Cancer Research and Treatment

Original Articles

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

Abstract PDF Supplementary Material PubReader ePub: 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.

2,931 View
151 Download

Hematologic malignancy

Clinical Impact of Drug Adherence of Tyrosine Kinase Inhibitors in Children with Ph-Positive Acute Lymphoblastic Leukemia: Jun-Xia Wang, Miao-Miao Yang, Li-Peng Liu, Hui-Min Zhang, Meng-Chuan Wang, Yu-Wen Chen, Xiao-Ying Zang, Fang Hu; Cancer Res Treat. 2023;55(3):1023-1030. Published online February 6, 2023; DOI: https://doi.org/10.4143/crt.2022.1618

Abstract PDF PubReader ePub: Purpose
This study aimed to explore the impact of ABL1–tyrosine kinase inhibitors (TKIs) adherence on the survival of chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) children and clarify the potential predictors of patients’ prognosis from TKIs intake practices.
Materials and Methods
Ninety newly diagnosed Ph+ ALL patients who received TKIs were enrolled. We collected the baseline characteristics and adverse events in all children; moreover, TKIs adherence was measured by an eight-item Morisky medication adherence scale (MMAS-8). Progression-free survival (PFS) and overall survival (OS) analysis were performed, and risk factors for PFS and OS were evaluated.
Results
Among all patients, 69 cases were regarded as adherers, while 21 were non-adherers. The median duration of TKIs interruption was significantly prolonged in the non-adherence group than in the adherence group (13 [0-101] vs. 56 [11-128], p < 0.001). Additionally, dose reduction occurred in 55.2% of non-adherers versus 23.0% of adherers (p=0.002). The PFS and OS in adherers were significantly higher versus non-adherers (p=0.020 and p=0.039). MMAS-8 score was an independent risk factor for PFS (p=0.010) and OS (p=0.031). Among non-adherers, the median OS was only 23.1% (4.2%-42%) in patients aged ≤ 10 years versus 54.4% (38.8%-70%) in adolescents. Most of the patients who experienced TKIs non-adherence suffered pancytopenia.
Conclusion
TKIs adherence during treatment significantly influenced the survival of pediatric Ph+ ALL patients, and non-adherers with age ≤ 10 years were more vulnerable to TKIs disruption. The cumulative TKIs dose should be especially emphasized to patients with age ≤ 10 years, which may result in an inferior achievement of relevant treatment milestones.

2,839 View
140 Download

Pediatric cancer

Outcome of Intensive Therapy for Children with Relapsed Acute Myeloid Leukemia: A Single Institution Korean Study: Jae Wook Lee, Jae Won Yoo, Seongkoo Kim, Pil-Sang Jang, Nack-Gyun Chung, Bin Cho; Cancer Res Treat. 2022;54(4):1230-1239. Published online December 17, 2021; DOI: https://doi.org/10.4143/crt.2021.1011

Abstract PDF Supplementary Material PubReader ePub

Purpose
Approximately 30%-40% of pediatric acute myeloid leukemia (AML) patients relapse. In this study, we analyzed the outcome and prognostic factors of relapsed AML patients who had previously received first-line therapy at our institution.
Materials and Methods
The study group consisted of 50 patients who had been diagnosed with AML from April 2009 to December 2018, and then showed first relapse. Thirty-two of the patients (64%) had previously received allogeneic hematopoietic stem cell transplantation (HSCT) in first complete remission (CR).
Results
Forty-five of the patients (90%) received intensive chemotherapy upon diagnosis of relapse, and 76% (34/45) of these patients achieved a second CR. Estimated 5-year overall survival for these 45 patients was 44.9%±7.6%. Time from diagnosis to relapse, extramedullary involvement (EMI) at diagnosis, core binding factor AML, and complex karyotype were significant prognostic factors; in multivariate study, both time from diagnosis to relapse and EMI at diagnosis proved significant. There was no difference in 5-year disease-free survival between patients previously treated with chemotherapy only and those who received HSCT in first CR (52.4%±14.9% vs. 52.6%±11.5%). Of the 19 patients who achieved second CR after previous allogeneic HSCT in first CR and subsequent relapse, 11 were treated with chemotherapy only, and seven survive disease-free.
Conclusion
Intensive therapy allowed for long-term survival in 40%-50% of patients, and 50% of patients who achieved second CR, regardless of prior treatment modalities in first CR. Intensive treatment may allow for salvage of a significant portion of patients with relapsed pediatric AML.

Citations

Citations to this article as recorded by

Diagnosis and Treatment of Pediatric Acute Megakaryoblastic Leukemia with NUP98::KDM5A Rearrangement: Case Report
Hyemin Kang, Suejung Jo, Jae Won Yoo, Seongkoo Kim, Jae Wook Lee, Nack-Gyun Chung, Bin Cho, Chae Yeon Lee, Myungshin Kim
Clinical Pediatric Hematology-Oncology.2024; 31(2): 56. CrossRef

5,177 View
112 Download
1 Crossref

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

Abstract PDF PubReader ePub

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.

