Comparable Outcomes of Allogeneic Peripheral Blood versus Bone Marrow Hematopoietic Stem Cell Transplantation in Major Thalassemia: A Multivariate Long-Term Cohort Analysis

Ghavamzadeh, Ardeshir; Kasaeian, Amir; Rostami, Tahereh; Kiumarsi, Azadeh

doi:10.1016/j.bbmt.2018.09.026

Cited by 14 publications

(11 citation statements)

References 36 publications

(40 reference statements)

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“…We speculate that use of peripheral blood stem cells may result in a higher incidence of aGVHD than bone marrow application. Ghavamzadeh et al concluded that peripheral blood stem cell transplantation is associated with higher incidence rates of aGVHD and cGVHD compared with bone marrow utilization, but blood cells are easier to harvest and associated with lower incidence of GR and shorter times to neutrophil and platelet engraftment, resulting in similar OS outcomes to bone marrow transplantation (27).…”

Section: Discussionmentioning

confidence: 99%

Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts

Huang¹,

Qu²,

Li³

et al. 2021

Transl Pediatr

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Background: Until now, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only effective method to cure Thalassemia major. However, it has not been determined whether similar results can be obtained with the same conditioning regimen for both fully-matched and mismatched donors grafts.We hypothesized that using modified NF-08-TM conditioning regimen could achieve similar results for both fully and mismatched donors grafts.Methods: This retrospective cohort study included patients with β-thalassemia major who underwent HSCT with modified NF-08-TM conditioning regimen at Guangzhou Women and Children's Medical

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Section: Discussionmentioning

confidence: 99%

Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts

Huang¹,

Qu²,

Li³

et al. 2021

Transl Pediatr

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“…First is the use of G-BM&PBSCs as the graft source in the present study. Ghavamzadeh et al [3] reported that patients receiving PBSCT have a shorter median time for neutrophil and platelet engraftments compared with the patients receiving BMT (3 days and 11 days shorter, respectively), and the GR rate is lower (5.7% versus 18.4%), showing higher incidence of acute GVHD (33.3% versus 50.8%) and chronic GVHD (26.1% versus 51.4%). Zhao et al [5] reported that coinfusion of G-BM&PBSC graft resulted in similar engraftment kinetics and lower rates of grade III-IV acute GVHD compared with PBSC grafts.…”

Section: Discussionmentioning

confidence: 99%

“…The grafts used in the present study were G-BM&PBSC grafts, which were intended to be used to combine the advantages of both elements. PBSCs have more CD34 + cells and T cells than steady-state BM, and they have been shown to accelerate engraftment and decrease GR [3,21]. Morton et al [22] reported that G-BM transplantation leads to reduced severity of acute GVHD and less subsequent chronic GVHD, and engraftment is comparable to that of PBSCT.…”

Section: Discussionmentioning

confidence: 99%

“…Traditionally, bone marrow (BM) is the standard stem cell source for transplantation in patients with TM, with cord blood and peripheral blood stem cells (PBSCs) as alternative sources. Although overall survival (OS) and TFS are similar in recipients of PBSC transplantation (PBSCT) and recipients of BM transplantation (BMT), PBSCT is associated with earlier engraftment, a lower rate of graft rejection (GR), greater convenience, and higher incidences of acute and chronic GVHD compared with BMT [3]. In the Beijing protocol for haploidentical hematopoietic stem cell transplantation, the coinfusion of G-CSF-mobilized BM and G-CSF-mobilized PBSCs (G-BM&PBSCs) at a 1:1 ratio with mononuclear cells (MNCs) is a standard procedure [4,5].…”

Section: Introductionmentioning

confidence: 99%

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G-CSF-Mobilized Blood and Bone Marrow Grafts as the Source of Stem Cells for HLA-Identical Sibling Transplantation in Patients with Thalassemia Major

