Treatment of acquired severe aplastic anemia: Bone marrow transplantation compared with immunosuppressive therapy-the European group for blood and marrow transplantation experience

Bacigalupo, Andrea; Brand, Ronald; Oneto, Rosi; Bruno, Benedetto; Sodé, Gerard; Passweg, Jakob; Locasciulli, Anna; Lint, Maria Teresa Van; Thewis, André; McCann, Shaun R.; Marsh, Judith; Ljungman, Per; Hows, Jill; Marı́n, Pedro; Schrezenmeier, Hubert

doi:10.1016/s0037-1963(00)90031-3

Cited by 240 publications

(224 citation statements)

References 34 publications

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“…Patients met the criteria for the diagnosis of severe AA [1,6]. All patients had failed to respond to conventional treatment with immunosuppression using ATG and/or cyclosporin and had been heavily transfused (see Table I).…”

Section: Materials and Methods Patientsmentioning

confidence: 99%

Allografting in patients with severe, refractory aplastic anemia using peripheral blood stem cells and a fludarabine-based conditioning regimen: The Mexican experience

et al. 2006

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We studied the effectiveness of a fludarabine/cyclophosphamide-based conditioning regimen without anti-thymocyte globulin in 23 aplastic anemia patients who had no response to previous conventional pharmacologic treatment. Patients received oral busulphan 4 mg/ kg/day/2 days, IV cyclophosphamide 350 mg/m 2 /day/3 days, and fludarabine 30 mg/m 2 /day/ 3 days. For GVHD prophylaxis, patients received MTX 5 mg/m 2 days +1, +3, +6, and +11 and oral cyclosporin A (CyA) 5 mg/kg/day, starting on day -1. Peripheral blood stem cell products were used with a median dose of 5.5 · 10 6 CD34 + /kg. The patients were followed for an average of 25 months. By a median of day +11, an ANC > 0.5 · 10 9 /L was reached; and by day +12, the platelet count had reached >20,000 · 10 9 /L. Acute grade I-II GVHD occurred in 4 patients, whereas limited chronic GVHD presented in 6 cases. Twenty-one patients (91.3%) achieved engraftment. Two patients failed to engraft, and 4 developed late rejection; 2 of these individuals died, 2 have survived with high transfusion requirements, whereas 2 received a second peripheral blood stem cell infusion and achieved sustained engraftment. Currently 21 (91%) of the 23 patients are alive, whereas 19 of 21 (90%) remain in complete remission. The average cost was about USD 15,000 for this kind of reducedintensity allotransplant. Reduced-intensity stem cell transplantation represents an affordable alternative to traditional more cytotoxic conditioning for severe aplastic anemia (SAA) patients. Long-term effects however, remain to be evaluated. Am.

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Section: Materials and Methods Patientsmentioning

confidence: 99%

Allografting in patients with severe, refractory aplastic anemia using peripheral blood stem cells and a fludarabine-based conditioning regimen: The Mexican experience

et al. 2006

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“…10,11 The incidence and severity of GVHD were based on a NIH consensus conference to determine GVHD grade. 12 Patients were evaluated for cGVHD if they survived for at least 100 days after HSCT.…”

Section: Definitions and Assessment Of Engraftmentmentioning

confidence: 99%

A novel protocol for haploidentical hematopoietic SCT without in vitro T-cell depletion in the treatment of severe acquired aplastic anemia

Liu

et al. 2012

Bone Marrow Transplant

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Mismatched related donors of hematopoietic SCT (HSCT) for severe aplastic anemia (SAA) present challenges mainly associated with graft failure and GVHD. The greater the HLA disparity, the poorer the OS. About 19 consecutive SAA/very SAA (VSAA) patients who received HSCT from haploidentical family donors in our center are reported in this study, 18/19 pairs had 2-3 loci mismatched. All 19 cases failed to respond to previous therapy and were heavily transfused before transplantation. The conditioning regimen before HSCT included BU, CY and thymoglobulin. The recipients received CsA, mycophenolate mofetil (MMF) and short-term MTX for GVHD prophylaxis. The source of stem cell grafts was a combination of G-CSF-primed BM and G-CSF-mobilized peripheral blood stem cells. All patients achieved 100% donor myeloid engraftment; the median time for myeloid engraftment was 12 days (ranging from 10-29 days) and for platelets was 18 days (ranging from 8-180 days) with a cumulative platelet engraftment incidence of 84.21 ± 10.53%. The cumulative incidence was 42.1 ± 11.3% for grade II-IV acute GVHD and 56.2 ± 12.4% for chronic GVHD. The OS was 64.6 ± 12.4% with a median 746-day (90-1970) follow-up for surviving patients. These limited retrospective analysis data suggest that HLA-haploidentical HSCT for SAA patients without an HLA-identical sibling donor might be feasible. Further research to increase OS by decreasing GVHD while maintaining stable engraftment will be needed in the future.

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“…8,19 Response to ALG þ CsA is usually slow, with median time to response of over 100 days, and 20-40% fail to respond to a first course of IST requiring a second course of ALG. This can rescue a significant proportion of nonresponders.…”

Section: Istmentioning

confidence: 99%

“…The estimated difference in the proportion of patients surviving event free after 5 years, after an HLA identical sibling BMT or IST as first-line therapy is based on severity of the disease and patient age. 8 Young patients with low neutrophil counts have a significant advantage if given a BMT first line. Older patients with higher neutrophil counts do better if given IST first line.…”

Section: Introductionmentioning

confidence: 99%

Treatment strategies for patients with severe aplastic anemia

Bacigalupo

2008

Bone Marrow Transplant

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Treatment of acquired severe aplastic anemia: Bone marrow transplantation compared with immunosuppressive therapy-the European group for blood and marrow transplantation experience

Cited by 240 publications

References 34 publications

Allografting in patients with severe, refractory aplastic anemia using peripheral blood stem cells and a fludarabine-based conditioning regimen: The Mexican experience

Allografting in patients with severe, refractory aplastic anemia using peripheral blood stem cells and a fludarabine-based conditioning regimen: The Mexican experience

A novel protocol for haploidentical hematopoietic SCT without in vitro T-cell depletion in the treatment of severe acquired aplastic anemia

Treatment strategies for patients with severe aplastic anemia

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