A5001066329- Neuroblastoma Research and Treatments
- Sarcoma Diagnosis and Treatment
- Cancer, Hypoxia, and Metabolism
- Cancer Genomics and Diagnostics
- Lymphoma Diagnosis and Treatment
- Cancer therapeutics and mechanisms
- Childhood Cancer Survivors' Quality of Life
- CAR-T cell therapy research
- Glioma Diagnosis and Treatment
- Acute Lymphoblastic Leukemia research
- Neuroendocrine Tumor Research Advances
- Adrenal and Paraganglionic Tumors
- Cardiac tumors and thrombi
- Lung Cancer Treatments and Mutations
- Protein Degradation and Inhibitors
- Histone Deacetylase Inhibitors Research
- Chromatin Remodeling and Cancer
- Lung Cancer Research Studies
- Cancer-related Molecular Pathways
- Testicular diseases and treatments
- Pancreatic and Hepatic Oncology Research
- Pharmaceutical studies and practices
- Acute Myeloid Leukemia Research
- Medical Imaging Techniques and Applications
- Renal and related cancers
Dana-Farber Cancer Institute
2016-2025
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
2016-2025
Harvard University
2016-2025
Commissariat à l'Énergie Atomique et aux Énergies Alternatives
2009-2025
CEA Paris-Saclay
2015-2025
Technologies pour la Santé
2021-2025
Université Paris-Saclay
2021-2025
Institut National de Recherche pour l'Agriculture, l'Alimentation et l'Environnement
2021-2025
Boston Children's Hospital
2014-2025
Dana-Farber Brigham Cancer Center
2024
Fusions involving one of three tropomyosin receptor kinases (TRK) occur in diverse cancers children and adults. We evaluated the efficacy safety larotrectinib, a highly selective TRK inhibitor, adults who had tumors with these fusions.We enrolled patients consecutively prospectively identified fusion-positive cancers, detected by molecular profiling as routinely performed at each site, into protocols: phase 1 study adults, 1-2 children, or 2 adolescents The primary end point for combined...
A MESSAGE FROM ASCO’S PRESIDENT I am pleased to present Clinical Cancer Advances 2017, which highlights the most promising advances in patient-oriented cancer research over past year. The report gives us an opportunity reflect on what exciting time it is for and how swiftly our understanding of has improved. One year ago, White House announced national Moonshot program accelerate progress against cancer. This shared vision reinvigorated community, identified new areas scientific...
Purpose Blinatumomab is a bispecific T-cell engager antibody construct targeting CD19 on B-cell lymphoblasts. We evaluated the safety, pharmacokinetics, recommended dosage, and potential for efficacy of blinatumomab in children with relapsed/refractory precursor acute lymphoblastic leukemia (BCP-ALL). Methods This open-label study enrolled < 18 years old BCP-ALL phase I dosage-escalation part II part, using 6-week treatment cycles. Primary end points were maximum-tolerated dosage (phase I)...
Purpose: The goal of this study was to determine the incidence metastatic sites in neuroblastoma and extent which correlate with age, tumor biology, survival. Patients Methods: All 648 patients stage IV IVS registered on Children's Cancer Group protocols 3881 3891 were analyzed. Metastatic site data provided by treating institutions reviewed central nervous system (CNS), intracranial, lung, or "other" metastases. Results: at diagnosis 70.5% bone marrow, 55.7% bone, 30.9% lymph nodes, 29.6%...
Osteosarcoma is a highly aggressive cancer for which treatment has remained essentially unchanged more than 30 years. characterized by widespread and recurrent somatic copy-number alterations (SCNA) structural rearrangements. In contrast, few point mutations in protein-coding genes have been identified, suggesting that within SCNAs are key oncogenic drivers this disease. rearrangements heterogeneous across osteosarcoma cases, the need genome-informed approach to targeted therapy. To identify...
Ewing sarcoma can arise in either bone or soft tissue. The purpose of this study was to investigate whether patient characteristics, treatment strategies, and outcomes differ between skeletal extraskeletal (EES).Patients <40 years age with peripheral primitive neuroectodermal tumor reported the United States Surveillance, Epidemiology, End Results Program database from 1973 2007 were evaluated based on (n = 1519) versus 683) site origin. Patient characteristics compared using Fisher exact...
Purpose Neuroblastoma (NB) is a heterogeneous tumor arising from sympathetic tissues. The impact of primary site in influencing the heterogeneity NB remains unclear. Patients and Methods Children younger than age 21 years diagnosed with or ganglioneuroblastoma between 1990 2002 known were identified International Risk Group database. Data compared sites respect to clinical biologic features, as well event-free survival (EFS) overall (OS). Results Among 8,369 children, 47% had adrenal tumors....
