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1.
Article in English | WPRIM (Western Pacific) | ID: wpr-1042341

ABSTRACT

Purpose@#We aimed to determine the current application and survival trends of hematopoietic stem cell transplantation (HSCT) among Korean children and adolescents with cancer. @*Materials and Methods@#Data of patients aged < 20 years with KCD-10 (Korean Classifications of Diseases, 10th revision) C codes and specific designation codes were collected from the National Health Insurance Service database. Thirty claim codes for HSCT were included, and data from 2009 to 2019 were analyzed. @*Results@#The operational definition of pediatric cancer yielded an annual average of 2,000, with annual cases decreasing. In 2019, 221 HSCTs were performed, a decrease from the ten-year average of 276. Allografts outnumbered autografts with a ratio of 1.5:1. The source of allograft was bone marrow in 15% of patients in 2009; however, it substantially decreased to 3.3% in 2019. Furthermore, 70.5% of allogeneic HSCT used peripheral blood stem cell (PBSC) grafts, which increased to 89.3% by 2015. Cord blood utilization markedly decreased to 2.7% in 2018. The 5-year overall survival (OS) rate of all patients was 85.1%. Overall mortality decreased among patients who underwent recent HSCT, and they exhibited a higher 5-year OS rate. @*Conclusion@#In Korea, the number of pediatric patients with cancer is declining; however, the ratio of transplants to all patients remains constant. Patients who recently underwent transplantation showed better survival rates, possibly due to HSCT optimization. Korea showed a substantially greater PBSC utilization in pediatric HSCT. An in-depth examination encompassing donor relations and cause of death with a prospective registry is required in future studies.

2.
Article in English | WPRIM (Western Pacific) | ID: wpr-1040225

ABSTRACT

Human taeniasis is presumed to have almost disappeared from Korea. Recently, we incidentally detected a Taenia saginata infection in an 8-year-old boy undergoing lymphoma diagnosis. The patient had been suffering for 4 months from intensifying snoring and obstructive sleep apnea. A neck computed tomography scan revealed a nasopharyngeal mass, and malignant B-cell lymphoma was supported by punch biopsy. On day 6 of the lymphoma workup period, the patient experienced anal itching, and two proglottids were detected in his stool. The patient had experienced four or five similar episodes within the past 2 years. He self-reported a history of raw beef and fish consumption and no history of traveling abroad. Laboratory findings revealed mild eosinophilia (eosinophil count: 791/μL).Two proglottids exhibited movement and possessed more than 15 branched uterine structures. Long segments approximately 84 cm in length were expelled after praziquantel treatment. Sequencing of the cytochrome oxidase 1 gene confirmed T. saginata, ruling out related Taenia species. After treatment, no proglottids or ova were detected in his stool, and the patient finally started chemotherapy for lymphoma. This case highlights the importance of timely diagnosis of hidden taeniasis in low-frequency endemic regions.

3.
Article in English | WPRIM (Western Pacific) | ID: wpr-1040599

ABSTRACT

22q11.2 deletion syndrome (22q11.2DS) is a disorder caused by microdeletions in the long arm of chromosome 22. It is one of the most common chromosomal microdeletion disorders. Clinical symptoms are caused by heterozygous deletion of chromosome 22q11.2 and include congenital heart diseases and palatal abnormalities.Bernard-Soulier syndrome (BSS) is a rare autosomal recessive bleeding disorder caused by deficiency of the glycoprotein Ib-IX-V complex (GPIb-IX-V), the receptor for von Willebrand factor. Glycoprotein Ib-beta (GPIbβ) gene mutation is one of the causes of BSS, and the GP1bβ gene is located on chromosome 22q11.2. Most 22q11DS patients do not have a bleeding issue since most of them have heterozygous deletion of the GPIbβ gene. However, we report a case in which a child with 22q11.2DS visited the hospital with subgaleal hemorrhage and was diagnosed with Bernard-Soulier syndrome with GPIbβ gene mutation by a platelet aggregation test and genetic sequencing.

