Article
Oncology
Mirjam Blattner-Johnson, David T. W. Jones, Elke Pfaff
Summary: Despite advances in diagnosing and treating pediatric cancers, it remains a leading cause of childhood death in developed countries. The heterogeneity of tumor types in children, which differ from adult carcinomas, has made understanding the underlying biology and developing targeted treatments difficult. Additionally, the pharmaceutical industry has been reluctant to develop products for this population due to market size. However, recent changes in science and regulation are addressing these challenges.
SEMINARS IN CANCER BIOLOGY
(2022)
Review
Oncology
Mohammed I. Quraishi
Summary: Precision oncology utilizes molecular profiling to guide therapeutic interventions, and radiomic analysis can depict molecular mechanisms and correlate with genetic alterations. Combining these two approaches can be beneficial in the management of colorectal cancer.
FRONTIERS IN ONCOLOGY
(2022)
Article
Biochemistry & Molecular Biology
Fabio Pastorino, Mario Capasso, Chiara Brignole, Serena Giglio, Veronica Bensa, Sueva Cantalupo, Vito Alessandro Lasorsa, Annalisa Tondo, Rossella Mura, Angela Rita Sementa, Alberto Garaventa, Mirco Ponzoni, Loredana Amoroso
Summary: Neuroblastoma is the most common extracranial solid tumor in childhood. The prognosis for patients with metastatic relapse or refractory disease is poor. The integration of genome sequencing into standard clinical practice is necessary for personalized therapy. In this study, whole exome sequencing was performed on two patients with relapsed NB, and actionable genetic variations were identified and treated accordingly. The results demonstrate the feasibility of incorporating clinical WES into pediatric oncology practice.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Review
Oncology
M. Aldea, L. Friboulet, S. Apcher, F. Jaulin, F. Mosele, T. Sourisseau, J. -c. Soria, S. Nikolaev, F. Andre
Summary: Precision medicine for cancer integrates multiple dimensions of biology to model cancer progression in each patient. Multiple drivers per tumor and the diversity of genomic alterations challenge medical decision-making. Drug sensitivity depends on actionable targets and genomic alterations. Artificial intelligence is indispensable for thorough analysis of complex biological data. Future perspectives involve acknowledging spatial and temporal heterogeneity in cancer.
Review
Oncology
Thai Hoa Tran, Stephen P. Hunger
Summary: Acute lymphoblastic leukemia (ALL) is the most common childhood cancer and has shown significant improvement in survival rates through risk-adapted chemotherapy, prevention measures, and molecular analysis. However, challenges remain for older patients and relapsed/refractory cases.
SEMINARS IN CANCER BIOLOGY
(2022)
Article
Oncology
Leonie W. Wahjudi, Stephan Bernhardt, Khalid Abnaof, Peter Horak, Simon Kreutzfeldt, Christoph Heining, Simone Borgoni, Corinna Becki, Daniela Berg, Daniela Richter, Barbara Hutter, Sebastian Uhrig, Katrin Pfuetze, Jonas Leichsenring, Hanno Glimm, Benedikt Brors, Christof von Kalle, Albrecht Stenzinger, Ulrike Korf, Stefan Froehling, Stefan Wiemann
Summary: The study demonstrates that integrated analysis of DNA, RNA, and protein data can optimize the therapeutic stratification of individual patients, potentially increasing the success rate of precision cancer therapy. Prospective validation studies are necessary to further incorporate proteomic analysis into precision oncology.
INTERNATIONAL JOURNAL OF CANCER
(2021)
Article
Oncology
Brendan Reardon, Nathanael D. Moore, Nicholas S. Moore, Eric Kofman, Saud H. AlDubayan, Alexander T. M. Cheung, Jake Conway, Haitham Elmarakeby, Alma Imamovic, Sophia C. Kamran, Tanya Keenan, Daniel Keliher, David J. Konieczkowski, David Liu, Kent W. Mouw, Jihye Park, Natalie Vokes, Felix Dietlein, Eliezer M. Van Allen
Summary: The MOAlmanac is a clinical interpretation algorithm and knowledge base for personalized cancer treatment, enabling decision-making and hypothesis generation. Testing showed an increase in the number of clinical hypotheses and nominated therapies for patients. This computational method provides support for integrated clinical interpretation of individualized molecular profiles.
