- Pediatric cancer
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Ten-Year Trends of Hematopoietic Stem Cell Transplantation in Korean Pediatric Cancer from the National Health Insurance Claims Data
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Hyery Kim, Hwa Jung Kim, Youngjun Jo, Su Hyun Yoon, Young Kwon Koh, Sunghan Kang, Kyung-Nam Koh, Ho Joon Im
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Cancer Res Treat. 2024;56(1):294-304. Published online September 7, 2023
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DOI: https://doi.org/10.4143/crt.2023.598
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Abstract
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- 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.
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Epidemiologic and Clinical Outcomes of Pediatric Renal Tumors in Korea: A Retrospective Analysis of The Korean Pediatric Hematology and Oncology Group (KPHOG) Data
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Kyung-Nam Koh, Jung Woo Han, Hyoung Soo Choi, Hyoung Jin Kang, Ji Won Lee, Keon Hee Yoo, Ki Woong Sung, Hong Hoe Koo, Kyung Taek Hong, Jung Yoon Choi, Sung Han Kang, Hyery Kim, Ho Joon Im, Seung Min Hahn, Chuhl Joo Lyu, Hee-Jo Baek, Hoon Kook, Kyung Mi Park, Eu Jeen Yang, Young Tak Lim, Seongkoo Kim, Jae Wook Lee, Nack-Gyun Chung, Bin Cho, Meerim Park, Hyeon Jin Park, Byung-Kiu Park, Jun Ah Lee, Jun Eun Park, Soon Ki Kim, Ji Yoon Kim, Hyo Sun Kim, Youngeun Ma, Kyung Duk Park, Sang Kyu Park, Eun Sil Park, Ye Jee Shim, Eun Sun Yoo, Kyung Ha Ryu, Jae Won Yoo, Yeon Jung Lim, Hoi Soo Yoon, Mee Jeong Lee, Jae Min Lee, In-Sang Jeon, Hye Lim Jung, Hee Won Chueh, Seunghyun Won, the Korean Pediatric Hematology and Oncology Group (KPHOG)
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Cancer Res Treat. 2023;55(1):279-290. Published online August 11, 2022
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DOI: https://doi.org/10.4143/crt.2022.073
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Abstract
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- 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.
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Citations
Citations to this article as recorded by 
- Hope and challenges in the diagnosis and treatment of Wilms tumor: a single-center retrospective study in China
Kongkong Cui, Peng Hong, Jie Lin, Zaihong Hu, Zhiqiang Gao, XiaoMao Tian, Tao Lin, Qinlin Shi, Guanghui Wei Frontiers in Pediatrics.2025;[Epub] CrossRef - Loss of YTHDF1 suppresses the progression of malignant rhabdoid tumor of the kidney by regulating Glutathione S-transferase Mu 2 (GSTM2)
Qian-Wen Xiong, Yuntao Liu, Min He, Xiao-Die Shen, Manli Zhao, Shuang-Ai Liu, Gensheng Zhang, Qian Liu, Jinhu Wang, Wan-Xin Peng Cell Biology and Toxicology.2025;[Epub] CrossRef - Congenital Mesoblastic Nephroma Mimic Wilms Tumor on 18F-FDG PET/CT and PET/MR
Wenzhu Hu, Chunxia Qin, Fuqiang Shao, Mengting Li, Xiaoli Lan Clinical Nuclear Medicine.2024; 49(4): 353. CrossRef - Progress towards Therapies for Solid Renal Tumors in Children
洁 林 Advances in Clinical Medicine.2024; 14(06): 245. CrossRef
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Clinical Characteristics and Treatment Outcomes of Childhood Acute Promyelocytic Leukemia in Korea: A Nationwide Multicenter Retrospective Study by Korean Pediatric Oncology Study Group
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Kyung Mi Park, Keon Hee Yoo, Seong Koo Kim, Jae Wook Lee, Nack-Gyun Chung, Hee Young Ju, Hong Hoe Koo, Chuhl Joo Lyu, Seung Min Han, Jung Woo Han, Jung Yoon Choi, Kyung Taek Hong, Hyoung Jin Kang, Hee Young Shin, Ho Joon Im, Kyung-Nam Koh, Hyery Kim, Hoon Kook, Hee Jo Baek, Bo Ram Kim, Eu Jeen Yang, Jae Young Lim, Eun Sil Park, Eun Jin Choi, Sang Kyu Park, Jae Min Lee, Ye Jee Shim, Ji Yoon Kim, Ji Kyoung Park, Seom Gim Kong, Young Bae Choi, Bin Cho, Young Tak Lim
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Cancer Res Treat. 2022;54(1):269-276. Published online April 20, 2021
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DOI: https://doi.org/10.4143/crt.2021.313
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Abstract
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- 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.
