Genotype-stratified treatment for monogenic insulin resistance: a systematic review.

The hormone insulin stimulates nutrient uptake from the bloodstream into tissues. In insulin resistance (IR), this action is blunted. Some rare gene alterations cause severe IR, diabetes that is difficult to control, and early complications. Many treatments have been suggested, but reliable evidence of their risks and benefits is sparse. We analysed all available reports describing treatment outcomes in severe IR. We found that the evidence is of low to very low quality overall. Injections of leptin, a hormone from fat tissue, or thiazolidinedione tablets that increase fat tissue both appear to improve diabetes control in people with reduced ability to make fat tissue. Injections of another treatment, insulin-like growth factor, appear to improve diabetes control in people with direct blockage of insulin action. There is a pressing need to improve evidence for treatment in these rare and severe conditions.