Review
Biochemistry & Molecular Biology
Stuart L. Rulten, Richard P. Grose, Susanne A. Gatz, J. Louise Jones, Angus J. M. Cameron
Summary: Our understanding of cancer has advanced rapidly, recognizing the variation between patients and leading to precision medicines that promise maximum efficacy, safety, and reduced costs. However, many patients still lack access to these personalized treatments, highlighting the need for new disease characteristics, accurate diagnosis, better clinical trials, and global accessibility to targeted therapies. This review discusses current technological trends and multidisciplinary efforts to ensure wider implementation of precision oncology.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Oncology
Mark Marshall, Jennifer Ivanovich, Morgan Schmitt, Amy Helvie, Lisa Langsford, Jennifer Casterline, Michael Ferguson
Summary: Precision oncology involves selecting effective treatments for cancer patients based on genomic profiling of their tumors. While progress has been slower in pediatric oncology compared to adult oncology, advancements made in adult cancer treatments over the past decade are now being applied to young high-risk and hard-to-treat patients. The FDA has approved 23 targeted drugs for pediatric cancer, making them a standard of care.
FRONTIERS IN ONCOLOGY
(2023)
Article
Clinical Neurology
Colin B. Josephson, Samuel Wiebe
Summary: Precision medicine aims to provide individualized treatment based on unique patient characteristics, but faces challenges in data integration, privacy issues, and acceptance by clinicians. Despite accumulating vast amounts of data, there is still a need to address the complexities of advanced analytics models and ensure patient consent and data security. Systematic approaches to data collection and education are necessary to facilitate the translation of precision medicine concepts into routine clinical practice for conditions like epilepsy.
Article
Biochemistry & Molecular Biology
Ruishan Liu, James Zou
Summary: An analysis of genomic data and medical records from over 40,000 cancer patients has identified hundreds of mutations that can predict how patients will respond to specific cancer therapies. These predictive biomarkers could provide guidance for personalized treatment planning.
Review
Health Care Sciences & Services
Vianney Gilard, Stephane Derrey, Stephane Marret, Soumeya Bekri, Abdellah Tebani
Summary: Neurosurgeons have always defined themselves as physicians with a surgical practice, and they are now striving to effectively embrace the era of data-driven medicine in the ongoing precision medicine surge that drives patient-centric healthcare. The convergence between surgery and precision medicine is best illustrated in neuro-oncology, paving the way for new medical perspectives. Prominent advances in healthcare and big data challenge the medical community to deeply rethink current and future medical practice.
JOURNAL OF PERSONALIZED MEDICINE
(2021)
Article
Oncology
P. Gargallo, F. Bautista, A. Juan-Ribelles, E. Izquierdo, A. Soriano, T. de Rojas, A. Escudero, C. Lavarino, P. Solano, R. Hladun, A. Rubio-San-Simon, I Martinez-Romera, I Calabria, N. G. Olaciregui, A. Castaneda-Heredia, E. de Alava, A. Perez-Martinez, I Astigarraga, A. Patino-Garcia, J. Alonso, A. Fernandez-Teijeiro, A. Canete, L. Moreno
Summary: The study analyzed the current status of personalized medicine in pediatric oncology in Spain, focusing on tumor molecular studies and genomic sequencing. Despite the implementation of precision medicine in many centers in Spain, there are unevenness and lack of protocol standardization in its implementation.
