• IMMUNE NETWORK
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• v.3 no.2
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• pp.83-88
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• 2003

The number of umbilical cord blood transplantation is increasing worldwide as it has expanded the ability of the transplantaion community to meet the growing needs of their patients. Clinical data over the last decade show promising results in transplantation using both related as well as unrelated cord bloods. Cord blood banks are essential for the clinical use for transplantation and are now established around the world with the major efforts to standardize banking in collection, processing and distribution of cord blood for providing the highest quality stem cells for the patients. In Korea, Medipost, Histostem and some regional cord blood banks were established some years ago and collected thousands of cord blood for public but it had some limitations and was not expanded as the cord blood transplantation was not covered by medical insurance. Recently with the change in the policy of medical insurance to cover the cord blood transplantation, several venture companies are showing great interests in cord blood banking and trying to establish private cord blood banks in Korea. This review article discusses the current status of cord blood transplantaion and also the clincial use of stem cells from cord blood.

• Clinical and Experimental Pediatrics
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• v.55 no.3
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• pp.93-99
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• 2012

Purpose: This study compared outcomes in children with acute leukemia who underwent transplantations with umbilical cord blood (UCB), bone marrow, or peripheral blood stem cells from a human leukocyte antigen (HLA)-matched related donor (MRD) or an unrelated donor (URD). Methods: This retrospective study included consecutive acute leukemia patients who underwent their first allogeneic hematopoietic stem cell transplantation (HSCT) at Samsung Medical Center between 2005 and 2010. Patients received stem cells from MRD (n=33), URD (n=46), or UCB (n=41). Results: Neutrophil and platelet recovery were significantly longer after HSCT with UCB than with MRD or URD ($p$ <0.01 for both). In multivariate analysis using the MRD group as a reference, the URD group had a significantly higher risk of grade III to IV acute graft-versus-host disease (GVHD; relative risk [RR], 15.2; 95% confidence interval [CI], 1.2 to 186.2; $p$=0.03) and extensive chronic GVHD (RR, 6.9; 95% CI, 1.9 to 25.2; $p$ <0.01). For all 3 donor types, 5-year event-free survival (EFS) and overall survival were similar. Extensive chronic GVHD was associated with fewer relapses (RR, 0.1; 95% CI, 0.04 to 0.6; $p$ <0.01). Multivariate analysis showed that lower EFS was associated with advanced disease at transplantation (RR, 3.2; 95% CI, 1.3 to 7.8; $p$ <0.01) and total body irradiation (RR, 2.1; 95% CI, 1.0 to 4.3; $p$=0.04). Conclusion: Survival after UCB transplantation was similar to survival after MRD and URD transplantation. For patients lacking an HLA matched donor, the use of UCB is a suitable alternative.

• Clinical and Experimental Pediatrics
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• v.52 no.6
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• pp.725-729
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• 2009

Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis (EBV-HLH) with central nervous system (CNS) involvement is usually fatal unless stem cell transplant (SCT) is offered. However, SCT with conventional intensity conditioning is associated with high transplant-related mortality. We describe our experience with unrelated SCTs after reduced-intensity conditioning (RIC) for patients with EBV-HLH with progressive CNS disease. This approach was associated with minimal toxicities and might be an effective option in patients with EBV-HLH with progressive CNS disease. Moreover, the addition of rituximab to RIC appears to be safe and effective in suppressing EBV in the patients with EBV-HLH.

• Asian Oncology Nursing
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• v.12 no.2
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• pp.132-138
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• 2012

Purpose: The purpose of this study was to identify the performance status and quality of life (QOL) of patients after hematopoietic stem cell transplantation (HSCT) according to period of survival. Methods: Participants consists of 83 HSCT patients who were being treated regularly at out-patient clinic in two general hospitals in D city. Data were collected using questionnaires that were modified by Functional Assessment of Cancer Therapy-Bone Marrow Transplabtation (FACT-BMT) scale and Eastern Cooperative Oncology Group (ECOG). Results: The unrelated HSCT group's survival period was significantly worse than related HSCT group and autologous HSCT group. Performance status of the group with more than 3 years survival was significantly higher than that of the group with less than a year survival. The mean score of total QOL of HSCT patients was 2.69 out of 4. Total QOL was not significantly different among period of survival less than 1 year, 1-3 years, and more than 3 years. But BMT QOL was shown that the group with more than 3 years survival was higher than the groups with less than a year survival. Conclusion: Performance status and BMT QOL of the group with less than 1 year survival was significantly lowered than the groups with more than 3 years survival.

