Allogeneic hematopoietic stem cell transplantation for aplastic anemia with pre‐transplant conditioning using fludarabine, reduced‐dose cyclophosphamide, and low‐dose thymoglobulin: A KSGCT prospective study

Kako, Shinichi; Kanda, Yoshinobu; Onizuka, Makoto; Aotsuka, Nobuyuki; Usuki, Kensuke; Tachibana, Takayoshi; Kobayashi, Takeshi; Kato, Jun; Yano, Shingo; Shimizu, Hiroaki; Shono, Katsuhiro; Tanaka, Masatsugu; Tsukamoto, Shokichi; Mori, Takehiko; Yamazaki, Etsuko; Najima, Yuho; Hangaishi, Akira; Hoshino, Takumi; Watanabe, Reiko; Matsumoto, Kenji; Okamoto, Shinichiro

doi:10.1002/ajh.25693

Cited by 9 publications

(6 citation statements)

References 28 publications

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“…A relatively high incidence of MC has been reported in patients receiving HSCT for AA [11,14,16], and MC has been found to be associated with GF [12,20]. In the present study, the probability of SGF was associated with the time that had elapsed from HSCT, as well as the number of RHCs present in the recipient at MC onset.…”

Section: Discussionsupporting

confidence: 53%

“…However, for patients in whom MC occurs early after transplantation and the RHCs reach level 3, the disease progresses quickly, and timely treatment is therefore of the essence. The impact of allo-HSCT and MC has been found to be associated with the conditioning regimen and donor source [11,20]. The present study suggested that donor/recipient sex-mismatching was a risk factor for MC, especially male donor to female patients in MSD-HSCT.…”

Section: Discussionmentioning

confidence: 56%

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Mixed chimerism after allogeneic hematopoietic stem cell transplantation for severe aplastic anemia

Zhang

et al. 2021

Hematology

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Background: Mixed chimerism (MC) frequently occurs in patients with severe aplastic anemia (SAA) after allogeneic hematological stem cell transplantation (allo-HSCT). Methods: A retrospective study on 287 patients with SAA who underwent allo-HSCT between October 2012 and January 2020 was conducted to explore the outcomes, risk factors and treatment options for MC. Among 287 AA patients who excluded Fanconi anemia (FA), Congenital dyskeratosis (DKC), Paroxysmal nocturnal hemoglobinuria (PNH), etc.112 underwent matched sibling donor (MSD)-HSCT, 91 matched unrelated donor-HSCT and 84 haploidentical-HSCT. Patients were divided into the following 4 groups: group 1: Donor chimerism (DC); group 2: MC without cytopenia; group 3: MC with cytopenia; group 4: secondary graft failure (SGF). Results: Compared with the other three groups, SGF predicted a poor prognosis of SAA (P< 0.001). In addition, SGF was associated with the early (within 3 months after transplantation) presence of MC and the high levels of MC. Uni-and multivariate logistic regression analysis showed that donor/recipient sex-mismatching and CTX + ATG regimen were high-risk factors for MC. Of note, in MC patients with cytopenia (group 3), the effective response rate reached 55% (6/11) following enhanced immunosuppression combined with cellular therapy, while only one of the four was effective who received enhanced immunosuppression alone. Conclusion: SGF was associated with poor prognosis, early presence of MC and increased levels of recipient chimerism. The donor/recipient sex-mismatching and CTX + ATG regimen based MSD-HSCT were risk factors for MC. Cellular therapy could improve the effective response rate of patients with progressive MC.

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Section: Discussionsupporting

confidence: 53%

Section: Discussionmentioning

confidence: 56%

Mixed chimerism after allogeneic hematopoietic stem cell transplantation for severe aplastic anemia

Zhang

et al. 2021

Hematology

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“…As a purine analog, Flu bears potential immune and lymphocyte clearance effects and has wide applicability and low toxicity. It has been applied to the SAA transplantation conditioning regimen and has achieved a substantial curative effect [24, 25]. Simultaneously, combined ATG therapy has achieved the effect of long-term removal of T lymphocytes in vivo.…”

Section: Discussionmentioning

confidence: 99%

Busulfan for Allogeneic Hematopoietic Stem Cell Transplantation in Children with Severe Aplastic Anemia: A Retrospective Study