CLINICAL & TRANSLATIONAL ONCOLOGY
(2022)
Article
Biotechnology & Applied Microbiology
Stefano Crabu
Summary: This article aims to contribute to the ongoing debate on how care professionals and biomedical investigators mobilize collective expertise to shape precise knowledge in cancer medicine. It discusses the importance of professional jurisdiction, organizing technologies, and cooperative work in the day-to-day practices of producing knowledge for precision oncology regimen.
NEW GENETICS AND SOCIETY
(2021)
Review
Oncology
Andrew Wahba, Russ Wolters, Jennifer H. Foster
Summary: Patients with high-risk neuroblastoma have a poor chance of survival and often develop significant late-effects. ALK inhibitors and anti-GD2 therapies have become mainstays of treatment, but the development of additional molecular targeted therapy has proven challenging due to the heterogeneity of high-risk neuroblastoma.
Article
Hematology
Swantje Buchmann, Martin Schrappe, Andre Baruchel, Andrea Biondi, Michael Borowitz, Myriam Campbell, Gunnar Cario, Giovanni Cazzaniga, Gabriele Escherich, Christine J. Harrison, Mats Heyman, Stephen P. Hunger, Csongor Kiss, Hsi-Che Liu, Franco Locatelli, Mignon L. Loh, Atsushi Manabe, Georg Mann, Rob Pieters, Ching-Hon Pui, Susana Rives, Kjeld Schmiegelow, Lewis B. Silverman, Jan Stary, Ajay Vora, Patrick Brown
Summary: Comparison of treatment strategies in de novo pediatric acute lymphoblastic leukemia (ALL) requires standardized measures of efficacy. Key parameters defining disease-related events, such as complete remission (CR), treatment failure (TF), and relapse, have significant heterogeneity in their definitions. A consensus definition was established by representatives of major international ALL clinical trial groups, which includes specific criteria for CR, TF, and relapse. These consensus definitions will improve the comparability of outcomes in pediatric ALL trials and promote the development of international collaborative trials.
Article
Oncology
Yingyi He, Jingliao Zhang, Yingchi Zhang, Zhengbin Hu, Pengfei Wang, Wenting Gan, Shao Xie, Maoxiang Qian, Ching-Hon Pui, Hua Jiang, Xiaofan Zhu, Hui Zhang, Weina Zhang
Summary: The prognosis of T-cell acute lymphoblastic leukemia (T-ALL) patients lags behind that of B-cell ALL patients, especially in refractory or relapsed cases. However, studies have suggested that dasatinib may have therapeutic potential in refractory T-ALL patients.
PEDIATRIC BLOOD & CANCER
(2022)
Article
Multidisciplinary Sciences
Valentin Barsan, Yuntao Xia, David Klein, Veronica Gonzalez-Pena, Sarah Youssef, Yuki Inaba, Ousman Mahmud, Sivaraman Natarajan, Vibhu Agarwal, Yakun Pang, Robert Autry, Ching-Hon Pui, Hiroto Inaba, William Evans, Charles Gawad
Summary: This study assessed the feasibility of using a hybrid capture next-generation sequencing panel to measure the clearance of molecular leukemic disease and abundance of microbial species in pediatric patients with ALL during induction chemotherapy. It provides a technical and conceptual framework for further expansion to other leukemia patients and cancer types.
Article
Oncology
Hope D. Swanson, Hana Hakim, Diego R. Hijano, Ted Morton, Shane Cross, Hiroto Inaba, Sima Jeha, Ching-Hon Pui, Seth E. Karol
Summary: This study aimed to evaluate the safety of SARS-CoV-2 vaccines in patients with anti-pegaspargase antibodies. The results showed that these patients tolerated the vaccines well, which also use polyethylene glycol as a stabilizing agent, with no significant adverse reactions observed.
