A Novel Conditioning Regimen Improves Outcomes in b-Thalassemia Major Patients Using Unrelated Donor Peripheral Blood Stem Cell Transplantation

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4514-4514
Author(s):  
Li Chunfu ◽  
Liu Huayin ◽  
Qifa Liu
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Peripheral Blood Stem Cell ◽  
Thalassemia Major ◽  
Blood Stem Cell ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Blood Stem Cell Transplantation

Abstract Abstract 4514 We used a novel NF-08-TM transplant protocol in 100 consecutive patients with β-thalassemia major (TM), including 66 with allogeneic peripheral blood stem cell transplantation (PBSCT) from unrelated donors (UD) with well-matched human leukocyte antigens (HLAs) and 34 with hematopoietic stem cell transplantation (HSCT) from matched sibling donors (MSD). The median age at transplantation was 6.5 yo (range: 0.6–15 yo), and the ratio of male to female patients was 68:32. The median follow-up time was 24 months (range: 12–39 months). The 3-year overall survival (OS) and TM-free survival (TFS) were 92.4% and 90.9% in the UD-PBSCT group and 88.2% and 82.4% in the MSD-HSCT group. The cumulative incidences of graft rejection (GR) and grade II–IV acute graft-versus-host disease (aGVHD) were 1.5% and 10.6%, respectively, in the UD-PBSCT group and 6.1% and 5.9%, respectively, in the MSD-HSCT group. The cumulative incidence of transplant-related mortality (TRM) was 7.6% in the UD-PBSCT group and 11.8% in the MSD-HSCT group. In conclusion, UD-PBSCTs using the well-tolerated NF-08-TM protocol show similar results to MSD-HSCTs and can be used to treat b thalassemia patients in the absence of MSD. Disclosures: No relevant conflicts of interest to declare.

A novel conditioning regimen improves outcomes in β-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation

Blood ◽  
2012 ◽  
Vol 120 (19) ◽  
pp. 3875-3881 ◽  
Author(s):  
Chunfu Li ◽  
Xuedong Wu ◽  
Xiaoqing Feng ◽  
Yuelin He ◽  
Huaying Liu ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Peripheral Blood Stem Cell ◽  
Thalassemia Major ◽  
Blood Stem Cell ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Blood Stem Cell Transplantation

Abstract We used a novel NF-08-TM transplant protocol based on intravenous busulfan, cyclophosphamide, fludarabine, and thiotepa in 82 consecutive patients with β-thalassemia major (TM), including 52 with allogeneic peripheral blood stem cell transplantation (PBSCT) from unrelated donors (UDs) with well-matched human leukocyte antigens and 30 with hematopoietic stem cell transplantation (HSCT) from matched sibling donors (MSDs). The median age at transplantation was 6.0 years (range, 0.6-15.0 years), and the ratio of male-to-female patients was 56:26. The median follow-up time was 24 months (range, 12-39 months). The estimated 3-year overall survival and TM-free survival were 92.3% and 90.4% in the UD-PBSCT group and 90.0% and 83.3% in the MSD-HSCT group. The cumulative incidences of graft rejection and grades III-IV acute graft-versus-host disease were 1.9% and 9.6%, respectively, in the UD-PBSCT group and 6.9% and 3.6%, respectively, in the MSD-HSCT group. The cumulative incidence of transplant-related mortality was 7.7% in the UD-PBSCT group and 10.0% in the MSD-HSCT group. In conclusion, UD-PBSCTs using the well-tolerated NF-08-TM protocol show similar results to MSD-HSCTs and can be used to treat β-thalassemia patients in the absence of MSDs.

Comparative Analysis of Outcomes of Allogeneic Peripheral Blood Stem Cell Transplantation From Related and Unrelated Donors for Adults with Hematologic Malignancies

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2302-2302
Author(s):  
Chunji Gao ◽  
Xiaohong Li ◽  
Honghua Li ◽  
Wenrong Huang ◽  
Xiaoxiong Wu ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Peripheral Blood Stem Cell ◽  
Hematologic Malignancies ◽  
Blood Stem Cell ◽  
Unrelated Donor ◽  
Gvhd Prophylaxis ◽  
Blood Stem Cell Transplantation

