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Pediatric cancer
Long-term Outcomes of Protocol-Based Treatment for Newly Diagnosed Medulloblastoma
Won Kee Ahn, Seung Min Hahn, Hong In Yoon, Jeongshim Lee, Eun Kyung Park, Kyu Won Shim, Dong Seok Kim, Chang-Ok Suh, Se Hoon Kim, Chuhl Joo Lyu, Jung Woo Han
Cancer Res Treat. 2024;56(2):652-664.   Published online November 30, 2023
DOI: https://doi.org/10.4143/crt.2023.865
AbstractAbstract PDFSupplementary MaterialPubReaderePub
Purpose
The Korean Society of Pediatric Neuro-Oncology (KSPNO) conducted treatment strategies for children with medulloblastoma (MB) by using alkylating agents for maintenance chemotherapy or tandem high-dose chemotherapy (HDC) with autologous stem cell rescue (ASCR) according to the risk stratification. The purpose of the study was to assess treatment outcomes and complications based on risk-adapted treatment and HDC.
Materials and Methods
Fifty-nine patients diagnosed with MB were enrolled in this study. Patients in the standard-risk (SR) group received radiotherapy (RT) after surgery and chemotherapy using the KSPNO M051 regimen. Patients in the high-risk (HR) group received two and four chemotherapy cycles according to the KSPNO S081 protocol before and after reduced RT for age following surgery and two cycles of tandem HDC with ASCR consolidation treatment.
Results
In the SR group, 24 patients showed 5-year event-free survival (EFS) and overall survival (OS) estimates of 86.7% (95% confidence interval [CI], 73.6 to 100) and 95.8% (95% CI, 88.2 to 100), respectively. In the HR group, more infectious complications and mortality occurred during the second HDC than during the first. In the HR group, the 5-year EFS and OS estimates were 65.5% (95% CI, 51.4 to 83.4) and 72.3% (95% CI, 58.4 to 89.6), respectively.
Conclusion
High intensity of alkylating agents for SR resulted in similar outcomes but with a high incidence of hematologic toxicity. Tandem HDC with ASCR for HR induced favorable EFS and OS estimates compared to those reported previously. However, infectious complications and treatment-related mortalities suggest that a reduced chemotherapy dose is necessary, especially for the second HDC.

Citations

Citations to this article as recorded by  
  • Ferroptosis Transcriptional Regulation and Prognostic Impact in Medulloblastoma Subtypes Revealed by RNA-Seq
    Christophe Desterke, Yuanji Fu, Jenny Bonifacio-Mundaca, Claudia Monge, Pascal Pineau, Jorge Mata-Garrido, Raquel Francés
    Antioxidants.2025; 14(1): 96.     CrossRef
  • 3,472 View
  • 161 Download
  • 1 Web of Science
  • 1 Crossref
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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
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)
Cancer Res Treat. 2023;55(1):279-290.   Published online August 11, 2022
DOI: https://doi.org/10.4143/crt.2022.073
AbstractAbstract PDFSupplementary MaterialPubReaderePub
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.

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
  • 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
  • 7,442 View
  • 195 Download
  • 1 Web of Science
  • 3 Crossref
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The Efficacy of Alternate Systemic Intravenous Chemotherapy and Intra-arterial Chemotherapy Approach for Eye Globe Salvage in Retinoblastoma
Jung Woo Han, Christopher Seungkyu Lee, Seung Min Hahn, Won Kee Ahn, Hyo Sun Kim, Hyeseon Yun, Sung Chul Lee, Byung Moon Kim, Dong Joon Kim, Chuhl Joo Lyu
Cancer Res Treat. 2023;55(1):270-278.   Published online May 24, 2022
DOI: https://doi.org/10.4143/crt.2021.1537
AbstractAbstract PDFSupplementary MaterialPubReaderePub
Purpose
The advances in the treatment of retinoblastoma have enabled salvaging the globe in advanced stages with intra-arterial chemotherapy (IAC). We developed a strategy of alternate application of systemic intravenous chemotherapy (IVC) and IAC (referred to as alternate systemic IVC and IAC; ASIAC) to reduce central nervous metastases during IAC and examined its efficacy and safety in eye globe salvage in this study.
Materials and Methods
Between January 2010 and February 2021, 43 eyes of 40 patients received ASIAC treatment for retinoblastoma at the Yonsei Cancer Center, Yonsei University Health System. Their medical records were reviewed retrospectively to evaluate the eye salvage rate (ESR), defined from diagnosis to enucleation. High-risk retinoblastoma was defined as group D or E by the International Classification of Retinoblastoma.
Results
The study enrolled 38 and five cases of high-risk and low-risk retinoblastoma, respectively. In total, 178 IAC and 410 IVC courses were administered, with a median of 4 (interquartile range [IQR], 3.0 to 5.0) IAC and 9 (IQR, 6.0 to 11) IVC courses per eye, respectively. The 5-year ESR was 60.4%±8.7% for the whole cohort, 100% for low-risk retinoblastoma, and 53.6%±9.8% for high-risk retinoblastoma. Among those diagnosed since 2015, the 5-year ESR for high-risk retinoblastoma was 63.5%±14.0%. Fifteen eyes underwent enucleation; no viable tumor was found in three enucleated eyes. There were no deaths in this cohort.
Conclusion
Primary IAC-IVC (i.e., ASIAC) for patients with retinoblastoma was tolerable and effective in salvaging the eye and maintaining survival.

