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termsTerm

Dose-limiting toxicity (DLT)

aka DLT, DLTs, dose-limiting, dose limiting toxicity, dose-limiting toxicities, DLT period, DLT window, no DLTs, DLT rate

A side effect in an early trial severe enough (usually grade 3 or worse, or forcing a long treatment pause) that the dose cannot safely go higher. Counting DLTs at each dose level is how phase 1 trials find the maximum tolerated dose.

Protocols define DLTs in advance (typically grade ≥3 non-haematological toxicity, prolonged grade 4 neutropenia, febrile neutropenia, or inability to deliver a set fraction of planned dose) and observe them over a fixed window, usually the first cycle. If a pre-set proportion of patients at a dose (2 of 6 in a 3+3 design) have DLTs, that dose exceeds tolerance. The one-cycle window misses cumulative and late toxicities (neuropathy, ILD, ocular effects), a recognised limitation for continuously dosed targeted drugs and ADCs that Project Optimus and time-to-event designs aim to correct.

Category
Trials & regulation

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