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Citations
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- Management of Acute Promyelocytic Leukemia at Extremes of Age
Sabine Kayser, Shannon E. Conneely Cancers.2023; 15(14): 3637. CrossRef - Current Challenges of Asian National Children's Cancer Study Groups on Behalf of Asian Pediatric Hematology and Oncology Group
Chi-kong Li, Purna Kurkure, Ramandeep Singh Arora, Bow Wen Chen, Kirill Kirgizov, Yasuhiro Okamoto, Panya Seksarn, Yongmin Tang, Keon Hee Yoo, Bharat Agarwal, Godfrey C.F. Chan, Rashmi Dalvi, Hiroki Hori, Muhammad Saghir Khan, Alice Yu, Akira Nakagawara JCO Global Oncology.2023;[Epub] CrossRef - Childhood acute promyelocytic leukemia in a pediatric cancer referral center in Baghdad, Iraq. Improved results with ATRA extended consolidation
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Lila Krebs-Drouot, Georgia Karpathiou, Virginie Scolan, Carolyne Bidat-Callet, Baptiste Boyer, Michel Péoc’h Forensic Science, Medicine and Pathology.2022; 19(1): 60. CrossRef - Successful Treatment of Isolated Central Nervous System Relapse with Intrathecal Chemotherapy in an Adolescent with Acute Promyelocytic Leukemia
Haerim Song, Eun Sang Yi Clinical Pediatric Hematology-Oncology.2022; 29(2): 70. CrossRef
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Effectiveness and Safety of Clofarabine Monotherapy or Combination Treatment in Relapsed/Refractory Childhood Acute Lymphoblastic Leukemia: A Pragmatic, Non-interventional Study in Korea
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Jung Yoon Choi, Che Ry Hong, Kyung Taek Hong, Hyoung Jin Kang, Seongkoo Kim, Jae Wook Lee, Pil Sang Jang, Nack-Gyun Chung, Bin Cho, Hyery Kim, Kyung-Nam Koh, Ho Joon Im, Jong Jin Seo, Seung Min Hahn, Jung Woo Han, Chuhl Joo Lyu, Eu Jeen Yang, Young Tak Lim, Keon Hee Yoo, Hong Hoe Koo, Hoon Kook, In Sang Jeon, Hana Cho, Hee Young Shin
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Cancer Res Treat. 2021;53(4):1184-1194. Published online January 4, 2021
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DOI: https://doi.org/10.4143/crt.2020.289
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Abstract
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- 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.
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Swathi Putta, Santhosh Kumar Chinnaiyan, Ramadevi Korni, Venkata Radha Gadela Journal of Drug Delivery Science and Technology.2025; 104: 106556. CrossRef - Proteomic analysis reveals chromatin remodeling as a potential therapeutical target in neuroblastoma
Zan Liu, Zitong Zhao, Longlong Xie, Zhenghui Xiao, Ming Li, Yong Li, Ting Luo Journal of Translational Medicine.2025;[Epub] CrossRef - 3′-O-β-Glucosylation of nucleoside analogues using a promiscuous bacterial glycosyltransferase
Jonathan P. Dolan, Tessa Keenan, Aisling Ní Cheallaigh, Martin A. Fascione, Gavin J. Miller RSC Chemical Biology.2025;[Epub] CrossRef - Opciones para el tratamiento de la recaída en leucemia linfoblástica aguda. Revisión de tema
Christian Omar Ramos-Peñafiel, Carlos Martínez-Murillo, Daniela Pérez-Sámano, Camila Terreros-Palacios, Adán Germán Gallardo-Rodríguez, Irma Olarte-Carrillo, Adolfo Martínez-Tovar Revista Médicas UIS.2024;[Epub] CrossRef - Clofarabine
Reactions Weekly.2023; 1958(1): 155. CrossRef - Patient-Level Meta-analysis of Clofarabine in Acute Lymphoblastic Leukemia
Sima Jeha, Hiroaki Goto, André Baruchel, Emmanuelle Boëlle-Le Corfec, Christine Geffriaud-Ricouard, Rob Pieters, Hee Young Shin Advances in Therapy.2023; 40(12): 5447. CrossRef - Novel Treatments for Pediatric Relapsed or Refractory Acute B-Cell Lineage Lymphoblastic Leukemia: Precision Medicine Era
Shang Mengxuan, Zhou Fen, Jin Runming Frontiers in Pediatrics.2022;[Epub] CrossRef
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- Central nervous system
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Atypical Teratoid/Rhabdoid Tumor of the Central Nervous System in Children under the Age of 3 Years
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Meerim Park, Jung Woo Han, Seung Min Hahn, Jun Ah Lee, Joo-Young Kim, Sang Hoon Shin, Dong-Seok Kim, Hong In Yoon, Kyung Taek Hong, Jung Yoon Choi, Hyoung Jin Kang, Hee Young Shin, Ji Hoon Phi, Seung-Ki Kim, Ji Won Lee, Keon Hee Yoo, Ki Woong Sung, Hong Hoe Koo, Do Hoon Lim, Hyung Jin Shin, Hyery Kim, Kyung-Nam Koh, Ho Joon Im, Seung Do Ahn, Young-Shin Ra, Hee-Jo Baek, Hoon Kook, Tae-Young Jung, Hyoung Soo Choi, Chae-Yong Kim, Hyeon Jin Park, Chuhl Joo Lyu
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Cancer Res Treat. 2021;53(2):378-388. Published online October 28, 2020
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DOI: https://doi.org/10.4143/crt.2020.756
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Abstract
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- 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.