Citations

Citations to this article as recorded by  
  • Intra-arterial chemotherapy for unilateral advanced intraocular retinoblastoma: A long-term review of a case series
    Yixiao Li, Minglei Han, Dan Song, Jing Li, Zhuang Liu, Xin Zhang, Liang Wang, Lei Guo
    Journal of Cancer Research and Therapeutics.2025; 21(2): 442.     CrossRef
  • Selective ophthalmic arterial injection using a balloon catheter for retinoblastoma: a seven-year clinical evaluation
    Sota Oguro, Yi Ning Chen, Takashi Yamane, Makoto Mohri, Shigenobu Suzuki
    Japanese Journal of Ophthalmology.2024; 68(4): 346.     CrossRef
  • Hematological Second Primary Malignancy in Pediatric Retinoblastoma: A Case Report and Systematic Review
    Seung Hyun Park, Hyun Young Park, Heejin Kim, Jung Woo Han, Jin Sook Yoon
    Ophthalmic Plastic & Reconstructive Surgery.2024; 40(5): 487.     CrossRef
  • 5,816 View
  • 137 Download
  • 3 Web of Science
  • 3 Crossref
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Breast cancer
Implication and Influence of Multigene Panel Testing with Genetic Counseling in Korean Patients with BRCA1/2 Mutation-Negative Breast Cancer
Ji Soo Park, Saeam Shin, Yoon Jung Lee, Seung-Tae Lee, Eun Ji Nam, Jung Woo Han, Sun Hwa Lee, Tae Il Kim, Hyung Seok Park
Cancer Res Treat. 2022;54(4):1099-1110.   Published online November 17, 2021
DOI: https://doi.org/10.4143/crt.2021.978
AbstractAbstract PDFSupplementary MaterialPubReaderePub
Purpose
The aim of the study was to evaluate the clinical implication of multigene panel testing of beyond BRCA genes in Korean patients with BRCA1/2 mutation-negative breast cancer.
Materials and Methods
Between 2016 and 2019, a total of 700 BRCA1/2 mutation-negative breast cancer patients received comprehensive multigene panel testing and genetic counseling. Among them, 347 patients completed a questionnaire about cancer worry, genetic knowledge, and preference for the method of genetic tests during pre- and post-genetic test counseling. The frequency of pathogenic and likely pathogenic variants (PV/LPV) were analyzed.
Results
At least one PV/LPV of 26 genes was found in 76 out of 700 patients (10.9 %). The rate for PV/LPV was 3.4% for high-risk genes (17 PALB2, 6 TP53, and 1 PTEN). PV/LPVs of clinical actionable genes for breast cancer management, high-risk genes and other moderate-risk genes such as ATM, BARD1, BRIP, CHEK2, NF1, and RAD51D, were observed in 7.4%. Patients who completed the questionnaire showed decreased concerns about the risk of additional cancer development (average score, 4.21 to 3.94; p < 0.001), influence on mood (3.27 to 3.13; p < 0.001), influence on daily functioning (3.03 to 2.94; p=0.006); and increased knowledge about hereditary cancer syndrome (66.9 to 68.8; p=0.025) in post-test genetic counseling. High cancer worry scales (CWSs) were associated with age ≤ 40 years and the identification of PV/LPV. Low CWSs were related to the satisfaction of the counselee.
Conclusion
Comprehensive multigene panel test with genetic counseling is clinically applicable. It should be based on interpretable genetic information, consideration of potential psychological consequences, and proper preventive strategies.