4.
Article in English | WPRIM (Western Pacific) | ID: wpr-966470

ABSTRACT

Purpose@#Renal tumors account for approximately 7% of all childhood cancers. These include Wilms tumor (WT), clear cell sarcoma of the kidney (CCSK), malignant rhabdoid tumor of the kidney (MRTK), renal cell carcinoma (RCC), congenital mesoblastic nephroma (CMN) and other rare tumors. We investigated the epidemiology of pediatric renal tumors in Korea. @*Materials and Methods@#From January 2001 to December 2015, data of pediatric patients (0–18 years) newly-diagnosed with renal tumors at 26 hospitals were retrospectively analyzed. @*Results@#Among 439 patients (male, 240), the most common tumor was WT (n=342, 77.9%), followed by RCC (n=36, 8.2%), CCSK (n=24, 5.5%), MRTK (n=16, 3.6%), CMN (n=12, 2.7%), and others (n=9, 2.1%). Median age at diagnosis was 27.1 months (range 0-225.5) and median follow-up duration was 88.5 months (range 0-211.6). Overall, 32 patients died, of whom 17, 11, 1, and 3 died of relapse, progressive disease, second malignant neoplasm, and treatment-related mortality. Five-year overall survival and event free survival were 97.2% and 84.8% in WT, 90.6% and 82.1% in RCC, 81.1% and 63.6% in CCSK, 60.3% and 56.2% in MRTK, and 100% and 91.7% in CMN, respectively (p < 0.001). @*Conclusion@#The pediatric renal tumor types in Korea are similar to those previously reported in other countries. WT accounted for a large proportion and survival was excellent. Non-Wilms renal tumors included a variety of tumors and showed inferior outcome, especially MRTK. Further efforts are necessary to optimize the treatment and analyze the genetic characteristics of pediatric renal tumors in Korea.

5.
Article in English | WPRIM (Western Pacific) | ID: wpr-976745

ABSTRACT

Hepatocellular adenoma is an uncommon, benign liver tumor usually occurring in patients using estrogen or anabolic androgens and in those with a genetic disease, including glycogen storage disease. Hepatocellular adenomas can sometimes induce pain. However, it is usually asymptomatic. Moreover, few studies have reported cases of hepatocellular adenomas presenting with iron deficiency anemia. Herein, we report a pediatric case of a large hepatocellular adenoma, presenting with iron therapy-refractory iron deficiency anemia. A 14-year-old boy was diagnosed with hepatocellular adenoma during an anemia work-up. Improvement in iron deficiency anemia was observed after tumor resection.

6.
Blood Research ; : 152-157, 2022.
Article in English | WPRIM (Western Pacific) | ID: wpr-937244

ABSTRACT

Background@#The incorporation of a reduced-intensity conditioning (RIC) regimen in hematopoietic cell transplantation (HCT) for patients with hemophagocytic lymphohistiocytosis (HLH) has decreased early mortality but is associated with a high rate of mixed chimerism and graft failure. Here, we present a successful single-center experience using busulfan and a fludarabine-based RIC regimen for the treatment of HLH. @*Methods@#The medical records of pediatric patients with HLH who underwent HCT using a busulfan/fludarabine-based RIC regimen between January 2008 and December 2017 were reviewed retrospectively. @*Results@#Nine patients received HCT with a busulfan/fludarabine-based RIC regimen. Three patients had primary HLH, and the other six patients had secondary HLH with multiple reactivations. All three patients with primary HLH had UNC13D mutations. All patients achieved neutrophil and platelet engraftment at a median of 11 days (range, 10‒21) and 19 days (range, 13‒32), and all eight evaluable patients had sustained complete donor chimerism at the last follow-up. Two patients (22%) experienced grade 2 acute graft-versus-host disease (GVHD). Two patients (22%) developed chronic GVHD, and one died from chronic GVHD. One patient (11%) experienced reactivation 4 months after HCT from a syngeneic donor and died of the disease. The 8-year overall survival and event-free survival rates were 78%. No early treatment-related mortality within 100 days after HCT was observed. @*Conclusion@#Our experience suggests that a busulfan/fludarabine-based RIC regimen is a viable option for pediatric patients with HLH who require HCT.