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Risk Factor Analysis for Secondary Malignancy in Dexrazoxane-Treated Pediatric Cancer Patients
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Hyery Kim, Hyoung Jin Kang, Kyung Duk Park, Kyung-Nam Koh, Ho Joon Im, Jong Jin Seo, Jae Wook Lee, Nack-Gyun Chung, Bin Cho, Hack Ki Kim, Jae Min Lee, Jeong Ok Hah, Jun Ah Lee, Young Ho Lee, Sang Kyu Park, Hee Jo Baek, Hoon Kook, Ji Yoon Kim, Heung Sik Kim, Hwang Min Kim, Hee Won Chueh, Meerim Park, Hoi Soo Yoon, Mee Jeong Lee, Hyoung Soo Choi, Hyo Seop Ahn, Yoshifumi Kawano, Ji Won Park, Seokyung Hahn, Hee Young Shin
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Cancer Res Treat. 2019;51(1):357-367. Published online May 14, 2018
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DOI: https://doi.org/10.4143/crt.2017.457
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Abstract
PDF Supplementary Material PubReader ePub
- 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.
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Veronika Keresteš, Jan Kubeš, Lenka Applová, Petra Kollárová, Olga Lenčová-Popelová, Iuliia Melnikova, Galina Karabanovich, Mushtaq M Khazeem, Hana Bavlovič-Piskáčková, Petra Štěrbová-Kovaříková, Caroline A Austin, Jaroslav Roh, Martin Štěrba, Tomáš Šimůn Toxicological Sciences.2024; 198(2): 288. CrossRef - Circ-0006332 stimulates cardiomyocyte pyroptosis via the miR-143/TLR2 axis to promote doxorubicin-induced cardiac damage
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Parya Rahimi, Behsheed Barootkoob, Ahmed ElHashash, Arun Nair Cureus.2023;[Epub] CrossRef - Inducing a Proinflammatory Response with Bioengineered Yeast Vacuoles with TLR2-Binding Peptides (VacT2BP) as a Drug Carrier for Daunorubicin Delivery
Wooil Choi, Woo-Ri Shin, Yang-Hoon Kim, Jiho Min ACS Applied Materials & Interfaces.2023; 15(35): 41258. CrossRef - Circulating Biomarkers for Monitoring Chemotherapy-Induced Cardiotoxicity in Children
Luigia Meo, Maria Savarese, Carmen Munno, Peppino Mirabelli, Pia Ragno, Ornella Leone, Mariaevelina Alfieri Pharmaceutics.2023; 15(12): 2712. CrossRef - Hyperhomocysteinemia as a Link of Chemotherapy-Related Endothelium Impairment
Ashot Avagimyan Current Problems in Cardiology.2022; 47(10): 100932. CrossRef - Late health outcomes after dexrazoxane treatment: A report from the Children's Oncology Group
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Unraveling the Genetic Landscape of Langerhans Cell Histiocytosis in Korean Patients: Comprehensive Insights from Mutational Profiles and Clinical Correlations
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Kyung-Nam Koh, Ha Ra Jun, Ji-Young Lee, Ji Young Kim, Su Hyun Yoon, Young Kwon Koh, Sung Han Kang, Hyery Kim, Ho Joon Im, Sung-Min Chun
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Received August 14, 2024 Accepted December 22, 2024 Published online December 24, 2024
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DOI: https://doi.org/10.4143/crt.2024.782
[Epub ahead of print]
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Abstract
PDF PubReader ePub
- Purpose
This study aimed to conduct a comprehensive genetic analysis of patients with Langerhans cell histiocytosis (LCH), focusing on the frequency of mitogen-activated protein kinase (MAPK) pathway mutations, detailed mutation profiles of MAPK pathway genes, and their correlation with clinical features and prognosis in Korean LCH patients.