Citations

Citations to this article as recorded by  
  • Evidence-based advancements in breast cancer genetic counseling: a review
    Zahra Batool, Mohammad Amjad Kamal, Bairong Shen
    Breast Cancer.2025; 32(2): 258.     CrossRef
  • Exploring Literacy and Knowledge Gaps and Disparities in Genetics and Oncogenomics Among Cancer Patients and the General Population: A Scoping Review
    Katerina Nikitara, Maria Luis Cardoso, Astrid Moura Vicente, Célia Maria Batalha Silva Rasga, Roberta De Angelis, Zeina Chamoun Morel, Arcangela De Nicolo, Maria Nomikou, Christina Karamanidou, Christine Kakalou
    Healthcare.2025; 13(2): 121.     CrossRef
  • Korean patients with hereditary cancer: a prospective multicentre cohort study protocol exploring psychosocial and health outcomes
    Jun-Kyu Kim, Mi-Ae Jang, Jong Eun Park, Dongju Won, Jung-Sook Ha, Kyoung-Bo Kim, Boyoung Park, Sun-Young Kong
    BMJ Open.2025; 15(2): e093905.     CrossRef
  • PALB2 germline pathogenic variants: frequency, clinical features, and functional analysis of c.3350+5G>A variant in 3987 Korean cancer patients
    M.-C. Kang, S. Lee, H. Kim, H.-S. Kang, S.-Y. Jung, J.-A. Hwang, J. Kwon, K.S. Lee, M.C. Lim, S.-Y. Park, S.H. Sim, W. Choi, J.E. Park, E.-H. Cho, S.-Y. Kong
    ESMO Open.2025; 10(3): 104132.     CrossRef
  • Human epidermal growth factor receptor-2 expression and subsequent dynamic changes in patients with ovarian cancer
    Yoo-Na Kim, Yun Soo Chung, Eunhyang Park, Seung Tae Lee, Jung-Yun Lee
    Scientific Reports.2024;[Epub]     CrossRef
  • Implementation of BRCA Test among Young Breast Cancer Patients in South Korea: A Nationwide Cohort Study
    Yung-Huyn Hwang, Tae-Kyung Yoo, Sae Byul Lee, Jisun Kim, Beom Seok Ko, Hee Jeong Kim, Jong Won Lee, Byung Ho Son, Il Yong Chung
    Cancer Research and Treatment.2024; 56(3): 802.     CrossRef
  • Germline RAD51C and RAD51D Mutations in High-Risk Chinese Breast and/or Ovarian Cancer Patients and Families
    Ava Kwong, Cecilia Yuen Sze Ho, Chun Hang Au, Sze Keong Tey, Edmond Shiu Kwan Ma
    Journal of Personalized Medicine.2024; 14(8): 866.     CrossRef
  • Prevalence Estimation of the PALB2 Germline Variant in East Asians and Koreans through Population Database Analysis
    Jong Eun Park, Min-Chae Kang, Taeheon Lee, Eun Hye Cho, Mi-Ae Jang, Dongju Won, Boyoung Park, Chang-Seok Ki, Sun-Young Kong
    Cancers.2024; 16(19): 3318.     CrossRef
  • Clinical Significance of PALB2 Pathogenic Germline Variant
    Min-Chae Kang, R.N., Jong Eun Park, Mi-Ae Jang, Dongju Won, Boyoung Park, Seeyoun Lee, Dong Ock Lee, Kum Hei Ryu, Yoon-Jung Chang, Sun-Young Kong
    Laboratory Medicine Online.2024; 14(4): 311.     CrossRef
  • Clinicopathological Features and Oncological Outcomes of Germline Partner and Localizer of Breast Cancer 2-Mutated Breast Cancer in Korea
    Chayanee Sae-lim, Seongyeon Jo, Shinyoung Park, Taeyong Kweon, Jeea Lee, Yoonjung Lee, Sun Hwa Lee, Dongju Won, Eun Ji Nam, Jung Woo Han, Tae Il Kim, Ji Soo Park, Hyung Seok Park
    Journal of Breast Cancer.2024; 27(6): 372.     CrossRef
  • Impact of High-to-Moderate Penetrance Genes on Genetic Testing: Looking over Breast Cancer
    Antonella Turchiano, Marilidia Piglionica, Stefania Martino, Rosanna Bagnulo, Antonella Garganese, Annunziata De Luisi, Stefania Chirulli, Matteo Iacoviello, Michele Stasi, Ornella Tabaku, Eleonora Meneleo, Martina Capurso, Silvia Crocetta, Simone Lattaru
    Genes.2023; 14(8): 1530.     CrossRef
  • 7,261 View
  • 195 Download
  • 10 Web of Science
  • 11 Crossref
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Pediatric cancer
Clinical Characteristics and Treatment Outcomes of Childhood Acute Promyelocytic Leukemia in Korea: A Nationwide Multicenter Retrospective Study by Korean Pediatric Oncology Study Group
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
Cancer Res Treat. 2022;54(1):269-276.   Published online April 20, 2021
DOI: https://doi.org/10.4143/crt.2021.313
AbstractAbstract PDFPubReaderePub
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.