Citations

Citations to this article as recorded by

Supratentorial ATRT in a young Infant: Expanding the diagnostic spectrum beyond medulloblastoma
Ali Msheik, Mohamad Aoun, Youssef Fares
Interdisciplinary Neurosurgery.2024; 35: 101857. CrossRef
Radiation Therapy Plays an Important Role in the Treatment of Atypical Teratoid/Rhabdoid Tumors: Analysis of the EU-RHAB Cohorts and Their Precursors
Sabine Frisch, Hanna Libuschewski, Sarah Peters, Joachim Gerß, Katja von Hoff, Rolf-Dieter Kortmann, Karolina Nemes, Stefan Rutkowski, Martin Hasselblatt, Torsten Pietsch, Michael C. Frühwald, Beate Timmermann
International Journal of Radiation Oncology*Biology*Physics.2024; 119(4): 1147. CrossRef
An adult with recurrent atypical teratoid rhabdoid tumor of the spine
Antoinette J Charles, Vanessa L Smith, C Rory Goodwin, Margaret O Johnson
CNS Oncology.2024;[Epub] CrossRef
Dynamic Survival Risk Prognostic Model and Genomic Landscape for Atypical Teratoid/Rhabdoid Tumors: A Population-Based, Real-World Study
Sihao Chen, Yi He, Jiao Liu, Ruixin Wu, Menglei Wang, Aishun Jin
Cancers.2024; 16(5): 1059. CrossRef
ESTRO-SIOPE guideline: Clinical management of radiotherapy in atypical teratoid/rhabdoid tumors (AT/RTs)
Beate Timmermann, Claire Alapetite, Karin Dieckmann, Rolf-Dieter Kortmann, Yasmin Lassen-Ramshad, John H. Maduro, Monica Ramos Albiac, Umberto Ricardi, Damien C. Weber
Radiotherapy and Oncology.2024; 196: 110227. CrossRef
Development and epigenetic regulation of Atypical teratoid/rhabdoid tumors in the context of cell-of-origin and halted cell differentiation
Laura Huhtala, Goktug Karabiyik, Kirsi J Rautajoki
Neuro-Oncology Advances.2024;[Epub] CrossRef
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
Russian Journal of Pediatric Hematology and Oncology.2023; 10(1): 11. 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
Survival and Malignant Transformation of Pineal Parenchymal Tumors: A 30-Year Retrospective Analysis in a Single-Institution
Tae-Hwan Park, Seung-Ki Kim, Ji Hoon Phi, Chul-Kee Park, Yong Hwy Kim, Sun Ha Paek, Chang-Hyun Lee, Sung-Hye Park, Eun Jung Koh
Brain Tumor Research and Treatment.2023; 11(4): 254. CrossRef
Atypical Teratoid/Rhabdoid Tumor in Taiwan: A Nationwide, Population-Based Study
Yen-Lin Liu, Min-Lan Tsai, Chang-I Chen, Noi Yar, Ching-Wen Tsai, Hsin-Lun Lee, Chia-Chun Kuo, Wan-Ling Ho, Kevin Li-Chun Hsieh, Sung-Hui Tseng, James S. Miser, Chia-Yau Chang, Hsi Chang, Wen-Chang Huang, Tai-Tong Wong, Alexander T. H. Wu, Yu-Chun Yen
Cancers.2022; 14(3): 668. CrossRef
Atypical Teratoid Rhabdoid Tumor: A Possible Oriented Female Pathology?
Cinzia Baiano, Rosa Della Monica, Raduan Ahmed Franca, Maria Laura Del Basso De Caro, Luigi Maria Cavallo, Lorenzo Chiariotti, Tamara Ius, Emmanuel Jouanneau, Teresa Somma
Frontiers in Oncology.2022;[Epub] CrossRef
Clinical predictors of survival for patients with atypical teratoid/rhabdoid tumors
Vismaya S. Bachu, Pavan Shah, Adrian E. Jimenez, Adham M. Khalafallah, Jignesh Tailor, Debraj Mukherjee, Alan R. Cohen
Child's Nervous System.2022; 38(7): 1297. CrossRef
Therapeutic Targeting of EZH2 and BET BRD4 in Pediatric Rhabdoid Tumors
Yukitomo Ishi, Yongzhan Zhang, Ali Zhang, Takahiro Sasaki, Andrea Piunti, Amreena Suri, Jun Watanabe, Kouki Abe, Xingyao He, Hiroaki Katagi, Pankaj Bhalla, Manabu Natsumeda, Lihua Zou, Ali Shilatifard, Rintaro Hashizume
Molecular Cancer Therapeutics.2022; 21(5): 715. CrossRef
Molecular targeted therapies for pediatric atypical teratoid/rhabdoid tumors
Chang Zhang, Hao Li
Pediatric Investigation.2022; 6(2): 111. CrossRef
The results of multicenter treatment of atypical teratoid/rhabdoid tumors of the central nervous system in children under 3 years
L. V. Olkhova, O. G. Zheludkova, L. S. Zubarovskaya, A. Yu. Smirnova, Yu. V. Dinikina, Yu. V. Kushel, A. G. Melikyan, S. K. Gorelyshev, M. V. Ryzhova, Yu. Yu. Trunin, E. I. Shults, A. G. Gevorgyan, S. V. Gorbatykh, A. N. Kislyakov, V. E. Popov, L. P. Priv
Pediatric Hematology/Oncology and Immunopathology.2021; 20(2): 121. CrossRef

