Three Hundred and Fifty Cases of Pediatric Allogeneic Hematopoietic Stem Cell Transplantation: Single Center Study in Korea.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 5139-5139
Author(s):  
Young-Shil Park ◽  
Pil-Sang Jang ◽  
Sangrhim Choi ◽  
Nak-Gyun Chung ◽  
Bin Cho ◽  
...  

Abstract Background: Hematopoietic stem cell transplantation (HST) was first introduced to Korean children in 1983. Since then HST has been a curative method for pediatric malignant or non-malignant hematologic disorders and the number of children receiving HST’s has been increasing steadily. Method: We analyzed three hundred and fifty cases of pediatric allogeneic hematopoietic stem cell transplantation between Nov. 1983 and Dec. 2003 in the Catholic Hematopoietic Stem Cell Transplantation Center of Korea. Results: HLA-matched sibling bone marrow transplantations were performed in 206 cases (103 males, 103 females, median age 12 years) with median follow-up of 73 months. The 5-year event-free survival (EFS) of ALL and AML was 70.5 % and 67.22 %, respectively. The 5-year EFS of severe aplastic anemia was 90.9 %. The 5-year EFS of CML/MDS and rare hematologic diseases was 65.5 % and 90.0 %, respectively. Eighty-one children (56 males, 25 females, median age 9 years) underwent unrelated bone marrow transplantation (UBMT), 42 (27 males, 15 females, median age 5 years) cord blood transplantation (CBT) and 21 (14 males, 7 females, median age 8 years) familial haploidentical HST (FHT). Three-year EFS of UBMT, CBT and FHT was 55.6 %, 48.0 % and 38.0 %, respectively. Conclusion: HLA-matched sibling allogeneic BMT showed better survival in children with hematopoietic stem cell disorders. Recently, transplants using alternative stem cell sources are increasing due to the lack of suitable sibling donors and continued efforts for reducing transplant-related complications are warranted for improved survival.

Hematology ◽  
2006 ◽  
Vol 2006 (1) ◽  
pp. 398-401 ◽  
Author(s):  
Franco Locatelli

Abstract The only well-established curative therapy for patients with hemoglobinopathies is allogeneic hematopoietic stem cell transplantation (HSCT), which, in the last 20 years, has been mainly performed from an HLA-matched, related donor, using bone marrow as source of hematopoietic progenitors. More recent studies indicate that HSCT from unrelated donors may offer results comparable to those obtained with HLA-identical family donors, provided that stringent criteria of compatibility are employed for selecting the donor. Cord blood transplantation was also suggested to be an equally effective, but safer, procedure than bone marrow transplantation, due to the lower incidence and severity of both acute and chronic graft-versus-host disease. In view of the early, as well as late, morbidity and mortality associated with conventional myeloablative transplantation in patients with hemoglobinopathies, it is not surprising that great interest and relevant expectations for patients with hemoglobinopathies have been raised by the introduction in the clinical practice of reduced-intensity preparative regimens. However, few reports have demonstrated the feasibility of using reduced-intensity preparative regimens for successfully treating these patients and many treatment failures, mainly due to the lack of sustained donor engraftment, have been reported. Despite these limitations, some of the concepts obtained from the use of reduced intensity regimens, such as the substitution of fludarabine for cyclophosphamide, may be important to further improve the outcome of patients with hemoglobinopathies, especially of those with poor prognostic characteristics, given HSCT.


2016 ◽  
Vol 8 ◽  
pp. 2016054 ◽  
Author(s):  
Hosein Kamranzadeh fumani ◽  
Mohammad Zokaasadi ◽  
Amir Kasaeian ◽  
Kamran Alimoghaddam ◽  
Asadollah Mousavi ◽  
...  

Background & objectives: Fanconi anemia (FA) is a rare genetic disorder caused by an impaired DNA repair mechanism which leads to an increased tendency toward malignancies and progressive bone marrow failure. The only curative management available for hematologic abnormalities in FA patients is hematopoietic stem cell transplantation (HSCT). This study aimed to evaluate the role of HSCT in FA patients.Methods: Twenty FA patients with ages of 16 or more who underwent HSCT between 2002 and 2015 enrolled in this study. All transplants were allogeneic and the stem cell source was peripheral blood and all patients had a full HLA-matched donor.Results: Eleven patients were female and 9 male (55% and 45%). Mean age was 24.05 years. Mortality rate was 50% (n=10) and the main cause of death was GVHD. Survival analysis showed an overall 5-year survival of 53.63% and 13 year survival of 45.96 % among patients.Conclusion: HSCT is the only curative management for bone marrow failure in FA patients and despite high rate of mortality and morbidity it seems to be an appropriate treatment with an acceptable long term survival rate for adolescent and adult group.


2014 ◽  
Vol 97 (12) ◽  
pp. e75-e77 ◽  
Author(s):  
Mathieu Meunier ◽  
Anne-Claire Manez ◽  
Aliénor Xhaard ◽  
Régis Peffault de Latour ◽  
Flore Sicre de Fontbrune ◽  
...  

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