Guidelines for the diagnosis and management of acquired aplastic anaemia

Marsh, Judith; Ball, Sarah E.; Darbyshire, P; Gordon‐Smith, E. C.; Keidan, A.J.; Martin, Angela; McCann, Shaun R.; Mercieca, Jane; Oscier, David; Roques, A. W. W.; Yin, John

doi:10.1046/j.1365-2141.2003.04721.x

Cited by 162 publications

(153 citation statements)

References 107 publications

(112 reference statements)

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“…Addition of fludarabine to cyclophosphamide and ATG has been recommended for unrelated transplant in severe aplastic anemia 13 and has been reported in limited series for severe aplastic anemia 4,15 and in Fanconi's anemia. 16 We chose to use this option in related transplants, because of the multiple risk factors for graft failure in our patients.…”

Section: Discussionmentioning

confidence: 99%

“…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%

“…Fludarabine-based regimens for HSCT in aplastic anemia have been described, but there has been considerable variation in doses and preparation. 4,13,15 Antithymocyte globulin given during conditioning is known to reduce both acute and chronic GVHD, especially if it is given nearer the day of transplant. 19 The circulating ATG produces an in vivo T-cell depletion of the infused donor T cells, as serum levels may be quite high at the time of transplant.…”

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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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%

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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“…included 77 patients who were tested for any correlation between the presence of a minor population in PNH-type cells and the response to IST in our previous study [14]. The severity of AA was classified according to the criteria proposed by Camitta et al [15,16]. All participants provided written, informed consent to all procedures associated with the study, which was approved by the Ethical Committee at our institution (study number 46).…”

Section: Introductionmentioning

confidence: 99%

Roles of DRB1∗1501 and DRB1∗1502 in the pathogenesis of aplastic anemia

Sugimori

Yamazaki

Feng

et al. 2007

Experimental Hematology

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Category Clinical investigations Word countsTotal text word count: 3412 words Abstract word count: 240 words 2 ABSTRACTObjective. Although a number of reports have documented a significantly increased incidence of HLA-DR15 in aplastic anemia (AA), the exact role of HLA-DR15 in the immune mechanisms of AA remains unclear. We herein clarify the difference between DRB1*1501 and DRB1*1502, the 2 DRB1 alleles which determine the presentation of HLA-DR15, in the pathophysiology of AA. Materials and Methods.We investigated the relationships of the patients' HLA-DRB1 allele with both the presence of a small population of CD55 -CD59 -(PNH-type) blood cells and the response to antithymocyte globulin (ATG) plus cyclosporine (CsA) therapy in 140 Japanese AA patients. Results. Of the 30 different DRB1 alleles, only DRB1*1501 (33.6% vs. 12.8%, P c <0.01) and DRB1*1502 (43.6% vs. 24.4%, P c <0.01) displayed significantly higher frequencies among the AA patients than among a control. AA patients possessing HLA-DR15 tended to be old, and especially, the frequency of DRB1*1502 in patients ≥40 years old (52.4%) was markedly higher than that in those <40 years old (16.2%, P c <0.01). Only DRB1*1501 was significantly associated with the presence of a small population of PNH-type cells and it also showed a good response to ATG plus CsA therapy in a univariate analysis. A multivariate analysis showed only the presence of a small population of PNH-type cells to be a significant factor associated with a good response to the immunosuppressive therapy (P<0.01). Conclusion.Although both DRB1*1501 and DRB1*1502 contribute to the development of AA, the methods of contribution differ between the two alleles.3

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“…SAA was defined by standard criteria. 14 No patient had clonal chromosomal abnormalities. Very SAA was considered if the patient met the criteria for SAA and had a neutrophil count less than 0.2 Â 10 9 /L.…”

Section: Patient and Donor Characteristicsmentioning

confidence: 99%

Unrelated cord blood transplantation for newly diagnosed patients with severe acquired aplastic anemia using a reduced-intensity conditioning: high graft rejection, but good survival

Liu

et al. 2012

Bone Marrow Transplant

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We report a single-center experience in treating 18 consecutive patients with severe aplastic anemia (SAA) who received unrelated cord blood transplantation (CBT). The median age was 17 years (range 5 --61 years). Sixteen cases received a reducedintensity regimen composed of CY (total dose1200 mg/m 2 ), rabbit antithymocyte globulin (ATG, total dose 30 mg/kg) and fludarabine (FLU, total dose 120 mg/m 2 ). CYA and mycophenolate mofetil were used as GVHD prophylaxis. Two patients were not evaluable for engraftment because of early death on day þ 21 and þ 22. Only one of the sixteen cases achieved engraftment, but experienced secondary graft failure 3 months post transplantation. Fifteen patients experienced primary graft rejection, but all of them acquired autologous recovery. The 3-month and 6-month cumulative incidence of response was 56% and 81%, respectively. So far, 16 patients have survived for 330 --1913 days (median, 750 days) after transplantation. The probability of OS at 2 years was 88.9%. Our data indicate that CBT for newly diagnosed SAA using no irradiation but FLU and ATG-based conditioning still seems to inevitably lead to the high risk of rejection, but may facilitate autologous recovery and improve survival with low risk of transplant-related mortality.

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Guidelines for the diagnosis and management of acquired aplastic anaemia

Cited by 162 publications

References 107 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

Roles of DRB1∗1501 and DRB1∗1502 in the pathogenesis of aplastic anemia

Unrelated cord blood transplantation for newly diagnosed patients with severe acquired aplastic anemia using a reduced-intensity conditioning: high graft rejection, but good survival

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