We focus on pharmacokinetic and pharmacodynamic properties of azathioprine, and on the efficacy and safety of the drug in multiple sclerosis patients. Azathioprine (AZA) is an immunosuppressive drug widely prescribed for the treatment of multiple sclerosis until the advent of interferon beta and glatiramer acetate. Data on its efficacy derive from old and often small clinical trials. The available results suggest that AZA reduces relapse rate, with a slight effect on disability progression. More recently, small studies detected a significant reduction in enhancing MRI lesions in patients treated with AZA. Several trials have suggested that the drug might be effective, at least in the short term, as an add-on treatment in patients breaking through monotherapy with conventional first-line drugs. Concerns about safety, mainly a possible increased risk of malignancy, have been raised. A review of the literature suggests that the safety profile of AZA is a cceptable, and that the risk of cancer does not significantly increase provided the cumulative dose is less than 300 g. Further large and well-conducted studies are needed to confirm the efficacy of the drug in monotherapy or in combination.
Azathioprine therapy for Multiple Sclerosis
CASETTA, Ilaria;GRANIERI, Enrico Gavino Giuseppe
2012
Abstract
We focus on pharmacokinetic and pharmacodynamic properties of azathioprine, and on the efficacy and safety of the drug in multiple sclerosis patients. Azathioprine (AZA) is an immunosuppressive drug widely prescribed for the treatment of multiple sclerosis until the advent of interferon beta and glatiramer acetate. Data on its efficacy derive from old and often small clinical trials. The available results suggest that AZA reduces relapse rate, with a slight effect on disability progression. More recently, small studies detected a significant reduction in enhancing MRI lesions in patients treated with AZA. Several trials have suggested that the drug might be effective, at least in the short term, as an add-on treatment in patients breaking through monotherapy with conventional first-line drugs. Concerns about safety, mainly a possible increased risk of malignancy, have been raised. A review of the literature suggests that the safety profile of AZA is a cceptable, and that the risk of cancer does not significantly increase provided the cumulative dose is less than 300 g. Further large and well-conducted studies are needed to confirm the efficacy of the drug in monotherapy or in combination.I documenti in SFERA sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.