8,893 View
297 Download
13 Web of Science
15 Crossref

Pediatric cancer

Effectiveness and Safety of Dabrafenib in the Treatment of 20 Chinese Children with BRAF^V600E-Mutated Langerhans Cell Histiocytosis: Ying Yang, Dong Wang, Lei Cui, Hong-Hao Ma, Li Zhang, Hong-Yun Lian, Qing Zhang, Xiao-Xi Zhao, Li-Ping Zhang, Yun-Ze Zhao, Na Li, Tian-You Wang, Zhi-Gang Li, Rui Zhang; Cancer Res Treat. 2021;53(1):261-269. Published online September 15, 2020; DOI: https://doi.org/10.4143/crt.2020.769

Abstract PDF Supplementary Material PubReader ePub

Purpose
We sought to investigate the effectiveness and safety of dabrafenib in children with BRAF^V600E-mutated Langerhans cell histiocytosis (LCH).
Materials and Methods
A retrospective analysis was performed on 20 children with BRAF^V600E-mutated LCH who were treated with dabrafenib.
Results
The median age at which the patients started taking dabrafenib was 2.3 years old (range, 0.6 to 6.5 years). The ratio of boys to girls was 2.3:1. The median follow-up time was 30.8 months (range, 18.9 to 43.6 months). There were 14 patients (70%) in the risk organ (RO)⁺ group and six patients (30%) in the RO^– group. All patients were initially treated with traditional chemotherapy and then shifted to targeted therapy due to poor control of LCH or intolerance to chemotherapy. The overall objective response rate and the overall disease control rate were 65% and 75%, respectively. During treatment, circulating levels of cell-free BRAF^V600E (cfBRAF^V600E) became negative in 60% of the patients within a median period of 3.0 months (range, 1.0 to 9.0 months). Grade 2 or 3 adverse effects occurred in five patients.
Conclusion
Some children with BRAF^V600E-mutated LCH may benefit from monotherapy with dabrafenib, especially high-risk patients with concomitant hemophagocytic lymphohistiocytosis and intolerance to chemotherapy. The safety of dabrafenib is notable. A prospective study with a larger sample size is required to determine the optimal dosage and treatment duration.