Citations

Citations to this article as recorded by  
  • 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
    Anna Maria Testi, Mazin Faisal Al-Jadiry, Hasanein Habeeb Ghali, Samaher Abdulrazzaq Fadhil, Amir Fadhil Al-Darraji, Raghad Majid Al-Saeed, Ahmed Hatem Sabhan, Safaa A. Faraj Al-Badri, Wisan Majeed Abed, Najiha Ahmed Ameen, Ruaa Zaki Al-Tameemi, Arabiya I
    Leukemia & Lymphoma.2022; 63(12): 2940.     CrossRef
  • Death due to unsuspected acute myeloid leukaemia: an unusual forensic diagnosis
    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
  • 8,096 View
  • 231 Download
  • 5 Web of Science
  • 5 Crossref
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Children’s Hepatic Tumors International Collaboration-Hepatoblastoma Stratification (CHIC-HS) System for Pediatric Patients with Hepatoblastoma: A Retrospective, Hospital-Based Cohort Study in South Korea
Pyeong Hwa Kim, Hyun Joo Shin, Hee Mang Yoon, Young Hun Choi, Jung-Man Namgoong, Dae Yeon Kim, Kyung-Nam Koh, Mi-Jung Lee, Haesung Yoon, Chuhl Joo Lyu, Jung Woo Han, Seung Min Hahn, Young Ah Cho
Cancer Res Treat. 2022;54(1):253-258.   Published online March 24, 2021
DOI: https://doi.org/10.4143/crt.2021.265
AbstractAbstract PDFPubReaderePub
Purpose
In 2017, the Children’s Hepatic Tumors International Collaboration-Hepatoblastoma Stratification (CHIC-HS) system was introduced. We aimed to evaluate the accuracy of CHIC-HS System for the prediction of event-free survival (EFS) in Korean pediatric patients with hepatoblastoma.
Materials and Methods
This two-center retrospective study included consecutive Korean pediatric patients with histopathologically confirmed hepatoblastoma from March 1988 through September 2019. We compared EFS among four risk groups according to the CHIC-HS system. Discriminatory ability of CHIC-HS system was also evaluated using optimism-corrected C-statistics. Factors associated with EFS were explored using multivariable Cox regression analysis.
Results
We included 129 patients (mean age, 2.6±3.3 years; female:male, 63:66). The 5-year EFS rates in the very low, low, intermediate, and high-risk groups, according to the CHIC-HS system were 90.0%, 82.8%, 73.5%, and 51.3%, respectively. The CHIC-HS system aligned significantly well with EFS outcomes (p=0.004). The optimism-corrected C index of CHIC-HS was 0.644 (95% confidence interval [CI], 0.561 to 0.727). Age ≥ 8 (vs. age ≤ 2; hazard ratio [HR], 2.781; 95% CI, 1.187 to 6.512; p=0.018), PRE-Treatment EXTent of tumor (PRETEXT) stage IV (vs. PRETEXT I or II; HR, 2.774; 95% CI, 1.228 to 5.974; p=0.009), and presence of metastasis (HR, 2.886; 95% CI, 1.457 to 5.719; p=0.002), which are incorporated as the first three nodes in the CHIC-HS system, were independently associated with EFS.
Conclusion
The CHIC-HS system aligned significantly well with EFS outcomes in Korean pediatric patients with hepatoblastoma. Age group, PRETEXT stage, and presence of metastasis were independently associated with EFS.

Citations

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  • The importance of age as a prognostic predictor of childhood hepatoblastoma: an analysis of single-center childhood hepatoblastoma in China
    Zhen Li, Hong Jiang, Yuan-Qi Wang, Wen-Qing Wang, Li-Bin Huang, Jun-Cheng Liu, Hong-Man Xue
    BMC Pediatrics.2025;[Epub]     CrossRef
  • Elevated serum uric acid is associated with the risk of advanced staging and vascular involvement in patients with hepatoblastoma: a 14-year retrospective study
    Yunlan Zhou, Jinning Li, Yanhui Ma, Mengjie Tang, Xiaojun Yuan, Lisong Shen
    Frontiers in Oncology.2023;[Epub]     CrossRef
  • 6,500 View
  • 213 Download
  • 2 Web of Science
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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
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
Cancer Res Treat. 2021;53(4):1184-1194.   Published online January 4, 2021
DOI: https://doi.org/10.4143/crt.2020.289
AbstractAbstract PDFSupplementary MaterialPubReaderePub
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.

