Second allogeneic hematopoietic stem cell transplantation in children with severe aplastic anemia

Kudo, Kazuko; Muramatsu, Hideki; Yoshida, Nao; Kobayashi, Ryōji; Yabe, Hiromasa; Tabuchi, Ken; Katô, Kôji; Koh, Katsuyoshi; Takahashi, Yoshiyuki; Hashii, Yoshiko; Kawano, Yoshifumi; Inoue, Masami; Cho, Y; Sakamaki, Hisashi; Kawa, Keisei; Suzuki, Ritsuro; Kojima, Seiji

doi:10.1038/bmt.2015.153

Cited by 15 publications

(8 citation statements)

References 18 publications

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“…The OS after the second HSCT in our group was 64%, while the second HSCT survival rate reported by the few small studies in non-malignant diseases varies from 77 to 90% [12][13][14]. The differences in OS between the studies may be explained by heterogeneous IEI included and patient selection for a second HSCT.…”

Section: Discussioncontrasting

confidence: 52%

“…One of important doubts remains the timing of a second HSCT. Indeed, a longer period between two transplants might result in recovery from toxic effects of the first transplant and better outcome [13,26,27]. However, persistent BM aplasia and secondary immunodeficiency lead to increased risk of infections, therefore an individual decision based on the patient's status must be made in every case.…”

Section: Discussionmentioning

confidence: 99%

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Second allogeneic hematopoietic stem cell transplantation in patients with inborn errors of immunity

Laberko

Sultanova

Idarmacheva

et al. 2022

Bone Marrow Transplant

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Graft failure (GF) remains a serious issue of hematopoietic stem cell transplantation (HSCT) in inborn errors of immunity (IEI). Second HSCT is the only salvage therapy for GF. There are no uniform strategies for the second HSCTs and limited data are available on the second HSCT outcomes. 48 patients with various IEI received second allogeneic HSCT from 2013 to 2020. Different conditioning regimens were used, divided into two main groups: containing myeloablative doses of busulfan/treosulfan (n = 19) and lymphoid irradiation 2–6 Gy (n = 22). Irradiation-containing conditioning was predominantly used in suspected immune-mediated rejection of the first graft. Matched unrelated donor was used in 28 patients, mismatched related in 18, and matched related in 1. 35 patients received TCRαβ/CD19 graft depletion. The median follow-up time was 2.4 years post-HSCT. One patient died at conditioning. The OS was 0.63 (95% CI: 0.41–0.85) after busulfan/treosulfan and 0.68 (95% CI: 0.48–0.88) after irradiation-based conditioning, p = 0.66. Active infection at HSCT significantly influenced OS: 0.43 (95% CI: 0.17–0.69) versus 0.73 (95% CI: 0.58–0.88) without infection, p = 0.004. The cumulative incidence of GF was 0.15 (95% CI: 0.08–0.29). To conclude, an individualized approach is required for the second HSCT in IEI. Low-dose lymphoid irradiation in suspected immune-mediated GF may be a feasible option.

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Section: Discussioncontrasting

confidence: 52%

Section: Discussionmentioning

confidence: 99%

Second allogeneic hematopoietic stem cell transplantation in patients with inborn errors of immunity

Laberko

Sultanova

Idarmacheva

et al. 2022

Bone Marrow Transplant

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“…If there is no MSD, the use of MUD-HSCT or IST should be decided according to whether there is a suitable donor, a graft resource, the patient’s status, the opinions of the pediatric patient’s family and so on. However, whether MSD-HSCT or MUD-HSCT is used, GF may occur and is one of the most serious complications after transplantation 6 . During 1998 and 2012, among 123 patients with HSCT in China, the incidence rate of GF was approximately 2.4% 7 .…”

Section: Discussionmentioning

confidence: 99%

Successful outcomes of second hematopoietic stem cell transplantation for graft failure in pediatric patients with severe aplastic anemia

Gui

et al. 2022

Sci Rep

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Severe aplastic anemia (SAA) is a life-threatening hematological disorder. The major therapies include matched sibling donor (MSD)- hematopoietic stem cell transplantation (HSCT), matched unrelated donor (MUD)-HSCT and immunosuppressive therapy (IST). However, there are many problems that can occur after HSCT, and graft failure (GF) is one of the most serious complications. To find an effective treatment, we analyzed 10 cases of second HSCT to treat SAA pediatric patients who suffered from GF and concluded that second haploidentical family donors HSCT is an effective treatment. Moreover, adding a small dose of busulfan or 2 ~ 3 Gy total body irradiation (TBI) in nonmyeloablative regimens (NMAs) can promote the engraftment. Although the study also showed that PBSCs, as a source of stem cells, can promote the implantation of neutrophil cells, due to small sample size, more research is still needed.

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“…Recently, FLU-based conditioning regimens for SAA patients receiving UD HSCT have shown promising results [7,[13][14][15][16][17], and a 95% failure-free survival rate has been reported with a FLU, Cy, and alemtuzumab conditioning regimen [16]. However, the exact dose of each conditioning medication has not yet been fully defined.…”

Section: Discussionmentioning

confidence: 99%

Improved Outcome of a Reduced Toxicity-Fludarabine, Cyclophosphamide, plus Antithymocyte Globulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia: Comparison of 2 Multicenter Prospective Studies

Kang

Hong

Lee

et al. 2016

Biology of Blood and Marrow Transplantation

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Hematopoietic stem cell transplantation (HSCT) is a curative therapy for severe aplastic anemia (SAA); however, the optimal conditioning regimen for HSCT with an unrelated donor has not yet been defined. A previous study using a fludarabine (FLU), cyclophosphamide (Cy), and antithymocyte globulin (ATG) conditioning regimen (study A: 50 mg/kg Cy once daily i.v. on days -9, -8, -7, and -6; 30 mg/m(2) FLU once daily i.v. on days -5, -4, -3, and -2; and 2.5 mg/kg of ATG once daily i.v. on days -3, -2, and -1) demonstrated successful engraftment (100%) but had a high treatment-related mortality rate (32.1%). Therefore, given that Cy is more toxic than FLU, we performed a new phase II prospective study with a reduced-toxicity regimen (study B: 60 mg/kg Cy once daily i.v. on days -8 and -7; 40 mg/m(2) FLU once daily i.v. on days -6, -5, -4, -3, and -2; and 2.5 mg/kg ATG once daily i.v. on 3 days). Fifty-seven patients were enrolled in studies A (n = 28) and B (n = 29), and donor type hematologic recovery was achieved in all patients in both studies. The overall survival (OS) and event-free survival (EFS) rates of patients in study B was markedly improved compared with those in study A (OS: 96.7% versus 67.9%, respectively, P = .004; EFS: 93.3% versus 64.3%, respectively, P = .008). These data show that a reduced-toxicity conditioning regimen with FLU, Cy, and ATG may be an optimal regimen for SAA patients receiving unrelated donor HSCT.

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Second allogeneic hematopoietic stem cell transplantation in children with severe aplastic anemia

Cited by 15 publications

References 18 publications

Second allogeneic hematopoietic stem cell transplantation in patients with inborn errors of immunity

Second allogeneic hematopoietic stem cell transplantation in patients with inborn errors of immunity

Successful outcomes of second hematopoietic stem cell transplantation for graft failure in pediatric patients with severe aplastic anemia

Improved Outcome of a Reduced Toxicity-Fludarabine, Cyclophosphamide, plus Antithymocyte Globulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia: Comparison of 2 Multicenter Prospective Studies

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