Si¹,

Luo²,

Qin³

et al. 2023

Acta Haematol

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Introduction This retrospective study aimed to compare a range of conditioning regimens in children with severe aplastic anemia (SAA) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) at the Seventh Medical Center of PLA General Hospital between January 2008 and June 2017. Methods Patients were categorized into the Bu (Bu + Flu + Cy + ATG-F regimen) and control (Flu + Cy + ATG-F) groups, with a median follow-up time after HSCT of 3.5 (range, 3.1–6.2) and 3.7 (3.2–5.9) years in the Bu and control groups, respectively. Results No differences were observed between the two groups regarding the median time of peripheral blood neutrophil and platelet engraftment (P = 0.538 and P = 0.491); the 28-day engraftment rates of neutrophils were similar (P = 0.199) although higher for platelets with Bu (P = 0.044). Additionally, graft failure was 0% and 20.0% in the Bu and control groups, respectively (P = 0.004). In both groups, the incidence of grades ⅡI–Ⅳ (or grades Ⅱ–Ⅳ) acute graft-versus-host disease (GVHD) and chronic GVHD was not significantly different (P > 0.05). Moreover, the 3-year overall survival and failure-free survival did not show significant differences (P = 0.670 and P = 0.908). Discussion In children with SAA undergoing allo-HSCT, conditioning regimen with Bu + Flu + Cy + ATG-F is capable of enhancing the myeloablation effect, promoting donor hematopoietic stem cell engraftment, and reducing the graft failure rate. Furthermore, it does not increase the incidence of complications, including GVHD.

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“…Pooled aGVHD and cGVHD were 55 and 33%, respectively. Although most of these GVHD were well-managed and not lethal, they certainly caused a longer hospitalization period, increased medical burden, and reduced the quality of life (38,39).…”

Section: Discussionmentioning

confidence: 99%

Comparisons Between Frontline Therapy and a Combination of Eltrombopag Plus Immunosuppression Therapy and Human Leukocyte Antigen-Haploidentical Hematopoietic Stem Cell Transplantation in Patients With Severe Aplastic Anemia: A Systematic Review

et al. 2021

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Background and Aims: This study aimed at comparing the efficacy and safety of eltrombopag (EPAG) plus immunosuppressive therapies (ISTs) and haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in the frontline treatment for severe aplastic anemia (SAA) patients.Methods: Four electronic databases and Clinicaltrials.gov were comprehensively searched from January 2010 to August 2020. Studies that aimed at evaluating the efficacy and safety of EPAG+IST or haplo-HSCT in SAA patients were included. One-/2-year overall survival (OS), complete response (CR), and overall response rates (ORRs) were indirectly compared between EPAG+IST and haplo-HSCT.Results: A total of 447 patients involved in 10 cohort studies were found to be eligible for this study. A narrative synthesis was performed due to lack of data directly comparing the outcome of EPAG+IST and haplo-HSCT. Consistent with the analysis results in the whole population, subgroup analyses in the age-matched population showed that there was no significant difference in ORR between EPAG+IST and haplo-HSCT groups. However, the CR rate was lower in the EPAG+IST group when compared with the haplo-HSCT group. The incidence rate of clonal evolution/SAA relapse ranged at 8–14 and 19–31% in the EPAG+IST group but not reported in the haplo-HSCT group. The incidence rate for acute graft vs. host disease (aGVHD) and chronic graft vs. host disease (cGVHD) ranged at 52–57 and 12–67%, respectively, for the haplo-HSCT group. The main causes of deaths were infections in the EPAG+IST group, and GVHD and infections in the haplo-HSCT group.Conclusion: EPAG+IST has a comparable ORR and 1-/2-year OS but lower CR rate when indirectly compared with haplo-HSCT in the frontline treatment of patients with SAA. Patients treated with haplo-HSCT may exhibit a high incidence of GVHD, whereas patients treated with EPAG+IST may experience more relapses or clone evolution.

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Allogeneic hematopoietic stem cell transplantation for aplastic anemia with pre‐transplant conditioning using fludarabine, reduced‐dose cyclophosphamide, and low‐dose thymoglobulin: A KSGCT prospective study

Cited by 9 publications

References 28 publications

Mixed chimerism after allogeneic hematopoietic stem cell transplantation for severe aplastic anemia

Mixed chimerism after allogeneic hematopoietic stem cell transplantation for severe aplastic anemia

Busulfan for Allogeneic Hematopoietic Stem Cell Transplantation in Children with Severe Aplastic Anemia: A Retrospective Study

Comparisons Between Frontline Therapy and a Combination of Eltrombopag Plus Immunosuppression Therapy and Human Leukocyte Antigen-Haploidentical Hematopoietic Stem Cell Transplantation in Patients With Severe Aplastic Anemia: A Systematic Review

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