Citations

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  • Revolutionizing cancer treatment: ROS-induced apoptosis via nanoformulated alkaloids
    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
  • 8,397 View
  • 200 Download
  • 4 Web of Science
  • 7 Crossref
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Central nervous system
Atypical Teratoid/Rhabdoid Tumor of the Central Nervous System in Children under the Age of 3 Years
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
Cancer Res Treat. 2021;53(2):378-388.   Published online October 28, 2020
DOI: https://doi.org/10.4143/crt.2020.756
AbstractAbstract PDFPubReaderePub
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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  • Comparative treatment results of children with atypical teratoid/rhabdoid tumor of the central nervous system in the younger age group
    L. V. Olkhova, O. G. Zheludkova, L. S. Zubarovskaya, A. S. Levashov, A. Yu. Smirnova, Yu. V. Dinikina, Yu. V. Kushel, A. G. Melikyan, S. K. Gorelyshev, M. V. Ryzhova, Yu. Yu. Trunin, A. G. Gevorgyan, O. B. Polushkina, V. E. Popov, L. P. Privalova, N. B. Y
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Loss of Heterozygosity at Chromosome 16q Is a Negative Prognostic Factor in Korean Pediatric Patients with Favorable Histology Wilms Tumor: A Report of the Korean Pediatric Hematology Oncology Group (K-PHOG)
Jun Eun Park, O Kyu Noh, Yonghee Lee, Hyoung Soo Choi, Jung Woo Han, Seung Min Hahn, Chuhl Joo Lyu, Ji Won Lee, Keon Hee Yoo, Hong Hoe Koo, Seon-Yong Jeong, Ki Woong Sung
Cancer Res Treat. 2020;52(2):438-445.   Published online September 10, 2019
DOI: https://doi.org/10.4143/crt.2019.313
AbstractAbstract PDFPubReaderePub
Purpose
Loss of heterozygosity (LOH) at chromosomes 1p and 16q is a poor prognostic factor in favorable histology Wilms tumor (FHWT). This study investigated the prevalence of LOH at 1p and 16q and evaluated its prognostic value in Korean children with FHWT. Materials and Methods We analyzed 101 FHWT patients who were diagnosed between 1996 and 2016 in Korean Society of Pediatric Hematology Oncology Group hospitals. Using paraffin-embedded kidney tissue samples sent from each center, we reviewed LOH at 1p and 16q in each patient and assessed the prognostic value of LOH status for clinical parameters affecting event-free survival (EFS).
Results
Of the 101 patients, 12 (11.9%) experienced recurrence; the 3-year EFS was 87.6%. LOH at 1p or 16q was detected in 19 patients (18.8%), with five having LOH at both 1q and 16q. The frequency of LOH at 1p was higher among younger patients (p=0.049), but there was no difference in LOH prevalence according to tumor stage. In the multivariate analysis, LOH at 16q was a significant negative prognostic factor affecting EFS (3-year EFS, 73.7% vs. 91.1%; hazard ratio, 3.95; p=0.037), whereas LOH at 1p was not (p=0.786). Conclusion LOH at 16q was a significant negative prognostic factor affecting outcome in Korean pediatric FHWT patients. Due to the small sample size of this study, large-scale multicenter trials are warranted to investigate the prognostic value of LOH at 1p and 16q in Korean children with FHWT.

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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 Pa
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    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
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Clinical Risk Factors Influencing Dental Developmental Disturbances in Childhood Cancer Survivors
Chung-Min Kang, Seung Min Hahn, Hyo Sun Kim, Chuhl Joo Lyu, Jae-Ho Lee, Jinae Lee, Jung Woo Han
Cancer Res Treat. 2018;50(3):926-935.   Published online October 10, 2017
DOI: https://doi.org/10.4143/crt.2017.296
AbstractAbstract PDFSupplementary MaterialPubReaderePub
Purpose
Although studies regarding dental developmental disturbances after childhood cancer treatment have increased, they have many limitations. Studies analyzing the significance of independent clinical risk factors with regard to the dental health status are also rare. We aimed to investigate the risk factors for dental developmental disturbances, particularly severe disturbances, in childhood cancer survivors (CCS).
Materials and Methods
Oral examinations and retrospective reviews of medical and panoramic radiographs were performed for 196 CCS (mean age, 15.6 years). Cancer type, age at diagnosis, treatment modality, type and accumulated dose of administered drugs, and dose and site of radiation were recorded. Dental developmental disturbances were diagnosed using panoramic radiographs and graded for severity according to the Modified Dental Defect Index (MDDI). Descriptive statistics and multivariate analyseswere performed to determine the association between dental abnormalities and clinical factors.
Results
In total, 109 CCS (55.6%) exhibited at least one dental anomaly, and the median value of MDDI was 2.5. Microdontia (30.6%) was the most prevalent anomaly, followed by tooth agenesis (20.4%), V-shaped roots (14.8%), and taurodontism (10.2%). Multivariate analysis revealed that a young age at diagnosis (≤ 3 years), a history of hematopoietic stem cell transplantation, the use of multiple classes of chemotherapeutic agents (≥ 4 classes), and the use of heavy metal agents were significant risk factors for severe dental disturbances.
Conclusion
CCS with any of the above risk factors for severe developmental disturbances should be comprehensively followed up to minimize adverse consequences to their dental development and preserve their future dental health.