7.
Blood Research ; : 41-50, 2022.
Article in English | WPRIM (Western Pacific) | ID: wpr-925644

ABSTRACT

Background@#Aggressive mature B-cell non-Hodgkin lymphoma (B-NHL) is the most common non-Hodgkin lymphoma in children. The outcome of chemotherapy for B-NHL has improved over decades. @*Methods@#We reviewed 82 children and adolescents with B-NHL diagnosed at Asan Medical Center between 1993 and 2020. The D-COMP/COMP (daunomycin–cyclophosphamide, doxorubicin, vincristine, and prednisolone), Pediatric Oncology Group (POG)-9219/9315/ 9317, R-CHOP/CHOP (rituximab–cyclophosphamide, doxorubicin, vincristine, and prednisolone), and Lymphomes Malins B 89 (LMB89)/LMB96 regimens were administered. In 2018, rituximab was added to the LMB protocol (R-LMB) for advanced-staged Burkitt lymphoma (BL). The patients’ clinical features and treatment outcomes were retrospectively analyzed. @*Results@#The most common subtype was BL (61%), followed by diffuse large B-cell lymphoma (DLBCL) (35%). The median age was 7.8 (range, 1.3‒16.4) years, and the most frequently used regimen was French‒American‒British (FAB)/LMB96 (58 patients, 70.7%). The 5-year overall survival (OS) and event-free survival (EFS) rates were 92.5% and 85.7%, respectively. The EFS rates of patients with BL and DLBCL were 90.0% and 79.3%, respectively. Among the FAB/LMB risk groups, group C (85.7%) had a significantly lower 5-year OS (P =0.037). Eleven events occurred (6 relapses, 3 deaths, and 2 secondary malignancies) during the median follow-up of 7.1 (range, 3.7‒118.5) months. Two patients treated with R-LMB had good outcomes without complications. @*Conclusion@#Various treatment regimens have favorable outcomes in pediatric patients with B-NHL.However, further studies are needed to improve survival in high-risk patients. In addition, careful monitoring for acute toxicity or secondary malignancy due to intensive multidrug chemotherapy is required.

8.
Article in English | WPRIM (Western Pacific) | ID: wpr-913833

ABSTRACT

Purpose@#Acute promyelocytic leukemia (APL) is a rare disease in children and there are some different characteristics between children and adult. We aimed to evaluate incidence, clinical characteristics and treatment outcomes of pediatric APL in Korea. @*Materials and Methods@#Seventy-nine pediatric APL patients diagnosed from January 2009 to December 2016 in 16 tertiary medical centers in Korea were reviewed retrospectively. @*Results@#Of 801 acute myeloid leukemia children, 79 (9.9%) were diagnosed with APL. The median age at diagnosis was 10.6 years (range, 1.3 to 18.0). Male and female ratio was 1:0.93. Thirty patients (38.0%) had white blood cell (WBC) count greater than 10×109/L at diagnosis. All patients received induction therapy consisting of all-trans retinoic acid and chemotherapy. Five patients (6.6%) died during induction chemotherapy and 66 patients (86.8%) achieved complete remission (CR) after induction chemotherapy. The causes of death were three intracranial hemorrhage, one cerebral infarction, and one sepsis. Five patients (7.1%) suffered a relapse during or after maintenance chemotherapy. The estimated 4-year event-free survival and overall survival (OS) rates were 82.1%±4.4%, 89.7%±5.1%, respectively. The 4-year OS was significantly higher in patients with initial WBC < 10×109/L than in those with initial WBC ≥ 10×109/L (p=0.020). @*Conclusion@#This study showed that the CR rates and survival outcomes in Korean pediatric APL patients were relatively good. The initial WBC count was the most important prognostic factor and most causes of death were related to serious bleeding in the early stage of treatment.

