Current status of allogeneic bone marrow transplantation in acquired aplastic anemia

Horowitz, Mary M.

doi:10.1016/s0037-1963(00)90028-3

Cited by 107 publications

(65 citation statements)

References 78 publications

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“…Addition of radiation to cyclophosphamide has reduced graft failure, but not improved survival owing to higher risk of GVHD, interstitial pneumonia and late complications of radiation. 2,10 Addition of ATG to cyclophosphamide has led to superior survival 12 and is considered standard therapy for multiply transfused patients. 13,14 However, the high graft rejections seen in India with these protocols 3 and subsequent poor outcome made us search for more intense conditioning regimens.…”

Section: Discussionmentioning

confidence: 99%

“…1,2 The use of G-CSF-mobilized peripheral blood stem cells (PBSC) for transplants results in a larger collection of stem cells, and presumably less graft failure, but with an increased risk of chronic GVHD. 2,17 The other advantage of PBSC transplant is more rapid neutrophil and platelet engraftment, 18 which would reduce the risk of early infections and cost of supportive therapy. As we do not have facilities of HEPA filter rooms, we hypothesized that early engraftment would minimize the risk of increased infections.…”

Section: Discussionmentioning

confidence: 99%

“…1 With improvement in transplant practices, the graft failure rates have fallen to about 10%. 2 In developing countries, most patients are multiply transfused before transplant. Owing to economic reasons, leukocyte filters are rarely used and occasionally blood from family donors is transfused because of blood shortage.…”

Section: Introductionmentioning

confidence: 99%

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Fludarabine, cyclophosphamide and horse antithymocyte globulin conditioning regimen for allogeneic peripheral blood stem cell transplantation performed in non-HEPA filter rooms for multiply transfused patients with severe aplastic anemia

Kumar

Prem

Mahapatra

et al. 2006

Bone Marrow Transplant

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Multiply transfused patients of severe aplastic anemia are at increased risk of graft rejection. Five such patients underwent peripheral blood stem cell transplantation from HLA-identical siblings with a fludarabine-based protocol. The conditioning consisted of fludarabine 30 mg/m 2 / day Â 6 days, cyclophosphamide 60 mg/kg/day Â 2 days and horse antithymocyte globulin (ATG) Â 4 days. Two different ATG preparations were used: ATGAM (dose 30 mg/kg/day Â 4 days) or Thymogam (dose 40 mg/kg/ day Â 4 days). Engraftment: median time to absolute neutrophil count (ANC) 40.5 Â 10 9 /l was 11 days (range: 8-17) and median time to platelet count 420 Â 10 9 /l was 11 days (range: 9-17). At a median follow-up of 171 days (range: 47-389), there has been no graft rejection and all patients are in complete remission. Acute GVHD (grade 1) occurred in one patient only. Chronic GVHD developed in two patients (extensive in one and limited in another). The transplants were performed in non-HEPA filter rooms. In only one patient, systemic antifungal therapy (voriconazole) was used. The use of Thymogam brand of ATG for conditioning is being reported for the first time. Our experience suggests that this fludarabine-based protocol allows rapid sustained engraftment in high-risk patients without significant immediate toxicity.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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Fludarabine, cyclophosphamide and horse antithymocyte globulin conditioning regimen for allogeneic peripheral blood stem cell transplantation performed in non-HEPA filter rooms for multiply transfused patients with severe aplastic anemia

Kumar

Prem

Mahapatra

et al. 2006

Bone Marrow Transplant

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“…Accordingly, it has been recognized than less than 15% of AA patients can remain untransfused at transplant. 30 Interestingly, in our study the number of patients with active hemorrhage apparently increased during the latest period, but whether this might reflect more optimized transfusion support in these patients is unknown.…”

Section: Radiationmentioning

confidence: 61%

CsA-based post-graft immunosuppression: the main factor for improving outcome of allografted patients with acquired aplastic anemia. A retrospective survey by the Spanish Group of Hematopoietic Transplantation

