COMBINED UMBILICAL CORD BLOOD AND BONE MARROW TRANSPLANT ATION IN THE TREATMENT OF BETA-THALASSEMIA MAJOR

2000 ◽  
Vol 17 (4) ◽  
pp. 307-314 ◽  
Author(s):  
Evgenios Goussetis ◽  
Julie Peristeri ◽  
Vasiliki Kitra ◽  
Antonios Kattamis ◽  
Demetrios Petropoulos ◽  
...  
Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4506-4506
Author(s):  
Wu Xuedong ◽  
Li Chunfu ◽  
Xiaoqin Feng ◽  
Yuelin He ◽  
Xiaohui Zhou ◽  
...  

Abstract Abstract 4506 Objective: To investigate the effect of transplantation using bone marrow plus umbilical cord blood from same sibling in children with β-thalassemia major (TM). Methods: Twenty three TM patients undergoing transplantation of bone marrow and umbilical cord blood of same sibling aged from 4.0 to 12 years, 13 boys and 10 girls, were recruited at the Department of Pediatrics, Nanfang Hospital, Southern Medical University from January 2005 to June 2012. The patients were classified into three classes, class¢ñ to class ¢ò 22 cases and class ¢ó 1 case. Donors ranged 1–4 years received 10 Ìg/kg per day of subcutaneous granulocyte colony-stimulating factor (G-CSF) for 5 consecutive days. Bone marrow was harvested on the fifth day. Bone marrow and umbilical cord blood of the same sibling then were transfused into the patient. Results: Recovery of hematopoiesis was gained in all patients 4 weeks following transplantation. Seventeen patients suffered from infection of different degree. Six patients developed mild venous occlusive disease. Four patients developed grade¢ñacute graft-versus-host disease (GVHD), and one developed grade¢ñchronic GVHD. Of twenty three patients, twenty survived, three died of whom, one died of lung infection and heart failure 32 days following transplantation, one died of organ failue on 47days after transplantation, and the other one died of lung fugal infection 22 months after transplantation. Conclusion: Combined transplantation of granulocyte colony-stimulating factor primed bone marrow and umbilical cord blood of same sibling in children with β-thalassemia major is safe and effective with promising results. However, complications should be paid high attention following transplantation. Disclosures: No relevant conflicts of interest to declare.


2016 ◽  
Vol 91 (9) ◽  
pp. E357-E358
Author(s):  
Gabriele Kropshofer ◽  
Sieghart Sopper ◽  
Michael Steurer ◽  
Wolfgang Schwinger ◽  
Roman Crazzolara

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 2538-2538
Author(s):  
Xuedong Wu ◽  
Jianyun Wen ◽  
Pei Fuyu ◽  
Libai Chen ◽  
Jianyun Liao ◽  
...  

Abstract Background: Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for thalassemia major (TM). Bone marrow (BM) and cord blood (CB) are biologically different stem cell sources. Methods: We analyed the results of a retrospective study of HSCT in 29 chlidren (median age at transplantation was 6 years old) with β-TM after the combined infusion of G-CSF primed bone marrow (BM) and cord blood (CB) from the same transplantation to outcomes in children with β-TM who had received BM (n=26).Patients treated with bone marrow transplant (BMT)were closely matched to the co-transplant group in terms of age, human leucocyte antigen (HLA) matching and duration of follow-up.Compared to BMT group, the donors in co-transplant group were younger (median age 2 vs. 4 years old, p=0.015) Results: In the co-transplant group,the mean total nucleated cells (TNC) was 2.63×108/kg(range,1.26-3.72×108/kg) and the CB was 0.39×108/kg(range,0.27-0.71×108/kg), respectively.The mean TNC (3.02 vs. 2.79×108/kg, p=0.532) and CD34+cells (7.55 vs. 6.94×106/kg, p=0.227) were insignificantly difference between the co-transplant group and BMT group. Of the 53 patients who had successful engraftment,patients who received a co-transplant had a lower incidence of ≥ grade II acute (3.3 vs. 20.8, p=0.047) and chronic(0vs.16.7%,p=0.022) graft versus host disease (GVHD) compared to BM transplant (BMT) recipients. There was no graft rejection (GR) after co-transplant, but GR happened two patients (7.7%) in BMT group(p=0.132).We found insignificant difference in neutrophils (18.7vs.19.9 days, p= 0.956) and platelet (24.7vs. 26.2 days, p=0.235) engraftment time between the co-transplant and BMT group. All patients were followed up until june 30, 2014, the 5 year probability of overall survival (OS), transplant free survival (TFS) and transplant-related mortality (TRM) were similar for the two groups. The 5-year probability of OS and TFS were 89.7% and 89.7% in the co-transplant group, 92.3%and 84.6% after BMT (P=0.740 and 0.573, respectively). Conclusions: Our data suggest that the lower risk of GVHD is retained with co-transplant group. The incidence of GR lower in the co-transplant group, although a larger cohort of patients will be needed to confirm this inital obser-vation.Here,we suggest transplantation of G-CSF primed BM a,nd CB of same sibling appears to be a feasible and effective strategy to further optimize outcomes of HSCT for TM with decreasing the risk of the occurrence of GVHD. Disclosures No relevant conflicts of interest to declare.


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