1. Transplant Outcomes in Beta-Thalassemia Major Patients Receiving Combined Granulocyte Colony-Stimulating Factor-Primed Bone Marrow and Cord Blood Graft Compared to Granulocyte Colony-Stimulating Factor-Primed Bone Marrow Alone
- Author
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Xiaoqin Feng, Libai Chen, Jianyun Wen, Yongsheng Ruan, Yue-lin He, Chunfu Li, Xuan Liu, Fuyu Pei, Xuedong Wu, and Qareen Haque
- Subjects
medicine.medical_specialty ,Adolescent ,medicine.medical_treatment ,Thalassemia ,Cytomegalovirus ,Graft vs Host Disease ,Bone Marrow Cells ,Hematopoietic stem cell transplantation ,Granulocyte ,Severity of Illness Index ,Gastroenterology ,Disease-Free Survival ,HLA Antigens ,Internal medicine ,Granulocyte Colony-Stimulating Factor ,medicine ,Humans ,Child ,Survival rate ,Retrospective Studies ,business.industry ,beta-Thalassemia ,Hematopoietic Stem Cell Transplantation ,Infant ,Cell Differentiation ,Hematology ,General Medicine ,Fetal Blood ,Hematopoietic Stem Cells ,medicine.disease ,Granulocyte colony-stimulating factor ,Transplantation ,surgical procedures, operative ,medicine.anatomical_structure ,Child, Preschool ,Cord blood ,Virus Activation ,Bone marrow ,business ,Immunosuppressive Agents - Abstract
Background: Hematopoietic stem cell transplantation (HSCT) is currently the only curative treatment for thalassemia majorTM. Graft rejection (GR) and graft-versus-host disease (GVHD) are the primary obstacles to a successful outcome. Methods: We conducted a retrospective study of HSCT in 29 children (median age at transplantation: 6 years) with Beta-thalassemia (β-TM) after the combined infusion of granulocyte colony-stimulating factor-primed bone marrow (G-BM) and cord blood (CB) from the human leukocyte antigen (HLA)-identical sibling donors. We also compared the outcomes of the co-transplanted children with those of children with β-TM who received G-BM alone from an HLA-identical sibling donor (n = 26). Results: Compared to the G-BM transplant (G-BMT) recipients, those who received a co-transplant had a lower incidence of grade ≥II acute (17.24 vs. 30.7%, p = 0.047) and limited chronic (0 vs.15.4%, p = 0.022) GVHD as well as a lower incidence of GR (0 vs. 7.7%, p = 0.132). Neutrophil recovery time was faster in the co-transplant group (18.5 vs. 21 days, p = 0.04). All the patients were monitored until December 31, 2016; the median follow-up time was 74 months, and the 5-year thalassemia-free survival rate was 89.7% in the co-transplant group and 84.6% in the G-BMT-alone group (p = 0.590). Conclusions: A combined CB and G-BM graft from an HLA-identical sibling donor is an effective treatment option for TM in children, with less acute and chronic GVHD.
- Published
- 2018