Luo

Zhang

et al. 2019

Biology of Blood and Marrow Transplantation

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As an inherited anemia, thalassemia major (TM) is currently only curable with allogeneic hematopoietic stem cell transplantation (allo-HSCT). Here we report an allo-HSCT protocol for patients with TM who received a combination of granulocyte colony-stimulating factor-primed bone marrow and peripheral blood stem cells (G-BM & PBSCs) from a matched sibling donor (MSD). The conditioning regimen consisted of i.v. busulfan, cyclophosphamide, fludarabine, and antithymocyte globulin. Chimerism analysis was performed for all patients. Immunosuppressive treatment was terminated if rejection was suspected, and donor lymphocyte infusion was administered once no response was observed. A total of 184 patients with TM were enrolled in the study between July 2007 and July 2018. The cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) was 13.1%, and that of moderate or severe chronic GVHD was 5.7%. The cumulative incidence of graft rejection was .6%. In the total cohort, the 3-year overall survival, thalassemia-free survival, and GVHD-free, relapse-free survival were 97.8%, 97.3%, and 89.5%, respectively. Collectively, our results indicate that G-BM & PBSCs from an MSD is be a good stem cell source for patients with TM undergoing allo-HSCT.

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“…3,4 Based on current guidelines, allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment option for eligible TM patients and can improve long-term survival. [5][6][7][8][9] It is reported that more than 90% of patients with thalassemia major survive after HSCT and disease-free survival (DFS) is about 80%. 6 Hepatic brosis is a common complication in transfusion-dependent thalassemia patients.…”

Section: Introductionmentioning

confidence: 99%

The Effect of Bone Marrow-Derived Mesenchymal Stem Cells Co-transplantation with Hematopoietic Stem Cells on Liver Fibrosis Alleviation and Survival in Patients with Class III β-thalassemia Major

Rostami

Kasaeian

Maleki

et al. 2021

Preprint

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Background: Hepatic fibrosis is a common complication in transfusion-dependent thalassemia patients. Data on the co-transplantation of mesenchymal stem cells (MSCs) with hematopoietic stem cells (HSCs) in beta-thalassemia major (BTM) patients are scarce. Therefore, we aimed to evaluate the effect of co-transplantation of bone marrow-derived MSC with HSCs on the liver fibrosis alleviation and transplant outcomes in class III BTM.Methods: Between April 1998 and January 2017, a total of 224 consecutive patients with class III BTM underwent allogeneic HSCT in the Research Institute for Oncology, Hematology and Cell Therapy, Tehran University of Medical Sciences, Tehran, Iran. To assess liver fibrotic changes after transplantation, 47 patients participated in the MSC plus HSC group and 30 patients in the HSC only group at the end of the follow-up period. All patients underwent laboratory tests, especially serum ferritin and liver function testing, hepatic T2* MRI, liver biopsy, and FibroScan before and two years after transplantation. Kaplan–Meier curves were derived to determine survival, and were compared using the log-rank test. Repeated-measure, mixed-effect linear regression models were used to examine changes in liver fibrosis over time.Results: The 10-year OS rate was 71.84% in the mesenchymal group and 61.89% in the non-mesenchymal group (P-value = 0.294), while the 10-year TFS rate was 63.64% in the mesenchymal group and 52.78% in the non-mesenchymal group (P-value = 0.285). No significant difference was observed in the 10-year NRM, rejection rate, ANC engraftment, platelet engraftment, acute GvHD, and chronic GvHD between the two groups. In addition, the results of repeated-measure, mixed-effect linear regression models showed that none of the variables determining hepatic fibrosis had a significant difference between patients receiving MSCs and patients who did not receive MSCs.Conclusions: Based on the results of this study, the co-transplantation of bone marrow-derived MSCs and HSCs to patients with class III beta-thalassemia major could not significantly improve the liver fibrosis alleviation and transplantation outcomes, including OS, TFS, TRM, rejection rate, ANC engraftment, platelet engraftment, acute GvHD, and chronic GvHD.

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Comparable Outcomes of Allogeneic Peripheral Blood versus Bone Marrow Hematopoietic Stem Cell Transplantation in Major Thalassemia: A Multivariate Long-Term Cohort Analysis

Cited by 14 publications

References 36 publications

Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts

Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts

G-CSF-Mobilized Blood and Bone Marrow Grafts as the Source of Stem Cells for HLA-Identical Sibling Transplantation in Patients with Thalassemia Major

The Effect of Bone Marrow-Derived Mesenchymal Stem Cells Co-transplantation with Hematopoietic Stem Cells on Liver Fibrosis Alleviation and Survival in Patients with Class III β-thalassemia Major

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