scholarly journals The European Paediatric Rare Tumours Network ‐ European Registry (PARTNER) project for very rare tumors in children

2021 ◽  
Author(s):  
Daniel Orbach ◽  
Andrea Ferrari ◽  
Dominik T. Schneider ◽  
Yves Reguerre ◽  
Jan Godzinski ◽  
...  
Keyword(s):  
Rare Tumors ◽  
European Paediatric ◽  
Rare Tumours ◽  
European Registry

scholarly journals Pancreatoblastoma in children: EXPeRT/PARTNER diagnostic and therapeutic recommendations.

2021 ◽  
Author(s):  
Ewa Bien ◽  
Jelena Roganovic ◽  
MALGORZATA Krawczyk ◽  
Jan Godziński ◽  
Daniel Orbach ◽  
...  
Keyword(s):  
Histopathological Examination ◽  
Elevated Serum ◽  
Abdominal Tumor ◽  
Cooperative Study Group ◽  
Rare Tumors ◽  
Complete Surgical Resection ◽  
Therapeutic Recommendations ◽  
Neo Adjuvant Chemotherapy ◽  
Upper Abdominal ◽  
European Registry

Pancreatoblastoma (PBL) is a rare malignant epithelial neoplasm affecting typically young children. Signs related to advanced upper-abdominal tumor accompanied by elevated serum α-fetoprotein levels in a young child suggest PBL, however histopathological examination is required for diagnosis. The mainstay of treatment is a complete surgical resection. Inoperable and/or metastatic PBL may become amenable to complete, delayed surgery after neo-adjuvant chemotherapy. This manuscript presents the internationally consensus recommendations for the diagnosis and treatment of children with PBL, established by the European Cooperative Study Group for Pediatric Rare Tumors (EXPeRT) within the EU-funded PARTNER (Paediatric Rare Tumors Network – European Registry) project.

European paediatric patients with neuromyelitis optica: a good prognosis for most and seronegative to NMO-IgG

2010 ◽  
Vol 41 (02) ◽  
Author(s):  
P Huppke ◽  
M Blüthner ◽  
O Bauer ◽  
W Stark ◽  
K Reinhardt ◽  
...  
Keyword(s):  
Neuromyelitis Optica ◽  
Good Prognosis ◽  
European Paediatric ◽  
Paediatric Patients

Inhalte und Ausbildung nach den Internationalen Leitlinien 2000 mit dem »European Paediatric Life Support-Kurs (EPLS)«

2005 ◽  
Vol 05 (01) ◽  
pp. 16-20
Author(s):  
Burkhard Wermter ◽  
Armin Wessel ◽  
Michael Sasse
Keyword(s):  
Life Support ◽  
Medizinisches Personal ◽  
European Paediatric

ZusammenfassungDie Reanimation im Kindesalter ist ein seltenes Ereignis, das medizinisches Personal oft unvorbereitet trifft und mit großer emotionaler Belastung einhergeht. Im Gegensatz zur Rettungsmedizin bei Erwachsenen befindet sich die Standardisierung von Handlungsweisen bei der Wiederbelebung von Kindern noch im Aufbau. Studien belegen, dass eine vereinheitlichte Ausbildung mit Algorithmen in der Kinderreanimation, die Letalität von Notfällen im Kindesalter um mehr als 30% (8, 18, 24) senken kann. Mit dem European Paediatric Life Support-Kurs steht ein Ausbildungsmodell zur Verfügung, das mit nachgewiesenem Erfolg zu einer flächendeckenden Verbreitung von Standards in der Kinderreanimation beitragen kann.

OVERSTITCH SX ENDOSCOPIC SUTURING SYSTEM FOR GASTROINTESTINAL APPLICATIONS: A MULTICENTER EUROPEAN REGISTRY

2020 ◽  
Author(s):  
R Maselli ◽  
R Palma ◽  
M Traina ◽  
A Granata ◽  
D Juzgado ◽  
...  
Keyword(s):  
Endoscopic Suturing ◽  
European Registry