Materials and Methods We performed targeted next-generation sequencing, capable of capturing exons from 382 cancer-related genes, on genomic DNA extracted from formaldehyde-fixed and paraffin-embedded samples of 45 pathologically confirmed LCH patients.
Results The majority of patients (91.1%) exhibited single-system disease, with bone being the most common location (84.4%). Initial treatments varied, and no patients died during a median follow-up of 6.8 years. Our genetic assays revealed that all patients had MAPK pathway alterations, including BRAF mutations in 51.2%, MAP2K1 mutations in 42.2%, RAF1 mutations in 4.4%, and a KRAS mutation in 2.2%. These mutations were mutually exclusive. Detailed mutation profiles indicated that among the BRAF mutations, there were 18 point mutations and five in-frame deletions, while most MAP2K1 mutations were in-frame deletions, with only one missense mutation. We detected previously unreported variations of point mutations in BRAF, MAP2K1, KRAS, and the first instance of a RAF1-KLC1 fusion in LCH. MAP2K1 mutations occurred more frequently in older patients, whereas BRAF V600 mutations were commonly associated with unifocal bone disease. Genetic mutations did not correlate with high-risk features or event-free survival.
Conclusion This study identified mutually exclusive MAPK pathway mutations in every LCH patient through comprehensive genetic analysis, highlighting the importance of inclusive testing in understanding the disease’s genetics.
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- Congenital Langerhans Cell Histiocytosis With Novel KCL1::RAF1 Gene Fusion Identified Through Routine Whole‐Genome Sequencing
Fiona E. Wright, Gemma Barnard, Shivani Bailey, C. Elizabeth Hook, Nicholas Coleman, Natasha Stembridge, Rowena Guermech, James Watkins, Jamie Trotman, Patrick Tarpey, Vasanta Nanduri, Matthew J. Murray Pediatric Blood & Cancer.2025;[Epub] CrossRef
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Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric and Young Adult Patients with Chronic Myeloid Leukemia in Tyrosine Kinase Inhibitor Era: A Study of the Korean Blood and Marrow Transplantation Registry
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Hee Young Ju, Hyoung Soo Choi, Hyeon Jin Park, Keon Hee Yoo, Chuhl Joo Lyu, Ho Joon Im, Min Kyoung Kim, Yeung-Chul Mun, Joon Ho Moon, Sung-Soo Yoon, Eunyoung Lee, Jae Hoon Lee, Je-Hwan Lee, So Young Chong, June-Won Cheong, Seunghyun Won, on behalf of the Korean Society of Blood and Marrow Transplantation
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Received December 10, 2024 Accepted April 29, 2025 Published online May 7, 2025
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DOI: https://doi.org/10.4143/crt.2024.1186
[Accepted]
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Abstract
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Chronic myeloid leukemia (CML) in children, adolescents, and young adults is rare and differs from older adults. This study evaluated the outcomes of allogeneic hematopoietic stem cell transplantation (HSCT) in young Korean CML patients during the tyrosine kinase inhibitor (TKI) era.
Materials and Methods
A retrospective analysis of 35 CML patients aged <40 years who underwent allogeneic HSCT from 2009 to 2019 was conducted using Korean Blood and Marrow Transplantation Registry data. Patients were grouped by age <20 years at HSCT (Group 1, n=15) and 20-40 years at HSCT (Group 2, n=20). Survival outcomes including overall survival (OS), relapse-free survival (RFS), and event-free survival (EFS) were analyzed using the Kaplan–Meier method.
Results
The median time between diagnosis and HSCT was 8.9 months. All the patients achieved engraftment but platelet recovery was significantly slower in Group 1 (p=0.034). Acute and chronic graft-versus-host disease occurred in 54.3% and 34.3%, respectively. Five-year OS, RFS, and EFS rates of total patients were 66.8%, 50.8%, and 47.6%, with better OS was observed in Group 1 by multivariable analysis (p=0.048). Disease status at HSCT was a significant predictor of OS (p=0.028), RFS (p=0.003) and EFS (p=0.004). Disease progression occurred in 13 out of 35 patients (37.1%); treatment-related mortality accounted for 63.6% of deaths (7 out of 11).
Conclusion
When performed at a younger age, allogeneic HSCT result in superior outcome in CML. Achieving remission before HSCT is critical for improved outcomes, highlighting the importance of pretransplant remission via optimal TKI strategies and minimal residual disease monitoring.
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