9.
Article in English | WPRIM (Western Pacific) | ID: wpr-924819

ABSTRACT

Health insurance big data provides real-world evidence of unmet needs in clinical practice and breakthroughs in the medical industry that will impact the future of health care. Big data is expected to revolutionize the current medical paradigm and usher in an era of personalized medicine. In Korea, the Health Insurance Review and Assessment Service and the National Health Insurance Service established large-capacity healthcare big data open systems in 2011 and 2013, respectively, and are providing researchers with secured healthcare big data. However, concerns have been raised regarding the quality of big data-based research. Thus, numerous obstacles remain in leveraging big data research to change medical practice. This paper describes the understanding and practical applications of healthcare big data in pediatric cancer research, ranging from clinical research design using health insurance big data to medical writing.

10.
Article in English | WPRIM (Western Pacific) | ID: wpr-889726

ABSTRACT

Purpose@#Atypical teratoid/rhabdoid tumor (ATRT) is a highly aggressive malignancy with peak incidence in children aged less than 3 years. Standard treatment for central nervous system ATRT in children under the age of 3 years have not been established yet. The objective of this study was to analyze characteristics and clinical outcomes of ATRT in children aged less than 3 years. @*Materials and Methods@#A search of medical records from seven centers was performed between January 2005 and December 2016. @*Results@#Forty-three patients were enrolled. With a median follow-up of 90 months, 27 patients (64.3%) showed at least one episode of disease progression (PD). The first date of PD was at 160 days after diagnosis. The 1- and 3-year progression-free survivals (PFS) were 51.2% and 28.5%, respectively. The 1- and 3-year overall survivals were 61.9% and 38.1%, respectively. The 3-year PFS was improved from 0% in pre-2011 to 47.4% in post-2011. Excluding one patient who did not receive any further therapy after surgery, 27 patients died due to PD (n=21), treatment-related toxicity (n=5), or unknown cause (n=1). In univariate analysis, factors associated with higher 3-year PFS were no metastases, diagnosis after 2011, early adjuvant radiotherapy, and high-dose chemotherapy (HDCT). In multivariate analysis, the use of HDCT and adjuvant radiotherapy remained significant prognostic factors for PFS (both p < 0.01). @*Conclusion@#Aggressive therapy including early adjuvant radiotherapy and HDCT could be considered to improve outcomes of ATRT in children under the age of 3 years.

11.
Cancer Research and Treatment ; : 1184-1194, 2021.
Article in English | WPRIM (Western Pacific) | ID: wpr-913813

ABSTRACT

Purpose@#Effectiveness and safety of clofarabine (one of the treatment mainstays in pediatric patients with relapsed/refractory acute lymphoblastic leukemia [ALL]) was assessed in Korean pediatric patients with ALL to facilitate conditional coverage with evidence development. @*Materials and Methods@#In this multicenter, prospective, observational study, patients receiving clofarabine as mono/combination therapy were followed up every 4-6 weeks for 6 months or until hematopoietic stem cell transplantation (HSCT). Response rates, survival outcomes, and adverse events were assessed. @*Results@#Sixty patients (2-26 years old; 65% B-cell ALL, received prior ≥ 2 regimen, 68.3% refractory to previous regimen) were enrolled and treated with at least one dose of clofarabine; of whom 26 (43.3%) completed 6 months of follow-up after the last dose of clofarabine. Fifty-eight patients (96.7%) received clofarabine combination therapy. Overall remission rate (complete remission [CR] or CR without platelet recovery [CRp]) was 45.0% (27/60; 95% confidence interval [CI], 32.4 to 57.6) and the overall response rate (CR, CRp, or partial remission [PR]) was 46.7% (28/60; 95% CI, 34.0 to 59.3), with 11 (18.3%), 16 (26.7%), and one (1.7%) patients achieving CR, CRp, and PR, respectively. The median time to remission was 5.1 weeks (95% CI, 4.7 to 6.1). Median duration of remission was 16.6 weeks (range, 2.0 to 167.6 weeks). Sixteen patients (26.7%) proceeded to HSCT. There were 24 deaths; 14 due to treatment-emergent adverse events. @*Conclusion@#Remission with clofarabine was observed in approximately half of the study patients who had overall expected safety profile; however, there was no favorable long-term survival outcome in this study.

