Comparison of Haploidentical Hematopoietic Stem Cell Transplantation and Immunosuppressive Therapy for the Treatment of Acquired Severe Aplastic Anemia in Pediatric Patients

Zhang, Yuan; Guo, Zhi; Liu, Xiao Dong; He, Xu; Yang, Kai; Chen, Peng; Chen, Hui‐Ren

doi:10.1097/mjt.0000000000000366

Cited by 27 publications

(13 citation statements)

References 13 publications

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“…A recent finding suggests that the OS of haplo-HSCT in pediatric SAA is comparable to IST. 29 Long-term FFS of the upfront therapy group in our trial was much better than that of IST mentioned above.…”

Section: Discussionmentioning

confidence: 49%

Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia

Zhang

Wang

et al. 2016

Bone Marrow Transplant

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Techniques for haploidentical hematopoietic stem cell transplantation (haplo-HSCT) to treat severe aplastic anemia (SAA) have recently improved, but no protocol has been evaluated in a large number of pediatric patients. Fifty-two children with SAA received haplo-HSCT in our center. The treatment protocol used G-CSF-primed bone marrow with G-CSF-mobilized PBSCs without in vitro T-cell depletion. The conditioning regimen included busulfan/cyclophosphamide and antithymocyte globulin. Fifty-one patients achieved primary engraftment; one child died of regimen-related toxicity on the day +1. Secondary graft failure occurred in three patients. The cumulative incidences of aGVHD grade II-IV and grade III-IV were 39.2±0.5 and 13.7±0.2%, respectively. The cumulative incidence of cGVHD was 34.2±0.5%. The 3-year overall and failure-free survival rates were 84.5±5.0 and 82.7±5.2%, respectively, with a median follow-up time of 744.5 days (100-3294) for surviving patients. The Eastern Cooperative Oncology Group score was the only predictor of overall and failure-free survival rates. Clinical outcomes were similar between the upfront and salvage group. This result suggests that both newly diagnosed and refractory pediatric SAA patients benefit from haplo-HSCT, especially when patients are in good general condition. Therefore, haplo-HSCT might be an alternative therapy for pediatric SAA patients without HLA-matched sibling donors.

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Section: Discussionmentioning

confidence: 49%

Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia

Zhang

Wang

et al. 2016

Bone Marrow Transplant

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“…Our results show no significant difference in 3-year OS between the frontline IST and frontline haplo-HSCT groups (79.3 § 7.5% versus 85.0 § 8.0%; x 2 = .110; P = .740); however, FFS was significantly higher in the haplo-HSCT group compared with the IST group (80.0 § 8.9% versus 35.9 § 10.9%; x 2 = 4.089; P = .043) and EFS was significantly higher in the haplo-HSCT group compared with the salvage HSCT group (80.0 § 8.9% versus 41.7 § 14.2%; x 2 = 3.992; P = .046). Zhang et al [18] compared the prognosis of 28 children with SAA treated with haplo-HSCT and IST and found OS rates of 66.7% and 70.0%, respectively (P not significant). These results are similar to our present findings.…”

Section: Discussionmentioning

confidence: 99%

Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor

Yang

Yuan

et al. 2019

Biology of Blood and Marrow Transplantation

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A B S T R A C TWe compared the outcomes of immunosuppressive therapy (IST) with those of T cell-replete haploidentical donor hematopoietic stem cell transplantation (haplo-HSCT) in children and adolescents with severe aplastic anemia (SAA). The medical records of 49 patients with SAA who received frontline IST (n = 29) or frontline haplo-HSCT (n = 20) between 2012 and 2016 were analyzed retrospectively. Fourteen patients responded after the first IST, and 1 patient responded after the second IST in the frontline IST group; 12 patients underwent salvage HSCT after IST failure. Sixteen of the 20 patients who underwent frontline haplo-HSCT survived without treatment failure. The 3-year overall survival of the frontline IST group was comparable to that of the frontline haplo-HSCT group (79.3 § 7.5% versus 85.0 § 8.0%; x 2 = 0.110; P = .740). The 3-year failure-free survival was lower in the frontline IST group compared with the frontline haplo-HSCT group (35.9 § 10.9% versus 80.0 § 8.9%; x 2 = 4.089; P = .043). Five patients of the IST group who underwent salvage HSCT achieved long survival without event. The event-free survival was lower in the salvage HSCT group compared with the haplo-HSCT group (41.7 § 14.2% versus 80.0 § 8.9%; x 2 = 3.992; P = .046), and the incidences of acute GVHD, grade II-IV acute GVHD, chronic GVHD, and severe infection were comparable between the 2 groups. Our results suggest that frontline haplo-HSCT may be a better treatment than IST for children and adolescents with SAA who lack an HLA age-matched familial donor.

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“…HLA-haploidentical relative HSCT has been greatly improved during the past decade 10 , 11 . Many cases of mismatched related donor transplants in SAA patients have been reported 9 , 12 – 21 . The latest 2015 British AA guidelines recommended alternative donor HSCT as an experimental treatment for patients who lacked suitably matched donors 1 .…”

Section: Introductionmentioning

confidence: 99%

Optimal donor for severe aplastic anemia patient requiring allogeneic hematopoietic stem cell transplantation: A large-sample study from China

Zeng

Wang

et al. 2018

Sci Rep

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HLA-haploidentical hematopoietic stem cell transplantation (HSCT) may be an option for severe aplastic anemia (SAA) patients. However, to date, no large-sample studies have been performed to determine which types of SAA patients are suitable for HLA-haploidentical HSCT. We retrospectively studied 189 consecutive patients with SAA who underwent HLA-identical or HLA-haploidentical HSCT at seven transplant centers in China. Propensity score matching (PSM) was applied in this study to reduce the influence of potential confounders. The 5-year overall survival (OS) rate was 72.0% in the HLA-haploidentical group and 76.5% in the HLA-identical group. The median time to achieve engraftment and the incidence of acute GVHD/chronic GVHD were not significantly different between the two groups. In the subgroup analysis, the outcome of patients older than 40 years in the HLA-haploidentical group was significantly poorer than that of patients younger than 40 years in the same group and that of patients older than 40 years in the HLA-identical group. Based on the above results, we suggest that HLA-haploidentical relative HSCT should be considered as a valid alternative option for patients younger than 40 years with SAA for whom no matched sibling donor is available.

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Comparison of Haploidentical Hematopoietic Stem Cell Transplantation and Immunosuppressive Therapy for the Treatment of Acquired Severe Aplastic Anemia in Pediatric Patients

Cited by 27 publications

References 13 publications

Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia

Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia

Comparison of Outcomes of Frontline Immunosuppressive Therapy and Frontline Haploidentical Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia Who Lack an HLA-Matched Sibling Donor

Optimal donor for severe aplastic anemia patient requiring allogeneic hematopoietic stem cell transplantation: A large-sample study from China

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