Economic implications of growth hormone use in patients with short bowel syndrome.

Objective: Short bowel syndrome is a rare, life-threatening condition that can result in nutritional malabsorption. Parenteral nutrition provides life-saving support but can lead to complications and affect quality of life. Recombinant human growth hormone, somatropin (rDNA origin), has been shown to significantly reduce dependence on nutritional support (p < 0.05). This study evaluates the economic impact of somatropin use in the management of short bowel syndrome.

Methods: A discrete event simulation (DES) model was developed to estimate the benefits and costs associated with somatropin use. Risks of treatment complications and of disease-related events were modeled in identical patient pairs--one receiving parenteral nutrition alone, the other receiving 4 weeks of somatropin--for 2 years following initiation of treatment. Life expectancy was assumed equivalent. Risk functions were estimated from the literature and one randomized clinical trial. Total and component costs associated with each strategy were determined. The distribution of patients reducing parenteral nutrition need and the final parenteral nutrition frequency were also estimated. Sensitivity analyses were completed for key inputs. Direct medical costs are reported in US 2004 dollars.

Results: The model predicted that 96.0% of patients receiving somatropin reduce or eliminate parenteral nutrition within 6 weeks: average use was reduced by 2.8 days and one-third weaned completely. Based on 1.9 L of parenteral nutrition per day, estimated costs were 118,098 dollars in year one and 132,935 dollars in year two. With somatropin, costs dropped to 84,309 dollars in year one--despite the 17,459 dollars cost of somatropin treatment--and 81,250 dollars in year two. Over 2 years savings totaled 85,474 dollars.

Limitations: Insufficient data required that assumptions be made for some inputs. DES is new in pharmacoeconomics and may be perceived as a limitation.

Conclusions: Somatropin use improves quality of life by reducing the need for parenteral nutrition and results in health care cost savings.