ECE2024 Oral Communications Oral Communications 5: Pituitary and Neuroendocrinology | Part I (6 abstracts)
B-RAF and MEK inhibitor argeted therapy in papillary craniopharyngiomas: results from the French national multicenter study
Dario De Alcubierre 1 , Grigorios Gkasdaris 2 , Claire Briet 3 , Fabien Almairac 4 , Julien Boetto 5 , Celine Mouly 6 , Delphine Larrieu-Ciron 7 , Anthony Joncour 8 , Margaux Mordrel 9 , Alexandre Vasiljevic 10 , Chiara Villa 11 , Camille Sergeant 12 , Francois Ducray 13 , Loic Feuvret 14 , Bertrand Baussart 15 , Gerald Raverot 2 & Emmanuel Jouanneau 2
1Sapienza University of Rome, Department of Experimental Medicine, Rome, Italy; 2Groupement Hospitalier Est, Hospices Civils de Lyon, Department of Neurosurgery, Bron, France; 3CHU d’Angers, Angers, France; 4Hôpital Pasteur II, University Hospital of Nice, Nice, France; 5Gui de Chauliac Hospital, Montpellier University Medical Center, Montpellier, France; 6CHU Toulouse, Toulouse, France; 7Institut Universitaire du Cancer Toulouse Oncopole, Institut Claudius Re-gaud, Department of Medical Oncology, Toulpouse, France; 8Poitiers University Hospital, Oncology Department, Poitiers, France; 9Université de Poitiers, CHU de Poitiers, ProDiCeT, Poitiers, France; 10Centre de Pathologie Est, Groupement Hospitalier Est, Hospices Civils de Lyon, Bron, France; 11Hôpital Universitaire Pitié-Salpêtrière, APHP, Sorbonne Université, Department of Neuropathology, Paris, France; 12Groupement Hospitalier Est, Hospices Civils de Lyon, Endocrinology Department, Bron, France; 13Groupement Hospitalier Est, Hospices Civils de Lyon, Department of Neuro-Oncology, Bron, France; 14Groupement Hospitalier Est, Hospices Civils de Lyon, Department of Radiation Oncology, Bron, France; 15La Pitié-Salpêtrière Hospital, AP-HP, Sorbonne University, Department of Neurosurgery, Paris, France
Background: Papillary craniopharyngiomas (PCPs) are driven by V600E BRAF mutations in 95% of cases. Recently, combined anti-BRAF/MEK targeted therapy (TT) has emerged as a potential treatment in aggressive PCPs. However, standardized data on large cohorts are still lacking. Our study aimed to assess the real-life efficacy and safety of TT in patients with PCPs.
Methods: This was a retrospective national multicenter study involving patients with V600E BRAF-mutated PCPs treated with anti-BRAF/MEK TT in France up to July 2023. Volumetric magnetic resonance imaging analysis, clinical and hormonal assessments were performed before TT, after 3 months, and at the last available follow-up during treatment. Radiological response was classified as either complete (lesion disappearance), subtotal (volume reduction >80%), partial (volume decrease 30-80%), stable disease (volume decrease <30% or increase <20%), or progressive disease (volume increase >20%).
Results: Sixteen patients (8 females, mean age 50.5±15.75 years) were included. Patients received either neoadjuvant therapy (NEO) for non-resectable tumors (n=6), adjuvant therapy (ADJ) post-surgery (n=8), or palliative therapy (PAL) after multimodal treatment (n=2).Before TT, symptoms included headache (5 patients), endocrine dysfunction (14), visual impairment (9), weight gain (3), and cognitive dysfunction (2). Mean tumor volume was 7429±2483 mm3. After 3 months of TT, mean volume reduction was 82.2±16.9%, 52.9±21.9%, and 60.8±17.3% [43.5-78.1] in the NEO, ADJ, and PAL groups, respectively. At the last follow-up (mean 7.6±5.3 months), 12 patients (6 NEO, 4 ADJ, 2 PAL) showed subtotal response, 3 ADJ patients exhibited partial response, and one ADJ patient maintained stable disease. Mean volume reduction was 88.9±4.4%, 73.3±23.4%, and 91.8±4.3% in the NEO, ADJ, and PAL groups, respectively.Clinically, TT resolved headaches in all patients and visual impairment in 6. Two patients had improved neurological symptoms, one presented weight loss, and two recovered endocrine function. Five patients stopped treatment due to adverse events. Two developed hepatic cytolysis, leading to temporary TT discontinuation and later reintroduction. One patient permanently discontinued TT due to peripheral oedema, another due to febrile pneumopathy, and the remaining one due to vomiting, skin rash, urinary tract infections, and hyperthermia.
Conclusions: Targeted therapy in PCP patients can induce impressive tumor volume reduction and clinical improvement within a few months from treatment initiation. Adverse events warrant careful monitoring. Further research is needed to establish standardized protocols, but present results advocate for reframing usual strategies.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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