12.
Article in English | WPRIM (Western Pacific) | ID: wpr-897430

ABSTRACT

Purpose@#Atypical teratoid/rhabdoid tumor (ATRT) is a highly aggressive malignancy with peak incidence in children aged less than 3 years. Standard treatment for central nervous system ATRT in children under the age of 3 years have not been established yet. The objective of this study was to analyze characteristics and clinical outcomes of ATRT in children aged less than 3 years. @*Materials and Methods@#A search of medical records from seven centers was performed between January 2005 and December 2016. @*Results@#Forty-three patients were enrolled. With a median follow-up of 90 months, 27 patients (64.3%) showed at least one episode of disease progression (PD). The first date of PD was at 160 days after diagnosis. The 1- and 3-year progression-free survivals (PFS) were 51.2% and 28.5%, respectively. The 1- and 3-year overall survivals were 61.9% and 38.1%, respectively. The 3-year PFS was improved from 0% in pre-2011 to 47.4% in post-2011. Excluding one patient who did not receive any further therapy after surgery, 27 patients died due to PD (n=21), treatment-related toxicity (n=5), or unknown cause (n=1). In univariate analysis, factors associated with higher 3-year PFS were no metastases, diagnosis after 2011, early adjuvant radiotherapy, and high-dose chemotherapy (HDCT). In multivariate analysis, the use of HDCT and adjuvant radiotherapy remained significant prognostic factors for PFS (both p < 0.01). @*Conclusion@#Aggressive therapy including early adjuvant radiotherapy and HDCT could be considered to improve outcomes of ATRT in children under the age of 3 years.

13.
Blood Research ; : 262-274, 2020.
Article in English | WPRIM (Western Pacific) | ID: wpr-889631

ABSTRACT

Background@#Lymphoblastic lymphoma (LBL) is the second most common subtype of pediatric non-Hodgkin lymphoma. Modified treatments derived from the LSA2-L2 regimen resulted in encouraging survival, but toxicities and long-term sequelae have been problematic. At present, the acute lymphoblastic leukemia (ALL)-type protocol has demonstrated efficacy in LBL. We analyzed the outcomes of children and adolescents with LBL treated with various regimens. @*Methods@#From 1991‒2018, this study enrolled 63 patients diagnosed with LBL at Asan Medical Center. Medical records were retrospectively analyzed. @*Results@#Among 63 patients, most patients (38.1%) presented with stage IV at diagnosis, and two had central nervous system (CNS) involvement. At a median follow-up of 160 months, the 5-year event free survival (EFS), overall survival (OS), and relapse free survival (RFS) were 68.8%, 79.3%, and 71.3%, respectively. Among 61 patients who received chemotherapy, 27 patients (44.3%) received the NY protocol, and 14 (23.0%) received the ALL-type protocol. There was no significant difference in 5-yr OS (85.2%/78.6%), EFS (73.5%/78.6%), and RFS (73.5%/78.6%) between the NY and ALL protocol groups, regardless of immunophenotype. Thirteen patients (21.3%) received prophylactic cranial radiotherapy with no difference in the incidence of CNS relapse based on irradiation. @*Conclusion@#This study showed no difference in outcome between the NY and ALL-type protocols, regardless of stage or immunophenotype. In addition to improving the effectiveness of treatment, it is necessary to continuously appraise the appropriate chemotherapy regimen, considering toxicities and long-term prognosis, for pediatric LBL.

