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Pediatric bone tumor

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https://www.readbyqxmd.com/read/28938163/tumor-cell-targeted-delivery-of-crispr-cas9-by-aptamer-functionalized-lipopolymer-for-therapeutic-genome-editing-of-vegfa-in-osteosarcoma
#1
Chao Liang, Fangfei Li, Luyao Wang, Zong-Kang Zhang, Chao Wang, Bing He, Jie Li, Zhihao Chen, Atik Badshah Shaikh, Jin Liu, Xiaohao Wu, Songlin Peng, Lei Dang, Baosheng Guo, Xiaojuan He, D W T Au, Cheng Lu, Hailong Zhu, Bao-Ting Zhang, Aiping Lu, Ge Zhang
Osteosarcoma (OS) is a highly aggressive pediatric cancer, characterized by frequent lung metastasis and pathologic bone destruction. Vascular endothelial growth factor A (VEGFA), highly expressed in OS, not only contributes to angiogenesis within the tumor microenvironment via paracrine stimulation of vascular endothelial cells, but also acts as an autocrine survival factor for tumor cell themselves, thus making it a promising therapeutic target for OS. CRISPR/Cas9 is a versatile genome editing technology and holds tremendous promise for cancer treatment...
September 13, 2017: Biomaterials
https://www.readbyqxmd.com/read/28935694/cellular-kinetics-of-ctl019-in-relapsed-refractory-b-cell-acute-lymphoblastic-leukemia-and-chronic-lymphocytic-leukemia
#2
Karen Thudium Mueller, Shannon L Maude, David L Porter, Noelle Frey, Patricia Wood, Xia Han, Edward Waldron, Abhijit Chakraborty, Rakesh Awasthi, Bruce L Levine, J Joseph Melenhorst, Stephan A Grupp, Carl H June, Simon F Lacey
Tisagenlecleucel (CTL019) is an investigational immunotherapy that involves reprogramming a patient's own T cells with a transgene encoding a chimeric antigen receptor to identify and eliminate CD19-expressing cells. We previously reported that CTL019 achieved impressive clinical efficacy in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) and chronic lymphocytic leukemia (CLL), including the expansion and persistence of CTL019 cells, which correlates with response to therapy. Here, we performed formal cellular kinetic analyses of CTL019 in a larger cohort of 103 patients treated with CTL019 in 2 different diseases (ALL and CLL)...
September 21, 2017: Blood
https://www.readbyqxmd.com/read/28933651/characterization-of-h3-3k36m-as-a-tool-to-study-h3k36-methylation-in-cancer-cells
#3
Saumya M Sankaran, Or Gozani
Recurrent mutations at key lysine residues in the histone variant H3.3 are thought to play an etiologic role in the development of distinct subsets of pediatric gliomas and bone and cartilage cancers. H3.3K36M is one such mutation that was originally identified in chondroblastomas, and its expression in these tumors contributes to oncogenic reprogramming by triggering global depletion of dimethylation and trimethylation at H3K36 with a concomitant increase in the levels of H3K27 trimethylation. H3.3K36M expression can also cause epigenomic changes in cell types beyond chondrocytic cells...
September 21, 2017: Epigenetics: Official Journal of the DNA Methylation Society
https://www.readbyqxmd.com/read/28913516/a-rare-erosive-orbital-mass-in-a-child-case-report-of-myofibroma
#4
Bahram Eshraghi, Shima Dehghani, Ghasem Saeedi-Anari
PURPOSE: To present the clinical, histological, and radiographic findings of a case of orbital myofibroma in an unusual location. The literature is reviewed and the clinical relevance discussed. METHODS: A 5-year-old boy was examined with a 1.5-month history of progressive swelling in the left supraorbital region. RESULTS: Examination revealed a firm, painless mass in the supralateral region of the left orbit with slight reddish discoloration of the overlying skin...
September 2017: Journal of Current Ophthalmology
https://www.readbyqxmd.com/read/28904574/multicentric-chordoma-in-a-child
#5
Shighakolli Ramesh, Raju Subodh, Srinadh Boppana, Erukkambattu Jayashankar
Chordomas are primary malignant bone tumors that arise in the axial skeleton, believed to originate from remnants of embryologic notochordal cell rests. Multicentric origin of chordoma is extremely rare. To our literature search, we found only three cases of multicentric chordoma in adults. We report a first case of multicentric chordoma in pediatric age group. A 14-month-old child presented with torticolis and left upper limb monoparesis, imaging showed expansile bony destructive lesion in clivus and dorsal spine simultaneously...
