Chapter 53

Treatment Interruption

Birgit Grund

53.1 Introduction

Interruption of active drug treatment in clinical trials can be classified broadly as therapeutic treatment interruption (TI), in which the potential therapeutic benefit of supervised interruption of treatment is investigated, and analytic TI, in which participants are randomized to stopping versus continuing active treatment and the TI arm serves as control group. Design issues for therapeutic TI trials are discussed in the example of a trial in HIV/AIDS.

Clinical trials that include interruption of active drug treatment have been conducted in many fields and have addressed a wide range of research questions. The spectrum ranges from therapeutic treatment interruption (TI), which tests the hypothesis that supervised interruption of treatment may provide a net benefit to the individual patient compared with continuous treatment, to analytic TI, in which the TI arm serves as control arm to evaluate an active drug. This article is restricted to randomized, controlled trials; it includes examples from HIV research, treatment of osteoporosis and hypertension, and a Phase II cancer trial.

53.2 Therapeutic TI Studies in HIV/AIDS

53.2.1 Overview

No cure for chronic HIV infection currently exists, but highly active antiretroviral therapy (ART) has substantially reduced morbidity and mortality caused by HIV and AIDS. However, regimens are often complex and burdensome to patients; drugs may have toxic side effects, including ...

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