Cord blood transplantation in aplastic anemia

Latour, Régis Peffault de; Rocha, Vanderson; Socié, Gèrard

doi:10.1038/bmt.2012.252

Cited by 38 publications

(18 citation statements)

References 9 publications

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“…3,[6][7][8][9][10] Higher risk HSCT for those who lack a histocompatible donor often include umbilical cord blood and haploidentical donor as a source of HSCs (HSCs). [11][12][13][14][15][16][17][18][19] The use of haploidentical donors has some advantages compared with matched unrelated donors (MUD) and cord blood units, as a donor is more widely and readily available usually within the family, abbreviating the time for stem cell collection and infusion into the recipient. 18 However, past reports of haploidentical HSCT as therapy in SAA have encountered important limitations with graft failure and risk of severe graft-versus host disease (GVHD).…”

Section: Introductionmentioning

confidence: 99%

Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study

Esteves

Bonfim

Pasqüini

et al. 2015

Bone Marrow Transplant

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138

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Patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor need new therapeutic approaches. Hematopoietic SCT (HSCT) using mismatched or haploidentical related donors has been used in the past, but was associated with a significant risk of GVHD and mortality. Recently, the use of post-transplant cyclophosphamide (Cy) has been shown to be an effective strategy to prevent GVHD in recipients of haploidentical HSCT, but the majority of reports have focused on patients with hematology malignancies. We describe the outcome of 16 patients who underwent haploidentical transplantation using a reduced-intensity conditioning regimen with post-transplant Cy. Stem cell sources were BM (N = 13) or PBSCs (N = 3). The rate of neutrophil engraftment was 94% and of platelet engraftment was 75%. Two patients had secondary graft failure and were successfully salvaged with another transplant. Three patients developed acute GVHD being grades 2-4 in two. Five patients have died and the 1-year OS was 67.1% (95% confidence interval: 36.5-86.4%). In our small series, the use of a reduced-intensity conditioning with post-transplant Cy in haploidentical BMT was associated with high rates of engraftment and low risk of GVHD in patients with relapsed/refractory SAA.

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Section: Introductionmentioning

confidence: 99%

Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study

Esteves

Bonfim

Pasqüini

et al. 2015

Bone Marrow Transplant

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138

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“…46 For patients with FA, only 1 CB is recommended with no more than 1 mismatch, because the use of 2 CB units in this situation was associated in our center with high rates of GVHD and consequently unacceptable toxicity). 48,49 Chimerism must also be followed up carefully post HSCT (Table 1). 8,15 • To reduce the risk of GVHD, BM is recommended for MMUDs for whom a FLU, CY, low-dose TBI 1 ATG or FCC regimen 1 low-dose TBI seems appropriate outside prospective clinical trials (Table 1).…”

Section: Alternative Donor Transplantation In Idiopathic Aamentioning

confidence: 99%

Transplantation for bone marrow failure: current issues

Latour

2016

Hematology

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The preferred treatment of idiopathic aplastic anemia (AA) is allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA)-identical sibling donor. Transplantation from a well-matched unrelated donor (MUD) may be considered for patients without a sibling donor after failure of immunosuppressive therapy, as may alternative transplantation (mismatched, cord blood or haplo-identical HSCT) for patients without a MUD. HSCT may also be contemplated for congenital disorders in cases of pancytopenia or severe isolated cytopenia. Currently, HSCT aims are not only to cure patients but also to avoid long-term complications, notably chronic graft-versus-host disease (GVHD), essential for a good quality of life long term. This paper summarizes recent advances in HSCT for idiopathic and inherited AA disorders. The effect of age on current transplantation outcomes, the role of transplantation in paroxysmal nocturnal hemoglobinuria, and the prevention of GVHD are also discussed. Emerging strategies regarding the role of up-front unrelated donor and alternative donor HSCT in idiopathic AA, along with advances in the treatment of clonal evolution in Fanconi anemia, are also examined. Learning Objectives• Understand the indications for HSCT and issues informing choice of donor and treatment regimen for inherited and acquired marrow failure • Recognize evolving strategies to minimize GVHD and longterm complications of HSCT for AA • Describe experimental approaches addressed in clinical trials for AA. Upfront matched unrelated HSCT and alternative HSCTs (mismatched unrelated, cord blood or haplo-identical) in AA are experimental procedures best tested within clinical trials

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“…28 The current EBMT protocol comprises fludarabine 30 mg/m 2 3 4, CY 30 mg/kg 3 4, ATG, and TBI 2 Gy, with CSA alone as postgraft immunosuppression. 29 Because CBT is still experimental, it should only be performed as part of prospective clinical study.…”

Section: Treatment Options If There Is No Suitable Udmentioning

confidence: 99%

Management of the refractory aplastic anemia patient: what are the options?

Marsh

Kulasekararaj

2013

Blood

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Refractory aplastic anemia (AA) is defined as a lack of response to first-line immunosuppressive therapy (IST) with antithymocyte globulin and cyclosporin and is manifested as persistence of severe cytopenias at 6 months after IST. Although supportive care is critical for AA patients, it is of paramount importance for refractory disease in view of the longer duration of pancytopenia and susceptibility to life-threatening infections due to IST. Improvements in supportive care have largely contributed to better outcome over the past 2 decades, with 5-year overall survival reaching 57% during 2002 to 2008 for patients with AA unresponsive to initial IST. Exclusion of hypocellular myelodysplastic syndrome and constitutional BM failure masquerading as apparent idiopathic AA should be done in conjunction with centers of excellence. Hematopoietic stem cell transplantation is indicated if refractory AA patients are fit and have a suitably matched donor, either a sibling (>40-50 years) or unrelated donor. Patients lacking a fully matched donor should be considered for a second course of antithymocyte globulin plus cyclosporin, although response in the refractory setting is only ∼30% to 35%. Response may also occur with alemtuzumab or the thrombopoietin mimetic eltrombopag in refractory AA. The emerging data for alternate donor (cord or haploidentical) transplantation in AA has provided additional therapeutic choices to consider in refractory disease.

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Cord blood transplantation in aplastic anemia

Cited by 38 publications

References 9 publications

Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study

Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study

Transplantation for bone marrow failure: current issues

Management of the refractory aplastic anemia patient: what are the options?

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