International Journal of Epidemiology, Volume 33, Number 1, pp. 15-17
IJE vol.33 no.1 © International Epidemiological Association 2004; all rights reserved.
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Commentary: Mendelian randomization—an update on its use to evaluate allogeneic stem cell transplantation in leukaemia
University of Birmingham Clinical Trials Unit, 1 Somerset Road, Birmingham B15 2RR, UK. E-mail: k.wheatley@bham.ac.uk
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In this issue of the International Journal of Epidemiology a Lancet letter by Martijn Katan is reprinted.1 Katan's letter introduced the notion that genotype–disease associations could be studied as a way of imputing the causal nature of the association between an environmentally influenceable intermediate phenotype and disease. This approach has come to be referred to as ‘Mendelian randomization’.2,3 This term had previously been introduced in a somewhat different context with reference to the evaluation of the efficacy of allogeneic stem cell transplantation (SCT) in leukaemia. In this commentary we elucidate this method and provide an update regarding its implementation.
Randomized controlled trials (RCT) are the gold standard for the evaluation treatment efficacy.4 Randomization to one treatment or another is necessary in order to avoid the selection biases that might be introduced if some patients were electively chosen to receive one treatment and different types of patient another treatment (e.g. young
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