Academic Journal

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: Francesco Calvanese, Timothée Jacquesson, Romain Manet, Alexandre Vasiljevic, Hélène Lasolle, Francois Ducray, Gerald Raverot, Emmanuel Jouanneau
المصدر: Frontiers in Endocrinology, Vol 13 (2022)
بيانات النشر: Frontiers Media S.A., 2022.
سنة النشر: 2022
المجموعة: LCC:Diseases of the endocrine glands. Clinical endocrinology
مصطلحات موضوعية: papillary craniopharyngiomas, tumor biopsy, V600E BRAF mutation, B-RAF and MEK inhibitor targeted therapy, neoadjuvant treatment, Diseases of the endocrine glands. Clinical endocrinology, RC648-665
الوصف: BackgroundSurgical 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.MethodWe 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.ResultThis 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.ConclusionOur 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
وصف الملف: electronic resource
اللغة: English
تدمد: 1664-2392
Relation: https://www.frontiersin.org/articles/10.3389/fendo.2022.882381/full; https://doaj.org/toc/1664-2392
DOI: 10.3389/fendo.2022.882381
URL الوصول: https://doaj.org/article/3ad4eae4f70d408e9f2c298d6dc36ee7
رقم الانضمام: edsdoj.3ad4eae4f70d408e9f2c298d6dc36ee7
قاعدة البيانات: Directory of Open Access Journals
الوصف
تدمد:16642392
DOI:10.3389/fendo.2022.882381