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Azathioprine in the 21st Century

Anja K Weidmann, MD, and Helen S Young, MD

Azathioprine was originally developed >50 years ago for use in solid organ transplantation and for the treatment of hematological malignancies. In clinical practice today, it is used in a variety of specialties and for a host of indications. It is available as a generic preparation at a relatively low cost. Importantly, an understanding of the metabolism of the drug via thiopurine S-methyltransferase (TPMT) has provided one of the first and most practical examples of pharmacogenomics. In conjunction with TPMT testing, the availability of techniques for monitoring active metabolites of azathioprine such as 6-thioguanine nucleotide (6-TGN) show promise for individualizing dosing, reducing the risk of side-effects, monitoring compliance, and predicting clinical response.

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