Article
Oncology
Andishe Attarbaschi, Anja Moericke, Christine J. Harrison, Georg Mann, Andre Baruchel, Barbara De Moerloose, Valentino Conter, Meenakshi Devidas, Sarah Elitzur, Gabriele Escherich, Stephen P. Hunger, Keizo Horibe, Atsushi Manabe, Mignon L. Loh, Rob Pieters, Kjeld Schmiegelow, Lewis B. Silverman, Jan Stary, Ajay Vora, Ching-Hon Pui, Martin Schrappe, Martin Zimmermann
Summary: This study aimed to investigate the prognostic factors and efficacy of allogeneic hematopoietic stem-cell transplantation (allo-HSCT) in the first remission of patients with noninfant childhood acute lymphoblastic leukemia (ALL) with 11q23/KMT2A rearrangements. The results showed that the prognosis of patients with noninfant 11q23/KMT2A-rearranged ALL has improved compared to historical data, but allo-HSCT did not improve outcomes. Targeted therapies are needed to reduce relapse and treatment-related mortality rates.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
Letter
Oncology
Kentaro Ohki, Ellie R. Butler, Nobutaka Kiyokawa, Shinsuke Hirabayashi, Anke K. Bergmann, Anja Moericke, Judith M. Boer, Helene Cave, Giovanni Cazzaniga, Allen Eng Juh Yeoh, Masashi Sanada, Toshihiko Imamura, Hiroto Inaba, Charles G. Mullighan, Mignon L. Loh, Ulrika Noren-Nystrom, Lee-Yung Shih, Marketa Zaliova, Ching-Hon Pui, Oskar A. Haas, Christine J. Harrison, Anthony V. Moorman, Atsushi Manabe
Article
Oncology
Wenting Hu, Yin Ting Cheung, Yanjing Tang, Li Hong, Yuan Zhu, Jing Chen, Zhuo Wang, Min Zhou, Yijin Gao, Jing Chen, Benshang Li, Huiliang Xue, Longjun Gu, Shuhong Shen, Jingyan Tang, Ching-Hon Pui, Hiroto Inaba, Jiaoyang Cai
Summary: Studies on the association between BMI at diagnosis and treatment outcomes in Chinese children with ALL have shown inconsistent results. While weight status was not associated with event-free survival or overall survival, overweight patients were at a higher risk of treatment-related mortality.
Meeting Abstract
Hematology
Shawn Lee, Wenjian Yang, Yoshihiro Gocho, August John, Lauren Rowland, Brandon Smart, Hannah Williams, Dylan Maxwell, Jeremy Hunt, Wentao Yang, Kristine R. Crews, Kathryn G. Roberts, Sima Jeha, Cheng Cheng, Seth E. Karol, Mary Relling, Gary Rosner, Hiroto Inaba, Charles G. Mullighan, Ching-Hon Pui, William E. Evans, Jun J. Yang
Meeting Abstract
Hematology
Yizhen Li, Takaya Moriyama, Satoshi Yoshimura, Xuijie Zhao, Zhenhua Li, Xu Yang, Jacob A. Steele, Shondra Miller, Elisabeth Paietta, Marina Konopleva, Jiyang Yu, Hiroto Inaba, Raul C. Ribeiro, Ching-Hon Pui, Jun J. Yang
Article
Hematology
David Spencer Mangum, Johnathon D. Bishop, Yinmei Zhou, Cheng Cheng, Seth E. Karol, Jeffrey E. Rubnitz, Raul C. Ribeiro, Jun J. Yang, Charles G. Mullighan, Sima Jeha, Ching-Hon Pui, Hiroto Inaba
Summary: Among children with acute lymphoblastic leukaemia, about 14.7% presented without peripheral blood blasts. While absence of blasts did not affect survival outcomes, these patients had distinct genetic and clinical characteristics, with a higher incidence of hyperdiploid B-ALL and lower rates of central nervous system involvement.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Article
Oncology
Chih-Hsiang Yu, Shiann-Tarng Jou, Ying-Hui Su, Elane Coustan-Smith, Gang Wu, Chao-Neng Cheng, Meng-Yao Lu, Kai-Hsin Lin, Kang-Hsi Wu, Shu-Huey Chen, Fang-Liang Huang, Hsiu-Hao Chang, Jinn-Li Wang, Hsiu-Ju Yen, Meng-Ju Li, Shu-Wei Chou, Wan-Ling Ho, Yen-Lin Liu, Chia-Ching Chang, Ze-Shiang Lin, Chien-Yu Lin, Hsuan-Yu Chen, Yu-Ling Ni, Dong-Tsamn Lin, Shu-Wha Lin, Jun J. Yang, Yen-Hsuan Ni, Ching-Hon Pui, Sung-Liang Yu, Yung-Li Yang
Summary: This study analyzed data from two consecutive protocols to determine the clinical impact of minimal/measurable residual disease (MRD) and tumor genetic subtypes in newly diagnosed pediatric acute lymphoblastic leukemia (ALL) patients. The results showed that MRD-directed therapy improved outcomes for pediatric ALL, especially for standard-risk patients. This study provides important data to inform the design of future clinical trials in Taiwan.
