scholarly journals Hematopoietic Stem Cell Transplantation From Unrelated Donor in Children with Beta Thalassemia Major

2018 ◽  
Vol 24 (3) ◽  
pp. S428-S429 ◽  
Author(s):  
Gulsun Karasu ◽  
Vedat Uygun ◽  
Hayriye Daloglu ◽  
Suar Kilic ◽  
Seda Ozturkmen ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Thalassemia Major ◽  
Beta Thalassemia ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Beta Thalassemia Major

scholarly journals Cost Comparison of Hematopoietic Stem Cell Transplantation and Conventional Therapy for Patients with Thalassemia Major in China

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 2082-2082 ◽  
Author(s):  
Wanxia Tan ◽  
Chunfu Li ◽  
Xuedong Wu ◽  
Yuelin He ◽  
Xiaoqin Feng ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Patient Characteristics ◽  
Thalassemia Major ◽  
Cost Comparison ◽  
Beta Thalassemia ◽  
Hematopoietic Stem

Abstract Background: The conventional treatment (CT) for beta- thalassemia major (TM) includes regular transfusion accompanied by iron-chelating therapy. However, this laborious treatment by given desferrioxamine (DFO) subcutaneously for 8 to12 hours per days, at least 5 days per week with/without deferiprone (DFP) has led to poor compliance, even though, the new medicine named deferasirox (DFX) has been introduced in China recent years. Nevertheless, the high cost makes it difficult to use widely for many families. Hematopoietic stem cell transplantation (HSCT) provides an alternative curative option for TM patients. To our knowledge, data from China has not been available in the literature on cost comparison between HSCT and CT. Aims: The principal aim of our study is to compare the lifetime undiscounted mean cost (UMC) of hematopoietic stem cell transplantation (HSCT) with UMC of conventional therapy (CT) in beta-thalassemia major (TM) patients and to investigate the relationship of the clinical features to cost outcomes of HSCT. Methods: We estimated UMC of 93 TM-HSCTs between 2011 and 2012 with a median age of 6 years (range, 3-16) and a median follow-up time of 3 years (range, 2.4 -3.8). The relationship between the UMC of HSCT and patient characteristics were analyzed. The UMC of 93 TM-HSCTs was compared with UMC of CT based on total 1526 TM patients. Age was used as matching variant, and the mean cost of each age was calculated, then cumulative cost was further adjusted for age in patients. Results: UMC of TM-HSCT was CNY (Chinese Yuan) 235,254/USD 37,664 (95% confidence interval (CI) CNY208, 081-262,719/USD 33,293-42,035) with mean hospital stay of 60.5 days (95% CI 49-71). UMC of 20 years of follow-up and total undiscounted lifetime (55 years) for patients underwent HSCT were CNY 345,317/USD55, 251 and CNY 465,975/USD 74,602 respectively. However, the corresponding costs of patients undergoing CT were CNY 2,101,488/ USD336, 238 and CNY 7,489,519/ USD1, 198,323 respectively. Patient characteristics were helpless to predict the costs. HLA-mismatched transplants increased significantly UMC of HSCT than matched transplants (USD 35,818 vs. USD 52,771; p<0.001). The development of GVHD were associated with higher costs (USD 63,933 vs. USD 44,547; p = 0.001). Conclusions: Cost comparison of HSCT and CT suggests that HSCT is an efficient and high cost-effective treatment for TM patients. Mismatched donor transplant increases the cost of HSCT. Keywords: hematopoietic stem cell transplantation, iron overload, cost analysis Figure 1. Cumulative lifetime treatment cost of CT and HSCT (USD) Figure 1. Cumulative lifetime treatment cost of CT and HSCT (USD) Disclosures No relevant conflicts of interest to declare.

scholarly journals Haploidentical Hematopoietic Stem-Cell Transplantation in Adults

2011 ◽  
Vol 2011 ◽  
pp. 1-10 ◽  
Author(s):  
Salem Alshemmari ◽  
Reem Ameen ◽  
Javid Gaziev
Keyword(s):  
Stem Cell ◽  
T Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Cell Depletion ◽  
Unrelated Donor ◽  
T Cell Depletion ◽  
Hematopoietic Stem

Haploidentical hematopoietic stem-cell transplantation is an alternative transplant strategy for patients without an HLA-matched donor. Still, only half of patients who might benefit from transplantation are able to find an HLA-matched related or unrelated donor. Haploidentical donor is readily available for many patients in need of immediate stem-cell transplantation. Historical experience with haploidentical stem-cell transplantation has been characterised by a high rejection rate, graft-versus-host disease, and transplant-related mortality. Important advances have been made in this field during the last 20 years. Many drawbacks of haploidentical transplants such as graft failure and significant GVHD have been overcome due to the development of new extensive T cell depletion methods with mega dose stem-cell administration. However, prolonged immune deficiency and an increased relapse rate remain unresolved problems of T cell depletion. New approaches such as partial ex vivo or in vivo alloreactive T cell depletion and posttransplant cell therapy will allow to improve immune reconstitution in haploidentical transplants. Results of unmanipulated stem-cell transplantation with using ATG and combined immunosuppression in mismatched/haploidentical transplant setting are promising. This paper focuses on recent advances in haploidentical hematopoietic stem-cell transplantation for hematologic malignancies.

Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan

Blood ◽  
2012 ◽  
Vol 120 (2) ◽  
pp. 473-476 ◽  
Author(s):  
Maria Ester Bernardo ◽  
Eugenia Piras ◽  
Adriana Vacca ◽  
Giovanna Giorgiani ◽  
Marco Zecca ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Cumulative Incidence ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Unrelated Donor ◽  
Hematopoietic Stem

Abstract Sixty thalassemia patients (median age, 7 years; range, 1-37) underwent allogeneic hematopoietic stem cell transplantation (HSCT) after a preparation combining thiotepa, treosulfan, and fludarabine. Before HSCT, 27 children were assigned to risk class 1 of the Pesaro classification, 17 to class 2, and 4 to class 3; 12 patients were adults. Twenty patients were transplanted from an HLA-identical sibling and 40 from an unrelated donor. The cumulative incidence of graft failure and transplantation-related mortality was 9% and 7%, respectively. Eight patients experienced grade II-IV acute GVHD, the cumulative incidence being 14%. Among 56 patients at risk, 1 developed limited chronic GVHD. With a median follow-up of 36 months (range, 4-72), the 5-year probability of survival and thalassemia-free survival are 93% and 84%, respectively. Neither the class of risk nor the donor used influenced outcome. This treosulfan-based preparation proved to be safe and effective for thalassemia patients given allogeneic HSCT.

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