14.
Blood Research ; : S5-S13, 2020.
Article in 0 | WPRIM (Western Pacific) | ID: wpr-830987

ABSTRACT

In recent decades, survival rates for childhood acute myeloid leukemia have remarkably improved, owing to chemotherapy intensification, allogeneic hematopoietic stem cell transplantation, and improved supportive care. Furthermore, treatment protocols have evolved and are currently better matched to prognostic factors and treatment responses. Recently, new molecular prognostic factors were discovered via leukemia genomic studies. Moreover, new tumor subtypes with independent gene expression profiles have been characterized. To broaden the therapeutic options for patients with poor prognoses, therapies that target specific candidate mutations are being identified. Additionally, new drugs are undergoing clinical trials, and immunotherapy is attracting significant interest as a treatment option for recurrent or refractory childhood acute myeloid leukemia.

15.
Jae-Min LEE; Jung-Yoon CHOI; Kyung-Taek HONG; Hyoung-Jin KANG; Hee-Young SHIN; Hee-Jo BAEK; Seongkoo KIM; Jae-Wook LEE; Nack-Gyun CHUNG; Bin CHO; Seok-Goo CHO; Kyung-Mi PARK; Eu-Jeen YANG; Young-Tak LIM; Jin-Kyung SUH; Sung-Han KANG; Hyery KIM; Kyung-Nam KOH; Ho-Joon IM; Jong-Jin SEO; Hee-Won CHO; Hee-Young JU; Ji-Won LEE; Keon-Hee YOO; Ki-Woong SUNG; Hong-Hoe KOO; Kyung-Duk PARK; Jeong-Ok HAH; Min-Kyoung KIM; Jung-Woo HAN; Seung-Min HAHN; Chuhl-Joo LYU; Ye-Jee SHIM; Heung-Sik KIM; Young-Rok DO; Jae-Won YOO; Yeon-Jung LIM; In-Sang JEON; Hee-won CHUEH; Sung-Yong OH; Hyoung-Soo CHOI; Jun-Eun PARK; Jun-Ah LEE; Hyeon-Jin PARK; Byung-Kiu PARK; Soon-Ki KIM; Jae-Young LIM; Eun-Sil PARK; Sang-Kyu PARK; Eun-Jin CHOI; Young-Bae CHOI; Jong-Hyung YOON; Hoon KOOK; Jae-Min LEE; Jung-Yoon CHOI; Kyung-Taek HONG; Hyoung-Jin KANG; Hee-Young SHIN; Hee-Jo BAEK; Seongkoo KIM; Jae-Wook LEE; Nack-Gyun CHUNG; Bin CHO; Seok-Goo CHO; Kyung-Mi PARK; Eu-Jeen YANG; Young-Tak LIM; Jin-Kyung SUH; Sung-Han KANG; Hyery KIM; Kyung-Nam KOH; Ho-Joon IM; Jong-Jin SEO; Hee-Won CHO; Hee-Young JU; Ji-Won LEE; Keon-Hee YOO; Ki-Woong SUNG; Hong-Hoe KOO; Kyung-Duk PARK; Jeong-Ok HAH; Min-Kyoung KIM; Jung-Woo HAN; Seung-Min HAHN; Chuhl-Joo LYU; Ye-Jee SHIM; Heung-Sik KIM; Young-Rok DO; Jae-Won YOO; Yeon-Jung LIM; In-Sang JEON; Hee-won CHUEH; Sung-Yong OH; Hyoung-Soo CHOI; Jun-Eun PARK; Jun-Ah LEE; Hyeon-Jin PARK; Byung-Kiu PARK; Soon-Ki KIM; Jae-Young LIM; Eun-Sil PARK; Sang-Kyu PARK; Eun-Jin CHOI; Young-Bae CHOI; Jong-Hyung YOON; Hoon KOOK.
Article in English | WPRIM (Western Pacific) | ID: wpr-831569

