The New England journal of medicine, 2020-04, Vol.382 (15), p.1430-1442
- Gross, Andrea M
- Wolters, Pamela L
- Dombi, Eva
- Baldwin, Andrea
- Whitcomb, Patricia
- Fisher, Michael J
- Weiss, Brian
- Kim, AeRang
- Bornhorst, Miriam
- Shah, Amish C
- Martin, Staci
- Roderick, Marie C
- Pichard, Dominique C
- Carbonell, Amanda
- Paul, Scott M
- Therrien, Janet
- Kapustina, Oxana
- Heisey, Kara
- Clapp, D. Wade
- Zhang, Chi
- Peer, Cody J
- Figg, William D
- Smith, Malcolm
- Glod, John
- Blakeley, Jaishri O
- Steinberg, Seth M
- Venzon, David J
- Doyle, L. Austin
- Widemann, Brigitte C
2020
Details
- Autor(en) / Beteiligte
- Gross, Andrea M
- Wolters, Pamela L
- Dombi, Eva
- Baldwin, Andrea
- Whitcomb, Patricia
- Fisher, Michael J
- Weiss, Brian
- Kim, AeRang
- Bornhorst, Miriam
- Shah, Amish C
- Martin, Staci
- Roderick, Marie C
- Pichard, Dominique C
- Carbonell, Amanda
- Paul, Scott M
- Therrien, Janet
- Kapustina, Oxana
- Heisey, Kara
- Clapp, D. Wade
- Zhang, Chi
- Peer, Cody J
- Figg, William D
- Smith, Malcolm
- Glod, John
- Blakeley, Jaishri O
- Steinberg, Seth M
- Venzon, David J
- Doyle, L. Austin
- Widemann, Brigitte C
- Titel
- Selumetinib in Children with Inoperable Plexiform Neurofibromas
- Ist Teil von
- The New England journal of medicine, 2020-04, Vol.382 (15), p.1430-1442
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2020
- Link zum Volltext
- Quelle
- Free E-Journal (出版社公開部分のみ）
- Beschreibungen/Notizen
- Neurofibromatosis involves activation of the RAS pathway. Inhibition of MEK, a component of the pathway, with selumetinib was performed in 50 children with inoperable disease. A total of 70% had a response, which was maintained in the majority for more than a year. Pain relief, improved function, and higher quality of life were also observed.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793eISSN: 1533-4406DOI: 10.1056/NEJMoa1912735Titel-ID: cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_7305659
- Format
- –
- Schlagworte
- Adolescent, Benzimidazoles - adverse effects, Benzimidazoles - therapeutic use, Cancer, Child, Child, Preschool, Children, Creatine, Creatine kinase, Deformities, Diarrhea, Diseases, Dosage, Drug therapy, Female, Genetic disorders, Humans, Kinases, Magnetic resonance imaging, Male, Medical research, Mitogen-Activated Protein Kinase Kinases - antagonists & inhibitors, Nausea, Nausea - chemically induced, Neurofibroma, Neurofibroma, Plexiform - complications, Neurofibroma, Plexiform - drug therapy, Neurofibroma, Plexiform - pathology, Neurofibromatosis, Neurofibromatosis 1 - complications, Neurofibromatosis 1 - drug therapy, Neurofibromatosis 1 - pathology, Neurological disorders, Pain, Pain - etiology, Patient outcomes, Patient Reported Outcome Measures, Patients, Pediatrics, Progression-Free Survival, Protein Kinase Inhibitors - adverse effects, Protein Kinase Inhibitors - therapeutic use, Quality of life, R&D, Recklinghausen's disease, Research & development, Response rates, Tumor Burden - drug effects, Tumors, Volumetric analysis, Vomiting