therapy (IST) in patients with aplastic anemia (AA) having no HLA-compatible bone marrow donor. Subjects and methods. The study covered 61 patients (34 men and 27 women...

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Main Authors: A A Ganapiyev, I K Golubovskaya, Yu R Zalyalov, M A Estrina, B V Afanasyev
Format: Article
Language:Russian
Published: "Consilium Medicum" Publishing house 2010-07-01
Series:Терапевтический архив
Subjects:
Online Access:https://ter-arkhiv.ru/0040-3660/article/view/30627
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author A A Ganapiyev
I K Golubovskaya
Yu R Zalyalov
M A Estrina
B V Afanasyev
author_facet A A Ganapiyev
I K Golubovskaya
Yu R Zalyalov
M A Estrina
B V Afanasyev
author_sort A A Ganapiyev
collection DOAJ
description Aim. To evaluate the efficiency of related and unrelated allogeneic bone marrow transplantation (allo-BMT) versus immunosuppressive therapy (IST) in patients with aplastic anemia (AA) having no HLA-compatible bone marrow donor. Subjects and methods. The study covered 61 patients (34 men and 27 women) diagnosed as having acquired AA. Of them, 51 patients were diagnosed as having severe AA, 5 had supersevere AA, and 5 had non-severe AA. Combined IST (antithymocyte globulin (ATG) + cyclosporin A (CsA)) was used in 43 patients; allo-BMT was performed in 18. The basic types of ATG (ATGAM (Pfizer), thymoglobulin (Genzim), ATG (Fresenius), and goat antilymphocyte globulin (ALG) (Research Institute of Gerontology, Ministry of Health of the Russian Federation) were administered. CsA was given in a dose of 5 mg/kg/day. The standard conditioning regimen (ATGAM + cyclophosphanum) and fludarabine-containing (fludarabine + cyclophosphanum + ATG; busulfan + fludarabine + ATG) programs were used in the allo-BMT group. A combination of CsA and metothrexate was given to prevent a graft-versus-host reaction. Results. Among the IST-receiving patients, overall survival (OS) was 71%. After the first course of IST by follow-up month 6, the response rate was 74%. The second course of IST was performed in 7 patients unresponsive after the first-line IST and in 8 patients with recurrent AA. After the second course of IST, the response rate was 46.7%. Four patients who failed to achieve remission after 2 courses of IST received its third course. A complete response was obtained in 3 patients. In 18 patients following allo-BMT (related and unrelated), OS was 86%; event-free survival was 65. In 12 patients after related allo-BMT, OS was 91.7%. Conclusion. Related allo-BMT is the method of choice if there is a HLA-compatible sibling. If there are contraindications to it or no related donor, IST with ATG + CsA is indicated. Ineffective IST is an indication for unrelated allo-BMT that may be recommended as life-saving therapy for young patients under 40 years of age.
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spelling doaj.art-b13bcf9bd7214261a3383b62b814b29d2022-12-21T19:40:09Zrus"Consilium Medicum" Publishing houseТерапевтический архив0040-36602309-53422010-07-01827485227654The use of allogeneic bone marrow transplantation and immunosuppressive therapy in the treatment of patients with acquired aplastic anemiaA A Ganapiyev0I K Golubovskaya1Yu R Zalyalov2M A Estrina3B V Afanasyev4Acad. I. P. Pavlov Saint Petersburg State Medical UniversityAcad. I. P. Pavlov Saint Petersburg State Medical UniversityAcad. I. P. Pavlov Saint Petersburg State Medical UniversityR. M. Gorbacheva Institute of Pediatric Hematology and Transplantology, Acad. I. P. Pavlov Saint Petersburg State Medical UniversityAcad. I. P. Pavlov Saint Petersburg State Medical UniversityR. M. Gorbacheva Institute of Pediatric Hematology and Transplantology, Acad. I. P. Pavlov Saint Petersburg State Medical UniversityAim. To evaluate the efficiency of related and unrelated allogeneic bone marrow transplantation (allo-BMT) versus immunosuppressive therapy (IST) in patients with aplastic anemia (AA) having no HLA-compatible bone marrow donor. Subjects and methods. The study covered 61 patients (34 men and 27 women) diagnosed as having acquired AA. Of them, 51 patients were diagnosed as having severe AA, 5 had supersevere AA, and 5 had non-severe AA. Combined IST (antithymocyte globulin (ATG) + cyclosporin A (CsA)) was used in 43 patients; allo-BMT was performed in 18. The basic types of ATG (ATGAM (Pfizer), thymoglobulin (Genzim), ATG (Fresenius), and goat antilymphocyte globulin (ALG) (Research Institute of Gerontology, Ministry of Health of the Russian Federation) were administered. CsA was given in a dose of 5 mg/kg/day. The standard conditioning regimen (ATGAM + cyclophosphanum) and fludarabine-containing (fludarabine + cyclophosphanum + ATG; busulfan + fludarabine + ATG) programs were used in the allo-BMT group. A combination of CsA and metothrexate was given to prevent a graft-versus-host reaction. Results. Among the IST-receiving patients, overall survival (OS) was 71%. After the first course of IST by follow-up month 6, the response rate was 74%. The second course of IST was performed in 7 patients unresponsive after the first-line IST and in 8 patients with recurrent AA. After the second course of IST, the response rate was 46.7%. Four patients who failed to achieve remission after 2 courses of IST received its third course. A complete response was obtained in 3 patients. In 18 patients following allo-BMT (related and unrelated), OS was 86%; event-free survival was 65. In 12 patients after related allo-BMT, OS was 91.7%. Conclusion. Related allo-BMT is the method of choice if there is a HLA-compatible sibling. If there are contraindications to it or no related donor, IST with ATG + CsA is indicated. Ineffective IST is an indication for unrelated allo-BMT that may be recommended as life-saving therapy for young patients under 40 years of age.https://ter-arkhiv.ru/0040-3660/article/view/30627aplastic anemiaallogenic bone marrow transplantationimmunosuppressive therapyantithymocyte globulin
spellingShingle A A Ganapiyev
I K Golubovskaya
Yu R Zalyalov
M A Estrina
B V Afanasyev
The use of allogeneic bone marrow transplantation and immunosuppressive therapy in the treatment of patients with acquired aplastic anemia
Терапевтический архив
aplastic anemia
allogenic bone marrow transplantation
immunosuppressive therapy
antithymocyte globulin
title The use of allogeneic bone marrow transplantation and immunosuppressive therapy in the treatment of patients with acquired aplastic anemia
title_full The use of allogeneic bone marrow transplantation and immunosuppressive therapy in the treatment of patients with acquired aplastic anemia
title_fullStr The use of allogeneic bone marrow transplantation and immunosuppressive therapy in the treatment of patients with acquired aplastic anemia
title_full_unstemmed The use of allogeneic bone marrow transplantation and immunosuppressive therapy in the treatment of patients with acquired aplastic anemia
title_short The use of allogeneic bone marrow transplantation and immunosuppressive therapy in the treatment of patients with acquired aplastic anemia
title_sort use of allogeneic bone marrow transplantation and immunosuppressive therapy in the treatment of patients with acquired aplastic anemia
topic aplastic anemia
allogenic bone marrow transplantation
immunosuppressive therapy
antithymocyte globulin
url https://ter-arkhiv.ru/0040-3660/article/view/30627
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