Results of Transplanting Bone Marrow from Genetically Identical Twins into Patients with Aplastic Anemia

Hinterberger, W; Rowlings, Philip; Hinterberger-Fischer, M; Gibson, John; Jacobsen, N; Klein, John P.; Kolb, Hans‐Jochem; Stevens, Don A.; Horowitz, Mary M.; Gale, Robert Peter

doi:10.7326/0003-4819-126-2-199701150-00004

Cited by 67 publications

(33 citation statements)

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“…[9][10][11][12] This finding was also confirmed in the CIBMTR cohort study of Hinterberger et al in which all 13 patients who had received conditioning and survived more than 30 days had stable engraftment, while only 12 of 23 transplants without conditioning engrafted successfully. 7 A significant proportion of retransplant after syngeneic transplant (38%) was also Syngeneic transplantation in aplastic anemia haematologica | 2013; 98(11) ; however, no data on conditioning was provided. 8 In both of these series, all patients received bone marrow as graft source.…”

Section: Discussionmentioning

confidence: 99%

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Syngeneic transplantation in aplastic anemia: pre-transplant conditioning and peripheral blood are associated with improved engraftment: an observational study on behalf of the Severe Aplastic Anemia and Pediatric Diseases Working Parties of the European Group for Blood and Marrow Transplantation

et al. 2013

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ARTICLES haematologica | 2013; 98(11) Aplastic Anemia Aplastic anemia is usually treated with immunosuppression or allogeneic transplant, depending on patient and disease characteristics. Syngeneic transplant offers a rare treatment opportunity with minimal transplant-related mortality, and offers an insight into disease mechanisms. We present here a retrospective analysis of all syngeneic transplants for aplastic anemia reported to the European Group for Blood and Marrow Transplantation. Between 1976 and 2009, 88 patients received 113 transplants. Most transplants (n=85) were preceded by a conditioning regimen, 22 of these including anti-thymocyte globulin. About half of transplants with data available (39 of 86) were followed by posttransplant immunosuppression. Graft source was bone marrow in the majority of cases (n=77). Transplant practice changed over time with more transplants with conditioning and anti-thymocyte globulin as well as peripheral blood stem cells performed in later years. Ten year overall survival was 93% with 5 transplant-related deaths. Graft failure occurred in 32% of transplants. Risk of graft failure was significantly increased in transplants without conditioning, and with bone marrow as graft source. Lack of posttransplant immunosuppression also showed a trend towards increased risk of graft failure, while anti-thymocyte globulin did not have an influence. In summary, syngeneic transplant is associated with a significant risk of graft failure when no conditioning is given, but has an excellent long-term outcome. Furthermore, our comparatively large series enables us to recommend the use of pre-transplant conditioning rather than not and possibly to prefer peripheral blood as a stem cell source. Syngeneic transplantation in aplastic anemia

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Section: Discussionmentioning

confidence: 99%

“…7,8 Here, we present an analysis of all syngeneic transplants performed for aplastic anemia reported to the European Group for Blood and Marrow Transplantation (EBMT) registry.…”

Section: Introductionmentioning

confidence: 99%

Syngeneic transplantation in aplastic anemia: pre-transplant conditioning and peripheral blood are associated with improved engraftment: an observational study on behalf of the Severe Aplastic Anemia and Pediatric Diseases Working Parties of the European Group for Blood and Marrow Transplantation

et al. 2013

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“…Aplastic anemia is a classical example of a serious disorder with an autoimmune pathogenesis in about two thirds of cases. 58,59 Replacement of the abnormal hemo-lymphatic system with normal or genetically modified stem cells may not only benefit patients with aplastic anemia, but also patients with other life-threatening AID. The therapeutic efficacy of SCTx in several serious disorders with an autoimmune pathogenesis is currently being explored.…”