Citations

Citations to this article as recorded by

Targeted therapy and immunotherapy for orbital and periorbital tumors: a major review
Emmanuel Lee Boniao, Richard C. Allen, Gangadhara Sundar
Orbit.2024; 43(5): 656. CrossRef
Treatment of children with refractory/relapse high risk langerhans cell histiocytosis with the combination of cytarabine, vindesine and prednisone
Wenqian Wang, Jian Ge, Honghao Ma, Hongyun Lian, Lei Cui, Yunze Zhao, Zhigang Li, Tianyou Wang, Rui Zhang
BMC Pediatrics.2024;[Epub] CrossRef
Vemurafenib combined with chemotherapy achieved sustained remission in pediatric LCH: a multi-center observational study
Jiaying Lei, Wenxia Wang, Danna Lin, Chengguang Zhu, Wenguang Jia, Wenjun Weng, Xiaoshan Liu, Yuhan Ma, Zhixuan Wang, Lihua Yang, Xiangling He, Yunyan He, Yang LI
Journal of Cancer Research and Clinical Oncology.2024;[Epub] CrossRef
Clinical features and treatment outcomes of liver involvement in paediatric Langerhans cell histiocytosis
Xinshun Ge, Wenxin Ou, Ang Wei, Hongyun Lian, Honghao Ma, Lei Cui, Dong Wang, Liping Zhang, Xiaoman Wang, Lejian He, Rui Zhang, Tianyou Wang
BMC Pediatrics.2024;[Epub] CrossRef
Refractory juvenile xanthogranuloma of the mastoid bone responsive to trametinib
Isaac Hauk, Ignacio Gonzalez‐Gomes, Deepak Chellapandian, Jonathan Metts, Peter H. Shaw
Pediatric Blood & Cancer.2024;[Epub] CrossRef
Advancements in the understanding and management of histiocytic neoplasms
Kyung-Nam Koh, Su Hyun Yoon, Sung Han Kang, Hyery Kim, Ho Joon Im
Blood Research.2024;[Epub] CrossRef
Real-world experience with targeted therapy in patients with histiocytic neoplasms in the Netherlands and in Belgium
Paul G. Kemps, F. J. Sherida H. Woei-A-Jin, Patrick Schöffski, Thomas Tousseyn, Isabelle Vanden Bempt, Friederike A. G. Meyer-Wentrup, Natasja Dors, Natasha K. A. van Eijkelenburg, Marijn A. Scheijde-Vermeulen, Ingrid M. Jazet, Maarten Limper, Margot Jak,
Blood Neoplasia.2024; 1(3): 100023. CrossRef
The clinical impact of serum soluble CD25 levels in children with Langerhans cell histiocytosis
Zi-Jing Zhao, Hong-Yun Lian, Wei-Jing Li, Qing Zhang, Hong-Hao Ma, Dong Wang, Yun-Ze Zhao, Ting Zhu, Hua-Lin Li, Xiao-Tong Huang, Tian-You Wang, Rui Zhang, Lei Cui, Zhi-Gang Li
Jornal de Pediatria.2024;[Epub] CrossRef
Liver transplantation in a child with sclerosing cholangitis due to Langerhans cell histiocytosis: a case report
Xue-Lian Wang, Chun-Xiao Fang, Min-Xia Chen, Hua-Mei Yang, Lan-Hui She, Yu Gong, Yi Xu, Wei-Qiang Xiao, Jin-Sheng Tian, Bin Ai, Li Huang, Xu-Fang Li
Frontiers in Pediatrics.2024;[Epub] CrossRef
Langerhans cell histiocytosis as a clonal disease of mononuclear phagocyte system
Evgeniy F. Khynku, Maria K. Monaenkova, Olga B. Tamrazova, Alexey V. Taganov, Мarina А. Gureeva, Gayane E. Bagramova, Anton V. Molochkov
Almanac of Clinical Medicine.2023; 50(7): 428. CrossRef
Lineage switching of the cellular distribution of BRAF V600E in multisystem Langerhans cell histiocytosis
Paul Milne, Simon Bomken, Olga Slater, Ashish Kumar, Adam Nelson, Somak Roy, Jessica Velazquez, Kshitij Mankad, James Nicholson, Dan Yeomanson, Richard Grundy, Ahmed Kamal, Anthony Penn, Jane Pears, Gerard Millen, Bruce Morland, James Hayden, Jason Lam, M
Blood Advances.2023; 7(10): 2171. CrossRef
Treatment of Langerhans Cell Histiocytosis and Histiocytic Disorders: A Focus on MAPK Pathway Inhibitors
Ashley V. Geerlinks, Oussama Abla
Pediatric Drugs.2023; 25(4): 399. CrossRef
Dabrafenib, alone or in combination with trametinib, in BRAF V600–mutated pediatric Langerhans cell histiocytosis
James A. Whitlock, Birgit Geoerger, Ira J. Dunkel, Michael Roughton, Jeea Choi, Lisa Osterloh, Mark Russo, Darren Hargrave
Blood Advances.2023; 7(15): 3806. CrossRef
Therapiestrategien bei Kindern und Jugendlichen mit Langerhanszell Histiozytosen
Anke Elisabeth Barnbrock, Caroline Hutter, Konrad Bochennek, Milen Minkov, Thomas Lehrnbecher
Klinische Pädiatrie.2023; 235(06): 342. CrossRef
Mutant PIK3CA is a targetable driver alteration in histiocytic neoplasms
Benjamin H. Durham, Oshrat Hershkovitz-Rokah, Omar Abdel-Wahab, Mariko Yabe, Young Rock Chung, Gilad Itchaki, Maayan Ben-Sasson, Vered A. Asher-Guz, David Groshar, Seyram A. Doe-Tetteh, Tina Alano, David B. Solit, Ofer Shpilberg, Eli L. Diamond, Roei D. M
Blood Advances.2023; 7(23): 7319. CrossRef
Validation of Liquid Chromatography Coupled with Tandem Mass Spectrometry for the Determination of 12 Tyrosine Kinase Inhibitors (TKIs) and Their Application to Therapeutic Drug Monitoring in Adult and Pediatric Populations
Marie Bellouard, Jean Donadieu, Pauline Thiebot, Etienne Giroux Leprieur, Philippe Saiag, Isabelle Etting, Pamela Dugues, Emuri Abe, Jean-Claude Alvarez, Islam-Amine Larabi
Pharmaceutics.2023; 16(1): 5. CrossRef
Langerhans cell histiocytosis: promises and caveats of targeted therapies in high-risk and CNS disease
Oussama Abla
Hematology.2023; 2023(1): 386. CrossRef
Characteristics and Treatment Outcomes of Pediatric Langerhans Cell Histiocytosis with Thymic Involvement
Ja-Feng Yao, Dong Wang, Hong-Hao Ma, Hong-Yun Lian, Li Zhang, Tian-You Wang, Zhi-Gang Li, Jin Jiang, Lei Cui, Rui Zhang
The Journal of Pediatrics.2022; 244: 194. CrossRef
Clinical features and treatment outcomes of pediatric Langerhans cell histiocytosis with macrophage activation syndrome-hemophagocytic lymphohistiocytosis
Dong Wang, Xi-Hua Chen, Ang Wei, Chun-Ju Zhou, Xue Zhang, Hong-Hao Ma, Hong-Yun Lian, Li Zhang, Qing Zhang, Xiao-Tong Huang, Chan-Juan Wang, Ying Yang, Wei Liu, Tian-You Wang, Zhi-Gang Li, Lei Cui, Rui Zhang
Orphanet Journal of Rare Diseases.2022;[Epub] CrossRef
Current perspectives on the role of liver transplantation for Langerhans cell histiocytosis: A narrative review
Jagadeesh Menon, Ashwin Rammohan, Mukul Vij, Naresh Shanmugam, Mohamed Rela
World Journal of Gastroenterology.2022; 28(30): 4044. CrossRef
Research Progress of BRAF V600E Gene Mutation in Papillary Thyroid Carcinoma
延泽刘
Advances in Clinical Medicine.2022; 12(09): 8499. CrossRef
Recent advances in the understanding of the molecular pathogenesis and targeted therapy options in Langerhans cell histiocytosis
Jin Kyung Suh, Sunghan Kang, Hyery Kim, Ho Joon Im, Kyung-Nam Koh
BLOOD RESEARCH.2021; 56(S1): S65. CrossRef
Improvement in Pituitary Imaging After Targeted Therapy in Three Children with BRAF-Mutated Langerhans Cell Histiocytosis with Pituitary Involvement