Arranz

Conde

Rodríguez-Salvanés

et al. 2002

Bone Marrow Transplant

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Summary:A retrospective multicenter study was performed to assess the clinical results in patients with acquired aplastic anemia (AA) allografted over a 19 year period and to identify prognostic factors influencing survival. From April 1978 to December 1997, 176 patients were transplanted. Records from 160 receiving related matched bone marrow transplantation (BMT) were reviewed. Fifty-two percent of the patients were older than 20 years, 5% older than 40; 6.3% were untransfused at BMT and 56.2% had received prior treatments. Conditioning regimens were with chemotherapy in 43.7% of the procedures and with additional irradiation in 56.3%. Graft-versus-host disease (GVHD) prophylaxis was based on cyclosporin A (CsA) in 58.1% of the patients while methotrexate (MTX) was administered to 41.9%. Transplantation earlier on, a longer interval from diagnosis to BMT, GVHD prophylaxis with MTX, graft failure/rejection and acute severe GVHD were adverse factors for survival. The use of CsA emerged as the main factor for the improvement, inducing a significant decrease in graft failure/rejection rate and severe acute GVHD when compared with MTX alone. Radiation-containing regimens decreased the graft failure/rejection rate without improving survival due to the increased risk of acute GVHD. Age and number of transfusions pretransplant did not influence outcome. Survival achieved since 1991 is 79.79%, and graft fail-

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“…Unrelated donor HSCT is regarded by most clinicians as a sequential option which, by definition, could not be compared with a preceding treatment. On the other hand, it was stated that "a prospective comparison of unrelated donor transplantation versus a second trial of immunosuppression is needed to address this issue", 9 and it was also stated that "an ongoing IBMTR/EBMT study will compare the outcome after MUD BMT versus second course of IST in patients failing the first course of IST". 34 Evidence base for second-line unrelated donor HSCT after failed IST In 2008, the results were published 26 of the first prospective multicenter study to compare the efficacy of repeated IST with HSCT from an alternative donor in children with acquired SAA who failed to respond to an initial course of IST.…”

Section: Lack Of Controlled Trialsmentioning

confidence: 99%

Decision-Making and Problem Solving

Saitis¹,

Saiti

2017

Initiation of Educators Into Educational Management Secrets

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Acquired severe aplastic anemia is a rare disease characterized by an immune-mediated functional impairment of hematopoietic stem cells. Transplantation of these cells from unrelated donors is a treatment option frequently offered to patients after failed immunosuppressive therapy. The aim was to investigate the outcome of these patients treated with unrelated donor transplants. Systematic literature searches were performed in MED-LINE, EMBASE, and The Cochrane Library. All databases were searched from inception to June 2009. Only full-text publications and studies including at least 10 patients were considered. The primary outcome was 5-year overall survival from the day of transplantation and the secondary outcomes were graft failure and graft-versus-host disease. A meta-analysis of survival estimates was conducted and heterogeneity was investigated. A total of 18 studies, one controlled trial and 17 case series were identified. The overall survival at five years and the corresponding confidence interval was stated in 8 studies and ranged from 28% to 94%. A meta-analysis revealed considerable heterogeneity between the studies that could not be explained and was also present in subgroups of the studies. The proportion of acute graft failure was 45% in one study using only umbilical cord blood, and it was reported to be 0-26% in 15 studies using mainly bone marrow as stem cell source after different follow-up periods. Acute GVHD grade II-IV was reported for 8-86% and extensive chronic GVHD for 0-38% of the evaluated patients in 16 studies. Recipient age, human leukocyte antigen match, performance status, year of transplantation, and conditioning with serotherapy were identified as significant factors for improved survival. Unrelated donor hematopoietic stem cell transplantation in patients with acquired severe aplastic anemia after failure to immunosuppressive therapy is a treatment option. A stable physical condition of the patients before receiving the transplant (for example, performance and age) may be associated with a better survival. Detailed HLA-matching facilitated by DNA-based typing, among other factors, may have contributed to recent improvements on survival after unrelated donor HSCT as a second-line treatment.Key words: donor stem cell transplantation, aplastic anemia, systematic review. Haematologica 2009. 94:1732-1742. doi:10.3324/haematol.2009 This is an open-access paper. ABSTRACT

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Current status of allogeneic bone marrow transplantation in acquired aplastic anemia

Cited by 107 publications

References 78 publications

Fludarabine, cyclophosphamide and horse antithymocyte globulin conditioning regimen for allogeneic peripheral blood stem cell transplantation performed in non-HEPA filter rooms for multiply transfused patients with severe aplastic anemia

Fludarabine, cyclophosphamide and horse antithymocyte globulin conditioning regimen for allogeneic peripheral blood stem cell transplantation performed in non-HEPA filter rooms for multiply transfused patients with severe aplastic anemia

CsA-based post-graft immunosuppression: the main factor for improving outcome of allografted patients with acquired aplastic anemia. A retrospective survey by the Spanish Group of Hematopoietic Transplantation

Decision-Making and Problem Solving

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