دورية أكاديمية

Neoadjuvant B-RAF and MEK Inhibitor Targeted Therapy for Adult Papillary Craniopharyngiomas: A New Treatment Paradigm

التفاصيل البيبلوغرافية
العنوان: Neoadjuvant B-RAF and MEK Inhibitor Targeted Therapy for Adult Papillary Craniopharyngiomas: A New Treatment Paradigm
المؤلفون: Calvanese, Francesco, Jacquesson, Timothée, Manet, Romain, Vasiljevic, Alexandre, Lasolle, Hélène, Ducray, Francois, Raverot, Gerald, Jouanneau, Emmanuel
المصدر: Frontiers in Endocrinology ; volume 13 ; ISSN 1664-2392
بيانات النشر: Frontiers Media SA
سنة النشر: 2022
المجموعة: Frontiers (Publisher - via CrossRef)
الوصف: Background Surgical and clinical management of craniopharyngiomas is associated with high long-term morbidity especially in the case of hypothalamic involvement. Improvements in knowledge of craniopharyngioma molecular biology may offer the possibility of safe and effective medical neoadjuvant treatments in a subset of patients harboring papillary subtype tumors with a BRAFV600E mutation. Method We report herein two cases of tubero-infundibular and ventricular Papillary Craniopharyngiomas in which BRAF/MEK inhibitor combined therapy was used as adjuvant (Case 1) or neoadjuvant (Case 2) treatment, with a 90% reduction in tumor volume observed after only 5 months. In Case 2 the only surgical procedure used was a minimal invasive biopsy by the trans-ventricular neuroendoscopic approach. As a consequence, targeted therapy was administered in purely neoadjuvant fashion. After shrinkage of the tumor, both patients underwent fractionated radiotherapy on the small tumor remnant to achieve long-term tumor control. A review of a previously reported case has also been performed. Result This approach led to tumor control with minimal long-term morbidity in both cases. No side effects or complications were reported after medical treatment and adjuvant radiotherapy. Conclusion Our experience and a review of the literature argue for a change in the current treatment paradigm for Craniopharyngiomas (CPs). In giant and invasive tumors, confirmation of BRAFV600E mutated PCPs by biopsy and BRAF/MEK inhibitor therapy before proposing other treatments may be useful to improve long term outcomes for patients.
نوع الوثيقة: article in journal/newspaper
اللغة: unknown
DOI: 10.3389/fendo.2022.882381
DOI: 10.3389/fendo.2022.882381/full
الإتاحة: https://doi.org/10.3389/fendo.2022.882381Test
حقوق: https://creativecommons.org/licenses/by/4.0Test/
رقم الانضمام: edsbas.C9AB41AD
قاعدة البيانات: BASE