Ying Yang, Dong Wang, Na Li, Honghao Ma, Hongyun Lian, Lei Cui, Qing Zhang, Xiaoxi Zhao, Liping Zhang, Yunze Zhao, Chanjuan Wang, Li Zhang, Tianyou Wang, Zhigang Li, Rui Zhang
OncoTargets and Therapy.2020; Volume 13: 12357. CrossRef

34,705 View
230 Download
18 Web of Science
23 Crossref

Pediatric malignancy

Genome-Wide Association Study for the Identification of Novel Genetic Variants Associated with the Risk of Neuroblastoma in Korean Children: Joon Seol Bae, Ji Won Lee, Jung Eun Yoo, Je-Gun Joung, Keon Hee Yoo, Hong Hoe Koo, Yun-Mi Song, Ki Woong Sung; Cancer Res Treat. 2020;52(4):1251-1261. Published online June 30, 2020; DOI: https://doi.org/10.4143/crt.2020.140

Abstract PDF Supplementary Material PubReader ePub

Purpose
Neuroblastoma (NB) is the most common extracranial solid tumor found in children. To identify significant genetic factors for the risk of NB, several genetic studies was conducted mainly for Caucasians and Europeans. However, considering racial differences, there is a possibility that genetic predispositions that contribute to the development of NB are different, and GWAS study has not yet been conducted on Korean NB patients.
Materials and Methods
To identify the genetic variations associated with the risk of pediatric NB in Korean children, we performed a genome-wide association analysis with 296 NB patients and 1000 unaffected controls (total n = 1,296) after data cleaning and filtering as well as imputation of non-genotyped SNPs using IMPUTE v2.3.2.
Results
After adjusting for multiple comparisons, we found 21 statistically significant SNPs associated with the risk of NB (P^corr < 0.05) within 12 genes (RPTN, MRPS18B, LRRC45, KANSL1L, ARHGEF40, IL15RA, L1TD1, ANO7, LAMA5, OR7G2, SALL4, and NEUROG2). Interestingly, out of these, 12 markers were nonsynonymous SNPs. The SNP rs76015112 was most significantly associated with the risk of NB (p = 8.1E-23, P^corr = 2.3E-17) and was located in the RPTN gene. In addition, significant nonsynonymous SNPs in ADGRE1 were found in patients with MYCN amplification (rs7256147, p = 2.6E-05). In high-risk group, rs7256147 was observed as a significant SNP (p = 5.9E-06).
Conclusion
Our findings might facilitate improved understanding of the mechanism of pediatric NB pathogenesis. However, functional evaluation and replication of these results in other populations are still needed.