• Clinical and Experimental Pediatrics
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• v.49 no.2
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• pp.173-180
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• 2006

Purpose : Cytomegalovirus(CMV) infection still remains as a major cause of morbidity and mortality after stem cell transplantation. In this study, we analyzed the results of antigenemia-guided preemptive therapy among children with allogeneic hematopoietic stem cell transplantation to determine the incidence and risk factors associated with CMV antigenemia, and evaluated the efficacy of the CMV antigenemia based preemptive therapy. Methods : We enrolled 213 pediatric patients following allogeneic hematopoietic stem cell transplantation(HSCT), at the Catholic HSCT center between October 1998 and December 2003. Pre-emptive ganciclovir was started when more than 5 CMV Ag-positive cells were detected in matched sibling HSCT, and when any Ag-positive cells were seen in unrelated allogenic HSCT. Results : CMV antigenemia was observed in 88(41.3 percent) of 213 patients on median day 28(day 11-99). In univariated analysis, use of unrelated donors(other than siblings), age of recipient(more than 5 years at transplant) at transplantation, the presence of recipient CMV-IgG before transplantation, TBI-based conditioning regimen and the presence of acute GvHD(grade ${\geq}II$) were the risk factors for positive CMV antigenemia. In multivariate analysis, unrelated bone marrow transplantation, positive recipient CMV serology and acute GvHD(grade ${\geq}II$) were the independent risk factors for positive CMV antigenemia. Conclusion : Risk factors of CMV infection in children were CMV serostatus of the recipient, the source of stem cells, and acute graft-versus-host disease. The pre-emptive therapy based on CMV antigenemia was effective in the prevention of CMV disease.

• Clinical and Experimental Pediatrics
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• v.58 no.6
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• pp.199-205
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• 2015

Severe aplastic anemia (SAA) is a life-threatening disorder for which allogeneic hematopoietic stem cell transplantation (HSCT) is the current available curative treatment. HSCT from matched sibling donors (MSDs) is the preferred therapy for children with acquired SAA. For patients who lack MSDs, immunosuppressive therapy (IST) is widely accepted as a first-line treatment before considering HCT from an unrelated donor (URD). Given the recent progress in HSCT using URDs for childhood SAA, well-matched URDs became a realistic alternative for pediatric patients who have no suitable related donors and who are refractory to IST. However, it is quite challenging to treat patients with refractory SAA who lack suitable related or URDs. Even though haploidentical HSCT from genetically mismatched family members seemed to be an attractive procedure with the amazing benefit of readily available donors for most patients, early attempts were disappointing because of refractory graft-versus-host disease (GVHD) and excessively high transplant-related mortality. Recent advances with effective ex vivo depletion of T cells or unmanipulated in vivo regulation of T cells, better supportive care, and optimal conditioning regimens have significantly improved the outcome of haploidentical transplant. Besides considerable progress in the treatment of malignant diseases, recent emerging evidences for haploidentical HSCT in SAA has provided additional therapeutic options for patients with refractory diseases. Further improvements to decrease the rates of graft failure, GVHD, and infectious complications will facilitate the emergence of haploidentical HSCT as a front-line therapy for treating acquired SAA in children and adolescents who have no suitably matched donors.

• Clinical and Experimental Pediatrics
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• v.50 no.6
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• pp.519-523
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• 2007

Aplastic anemia is a rare disease, which is characterized by pancytopenia and hypocellular bone marrow without infiltration of abnormal cells or fibrosis. The incidence in Asia is higher than in the West and new cases are diagnosed at a rate of 5.1 per million pediatric populations per year in Korea. The pathophysiology is understood roughly by defective hematopoiesis, impaired bone marrow micro-environment and immune mechanism. Treatments are performed on basis of pathogenesis and selected depending on the severity. Immunosuppressive therapy with antilymphocyte or antithymocyte globulin and cyclosporine is effective in the majority of patients but has some problems including relapse or clonal evolution. Recently, there have been clinical trials of immunosuppression with hematopoietic growth factors or other drugs. Allogeneic hematopoietic stem cell transplantation (HSCT) is curative in children with severe aplastic anemia. The overall survival in HSCT from HLA-identical sibling is higher than alternative donor, including HLA matched unrelated donor or cord blood. We have to consider quality of life after HSCT because of high survival rate. However, chronic graft versus host disease and graft failure are important factors that affect the quality of life and overall survival. We need further investigation to make new regimens aimed at overcoming these risk factors and perform clinical trials.