Proteomics of Pancreatic Neuroendocrine Tumors: A Systematic Review

2020 ◽  
Vol 27 (12) ◽  
pp. 1276-1287
Author(s):  
Brigida Anna Maiorano ◽  
Giovanni Schinzari ◽  
Sabrina Chiloiro ◽  
Felicia Visconti ◽  
Domenico Milardi ◽  
...  
Keyword(s):  
Systematic Review ◽  
Neuroendocrine Tumors ◽  
Prognostic Markers ◽  
State Of The Art ◽  
Therapeutic Targets ◽  
The State ◽  
Pancreatic Neuroendocrine Tumors ◽  
Rare Tumors ◽  
Personalized Approach ◽  
Future Management

Pancreatic neuroendocrine tumors (PanNETs) are rare tumors having usually an indolent behavior, but sometimes with unpredictable aggressiveness. PanNETs are more often non-functioning (NF), unable to produce functioning hormones, while 10-30% present as functioning (F) - PanNETs, such as insulinomas , gastrinomas , and other rare tumors. Diagnostic and prognostic markers, but also new therapeutic targets, are still lacking. Proteomics techniques represent therefore promising approaches for the future management of PanNETs. We conducted a systematic review to summarize the state of the art of proteomics in PanNETs. A total of 9 studies were included, focusing both on NF- and F-PanNETs. Indeed, proteomics is useful for the diagnosis, the prognosis and the detection of therapeutic targets. However, further studies are required. It is also warranted to standardize the analysis methods and the collection techniques, in order to validate proteins with a relevance in the personalized approach to PanNETs management.

scholarly journals Platform study of genotyping-guided precision medicine for rare solid tumours: a study protocol for a phase II, non-randomised, 18-month, open-label, multiarm, single-centre clinical trial testing the safety and efficacy of multiple Chinese-approved targeted drugs and PD-1 inhibitors in the treatment of metastatic rare tumours

BMJ Open ◽  
2021 ◽  
Vol 11 (6) ◽  
pp. e044543
Author(s):  
Shuhang Wang ◽  
Hui-Yao Huang ◽  
Dawei Wu ◽  
Hong Fang ◽  
Jianming Ying ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Targeted Therapy ◽  
Single Agent ◽  
Solid Tumours ◽  
Targeted Drugs ◽  
Gene Fusions ◽  
Single Centre ◽  
Open Label ◽  
Safety And Efficacy ◽  
Rare Tumours

IntroductionLimited clinical studies have been conducted on rare solid tumours, and there are few guidelines on the diagnosis and treatment, including experiences with targeted therapy and immunotherapy, of rare solid tumours in China, resulting in limited treatment options and poor outcomes. This study first proposes a definition of rare tumours and is designed to test the preliminary efficacy of targeted and immunotherapy drugs for the treatment of rare tumours.Methods and analysisThis is a phase II, open-label, non-randomised, multiarm, single-centre clinical trial in patients with advanced rare solid tumours who failed standard treatment; the study aims to evaluate the safety and efficacy of targeted drugs in patients with advanced rare solid tumours with corresponding actionable alterations, as well as the safety and efficacy of immune checkpoint (programmed death receptor inhibitor 1, PD-1) inhibitors in patients with advanced rare solid tumours without actionable alterations. Patients with advanced rare tumours who fail standardised treatment and carry actionable alterations (Epidermal growth factor receptor (EGFR) mutations, ALK gene fusions, ROS-1 gene fusions, C-MET gene amplifications/mutations, BRAF mutations, CDKN2A mutations, BRCA1/2 mutations, HER-2 mutations/overexpressions/amplifications or C-KIT mutations) will be enrolled in the targeted therapy arm and be given the corresponding targeted drugs. Patients without actionable alterations will be enrolled in the PD-1 inhibitor arm and be treated with sintilimab. After the patients treated with vemurafenib, niraparib and palbociclib acquire resistance, they will receive combination treatment with sintilimab or atezolizumab. With the use of Simon’s two-stage Minimax design, and the sample size was estimated to be 770. The primary endpoint of this study is the objective response rate. The secondary endpoints are progression-free survival in the targeted treatment group and single-agent immunotherapy group; the duration of response in the targeted therapy and single-agent immunotherapy groups; durable clinical benefit in the single-agent immunotherapy group; and the incidence of adverse events.Ethics and disseminationEthics approval was obtained from the Chinese Academy of Medical Sciences (ID: 20/132-2328). The results from this study will be actively disseminated through manuscript publications and conference presentations.Trial registration numbersNCT04423185; ChiCTR2000039310.

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