Citations

Citations to this article as recorded by

Expression of anoctamin 7 (ANO7) is associated with poor prognosis and mucin 2 (MUC2) in colon adenocarcinoma: a study based on TCGA data
Chen Chen, Siripat Aluksanasuwan, Keerakarn Somsuan
Genomics & Informatics.2023; 21(4): e46. CrossRef
An Overview of Long Non-Coding (lnc)RNAs in Neuroblastoma
Francesca Baldini, Matilde Calderoni, Laura Vergani, Paola Modesto, Tullio Florio, Aldo Pagano
International Journal of Molecular Sciences.2021; 22(8): 4234. CrossRef

6,836 View
154 Download
4 Web of Science
2 Crossref

Induction Chemotherapy Plus Concurrent Chemoradiotherapy Versus Concurrent Chemoradiotherapy Alone in Locoregionally Advanced Nasopharyngeal Carcinoma in Children and Adolescents: A Matched Cohort Analysis: Yang Li, Lin-Quan Tang, Li-Ting Liu, Shan-Shan Guo, Yu-Jing Liang, Xue-Song Sun, Qing-Nan Tang, Jin-Xin Bei, Jing Tan, Shuai Chen, Jun Ma, Chong Zhao, Qiu-Yan Chen, Hai-Qiang Mai; Cancer Res Treat. 2018;50(4):1304-1315. Published online January 8, 2018; DOI: https://doi.org/10.4143/crt.2017.463

Abstract PDF Supplementary Material PubReader ePub

Purpose
The purpose of this study was to evaluate the long-term clinical outcome and toxicity of induction chemotherapy (IC) followed by concomitant chemoradiotherapy (CCRT) compared with CCRT alone for the treatment of children and adolescent locoregionally advanced nasopharyngeal carcinoma (LACANPC).
Materials and Methods
A total of 194 locoregionally advanced nasopharyngeal carcinoma patients youngerthan 21 years who received CCRT with or without IC before were included in the study population. Overall survival (OS) rate, progression-free survival (PFS) rate, locoregional recurrence-free survival (LRFS) rate, and distant metastasis-free survival (DMFS) rate were assessed by the Kaplan-Meier method and a log-rank test. Treatment toxicities were clarified and compared between two groups.
Results
One hundred and thiry of 194 patients received IC+CCRT. Patients who were younger and with more advanced TNM stage were more likely to receive IC+CCRT and intensive modulated radiotherapy. The addition of IC before CCRT failed to improve survival significantly. The matched analysis identified 43 well-balanced patients in both two groups. With a median follow-up of 51.5 months, no differences were found between the IC+CCRT group and the CCRT group in 5-year OS (83.7% vs. 74.6%, p=0.153), PFS (79.2% vs. 73.4%, p=0.355), LRFS (97.7% vs. 88.2%, p=0.083), and DMFS (81.6% vs. 81.6%, p=0.860). N3 was an independent prognostic factor predicting poorer OS, PFS, and DMFS. The addition of IC was associated with increased rates of grade 3 to 4 neutropenia.
Conclusion
This study failed to demonstrate that adding IC before CCRT could provide a significant additional survival benefit for LACANPC patients. Further investigations are warranted.

