Analysis of recurrent events: application to a clinical trial of colony stimulating factor with the endpoint of febrile neutropenia
✍ Scribed by Shigeyuki Matsui; Yasuo Ohashi
- Publisher
- John Wiley and Sons
- Year
- 1999
- Tongue
- English
- Weight
- 109 KB
- Volume
- 18
- Category
- Article
- ISSN
- 0277-6715
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✦ Synopsis
This paper discusses the analysis of randomized clinical trials where the primary endpoint is a recurrent event observed during a time period which can vary among patients. In particular it describes a method of estimating robust con"dence limits for clinically relevant parameters. As an illustrative example, a clinical trial of a macrophage colony stimulating factor (M-CSF) for patients with acute myeloblastic leukaemia (AML) is introduced where the endpoint is the incidence and duration of febrile neutropenia. The causal e!ects in the framework of Rubin's deterministic model are de"ned as estimands. A non-parametric method based on the permutation test, using an accelerated search procedure proposed by Garthwaite, is compared with other standard methods by simulation and by application to the AML trial data.