April 2017: Journal of Pediatric Neurosciences
https://www.readbyqxmd.com/read/28903844/bone-marrow-involvement-in-metastatic-pediatric-ewing-sarcoma
#6
Saadiya Javed Khan, Irfana Ishaq, Haleema Saeed, Muhammad Bilal Fayyaz, Syed Ali Shazif Baqari, Rabia Mohammad Wali
OBJECTIVE: To evaluate the frequency of bone marrow involvement with metastatic lung and bone sites in newly-diagnosed pediatric patients with Ewing sarcoma (ES). STUDY DESIGN: An observational study. PLACE AND DURATION OF STUDY: Shaukat Khanum Memorial Cancer Hospital, Lahore, Pakistan, from January 2010 to October 2015. METHODOLOGY: Newly-diagnosed pediatric-age patients with ES were inducted. Ten patients were excluded because bone marrow aspiration/biopsy (BMAB) was not done...
August 2017: Journal of the College of Physicians and Surgeons—Pakistan: JCPSP
https://www.readbyqxmd.com/read/28888615/who-cares-for-adolescents-and-young-adults-with-cancer-in-brazil
#7
Helena T G Martins, Nathalie V Balmant, Neimar de Paula Silva, Marceli de O Santos, Rejane de S Reis, Beatriz de Camargo
OBJECTIVE: Approximately 6% of all cancers arise in adolescents and young adults. Currently, the ward type best placed to treat this patient group remains controversial. The aim of this study was to evaluate exactly where adolescents and young adults with cancer are treated in Brazil. METHODS: Data were extracted from 271 Brazilian hospital-based cancer registries (2007-2011), including all five national regions (North, Northeast, Midwest, South, and Southeast)...
September 6, 2017: Jornal de Pediatria
https://www.readbyqxmd.com/read/28887617/fdg-pet-ct-appearance-of-local-osteosarcoma-recurrences-in-pediatric-patients
#8
Susan E Sharp, Barry L Shulkin, Michael J Gelfand, M Beth McCarville
BACKGROUND: Osteosarcoma is the most common pediatric malignant bone tumor, frequently surgically managed with limb salvage rather than amputation. Local recurrences are seen in up to 9% of osteosarcoma patients, with CT and MRI imaging often limited by metal artifacts. OBJECTIVE: To describe the [F-18]2-fluoro-2-deoxyglucose (FDG) PET/CT appearance of local osteosarcoma recurrences with correlation to findings on other imaging modalities. MATERIALS AND METHODS: A retrospective review of pediatric osteosarcoma patients imaged with FDG PET/CT was performed in patients with pathologically proven local recurrences...
September 8, 2017: Pediatric Radiology
https://www.readbyqxmd.com/read/28885439/multiple-recurrent-fibromatosis-with-cranial-fasciitis-characteristics-in-a-pediatric-patient
#9
Rui Dai, Irene J Pien, David A Brown, Andre Marshall, Herbert E Fuchs, Jeffrey R Marcus
Cranial fasciitis is a rare, rapidly growing, but benign fibroblastic tumor of the skull that generally presents in childhood. Local resection or curettage of the affected bone is generally curative and the tumor is thought not to recur. Cranial fasciitis is distinguished by positive cytoplasmic and nuclear beta-catenin staining. Fibromatosis is a clonal myofibroblastic nonmalignant proliferation that generally demonstrates positive nuclear beta-catenin staining. In this report, the authors present a patient with fibromatosis with cranial fasciitis characteristics in a 2...
September 6, 2017: Journal of Craniofacial Surgery
https://www.readbyqxmd.com/read/28881804/altered-erythropoiesis-and-decreased-number-of-erythrocytes-in-children-with-neuroblastoma
#10
Fabio Morandi, Sebastiano Barco, Sara Stigliani, Michela Croce, Luca Persico, Corrado Lagazio, Francesca Scuderi, Maria Luisa Belli, Mariapina Montera, Giuliana Cangemi, Sarah Pozzi, Valentina Rigo, Paola Scaruffi, Loredana Amoroso, Giovanni Erminio, Vito Pistoia, Silvano Ferrini, Maria Valeria Corrias
Neuroblastoma (NB) is a pediatric tumor presenting at diagnosis either as localized or metastatic disease, which mainly involves the bone marrow (BM). The physical occupancy of BM space by metastatic NB cells has been held responsible for impairment of BM function. Here, we investigated whether localized or metastatic NB may alter hematopoietic lineages' maturation and release of mature cells in the periphery, through gene expression profiling, analysis of BM smears, cell blood count and flow cytometry analysis...