• Clinical and Experimental Pediatrics
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• v.55 no.3
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• pp.100-106
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• 2012

Purpose: The survival rate for childhood acute lymphoblastic leukemia (ALL) has improved significantly. However, overall prognosis for the 20 to 25% of patients who relapse is poor, and allogeneic hematopoietic stem cell transplantation (HSCT) offers the best chance for cure. In this study, we identified significant prognostic variables by analyzing the outcomes of allogeneic HSCT in ALL patients in second complete remission (CR). Methods: Fifty-three ALL patients (42 men, 79%) who received HSCT in second CR from August 1991 to February 2009 were included (26 sibling donor HSCTs, 49%; 42 bone marrow transplantations, 79%). Study endpoints included cumulative incidence of acute and chronic graft-versus-host disease (GVHD), relapse, 1-year transplant-related mortality (TRM), disease-free survival (DFS), and overall survival (OS). Results: Cumulative incidences of acute GVHD (grade 2 or above) and chronic GVHD were 45.3% and 28.5%, respectively. The estimated 5-year DFS and OS for the cohort was $45.2{\pm}6.8%$ and $48.3{\pm}7%$, respectively. Only donor type, i.e., sibling versus unrelated, showed significant correlation with DFS in multivariate analysis ($p$=0.010). The rates of relapse and 1 year TRM were $28.9{\pm}6.4%$ and $26.4{\pm}6.1%$, respectively, and unrelated donor HSCT ($p$=0.002) and HLA mismatch ($p$=0.022) were significantly correlated with increased TRM in univariate analysis. Conclusion: In this single institution study spanning more than 17 years, sibling donor HSCT was the only factor predicting a favorable result in multivariate analysis, possibly due to increased TRM resulting from unrelated donor HSCT.

• Clinical and Experimental Pediatrics
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• v.45 no.7
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• pp.912-916
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• 2002

Cord blood is a useful source of allogeneic hematopoietic stem cells for bone marrow reconstitution. The number of umbilical cord blood transplants is increasing worldwide. In this a case 15-month-old boy with acute myeloid leukemia was treated with umbilical cord blood transplant from an HLA-3 loci mismatched unrelated donor. Granulocyte recovery greater than $500/mm^3$ occurred at day 49, and the platelet recovered greater than $20,000/mm^3$ independent of transfusion at day 81 after stem cell infusion.

• Clinical and Experimental Pediatrics
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• v.45 no.3
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• pp.370-375
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• 2002

Purpose : In most cases, myelodysplastic syndrome(MDS) transforms into a more aggressive state or acute myelogenous leukemia; it's prognosis is very poor. It is believed that hematopoietic stem cell transplantation(HSCT) is the only curative treatment of MDS, but available data in children are very sparse. In this report, the short term outcome of HSCT in childhood MDS was analyzed. Methods : Ten children with MDS(CMMoL 5, RAEB 3, RAEBt 2) underwent HSCT(HLA-matched sibling transplantation 4, HLA-matched unrelated transplantation 4, cord blood transplantation 1, HLA-mismatched familial transplantation 1) between November 1995 and January 2001 at St. Mary's Hospital. Median follow-up duration was 11 months. Results : Engraftment was successful in all cases and 8 patients are alive without disease. Three cases of VOD were observed and improved without complication. Four cases of grade II and 1 case of grade III acute GVHD were observed and well controlled with treatment. Three patients relapsed after transplantation. One patient is alive without disease after cytoreduction with allogenic stem cell rescue and 2 patients died of relapse. Conclusion : HSCT is a curative strategy of MDS and the survival rate is relatively higher than that of adults. But there is an obvious need for more studies because of the small number of patients and the short duration of the follow-up.