Standardized nursing management of enzyme replacement therapy for late-onset Pompe disease.

Abstract

Pompe disease or glycogen storage disease type II is a rare autosomal recessive disorder caused by a deficiency of the lysosomal enzyme a-glucosidase. Although enzyme replacement therapy (ERT) with 2 weekly intervals following was considered an effective treatment for Pompe disease in 2006, few patients can afford to receive treatment in China because of the high cost. This study aimed to examine the standard management of enzyme replacement therapy for late-onset Pompe disease among patients over the age of 14 years from a nursing perspective in order to assess operating procedures. ERT injection fluid dispensing and infusion procedures using different methods were analyzed and compared in 3 patients with advanced Pompe disease for forming standard operation procedures. In addition, the impact of different methods on time consumption was analyzed by 1-way analysis of variance. There were significant differences in time consumption between different dispensing and infusion methods. The time of dispensing and infusing the injection fluids using the cooperative method was 15.97 minutes shorter than that using the conventional method (95% CI: 4.51–27.43, P = .012); the time using the modified method was 20.93 minutes shorter than that using the conventional method (95% CI: 9.47–32.39, P = .012); and there was no significant difference between the cooperative and modified methods (P = .431). Enzyme replacement therapy entails frequent treatment and strict nursing requirements related to the intravenous infusion process. In this context, a standard operating procedure can be used to control nursing times and labor costs effectively while ensuring a safe and effective infusion process.