Unrelated cord blood transplantation in aplastic anemia: is anti-thymocyte globulin indispensable for conditioning?

Kudo, Kazuko; Muramatsu, Hideki; Narita, Atsushi; Yoshida, Nao; Kobayashi, Ryōji; Yabe, Hiromasa; Inoue, Masami; Hara, Jun; Kounami, Shinji; Inagaki, Jiro; Hashii, Yoshiko; Kato, Koji; Tabuchi, Ken; Kojima, Seiji

doi:10.1038/bmt.2017.169

Cited by 17 publications

(6 citation statements)

References 12 publications

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“…Cord blood transplantation is probably inferior to HLAhaploidentical marrow grafts because of the low cell dose infused, the high risk for graft rejection, and delayed engraftment/immune recovery. 65,66 Nontransplant IST…”

Section: Hla-haploidentical Related Marrow Transplantationmentioning

confidence: 99%

Severe aplastic anemia: allogeneic bone marrow transplantation as first-line treatment

2018

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Treatment of severe aplastic anemia has improved significantly over the past 4 decades. This review will summarize the key areas of progress in the use of allogeneic hematopoietic cell transplantation and nontransplant immunosuppressive therapy (IST) for the treatment of aplastic anemia and then summarize the recommendations for first-line treatment. Based on recent data, we argue that guidelines for the initial treatment of patients with newly diagnosed severe aplastic anemia require revision. At the time of diagnosis, before beginning treatment, HLA typing should be done to identify a marrow donor among family members or in the unrelated donor registries, and a marrow transplant should be considered first-line therapy. The priority order of donor source for bone marrow transplantation is: (1) HLA-identical sibling, (2) HLA-matched unrelated donor, and (3) HLA-haploidentical donor if an HLA-matched unrelated donor is not rapidly available. Each of these donor marrow sources may be preferable to nontransplant IST. We make this recommendation because of the long-term persistent risk for disease relapse and secondary myelodysplastic syndrome or acute myeloid leukemia with the use of nontransplant IST for patients with aplastic anemia. In contrast, marrow transplantation is associated with high cure rates of aplastic anemia and a relatively low risk for graft-versus-host disease, with many patients now living for decades without the risk for disease recurrence or the development of clonal disorders. Implementation of this first-line treatment strategy will provide patients with severe aplastic anemia the best chance of long-term disease-free survival.

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Section: Hla-haploidentical Related Marrow Transplantationmentioning

confidence: 99%

Severe aplastic anemia: allogeneic bone marrow transplantation as first-line treatment

2018

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“…Limitations of this study include its small cohort, retrospective nature, and short duration of follow-up. However, the other specific regimens described in previous studies of UCBT in patients with AA were also based on a small number of cases [12,13,15,19]. Although this study investigated only 6 patients, findings of their successful outcomes might be helpful when considering the optimal regimen for UCBT, especially in adult patients with AA.…”

Section: Discussionmentioning

confidence: 97%

“…A recent study using registry data from pediatric patients with AA suggested a negative impact of ATG in conditioning for UCBT, because primary GF was observed in 8 of 15 patients receiving the ATG regimen [19]. The use of ATG in conditioning might reduce donor T cells, which would adversely affect achievement of sustained engraftment with complete donor chimerism.…”

Section: Discussionmentioning

confidence: 99%

Umbilical Cord Blood Transplantation Using Reduced-Intensity Conditioning without Antithymocyte Globulin in Adult Patients with Severe Aplastic Anemia

Ochi

Onishi

Nasu

et al. 2019

Biology of Blood and Marrow Transplantation

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A B S T R A C T Umbilical cord blood transplantation (UCBT) is a possible option for patients with aplastic anemia (AA) without a related or unrelated HLA-matched donor, particularly if immunosuppressive therapy (IST) has failed or transplantation is urgently needed. However, a higher rate of graft failure after UCBT remains a major problem, and the optimal conditioning regimen for stable engraftment after UCBT has not been established. Here we investigated 6 adult patients with AA who underwent UCBT using a reduced-intensity conditioning (RIC) regimen comprising fludarabine 125 mg/m 2 , cyclophosphamide 120 mg/kg, and 4 Gy of total body irradiation (Flu/CY/TBI4Gy) without antithymocyte globulin (ATG). Five patients underwent UCBT after IST failure, and 1 patient underwent UCBT as a first-line treatment due to a fulminant clinical finding of a neutrophil count of 0, despite granulocyte colony-stimulating factor administration. Regarding graft-versus-host disease (GVHD) prophylaxis, 2 patients received tacrolimus plus short-term methotrexate and 4 patients received tacrolimus plus mycophenolate mofetil, and all patients achieved sustained engraftment of both neutrophils and platelets, at a median of 17.5 days (range, 14 to 37 days) and 38.5 days (range, 31 to 86 days), respectively, with complete donor chimerism confirmed in all patients at a median of 14 days (range, 14 to 32 days). Three patients developed grade II acute GVHD (aGVHD), but grade III/IV aGVHD was not observed, whereas 4 patients developed chronic GVHD involving only skin. At the time of this report, all 6 patients were alive without the need for blood transfusion, at a median follow-up of 16 months (range, 12 to 131 months). Although further study is needed, our findings suggest that conditioning with Flu/CY/TBI4Gy without ATG might allow stable engraftment in UCBT for adults with AA.

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“…Although early studies on UCBT in patients with acquired AA showed limited success, new studies have shown promising results. Kudo et al, demonstrated the excellent OS in patients treated with the conditioning regimen comprising fludarabine, cyclophosphamide or melphalan, and low dose irradiation without anti-thymocyte globulin 17 . Similar results were demonstrated by the French group in a prospective study using conditioning with FLU, CY and 2 Gy of TBI with ATG; this group reported a 2-year OS rate of 81% and engraftment of 88% 18 .but results from previous studies are not encouraging.…”

Section: Second-line Treatment Of Acquired Saamentioning

confidence: 99%

Brazilian Consensus Meeting on Pediatric Hematopoietic Stem Cell Transplantation for Acquired Aplastic Anemia and Inherited Bone Marrow Failure Syndromes

Darrigo¹,

Loth²,

Scheinberg³

et al. 2021

jbmtct

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Unrelated cord blood transplantation in aplastic anemia: is anti-thymocyte globulin indispensable for conditioning?

Cited by 17 publications

References 12 publications

Severe aplastic anemia: allogeneic bone marrow transplantation as first-line treatment

Severe aplastic anemia: allogeneic bone marrow transplantation as first-line treatment

Umbilical Cord Blood Transplantation Using Reduced-Intensity Conditioning without Antithymocyte Globulin in Adult Patients with Severe Aplastic Anemia

Brazilian Consensus Meeting on Pediatric Hematopoietic Stem Cell Transplantation for Acquired Aplastic Anemia and Inherited Bone Marrow Failure Syndromes

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