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    K. Seremidi, K. Kavvadia, A. Kattamis, A. Polychronopoulou
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Detection of Germline Mutations in Patients with Epithelial Ovarian Cancer Using Multi-gene Panels: Beyond BRCA1/2
Kyung Jin Eoh, Ji Eun Kim, Hyung Seok Park, Seung-Tae Lee, Ji Soo Park, Jung Woo Han, Jung-Yun Lee, Sunghoon Kim, Sang Wun Kim, Jae Hoon Kim, Young Tae Kim, Eun Ji Nam
Cancer Res Treat. 2018;50(3):917-925.   Published online September 27, 2017
DOI: https://doi.org/10.4143/crt.2017.220
AbstractAbstract PDFSupplementary MaterialPubReaderePub
Purpose
Next-generation sequencing (NGS) allows simultaneous sequencing of multiple cancer susceptibility genes and may represent a more efficient and less expensive approach than sequential testing. We assessed the frequency of germline mutations in individuals with epithelial ovarian cancer (EOC), using multi-gene panels and NGS.
Materials and Methods
Patients with EOC (n=117) with/without a family history of breast or ovarian cancer were recruited consecutively, from March 2016 toDecember 2016.GermlineDNAwas sequenced using 35-gene NGS panel, in order to identify mutations. Upon the detection of a genetic alteration using the panel, results were cross-validated using direct sequencing.
Results
Thirty-eight patients (32.5%) had 39 pathogenic or likely pathogenic mutations in eight genes, including BRCA1 (n=21), BRCA2 (n=10), BRIP1 (n=1), CHEK2 (n=2), MSH2 (n=1), POLE (n=1), RAD51C (n=2), and RAD51D (n=2). Among 64 patients with a family history of cancer, 27 (42.2%) had 27 pathogenic or likely pathogenic mutations, and six (9.3%) had mutations in genes other than BRCA1/2, such as CHECK2, MSH2, POLE, and RAD51C. Fifty-five patients (47.0%) were identified to carry only variants of uncertain significance.
Conclusion
Using the multi-gene panel test, we found that, of all patients included in our study, 32.5% had germline cancer-predisposing mutations. NGS was confirmed to substantially improve the detection rates of a wide spectrum of mutations in EOC patients compared with those obtained with the BRCA1/2 testing alone.

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Identification of a Novel BRCA1 Pathogenic Mutation in Korean Patients Following Reclassification of BRCA1 and BRCA2 Variants According to the ACMG Standards and Guidelines Using Relevant Ethnic Controls
Ji Soo Park, Eun Ji Nam, Hyung Seok Park, Jung Woo Han, Jung-Yun Lee, Jieun Kim, Tae Il Kim, Seung-Tae Lee
Cancer Res Treat. 2017;49(4):1012-1021.   Published online January 17, 2017
DOI: https://doi.org/10.4143/crt.2016.433
AbstractAbstract PDFSupplementary MaterialPubReaderePub
Purpose
Comparison of variant frequencies in the general population has become an essential part of the American College of Medical Genetics and Genomics (ACMG) standards and guidelines for interpreting sequence variants. We determined the optimal number of relevant ethnic controls that should be used to accurately calculate the odds ratio (OR) of genetic variants.
Materials and Methods
Using the ACMG guidelines, we reclassified BRCA1 and BRCA2 mutations and variants of unknown significance in 745 Korean patients susceptible to hereditary breast and ovarian cancer compared with 1,314 Korean population controls.
Results
We observed that the ORs were falsely inflated when we analyzed several variants using non-Korean population data. Our simulation indicated that the number of controls needed for the lower limit of a 95% confidence interval to exceed 1.0 varied according to the frequency of the variant in each patient group, with more than 820 controls needed for a variant existing in 1% of cases. Using a sufficient number of relevant population data, we could efficiently classify variants and identified the BRCA1 p.Leu1780Pro mutation as a possible pathogenic founder mutation in Korean patients.
Conclusion
Our study suggests that BRCA1 p.Leu1780Pro is a novel pathogenic mutation found in Korean patients. We also determined the optimal number of relevant ethnic controls needed for accurate variant classification according to the ACMG guidelines.