Abstract Abstract 2302 Although allogeneic peripheral blood stem cell transplantation from a matched related donor (RD-PBSCT) presents the best curable opportunity for many patients with hematologic malignancies, only about one third of individuals have HLA matched sibling donors. Fortunately, from unrelated donor peripheral blood stem cell transplantation has been acceptable and expanded recently. In order to evaluate the effect of allogeneic peripheral blood stem cell transplantation from unrelated donor (URD-PBSCT), we compare results of URD-PBSCT and RD-PBSCT that were done for 172 consecutive adult patients with hematologic maligancies from Jan 2002 to Dec 2008 at a single-center. Among these patients, 62 cases underwent URD-PBSCT and 110 cases underwent RD-PBSCT. Myeloablative conditioning was adopted for all patients. In graft versus host disease (GVHD) prophylaxis, 49 URD-PBSCT recipients received CSA, MTX, MMF and ATG (URD-ATG group), 13 recipients were given simulect more in base of URD-ATG group (URD-ATG+CD25 group) while RD-PBSCT recipients (RD group) received CSA, MTX and MMF. Engraftment was achieved in 98.4% of URD-PBSCT patients and 98.2% of RD-PBSCT patients (100% for patients surviving beyond 28 days after transplant). The cumulative incidence of acute GVHD (grade II-IV) was 15.4%, 36.7% and 29.1% respectively in the URD-ATG+CD25, URD-ATG and RD groups (P = 0.275). The cumulative incidence of chronic GVHD was 0%, 45.6%, 39.6% respectively and significant lower in URD-ATG+CD25 group than the other two groups (P = 0.0134). Relapse incidence was 53.8%, 12.2%, 14.5% respectively and significant higher in URD-ATG+CD25 group than the other s (P = 0.0059), while there was no different between the URD-ATG and RD groups (P = 0.610). Estimated overall survival (OS) at 5 years was 30.8%, 69.4% and 67.3% respectively and no significant difference between URD-ATG group and RD group (p=0.898). Adverse prognosis factors for relapse and OS included transplant not in remission and GVHD prophylaxis with simulect. Our results indicate PBSCT from unrelated donor may be considered comparable to those from related donor. Disclosures: No relevant conflicts of interest to declare.

Twenty-five years of peripheral blood stem cell transplantation

Blood ◽  
2011 ◽  
Vol 117 (24) ◽  
pp. 6411-6416 ◽  
Author(s):  
Martin Körbling ◽  
Emil J Freireich
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Peripheral Blood Stem Cell ◽  
Blood Stem Cell ◽  
Cxcr4 Antagonist ◽  
Hematopoietic Stem ◽  
Blood Stem Cell Transplantation

Abstract Peripheral blood stem cell transplantation (PBSCT) is the most common transplantation procedure performed in medicine. Its clinical introduction in 1986 replaced BM as a stem-cell source to approximately 100% in the autologous and to approximately 75% in the allogeneic transplantation setting. This historical overview provides a brief insight into the discovery of circulating hematopoietic stem cells in the early 1960s, the development of apheresis technology, the discovery of hematopoietic growth factors and small molecule CXCR4 antagonist for stem- cell mobilization, and in vivo experimental transplantation studies that eventually led to clinical PBSCT. Also mentioned are the controversies surrounding the engraftment potential of circulating stem cells before acceptance as a clinical modality. Clinical trials comparing the outcome of PBSCT with BM transplantation, registry data analyses, and the role of the National Marrow Donor Program (NMDP) in promoting unrelated blood stem-cell donation are addressed.

Unrelated Donor Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5430-5430
Author(s):  
Chunji Gao ◽  
Wanming Da ◽  
Bolong Zhang ◽  
Xiaoping Han ◽  
Jing Yu ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Peripheral Blood Stem Cell ◽  
Hematologic Malignancies ◽  
Blood Stem Cell ◽  
Unrelated Donor ◽  
Post Transplantation ◽  
Blood Stem Cell Transplantation

Abstract Objiective To explore feasibility and efficacy of unrelated donor peripheral blood stem cell transplantation (UD-PBSCT) in treatment of hematologic malignancies. Methods Ten patients with hematologic malignancies undewent high resolution DNA based typing HLA-matched or 1 locus mismatched UD-PBSCT. Busulfan, cyclophosphamide, Ara-C, MeCCNU and antithymocyte globulin (ATG) were used for preparative regimen in all cases. All patients received mycophenolate mofetile, cyclosporin A and short-term methotrexate with CD25 antibody as the graft-versus-host disease (GVHD) prophylaxis. Results The results showed that rapid engraftment was observed in all cases who presented full donor chimerism at 28 days post transplantation by STR-PCR. The median time of neutrophil recovery >0.5×109/L, platelet recovery >20×109/L was observed at 13, 17.5days respectively post transplantation. The incidence of acute GVHD was 3/10 cases (1case with grade I was recovery by himself, 1case with grade III was cured, 1case with grade VI was died). Conclusion It is concluded that above-mentioned conditioning and GVHD prophylaxis are effective approaches for unrelated donor peripheral blood stem cell transplantation in treatment of hematopoietic malignancies.

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