ABSTRACT

Background@#Hodgkin's lymphoma (HL) constitutes 10%–20% of all malignant lymphomas and has a high cure rate (5-year survival, around 90%). Recently, interest has increased concerning preventing secondary complications (secondary cancer, endocrine disorders) in long-term survivors. We aimed to study the epidemiologic features and therapeutic outcomes of HL in children, adolescents, and young adults in Korea. @*Methods@#We performed a multicenter, retrospective study of 224 patients aged < 25 years diagnosed with HL at 22 participating institutes in Korea from January 2007 to August 2016. @*Results@#A higher percentage of males was diagnosed at a younger age. Nodular sclerosis histopathological HL subtype was most common, followed by mixed cellularity subtype.Eighty-one (36.2%), 101 (45.1%), and 42 (18.8%) patients were classified into low, intermediate, and high-risk groups, respectively. Doxorubicin, bleomycin, vinblastine, dacarbazine was the most common protocol (n = 102, 45.5%). Event-free survival rate was 86.0% ± 2.4%, while five-year overall survival (OS) rate was 96.1% ± 1.4%: 98.7% ± 1.3%, 97.7% ± 1.6%, and 86.5% ± 5.6% in the low, intermediate, and high-risk groups, respectively (P = 0.021). Five-year OS was worse in patients with B-symptoms, stage IV disease, highrisk, splenic involvement, extra-nodal lymphoma, and elevated lactate dehydrogenase level.In multivariate analysis, B-symptoms and extra-nodal involvement were prognostic factors for poor OS. Late complications of endocrine disorders and secondary malignancy were observed in 17 and 6 patients, respectively. @*Conclusion@#This is the first study on the epidemiology and treatment outcomes of HL in children, adolescents, and young adults in Korea. Future prospective studies are indicated to develop therapies that minimize treatment toxicity while maximizing cure rates in children, adolescents, and young adults with HL.

16.
Article in 0 | WPRIM (Western Pacific) | ID: wpr-832096

ABSTRACT

Hepatoblastoma is the most common malignant hepatic tumor in infants and young children and accounts for approximately 1% of all pediatric malignancies. A treatment strategy incorporating chemotherapy and surgical resection has evolved based on the results of the multicenter clinical trials performed by the major liver study groups during the last two decades and led to significantly improved survival outcomes. The alpha-fetoprotein level, PRE-Treatment EXTent tumor stage, and histological category are well-known prognostic factors that are used for risk stratification. Platinum-based chemotherapy regimens are effective in terms of increasing the likelihood of surgical resectability. Refinement of surgical techniques and the advent of liver transplantation have improved the outcomes in patients with advanced tumors. However, the optimal treatment strategy for advanced hepatoblastoma remains unclear. Unanswered questions include the optimal timing and indications for pulmonary metastasectomy and when the surgical strategy should be complex liver resection or liver transplantation. The major liver study groups have now formed a global coalition known as the Children’s Hepatic tumors International Collaboration and developed an international staging system. The aim of this article is to review current treatment strategies of hepatoblastoma focusing on high risk patients.