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Outcomes of Treatment for Malignant Peripheral Nerve Sheath Tumors: Different Clinical Features Associated with Neurofibromatosis Type 1
In Kyung Hwang, Seung Min Hahn, Hyo Sun Kim, Sang Kyum Kim, Hyo Song Kim, Kyoo‑Ho Shin, Chang Ok Suh, Chuhl Joo Lyu, Jung Woo Han
Cancer Res Treat. 2017;49(3):717-726.   Published online December 1, 2016
DOI: https://doi.org/10.4143/crt.2016.271
AbstractAbstract PDFSupplementary MaterialPubReaderePub
Purpose
Malignant peripheral nerve sheath tumors (MPNSTs) are a rare subtype of sarcoma that occur spontaneously or in association with neurofibromatosis type 1 (NF-1). This study aimed to clinically differentiate these types of MPNSTs.
Materials and Methods
The study reviewed 95 patients diagnosed with and treated for MPNST at Yonsei University Health System, Seoul, Korea over a 27-year period. The clinical characteristics, prognostic factors, and treatment outcomes of sporadic MPNST (sMPNST) and NF-1 associated MPNST (NF-MPNST) cases were compared.
Results
Patients with NF-MPNST had a significantly lower median age (32 years vs. 45 years for sMPNST, p=0.012), significantly larger median tumor size (8.2 cm vs. 5.0 cm for sMPNST, p < 0.001), and significantly larger numbers of imaging studies and surgeries (p=0.004 and p < 0.001, respectively). The 10-year overall survival (OS) rate of the patients with MPNST was 52±6%. Among the patients with localized MPNST, patients with NF-MPNST had a significantly lower 10-year OS rate (45±11% vs. 60±8% for sMPNST, p=0.046). Univariate analysis revealed the resection margin, pathology grade, and metastasis to be significant factors affecting the OS (p=0.001, p=0.020, and p < 0.001, respectively). Multivariate analysis of the patients with localized MPNST identified R2 resection and G1 as significant prognostic factors for OS.
Conclusion
NF-MPNST has different clinical features from sMPNST and requires more careful management. Further study will be needed to develop specific management plans for NF-MPNST.

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    Zhenyu Cai, Xiaodong Tang, Haijie Liang, Rongli Yang, Taiqiang Yan, Wei Guo
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    Jordan L. Kohlmeyer, Courtney A. Kaemmer, Casey Pulliam, Chandra K. Maharjan, Allison Moreno Samayoa, Heather J. Major, Kendall E. Cornick, Vickie Knepper-Adrian, Rajesh Khanna, Jessica C. Sieren, Mariah R. Leidinger, David K. Meyerholz, K.D. Zamba, Jill
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    Enrico Martin, Ivo S Muskens, J H Coert, Timothy R Smith, Marike L D Broekman
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    Chang Yeon Jung, Jung Min Bae, Joon Hyuk Choi, Ki Hoon Jung
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Patterns of Failure Following Multimodal Treatment for Medulloblastoma: Long-Term Follow-up Results at a Single Institution
Dong Soo Lee, Jaeho Cho, Se Hoon Kim, Dong-Seok Kim, Kyu Won Shim, Chuhl Joo Lyu, Jung Woo Han, Chang-Ok Suh
Cancer Res Treat. 2015;47(4):879-888.   Published online December 8, 2014
DOI: https://doi.org/10.4143/crt.2014.067
AbstractAbstract PDFPubReaderePub
Purpose
The purpose of this study is to investigate the long-term results and appropriateness of radiation therapy (RT) for medulloblastoma (MB) at a single institution. Materials and Methods We analyzed the clinical outcomes of 106 patients with MB who received RT between January 1992 and October 2009. The median age was 7 years (range, 0 to 50 years), and the proportion of M0, M1, M2, and M3 stages was 60.4%, 8.5%, 4.7%, and 22.6%, respectively. The median total craniospinal irradiation (CSI) and posterior fossa tumor bed dose in 102 patients (96.2%) treated with CSI was 36 Gy and 54 Gy, respectively. Results The median follow-up period in survivors was 132 months (range, 31 to 248 months). A gradual improvement in survival outcomes was observed, with 5-year overall survival rates of 61.5% in 1990s increasing to 73.6% in 2000s. A total of 29 recurrences (27.4%) developed at the following sites: five (17.2%) in the tumor bed; five (17.2%) in the posterior fossa other than the tumor bed; nine (31%) in the supratentorium; and six (20.7%) in the spinal subarachnoid space only. The four remaining patients showed multiple site recurrences. Among 12 supratentorial recurrences, five cases recurred in the subfrontal areas. Although the frequency of posterior fossa/tumor bed recurrences was significantly high among patients treated with subtotal resection, other site (other intracranial/spinal) recurrences were more common among patients treated with gross tumor removal (p=0.016). There was no case of spinal subarachnoid space relapse from desmoplastic/extensive nodular histological subtypes. Conclusion Long-term follow-up results and patterns of failure confirmed the importance of optimal RT dose and field arrangement. More tailored multimodal strategies and proper CSI technique may be the cornerstones for improving treatment outcomes in MB patients.

