Novel molecular targeted therapies for patients with neurofibromatosis type 1 with inoperable plexiform neurofibromas: a comprehensive review
| العنوان: | Novel molecular targeted therapies for patients with neurofibromatosis type 1 with inoperable plexiform neurofibromas: a comprehensive review |
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| المؤلفون: | J. Feliu, I. Solares, D. Viñal, N. Rodriguez-Salas, M. Morales-Conejo |
| المساهمون: | UAM. Departamento de Medicina |
| المصدر: | Biblos-e Archivo. Repositorio Institucional de la UAM instname ESMO Open |
| بيانات النشر: | Bmj Publishing Group, 2021. |
| سنة النشر: | 2021 |
| مصطلحات موضوعية: | Adult, MAPK/ERK pathway, Oncology, Cancer Research, medicine.medical_specialty, Neurofibromatosis 1, Cabozantinib, molecular targeted therapy, Medicina, neoplasms, Review, neurofibromatosis type 1, chemistry.chemical_compound, Internal medicine, medicine, Humans, Neurofibromatosis, Child, Adverse effect, Protein Kinase Inhibitors, Neurofibroma, Plexiform, Trametinib, business.industry, Genetic disorder, Binimetinib, medicine.disease, Tumor Burden, plexiform neurofibromas, chemistry, Selumetinib, business |
| الوصف: | Neurofibromatosis type 1 (NF1) is a genetic disorder that carries a higher risk of tumor development. Plexiform neurofibromas (PNs) are present in 50% of NF1 and cause significant morbidity when surgery is not feasible. Systemic therapies had not succeeded to reduce PN tumor volume until 2016 when the first trial with an MAPK/extracellular-signal-regulated kinase (MEK) inhibitor was published. We performed a systematic research on novel targeted therapies for patients with NF1 and PNs in PubMed, EMBASE, and conference abstracts with the last update in February 2021. Since 2016, seven trials have reported positive results with MEK inhibitors and other molecular targeted therapies (cabozantinib). Selumetinib has shown an overall response rate of 68% in children with NF1 and symptomatic inoperable PNs, and was associated with pain improvement and a manageable adverse events profile. This led to Food and Drug Administration (FDA) approval of selumetinib in May 2020. Recently, cabozantinib and mirdametinib have also proven their efficacy in adult population. Other MEK inhibitors such as trametinib and binimetinib have also communicated promising preliminary results. Ongoing trials in different populations and with intermittent dosing strategies are underway. Highlights • PNs are a major cause of morbidity in NF1. • Promising efficacy results with MEK inhibitors and cabozantinib have been reported. • MEK inhibitors are associated with a manageable toxicity profile. • Selumetinib is now FDA approved for the treatment of PNs. |
| وصف الملف: | application/pdf |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::53658ed99a30c8448c813dab7572f56bTest http://hdl.handle.net/10486/701320Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....53658ed99a30c8448c813dab7572f56b |
| قاعدة البيانات: | OpenAIRE |
| الوصف غير متاح. |