17.
Blood Research ; : 262-274, 2020.
Article in English | WPRIM (Western Pacific) | ID: wpr-897335

ABSTRACT

Background@#Lymphoblastic lymphoma (LBL) is the second most common subtype of pediatric non-Hodgkin lymphoma. Modified treatments derived from the LSA2-L2 regimen resulted in encouraging survival, but toxicities and long-term sequelae have been problematic. At present, the acute lymphoblastic leukemia (ALL)-type protocol has demonstrated efficacy in LBL. We analyzed the outcomes of children and adolescents with LBL treated with various regimens. @*Methods@#From 1991‒2018, this study enrolled 63 patients diagnosed with LBL at Asan Medical Center. Medical records were retrospectively analyzed. @*Results@#Among 63 patients, most patients (38.1%) presented with stage IV at diagnosis, and two had central nervous system (CNS) involvement. At a median follow-up of 160 months, the 5-year event free survival (EFS), overall survival (OS), and relapse free survival (RFS) were 68.8%, 79.3%, and 71.3%, respectively. Among 61 patients who received chemotherapy, 27 patients (44.3%) received the NY protocol, and 14 (23.0%) received the ALL-type protocol. There was no significant difference in 5-yr OS (85.2%/78.6%), EFS (73.5%/78.6%), and RFS (73.5%/78.6%) between the NY and ALL protocol groups, regardless of immunophenotype. Thirteen patients (21.3%) received prophylactic cranial radiotherapy with no difference in the incidence of CNS relapse based on irradiation. @*Conclusion@#This study showed no difference in outcome between the NY and ALL-type protocols, regardless of stage or immunophenotype. In addition to improving the effectiveness of treatment, it is necessary to continuously appraise the appropriate chemotherapy regimen, considering toxicities and long-term prognosis, for pediatric LBL.

19.
Article in English | WPRIM (Western Pacific) | ID: wpr-763508

ABSTRACT

In recent decades, survival rates for childhood acute lymphoblastic leukemia have improved remarkably, as demonstrated by risk-stratified, prospective multicenter studies. Treatment protocols have evolved and become better matched to both prognostic factors and treatment responses. Recently, new molecular prognostic factors have been discovered in leukemia genomic studies. New tumor subtypes with independent gene expression profiles have also been characterized. Furthermore, therapies targeted to specific candidate mutations are being identified to broaden therapeutic options for patients with poor prognoses. Many new drugs are in clinical trials and immunotherapy is attracting significant interest for the treatment of recurrent or refractory disease in childhood acute lymphoblastic leukemia.


Subject(s)
Humans , Clinical Protocols , Immunotherapy , Leukemia , Precursor Cell Lymphoblastic Leukemia-Lymphoma , Prognosis , Prospective Studies , Survival Rate , Transcriptome
20.
Article in English | WPRIM (Western Pacific) | ID: wpr-719418

ABSTRACT

PURPOSE: Dexrazoxane has been used as an effective cardioprotector against anthracycline cardiotoxicity. This study intended to analyze cardioprotective efficacy and secondary malignancy development, and elucidate risk factors for secondary malignancies in dexrazoxane-treated pediatric patients. MATERIALS AND METHODS: Data was collected from 15 hospitals in Korea. Patients who received any anthracyclines, and completed treatment without stem cell transplantation were included. For efficacy evaluation, the incidence of cardiac events and cardiac event-free survival rates were compared. Data about risk factors of secondary malignancies were collected. RESULTS: Data of total 1,453 cases were analyzed; dexrazoxane with every anthracyclines group (D group, 1,035 patients) and no dexrazoxane group (non-D group, 418 patients). Incidence of the reported cardiac events was not statistically different between two groups; however, the cardiac event-free survival rate of patients with more than 400 mg/m2 of anthracyclines was significantly higher in D group (91.2% vs. 80.1%, p=0.04). The 6-year cumulative incidence of secondary malignancy was not different between both groups after considering follow-up duration difference (non-D, 0.52%±0.37%; D, 0.60%±0.28%; p=0.55). The most influential risk factor for secondary malignancy was the duration of anthracycline administration according to multivariate analysis. CONCLUSION: Dexrazoxane had an efficacy in lowering cardiac event-free survival rates in patients with higher cumulative anthracyclines. As a result of multivariate analysis for assessing risk factors of secondary malignancy, the occurrence of secondary malignancy was not related to dexrazoxane administration.


Subject(s)
Humans , Anthracyclines , Cardiotoxicity , Dexrazoxane , Disease-Free Survival , Follow-Up Studies , Incidence , Korea , Multivariate Analysis , Neoplasms, Second Primary , Risk Factors , Stem Cell Transplantation
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