Pediatric cancers represent a unique case with respect to cancer genomics and precision medicine, as the mutation frequency is low, targeted therapies are less available. Consequently, it unknown whether clinical sequencing can be of benefit.To assess feasibility identifying actionable alterations making individualized therapy (iCat) recommendations in pediatric patients extracranial solid tumors.Clinical study at 4 academic medical centers enrolling between September 5, 2012, November 19,...
NUT midline carcinoma, renamed carcinoma (NC), is an aggressive squamous cancer defined by rearrangement of the
Larotrectinib is a first-in-class, highly selective tropomyosin receptor kinase (TRK) inhibitor approved to treat adult and pediatric patients with TRK fusion-positive cancer. The aim of this study was evaluate the efficacy safety larotrectinib in primary central nervous system (CNS) tumors.Patients CNS tumors from two clinical trials (NCT02637687, NCT02576431) were identified. endpoint investigator-assessed objective response rate (ORR).As July 2020, 33 identified (median age: 8.9 years;...
Larotrectinib is a highly selective tropomyosin receptor kinase (TRK) inhibitor with efficacy in children TRK fusion tumors. We evaluated patient outcomes after elective discontinuation of larotrectinib the absence disease progression protocol-defined wait-and-see subset analysis eligible patients where treatment resumption was allowed if progressed. also assessed safety and all pediatric sarcoma. This cohort included 91 (younger than 18 years) from two clinical trials: infantile...
BACKGROUND Patients with Ewing sarcoma require local primary tumor control surgery, radiation, or both. The optimal choice of for overall and disease remains unclear. METHODS localized bone who were treated on 3 consecutive protocols standard‐dose, 5‐drug chemotherapy every weeks included (n=465). Propensity scores used to differences between groups by constructing multivariate models assess the impact type clinical endpoints (event‐free survival [EFS], survival, failure, distant failure)...
Purpose: The cyclin-dependent kinase (CDK) 4/6 inhibitor, ribociclib (LEE011), displayed preclinical activity in neuroblastoma and malignant rhabdoid tumor (MRT) models. In this phase I study, the maximum tolerated dose (MTD) recommended II (RP2D), safety, pharmacokinetics (PK), preliminary of single-agent were investigated pediatric patients with neuroblastoma, MRT, or other cyclin D-CDK4/6-INK4-retinoblastoma pathway-altered tumors.Experimental Design: Patients (aged 1-21 years) received...
Abstract BACKGROUND: Microphthalmia transcription factor (MITF)‐associated (MiT) tumors are a family of rare malignancies, including alveolar soft part sarcoma (ASPS), clear cell (CCS), and translocation‐associated renal carcinoma (tRCC) that have dysregulated expression oncogenic MITF proteins. The MET receptor tyrosine kinase gene is transcriptionally activated by proteins, making potential therapeutic target for MiT tumors. This study assessed the activity tivantinib (ARQ 197), selective...
BACKGROUND The authors describe the incidence and characteristics of secondary malignant neoplasms (SMNs) in adolescent young adult (AYA) cancer survivors compared with those younger older survivors. METHODS Children aged ≤14 years, AYAs 15 to 39, adults ≥40 years at time primary diagnosis who were reported as Surveillance, Epidemiology, End Results (SEER) program between 1973 2011 this population‐based analysis. analysis was risk that an SMN would occur ≥5 after original for patients had...
Purpose Alisertib is an oral Aurora A kinase inhibitor with preclinical activity in neuroblastoma. Irinotecan and temozolomide have patients advanced The goal of this phase I study was to determine the maximum tolerated dose (MTD) alisertib irinotecan population. Patients Methods age 1 30 years relapsed or refractory neuroblastoma were eligible. received tablets at levels 45, 60, 80 mg/m 2 per day on days 7 along 50 intravenously 100 orally 5. Dose escalation followed rolling six design....
The highly selective oral tropomyosin receptor kinase (TRK) inhibitor larotrectinib has demonstrated significant activity in adult and pediatric TRK fusion cancers. In the current study, authors describe clinical course of children with locally advanced sarcoma who were treated preoperatively underwent subsequent surgical resection.A total 24 on a phase 1 trial (ClinicalTrials.gov identifier NCT02637687). Five had documented resection included analysis. Tumor response (Response Evaluation...
Pediatric mesenchymal tumors harboring variant NTRK fusions (ETV6-negative) are being increasingly described; however, the histologic and clinical features of these their relationship to classic infantile fibrosarcoma not well characterized. A better understanding clinicopathologic is necessary, would aid in both early diagnosis treatment. Therefore, aim this study was characterize a series pediatric NTRK-rearranged tumors, including ETV6-NTRK3 fused with (non-ETV6) fusions. The features,...