TRAM-01: A phase 2 study of trametinib for pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Sadat Kiaei Dorsa, Caru Maxime, Larouche Valérie, Clini Emma, Pugalenthi Saravanan Deepika, Lippé Sarah, Théoret Yves, Décarie Jean-Claude, Tabori Uri, Hawkins Cynthia, Ellezam Benjamin, Ospina Luis H, Desjardins Léandra, Métras Marie-Élaine, Sultan Serge, Cantin Édith, Routhier Marie-Ève, Mailloux Chantal, Bertrand Marie-Claude, Estephan Karim, McKeown Tara, Vairy Stéphanie, Legault Geneviève, Renzi Samuele, Bouffet Éric, Ramaswamy Vijay, Coltin Hallie, Lafay-Cousin Lucie, Hukin Juliette, Alves Mélanie, Rent Tiffany, Erker Craig, Jabado Nada, Dehaes Mathieu, Perreault Sébastien
Аннотация
METHODS: Patients with NF1 and inoperable PNs aged ≥1 month to ≤25 years when starting trametinib were eligible. Patients received trametinib once daily orally continuously for a maximum of 18 cycles of 28 days. Patients completed patient-reported outcome measures. Response evaluations were performed after cycle 3, 6, 12 and at the end of treatment and then every 6 months. The PN volumes were centrally quantified using a semi-automatic 3D segmentation method.
RESULTS: Forty-six patients were recruited. The median age was 11.1 years (range 0.7-19.8). The median baseline tumor volume was 104.6 ml (range 16.3 to 650.1 ml). Volumetric assessment demonstrated an overall response rate (volume decrease of ≥20%) in 47.8% (22/46) of patients. Median volume change was -19.3% (range -80.5 to 3.5). Forty-one patients (89.1%) completed the planned treatment. The 3 years progression free survival and the event free survival were respectively 73.1% (95% CI 53.3%-85.5%) and 47.1% (95% CI 28.8-63.5).
CONCLUSIONS: We report outcomes and volumetric response of PNs treated with trametinib within a phase 2 trial. Based on these results, trametinib is effective and overall well tolerated.