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Incidence and Survival of Pediatric Soft Tissue Sarcomas: Comparison between Adults and Children
Sun Min Lim, Cheol Joo Yoo, Jung Woo Han, Yong Jin Cho, Soo Hee Kim, Joong Bae Ahn, Sun Young Rha, Sang Joon Shin, Hyun Cheol Chung, Woo Ick Yang, Kyoo-Ho Shin, Jae Kyung Rho, Hyo Song Kim
Cancer Res Treat. 2015;47(1):9-17.   Published online August 21, 2014
DOI: https://doi.org/10.4143/crt.2013.157
AbstractAbstract PDFPubReaderePub
Purpose
Pediatric-type sarcomas such as rhabdomyosarcoma (RMS), Ewing sarcoma (EWS), primitive neuroectodermal tumor (PNET), and desmoplastic small round-cell tumor (DSRCT) are rare in adults, with limited studies on their prognosis and optimal treatment strategies. We aimed to examine the outcome of children and adult patients with RMS, EWS, PNET, and DSRCT and relevant prognostic factors. Materials and Methods We retrospectively reviewed 220 pediatric-type sarcoma patients at a single institution between 1985 and 2011. Comparisons were made in order to examine differences in demographics, disease characteristics, and survival. Survival analyses were performed using the Kaplan-Meier method with log-rank tests and Cox proportional hazards models. Results A total of 220 consecutive patients were identified at our institute. Median age was 15.6 years (range, 0 to 81 years) and there were 108 children (49%) and 112 adult patients (51%). According to histological classification, 106 patients (48.2%) had RMS, 60 (27.3%) had EWS, 50 (22.7%) had PNET, and 4 (1.8%) had DSRCT. With a median follow-up period of 6.6 years, the estimated median overall survival (OS) of all patients was 75 months (95% confidence interval [CI], 27.2 to 122.8 months) and median event-free survival (EFS) for all patients was 11 months (95% CI, 8.8 to 13.2 months). No significant difference in OS and EFS was observed between adults and children. In multivariate analysis, distant metastasis (hazard ratio [HR], 1.617; 95% CI, 1.022 to 2.557; p=0.040) and no debulking surgery (HR, 1.443; 95% CI, 1.104 to 1.812; p=0.012) showed independent association with worse OS. Conclusion Metastatic disease and no surgical treatment are poor prognostic factors for OS among pediatric-type sarcomas for both adults and children.

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  • Clinicopathological and treatment response characteristics of updated rhabdomyosarcoma histomolecular subtypes: An Asian population‐based study
    Guo Yuan How, Chik Hong Kuick, Min Hwee Yong, Shui Yen Soh, Esther XY Hee, Meng Kang Wong, Richard Quek, Mohd Farid Harunal, Sathiyamoorthy Selvarajan, Kesavan Sittampalam, Chetan Anil Dhamne, Victor Lee, Kenneth TE Chang, Amos HP Loh
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    Peter M. Fantozzi, Gina Sprint, Anna Marie Medina
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    Vassiliki Lyra, Sofia Chatziioannou, Maria Kallergi
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    Laura Olivier-Gougenheim, Daniel Orbach, Vincent Atallah, Perrine Marec-Berard, Amandine Bertrand
    Journal of Pediatric Hematology/Oncology.2022; 44(3): e637.     CrossRef
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    Antonio Juan Ribelles, Pablo Gargallo, Pablo Berlanga, Vanessa Segura, Yania Yáñez, Bárbara Juan, Marta Salom, Margarita Llavador, Jaime Font de Mora, Victoria Castel, Adela Cañete
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    Hee Young Ju
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Weekly Gemcitabine and Docetaxel in Refractory Soft Tissue Sarcoma: A Retrospective Analysis
Ha-young Lee, Sang Joon Shin, Hyo Song Kim, Soo Jung Hong, Jung Woo Han, Seung Taek Lim, Jae Kyung Roh, Sun Young Rha
Cancer Res Treat. 2012;44(1):43-49.   Published online March 31, 2012
DOI: https://doi.org/10.4143/crt.2012.44.1.43
AbstractAbstract PDFPubReaderePub
PURPOSE
The combination of gemcitabine and docetaxel (GD) is used to effectively treat patients with soft tissue sarcoma (STS). It is widely considered that the conventional doses used are too high for long term use and many patients must discontinue GD treatment due to its toxicity. Therefore, to determine the appropriate dose meeting acceptable efficacy results, while minimizing toxic side effects, we treated patients with a weekly infusion of GD (weekly GD).
MATERIALS AND METHODS
A total of 22 patients presenting a variety of STSs were treated at Yonsei Cancer Center. All patients had metastatic or recurrent cancer and had previously received doxorubicin and ifosfamide combination chemotherapy. In all cases, gemcitabine (1,000 mg/m2) and docetaxel (35 mg/m2) were administered intravenously on days 1 and 8 of a 21-day cycle. We retrospectively reviewed the medical records of these patients.
RESULTS
The response rate was 4.5%, with one patient diagnosed with leiomyosarcoma having a partial response, and the disease control rate was 40.9%. The median progression-free survival (PFS) duration was 2.7 months and the PFS was correlated with the treatment response to a weekly GD. The median overall survival (OS) duration was 7.8 months and the OS was correlated with histology. There was no significant difference in OS between patients who received weekly GD as a 2nd line chemotherapy and those who received 3rd line or more. Treatment was generally well tolerated.
CONCLUSION
Weekly GD was well tolerated and showed moderate efficacy, indicating that this could be a reasonable option as a salvage treatment for metastatic STS.

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