Section: Discussionmentioning

confidence: 99%

“…Hemopoietic stem cell transplants utilizing identical twin donors have only rarely been performed in proven or suspected AID. 16,34,57,58 Only two leukemic recipients exhibiting a concomitant AID (IDDM and autoimmune thyroiditis) received identical twin marrow grafts. The AID of these patients obviously had not been expected to respond.…”

Section: Discussionmentioning

confidence: 99%

Clinically demonstrable anti-autoimmunity mediated by allogeneic immune cells favorably affects outcome after stem cell transplantation in human autoimmune diseases

Hinterberger

Hinterberger-Fischer

Marmont

2002

Bone Marrow Transplant

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Summary:To determine the role of allogeneic, autologous and syngeneic hemopoietic stem cell transplantation (SCTx) as a treatment for severe autoimmune disease (AID) we performed a literature search employing Medline, Cancer Lit and abstract books for reports on transplants for blood disorders and a concomitant AID. All reviews, case reports and abstracts available between June 1977 and September 2001 were used and attempts made to update them by e-mail by the corresponding authors. Disease-free survival (DFS) after allogeneic SCTx for 23 patients with severe aplastic anemia was 78% at 16 years and survival in unmaintained remission of concomitant AID was 64% at 13 years. DFS after allogeneic SCTx for 24 patients with hematologic malignancies was 87% at 15 years and survival in unmaintained remission for concomitant AID was 70% at 11 years. DFS after autologous SCTx for 24 patients with hematologic malignancies was 48% at 6 years and survival in unmaintained remission for concomitant AID was 29% at 3 years. Among 30 patients given allogeneic SCTx 19 developed graft-versus-host disease (GVHD) and 11 did not. Upon clinically justified discontinuation of all immunosuppressive therapy, 3/11 patients without GVHD relapsed with their concomitant AID (freedom of AID-relapse 69% at 9 years), whereas none of 19 patients with GVHD did so (log rank: P ‫؍‬ 0.0135). Freedom of AID-relapse was superior after allo SCTx compared to autologous SCTx (89% at 18 years vs 38% at 5 years; log rank: P ‫؍‬ 0.0002). Our data suggest that a graft-versus-autoimmunity effect after allogeneic hemopoietic SCTx mediates elimination of autoimmunity.

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“…For example, in a study of persons with aplastic anaemia receiving a transplant from a genetically identical twin, about one-half of subjects recovered normal bone marrow function after the twin haematopoietic cells were infused. 1 These persons were presumed to have aplastic anaemia from abnormal or absent haematopoietic stem or precursor cells, but a modern interpretation (which I regard as unlikely, but then again who am I to say?) is that their bone marrow microenvironment was defective and restored by unspecified cells in the graft other than haematopoietic stem or progenitor cells such as mesenchymal stromal cells.…”

mentioning

confidence: 99%

Early and late graft-failure after transplants

Gale

2015

Bone Marrow Transplant

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Results of Transplanting Bone Marrow from Genetically Identical Twins into Patients with Aplastic Anemia

Abstract: Most patients with aplastic anemia recover bone marrow function after receiving a transplant from a genetically identical twin. Pretransplantation conditioning may increase the chance of bone marrow recovery but does not seem to improve survival.

Cited by 67 publications

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Syngeneic transplantation in aplastic anemia: pre-transplant conditioning and peripheral blood are associated with improved engraftment: an observational study on behalf of the Severe Aplastic Anemia and Pediatric Diseases Working Parties of the European Group for Blood and Marrow Transplantation

Syngeneic transplantation in aplastic anemia: pre-transplant conditioning and peripheral blood are associated with improved engraftment: an observational study on behalf of the Severe Aplastic Anemia and Pediatric Diseases Working Parties of the European Group for Blood and Marrow Transplantation

Clinically demonstrable anti-autoimmunity mediated by allogeneic immune cells favorably affects outcome after stem cell transplantation in human autoimmune diseases

Early and late graft-failure after transplants

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