Citations

Citations to this article as recorded by

Nasopharyngeal Carcinoma in Children, Current Treatment Approach
Tal Ben-Ami
Journal of Pediatric Hematology/Oncology.2024; 46(3): 117. CrossRef
Concurrent chemoradiotherapy with or without neoadjuvant chemotherapy in pediatric patients with stage III-IVa nasopharyngeal carcinoma: a real-world propensity score-matched cohort study
Ya-Nan Jin, Zhao-Hui Ruan, Wan-Wei Cao, Lin Yang, Wei Yao, Xiao-Feng Pei, Wang-Jian Zhang, Tia Marks, Ji-Jin Yao, Liang-Ping Xia
Journal of Cancer Research and Clinical Oncology.2023; 149(13): 11929. CrossRef
Epidemiology and treatment of head and neck malignancies in the AYA generation
Takahiro Asakage
International Journal of Clinical Oncology.2022; 27(3): 465. CrossRef
Association of Treatment Advances With Survival Rates in Pediatric Patients With Nasopharyngeal Carcinoma in China, 1989-2020
Yu-Jing Liang, Li-Ting Liu, Yang Li, Pan Wang, Mei-Juan Luo, Dong-Xiang Wen, Qiu-Yan Chen, Hai-Qiang Mai
JAMA Network Open.2022; 5(3): e220173. CrossRef
LHX2 facilitates the progression of nasopharyngeal carcinoma via activation of the FGF1/FGFR axis
Tao Xie, Kunpeng Du, Wei Liu, Chunshan Liu, Baiyao Wang, Yunhong Tian, Rong Li, Xiaoting Huang, Jie Lin, Haifeng Jian, Jian Zhang, Yawei Yuan
British Journal of Cancer.2022; 127(7): 1239. CrossRef
Exploring the Optimal Chemotherapy Strategy for Locoregionally Advanced Children and Adolescent Nasopharyngeal Carcinoma Based on Pretreatment Epstein-Barr Virus DNA Level in the Era of Intensity Modulated Radiotherapy
Ziyi Zeng, Chen Chen, Lanlan Guo, Cheng Zhang, Lei Chen, Chuanping Yuan, Lixia Lu
Frontiers in Oncology.2021;[Epub] CrossRef
Optimal cumulative cisplatin dose during concurrent chemoradiotherapy among children and adolescents with locoregionally advanced nasopharyngeal carcinoma: A real-world data study
Ya-Nan Jin, Ji-Jin Yao, Ya-Fei You, Hui-Jiao Cao, Zi-Zi Li, Dan-Ling Dai, Wang-Jian Zhang, Tia Marks, Bei Zhang, Liang-Ping Xia
Radiotherapy and Oncology.2021; 161: 83. CrossRef
Nasopharyngeal carcinoma in children: Multimodal treatment and long‐term outcome of 92 patients in a single center over a 28‐year period
Rejin Kebudi, Sema Bay Buyukkapu, Omer Gorgun, Kübra Ozkaya, Rasim Meral, Inci Ayan, Musa Altun
Pediatric Blood & Cancer.2021;[Epub] CrossRef
Induction or adjuvant chemotherapy plus concurrent chemoradiotherapy versus concurrent chemoradiotherapy alone in paediatric nasopharyngeal carcinoma in the IMRT era: A recursive partitioning risk stratification analysis based on EBV DNA
Yu-Jing Liang, Dong-Xiang Wen, Mei-Juan Luo, Lin-Quan Tang, Shan-Shan Guo, Pan Wang, Qiu-Yan Chen, Li-Ting Liu, Hai-Qiang Mai
European Journal of Cancer.2021; 159: 133. CrossRef
Time-to-Event Supervised Genetic Algorithm Enables Induction Chemotherapy Decision Making for Nasopharyngeal Carcinoma
Demin Liu, Haojiang Li, Liyang Wu, Shuchao Chen, Tianqiao Zhang, Wenjie Huang, Guangying Ruan, Sai Li, Lizhi Liu, Hongbo Chen
IEEE Access.2021; 9: 98701. CrossRef
LHX2 Facilitates the Progression of Nasopharyngeal Carcinoma via Activation of the FGF1/FGFR Axis
Tao Xie, Kunpeng Du, Wei Liu, Chunshan Liu, Baiyao Wang, Yunhong Tian, Rong Li, Xiaoting Huang, Jie Lin, Haifeng Jian, Jian Zhang, Yawei Yuan
SSRN Electronic Journal .2021;[Epub] CrossRef
Prognostic impact of immunohistopathologic features in definitive radiation therapy for nasopharyngeal cancer patients
Naoya Murakami, Taisuke Mori, Yuko Kubo, Seiichi Yoshimoto, Kimiteru Ito, Yoshitaka Honma, Takao Ueno, Kenya Kobayashi, Hiroyuki Okamoto, Narikazu Boku, Kana Takahashi, Koji Inaba, Kae Okuma, Hiroshi Igaki, Yuko Nakayama, Jun Itami
Journal of Radiation Research.2020; 61(1): 161. CrossRef
The efficacy and safety of induction chemotherapy combined with concurrent chemoradiotherapy versus concurrent chemoradiotherapy alone in nasopharyngeal carcinoma patients: a systematic review and meta-analysis
Bi-Cheng Wang, Bo-Ya Xiao, Guo-He Lin, Chang Wang, Quentin Liu
BMC Cancer.2020;[Epub] CrossRef
Update in pediatric nasopharyngeal undifferentiated carcinoma
Line Claude, Emmanuel Jouglar, Loig Duverge, Daniel Orbach
The British Journal of Radiology.2019;[Epub] CrossRef

8,462 View
210 Download
18 Web of Science
14 Crossref

Case report

Secretory Carcinoma of the Breast in Three Year-old Girl: A Case report: Woo Chul Noh, Nam Sun Paik, Kyung Ja Cho, Jin Haeng Chung, Seung Kon Nam, Doo Hwan Choe, Nan Mo Moon; J Korean Cancer Assoc. 2000;32(4):822-826.

