On the analysis of tuberculosis studies with intermittent missing sputum data. (English) Zbl 1397.62471

Scharfstein, Daniel; Rotnitzky, Andrea; Abraham, Maria; McDermott, Aidan; Chaisson, Richard; Geiter, Lawrence

Ann. Appl. Stat. 9, No. 4, 2215-2236 (2015).

Summary: In randomized studies evaluating treatments for tuberculosis (TB), individuals are scheduled to be routinely evaluated for the presence of TB using sputum cultures. One important endpoint in such studies is the time of culture conversion, the first visit at which a patient’s sputum culture is negative and remains negative. This article addresses how to draw inference about treatment effects when sputum cultures are intermittently missing on some patients. We discuss inference under a novel benchmark assumption and under a class of assumptions indexed by a treatment-specific sensitivity parameter that quantify departures from the benchmark assumption. We motivate and illustrate our approach using data from a randomized trial comparing the effectiveness of two treatments for adult TB patients in Brazil.

MSC:

62P10

Applications of statistics to biology and medical sciences; meta analysis

Keywords:

culture conversion; curse of dimensionality; exponential tilting; reverse-time hazard; sensitivity analysis

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