MEK inhibitors are an emerging therapy with increasing use in mitogen-activated protein kinase-driven central nervous system (CNS) tumors. There is limited data regarding efficacy and toxicity in pediatric patients. We report our clinical experience with trametinib-based therapy for the treatment of 14 consecutive pediatric patients with recurrent low-grade glioma (N = 11) or highgrade CNS tumors (N = 3) with MAP kinase pathway mutations. Patients received trametinib as monotherapy (N = 9) or in combination (N = 5) with another antineoplastic agent. Nine patients (64%) were progression free during treatment. Five patients showed a partial response, while 4 had stable disease. Two patients (14%) progressed on therapy. All partial responses were in patients with low-grade tumors. The remaining 3 patients were not evaluable due to toxicity limiting duration of therapy. Two of 3 patients with lowgrade glioma with leptomeningeal dissemination showed radiographic treatment response. Five patients reported improved clinical symptoms while on trametinib. Adverse events on trametinib-based therapy included dermatologic, mouth sores, fever, gastrointestinal, infection, neutropenia, headache, and fatigue, and were more common in patients using combination therapy. Trametinib-based therapy demonstrated signals of efficacy in our single institutional cohort of pediatric patients with mitogen-activated protein kinasedriven CNS tumors. Our observations need to be confirmed in a clinical trial setting.
MET PET image fusion may facilitate the targeting of anaplastic foci in homogeneous MRI non-enhancing gliomas for biopsy, may identify oligodendroglial histology preoperatively as well as characterize biologically active tumor volumes within MRI T(1)/FLAIR tumor areas of candidate patients for resection.
The use of Outdoor Behavioral Healthcare (OBH) as a viable treatment modality for adolescents with behavioral, emotional, and substance use issues has been gaining increased attention. This research builds upon the literature by utilizing a longitudinal study to explore clinical changes, measured using the Youth Outcome Questionnaire (YOQ), and changes in family functioning as measured by the general functioning scale of the Family Assessment Device (FAD). Both clinically and statistically significant positive results with youth, mothers, and fathers at points of intake, discharge, and six months post discharge were found; however, parent and youth reports differed especially at six months post discharge. In addition, regression analyses showed that mothers and youth were more aligned than fathers in their perceptions of changes in family functioning post OBH treatment. This research fills a gap in the behavioral healthcare literature concerning the outcomes of using wilderness therapy and their association with family involvement in maintaining clinical change and improved family functioning.
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