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Real life data of ONC201 (dordaviprone) in pediatric and adult H3K27-altered recurrent diffuse midline glioma: Results of an international academia-driven compassionate use program Open

Daniela Di Carlo, Maxime Annereau, Marina Vignes, Lucas Denis, Nicolas Epaillard , et al. · 2024

While the efficacy of ONC201 needs validation in a controlled randomized clinical trial, our real-life data support a better outcome for patients with thalamic tumors treated with ONC201. We demonstrated furthermore the feasibility of a su…

Lenvatinib Plus Ifosfamide and Etoposide in Children and Young Adults With Relapsed Osteosarcoma Open

Nathalie Gaspar, Giun-Yi Hung, Sandra J. Strauss, Quentin Campbell‐Hewson, Filemon S. Dela Cruz , et al. · 2024

Importance The combination of ifosfamide and etoposide (IE) is commonly used to treat relapsed or refractory osteosarcoma; however, second-line treatment recommendations vary across guidelines. Objective To evaluate whether the addition of…

Biological Sample Collection to Advance Research and Treatment: A Fight Osteosarcoma Through European Research and Euro Ewing Consortium Statement Open

Darrell Green, Roelof van Ewijk, Elisa Tirtei, Dimosthenis Andreou, Fredrik Baecklund , et al. · 2024

Osteosarcoma and Ewing sarcoma are bone tumors mostly diagnosed in children, adolescents, and young adults. Despite multimodal therapy, morbidity is high and survival rates remain low, especially in the metastatic disease setting. Trials i…

Supplementary Table S4 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S4

Supplementary Table S4 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S4

Supplementary Table S6 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S6

Data from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Infant high-grade gliomas appear clinically distinct from their counterparts in older children, indicating that histopathologic grading may not accurately reflect the biology of these tumors. We have collected 241 cases under 4 years of ag…

Supplementary Data from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Figures and Legends

Supplementary Table S3 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S3

Supplementary Table S2 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S2

Supplementary Table S5 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S5

Supplementary Table S7 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S7

Supplementary Table S1 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S1

Supplementary Data from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Figures and Legends

Supplementary Table S7 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S7

Supplementary Table S5 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S5

Supplementary Table S2 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S2

Supplementary Table S1 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S1

Supplementary Table S6 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S6

Data from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Infant high-grade gliomas appear clinically distinct from their counterparts in older children, indicating that histopathologic grading may not accurately reflect the biology of these tumors. We have collected 241 cases under 4 years of ag…

Supplementary Table S3 from Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2023

Supplementary Table S3

Sodium thiosulfate as cisplatin otoprotectant in children: The challenge of when to use it Open

Penelope Brock, Annelot J. M. Meijer, Per Kogner, Marc Ansari, Michael Capra , et al. · 2023

Cisplatin is an essential chemotherapeutic agent for children treated with hepatic tumors, neuroblastoma, medulloblastoma, germ cell tumors, and osteosarcoma. Unfortunately, cisplatin-induced hearing loss (CIHL) occurs in about 50% of trea…

The Global Retinoblastoma Outcome Study: a prospective, cluster-based analysis of 4064 patients from 149 countries Open

Ido Didi Fabian, Abdallah Elhassan, Shehu U. Abdullahi, Rula Ahmed Abdulqader, Aminatu A Abdulrahaman , et al. · 2022

Efficacy and safety of larotrectinib in TRK fusion-positive primary central nervous system tumors Open

François Doz, Cornelis M. van Tilburg, Birgit Geoerger, Martin Højgaard, Ingrid Øra , et al. · 2021

Background 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 to evaluate the efficacy a…

CTNI-58. EFFICACY AND SAFETY OF LAROTRECTINIB IN ADULT AND PEDIATRIC PATIENTS WITH TROPOMYOSIN RECEPTOR KINASE (TRK) FUSION-POSITIVE PRIMARY CENTRAL NERVOUS SYSTEM (CNS) TUMORS Open

François Doz, Cornelis M. van Tilburg, Birgit Geoerger, Karsten Nysom, Ingrid Øra , et al. · 2021

BACKGROUND NTRK gene fusions are oncogenic drivers in various CNS and non-CNS tumors. Larotrectinib is a first-in-class, highly selective TRK inhibitor approved for patients with TRK fusion cancer, with a 75% objective response rate (ORR) …

RARE-07. EFFICACY AND SAFETY OF LAROTRECTINIB IN PEDIATRIC PATIENTS WITH TROPOMYOSIN RECEPTOR KINASE (TRK) FUSION-POSITIVE PRIMARY CENTRAL NERVOUS SYSTEM (CNS) TUMORS Open

Sébastien Perreault, François Doz, Birgit Geoerger, Karsten Nysom, Ingrid Øra , et al. · 2021

Background NTRK gene fusions are oncogenic drivers in various CNS and non-CNS tumors. Larotrectinib is a highly selective TRK inhibitor approved to treat patients with TRK fusion cancer, with an objective response rate (ORR) of 78% across …

Incidence of Retinoblastoma Has Increased: Results from 40 European Countries Open

Andrew W. Stacey, Richard Bowman, Allen Foster, Tero Kivelä, Francis L. Munier , et al. · 2021

EPCT-08. ACTIVITY OF LAROTRECTINIB IN PEDIATRIC TROPOMYOSIN RECEPTOR KINASE (TRK) FUSION CANCER PATIENTS WITH PRIMARY CENTRAL NERVOUS SYSTEM (CNS) TUMORS Open

Hiroaki Goto, Birgit Geoerger, Steven G. DuBois, Juneko E. Grilley‐Olson, Cornelis M. van Tilburg , et al. · 2020

BACKGROUND TRK fusions are oncogenic drivers in a variety of tumors, many involving the CNS. Larotrectinib, a selective FDA- and EMA-approved TRK inhibitor, demonstrated a 79% objective response rate (ORR) and a 35.2-month median duration …

Travel burden and clinical presentation of retinoblastoma: analysis of 1024 patients from 43 African countries and 518 patients from 40 European countries Open

Ido Didi Fabian, Andrew W. Stacey, Allen Foster, Tero Kivelä, Francis L. Munier , et al. · 2020

Background The travel distance from home to a treatment centre, which may impact the stage at diagnosis, has not been investigated for retinoblastoma, the most common childhood eye cancer. We aimed to investigate the travel burden and its …

Infant High-Grade Gliomas Comprise Multiple Subgroups Characterized by Novel Targetable Gene Fusions and Favorable Outcomes Open

Matthew Clarke, Alan Mackay, Britta Ismer, Jessica C. Pickles, Ruth Tatevossian , et al. · 2020

Infant high-grade gliomas appear clinically distinct from their counterparts in older children, indicating that histopathologic grading may not accurately reflect the biology of these tumors. We have collected 241 cases under 4 years of ag…

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