August 8, 2017: Oncotarget
https://www.readbyqxmd.com/read/28867113/precision-medicine-and-pet-computed-tomography-in-pediatric-malignancies
#11
REVIEW
Yasemin Sanli, Ebru Yilmaz, Rathan M Subramaniam
Fluorine-18 fluorodeoxyglucose ((18)F-FDG) PET-computed tomography (CT) plays a significant role in diagnosis, staging, therapy selection, and therapy assessment of multiple pediatric malignancies and facilitating precision medicine delivery in pediatric patients. In patients with Hodgkin lymphoma, interim fludeoxyglucose (18)F-FDG PET/CT is highly sensitive and specific for predicting survival and multiple trials with FDG PET/CT-based adaptive therapies are currently ongoing. It is superior to iodine-131 metaiodobenzylguanidine ((131)I-MIBG) scintigraphy and bone scintigraphy for detecting metastases in neuroblastoma patients and sarcoma patients...
October 2017: PET Clinics
https://www.readbyqxmd.com/read/28864350/immunohistochemical-analysis-of-nkx2-2-etv4-and-bcor-in-a-large-series-of-genetically-confirmed-ewing-sarcoma-family-of-tumors
#12
Isidro Machado, Akihiko Yoshida, José Antonio López-Guerrero, María Gema Nieto, Samuel Navarro, Piero Picci, Antonio Llombart-Bosch
Ewing sarcoma is an aggressive neoplasm of pediatric and adolescent patients. Immunohistochemistry (IHC) can be used to support the morphologic diagnosis of Ewing sarcoma family of tumors (ESFT) in a convincing clinical/radiological context. Although neither NKX2.2 nor CD99 alone are entirely specific, when combined, the diagnostic specificity is high. The aim of the present study was to investigate the IHC expression of NKX2.2, ETV4 and BCOR in a large series of genetically confirmed ESFT. The results for CD99 and CAV-1 immunoreactivity, and the histological and fusion gene subtypes were retrieved from our previous study...
August 25, 2017: Pathology, Research and Practice
https://www.readbyqxmd.com/read/28853528/-neuro-oncology-a-new-field-in-davidoff-cancer-center-at-rabin-medical-center
#13
REVIEW
Shlomit Yust-Katz, Dror Limon, Ramez Abu-Shkara, Tali Siegal
Neuro-oncology is a subspecialty attracting physicians from medical disciplines such as neurology, neurosurgery, pediatrics, oncology, and radiotherapy. It deals with diagnosis and management of primary brain tumors, as well as metastatic and non-metastatic neurological manifestations that frequently affect cancer patients including brain metastases, paraneoplastic syndromes and neurological complications of cancer treatment. A neuro-oncology unit was established in Davidoff Cancer Center at Rabin Medical Center...
August 2017: Harefuah
https://www.readbyqxmd.com/read/28852349/an-osteological-study-on-the-prevalence-of-osteochondromas
#14
Gregory R Gaumer, Doug S Weinberg, Christopher D Collier, Patrick J Getty, Raymond W Liu
BACKGROUND: Osteochondromas are frequently reported to be the most common benign tumor of bone. However, there is not definitive data on their prevalence rate as they are often clinically silent, with previous studies estimating 1-3%. METHODS: We examined a large osteologic collection of 2954 skeletons to identify likely osteochondromas. One author examined all bones excluding the skull in each skeleton for atypical cortical projections. These candidates were then evaluated by an orthopedic resident and then a fellowship trained pediatric orthopedic surgeon for final inclusion using strict criteria...
2017: Iowa Orthopaedic Journal
https://www.readbyqxmd.com/read/28807769/reduced-intensity-haploidentical-bone-marrow-transplantation-with-post-transplant-cyclophosphamide-for-solid-tumors-in-pediatric-and-young-adult-patients
#15
Nicolas J Llosa, Kenneth R Cooke, Allen Chen, Christopher Gamper, Orly Klein, Elias Zambidis, Brandon Luber, Gary Rosner, Nicholas Siegel, Mary Jo Holuba, Nancy Robey, Masanori Hayashi, Richard J Jones, Ephraim Fuchs, Matthias Holdhoff, David M Loeb, Heather J Symons
High-risk, recurrent, or refractory solid tumors in pediatric, adolescent, and young adult (AYA) patients have an extremely poor prognosis despite current intensive treatment regimens. We piloted an allogeneic bone marrow transplant platform using reduced-intensity conditioning (RIC) and partially HLA-mismatched (haploidentical) related donors for this population of pediatric and AYA solid tumor patients. Sixteen patients received fludarabine, cyclophosphamide, melphalan, and low-dose total body irradiation RIC haploidentical BMT (haploBMT) followed by post-transplantation cyclophosphamide (PTCy), mycophenolate mofetil, and sirolimus...