Article
Biochemistry & Molecular Biology
Shawn H. R. Lee, Wenjian Yang, Yoshihiro Gocho, August John, Lauren Rowland, Brandon Smart, Hannah Williams, Dylan Maxwell, Jeremy Hunt, Wentao Yang, Kristine R. R. Crews, Kathryn G. G. Roberts, Sima Jeha, Cheng Cheng, Seth E. E. Karol, Mary V. V. Relling, Gary L. L. Rosner, Hiroto Inaba, Charles G. G. Mullighan, Ching-Hon Pui, William E. E. Evans, Jun J. J. Yang
Summary: Contemporary chemotherapy for childhood acute lymphoblastic leukemia is personalized based on clinical features, leukemia genomics, and minimal residual disease. However, the pharmacological basis of these prognostic variables is unclear. A study analyzing samples from 805 children with newly diagnosed leukemia identified variations in drug response and determined that certain subtypes were more sensitive to specific agents. The findings suggest opportunities for individualizing therapy and exploring alternative strategies for childhood acute lymphoblastic leukemia.
Article
Oncology
Georgios E. Christakopoulos, Kendra N. Walker, Jesse Smith, Clifford M. Takemoto, Yan Zheng, Ching-Hon Pui, Raul C. Ribeiro, Lei Wang, Stanley B. Pounds, Jeffrey E. Rubnitz, Hiroto Inaba
Summary: This study investigated the clinical management strategies and effects of cytoreduction in pediatric patients with acute myeloid leukemia (AML) and hyperleukocytosis. The results showed that LD-cytarabine treatment was safe and effective in reducing leukocyte counts in children with AML and hyperleukocytosis.
Meeting Abstract
Oncology
Shawn Lee, Federico Antillon, Deqing Pei, Wenjian Yang, Kathryn G. Roberts, Zhenhua Li, Meenakshi Devidas, Wentao Yang, Cesar Najera, Hai Peng Lin, Ah Moy Tan, Hany Ariffin, Cheng Cheng, William E. Evans, Stephen P. Hunger, Sima Jeha, Charles G. Mullighan, Mignon L. Loh, Allen Ej Yeoh, Ching-Hon Pui, Jun J. Yang
CANCER EPIDEMIOLOGY BIOMARKERS & PREVENTION
(2022)
Article
Oncology
Kellen Gandy, Matthew A. Scoggins, Nicholas Phillips, Ellen van der Plas, Slim Fellah, Lisa M. Jacola, Ching-Hon Pui, Melissa M. Hudson, Wilburn E. Reddick, Ranganatha Sitaram, Kevin R. Krull
Summary: This study found sex-based differences in functional neuroimaging outcomes among survivors of pediatric acute lymphoblastic leukemia treated with chemotherapy alone. Female survivors exhibited lower brain activation and slower response speed during increased working memory load compared to male survivors. Performance accuracy was negatively correlated with fMRI activity in female survivors but not in males.
JNCI CANCER SPECTRUM
(2022)