Primary malignant brain tumours (PMBT) in phase I studies: Barriers to treatment and patient outcomes.

2024 Background: Primary malignant brain tumours (PMBT) are a rare tumour group with a poor outcome and limited options at relapse. Despite recent incremental gains from multimodal therapy and an emerging understanding of underlying genetic alterations, patients (pts) with PMBT have traditionally been excluded from phase I trials due to a number of concerns including blood-brain barrier penetration; it is recognised that such concerns are less relevant in the molecular era. We examined the outcomes of and barriers to participation of these pts on phase I studies. Methods: We reviewed the case records of pts with PMBT referred to the Neuro Oncology and Clinical Pharmacology Units of the Royal Marsden Hospital between 2004 and 2013 for consideration of a phase I study. Pt characteristics including demographics and details of prior treatment were examined. Kaplan-Meier estimator and Cox regression were used to evaluate progression free survival (PFS) and overall survival (OS). All tests performed were two-si...