August 12, 2017: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/28806574/quantitative-diffusion-weighted-magnetic-resonance-imaging-assessment-of-chemotherapy-treatment-response-of-pediatric-osteosarcoma-and-ewing-sarcoma-malignant-bone-tumors
#16
Andrew J Degnan, Chul Y Chung, Amisha J Shah
OBJECTIVE: Assessment of tumor response to chemotherapy is essential in managing malignant pediatric bone tumors prior to resection. SUBJECTS AND METHODS: Pre-chemotherapy and post-chemotherapy osteosarcoma and Ewing sarcoma cases (n=18) were analyzed with apparent diffusion coefficient (ADC) values measured by two readers. RESULTS: Treated tumors demonstrated significantly greater ADC values compared to untreated tumors (p<0.001). Intraclass correlation coefficients ranged between 0...
August 5, 2017: Clinical Imaging
https://www.readbyqxmd.com/read/28803261/advantages-in-prognosis-of-adult-patients-with-ewing-sarcoma-11-years-experiences-and-current-treatment-management
#17
Dagmar Adamkova Krakorova, Katerina Kubackova, Ladislav Dusek, Tomas Tomas, Pavel Janicek, Stepan Tucek, Jana Prausova, Igor Kiss, Iva Zambo
Ewing sarcoma (ES) is an exceptionally rare tumor in adults. Data regarding outcomes of adult patients with ES and experiences with age-adapted therapeutic strategies are very limited. The aim of this study was to evaluate prognostic factors and clinical outcome in a cohort of adult patients treated according to pediatric protocols in the Czech Republic. The records of 58 adult ES patients diagnosed between 2002 and 2013 were reviewed and factors relevant to prognosis and survival were analyzed. The median age of study cohort was 29 years (range, 18-59)...
August 12, 2017: Pathology Oncology Research: POR
https://www.readbyqxmd.com/read/28783147/marrow-ablative-chemotherapy-followed-by-tandem-autologous-hematopoietic-cell-transplantation-in-pediatric-patients-with-malignant-brain-tumors
#18
J A Guerra, G Dhall, A Marachelian, E Castillo, J Malvar, K Wong, R Sposto, J L Finlay
To improve survival in young children with malignant brain tumors, irradiation-avoiding or -minimizing marrow-ablative chemotherapy (HDCx) with autologous hematopoietic cell transplantation (AuHCT) has been investigated. We evaluated the outcome of 44 children with malignant brain tumors treated with HDCx and tandem AuHCT at Children's Hospital Los Angeles between June 1999 and July 2012. Forty-four children with malignant brain tumors were studied. Twenty-one had medulloblastoma/primitive neuro-ectodermal tumor, eight atypical teratoid/rhabdoid tumor (ATRT), five high-grade glioma, four malignant germ cell tumor, three ependymoma and three choroid plexus carcinoma...
August 7, 2017: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/28781793/activation-of-the-arachidonic-acid-metabolic-pathway-and-induction-of-sleep-disturbance-by-sacral-osteoblastoma-a-case-report
#19
Mitsunori Ozaki, Kazuya Nishioka, Akihiko Kimura, Toshikazu Kondo, Naoyuki Nakao
Osteoblastomas are benign bone tumors that produce prostaglandin and promote inflammation. The aim of the present study was to describe the clinical and radiological characteristics of a pediatric osteoblastoma case over an 8-month postoperative follow-up. The case involved an 11-year-old female patient with normal somatic development, presenting with a chief complaint of sleep disturbance. The patient had no spontaneous pain or other readily evident possible causes. Magnetic resonance imaging (MRI) revealed a neoplastic lesion in the sacrum, with peritumoral edema...
August 2017: Molecular and Clinical Oncology
https://www.readbyqxmd.com/read/28780023/shared-acvr1-mutations-in-fop-and-dipg-opportunities-and-challenges-in-extending-biological-and-clinical-implications-across-rare-diseases
#20
Harry J Han, Payal Jain, Adam C Resnick
Gain-of-function mutations in the Type I Bone Morphogenic Protein (BMP) receptor ACVR1 have been identified in two diseases: Fibrodysplasia Ossificans Progressiva (FOP), a rare autosomal dominant disorder characterized by genetically driven heterotopic ossification, and in 20-25% of Diffuse Intrinsic Pontine Gliomas (DIPGs), a pediatric brain tumor with no effective therapies and dismal median survival. While the ACVR1 mutation is causal for FOP, its role in DIPG tumor biology remains under active investigation...
August 2, 2017: Bone
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