Abstract PDF: Secretory carcinoma is one of the least common forms of breast cancer and demonstrates distinctive clinical and pathological characteristics. We herein report a case of secretory carcinoma of the breast in 3 year and 1 month-old girl. At presentation, the patient had a 2.5cm sized mass on her left breast which was firmly attached to the overlying nipple. The aspiration cytologic findings of the tumor were consistent with a secretory carcinoma. After confirming malignancy by frozen section diagnosis, a modified radical mastectomy was performed and secretory carcinoma was finally diagnosed. To our knowledge, secretory breast carcinoma in children has not been reported previously in Korea and this seems to be the youngest case of secretory carcinoma of the breast which had been reported in English literature.

2,928 View
42 Download

Original Articles

A Clinical Study of Pediatric Myelodysplastic Syndrome: Application of International Prognostic Scoring System and the Review of the Korean Literature: Hoon Kook, Chan Jong Kim, Weon Sang Yoon, Na Eun Ryu, Kyoung Joong Chung, Tai Ju Hwang; J Korean Cancer Assoc. 2000;32(1):178-190.

Abstract PDF: PURPOSE
Myelodysplastic syndrome (MDS) in children needs to be elucidated in terms of clinical characteristics, natural history, the most effective treatment and prognostic factors, as the disease is very rare and its definition and classification has not reached a consensus by many physician. This study was aimed to describe the characteristics and the disease courses of Korean children with MDS, and to analyze the usefulness of prognostic scoring systems in the prediction of transformation to acute myelogenous leukemia (AML) and overall survival among subgroups.
MATERIALS AND METHODS
Fourteen children with MDS seen at Chonnam University Hospital and additional 59 patients identified by the review of Korean literature were evaluated to define clinical characteristics and disease courses. Kaplan-Meier (K-M) probability of leukemic transformation and overall survival were plotted. FAB subtypes, subgroups by Boumemouth Scoring System (BSS), and International Prognostic Scoring System (IPSS) risk groups were compared to predict transformation to AML and overall survival.
RESULTS
The median age of 14 patients was 36.5 months. The sex ratio was 3.7:1 (M: F). The frequency of FAB subtypes in Korea was similar to that of other countries except for higher proportion of RA (37%). K-M 3-yr probability of AML transformation and survival for Korean patients were 54.7%, and 49.8%, respectively. Although FAB system, BMS and IPSS were all capable of discriminating subgroups in the prediction of AML transformation and survival, they did not reach the significant level possibly due to small number of patients assigned to each subgroup.
CONCLUSION
The clinical characteristics of Korean children with MDS were not different from those of other countries. This study showed the high rate of AML transformation and poor survival in children with MDS.

2,824 View
31 Download

Pediatric Pancreatic Tumors: Dae Yeon Kim, Min Gew Choi, Ki Hong Kim, In One Kim, Sung Eun Jung, Seong Cheol Lee, Kwi Won Park, Woo Ki Kim; J Korean Cancer Assoc. 1999;31(4):830-835.

Abstract PDF: PURPOSE
Pancreatic tumors are relatively rare in children. Until now more than 150 cases have been reported in the English literature. In this paper, the authors report the tumors clinical characteristics and the results of surgery in eleven children.
MATERIALS AND METHODS
Eleven cases of pancreatic tumor pathologically verified at Seoul National University Children's Hospital between 1984 to 1998 were retrospectively analyzed. Four were boys and seven were girls, and their mean age at diagnosis was 7.7 (range, 2 13) years.
RESULTS
There were six solid and papillary epithelial neoplasms of the pancreas (SPENP) and five pancreatoblastomas. All children came to medical attention because of abdominal masses. Tumors ranged in size from 6.0X 5.0 cm to 10.5 x 8.0 cm. Eight tumors were located in head and three in tail. Complete excision was performed in eight cases (six in SPENP and two in pancreatoblastoma). Incomplete excision was performed in two cases of pancreatoblastoma. One patient with pancreatoblastoma had an unresectable tumor at the time of diagnosis and needle aspiration biopsy was done under the ultrasound guidance. No patient died during surgery. After a mean follow-up period of 4.1 years, all patients with SPENP were alive and there had been no recurrence. However, of two patients who received complete excision in pancreatoblastoma, one presented with liver metastasis at 4 months after operation and received chemotherapy, but died 6 months after operation. The other patient had local recurrence 1 year after operation. Reoperation and chemotherapy were performed and the child is now alive without evidence of disease montbs after the initial operation. All three patients with unresectable tumor died in spite of adjuvant radiotherapy and chemotherapy.
CONCLUSION
Pediatric pancreatic tumors comprise rare heterogenous groups of malignancies with their prognosis dependent upon adequate resection and pathologic classification. Complete resection of pancreatic tumors arising anywhere in the pancreas was recommended.

2,677 View
17 Download

First
Prev
Page of 1
Next
Last

Search