Impact of Enzyme Replacement Therapy on Linear Growth in Korean Patients with Mucopolysaccharidosis Type II (Hunter Syndrome)

Hunter syndrome (or mucopolysaccharidosis type II [MPS II]) arises because of a deficiency in the lysosomal enzyme iduronate-2-sulfatase. Short stature is a prominent and consistent feature in MPS II. Enzyme replacement therapy (ERT) with idursulfase (Elaprase®) or idursulfase beta (Hunterase®) have been developed for these patients. The effect of ERT on the growth of Korean patients with Hunter syndrome was evaluated at a single center. This study comprised 32 patients, who had received ERT for at least 2 yr; they were divided into three groups according to their ages at the start of ERT: group 1 (<6 yr, n=14), group 2 (6-10 yr, n=11), and group 3 (10-20 yr, n=7). The patients showed marked growth retardation as they got older. ERT may have less effect on the growth of patients with the severe form of Hunter syndrome. The height z-scores in groups 2 and 3 revealed a significant change (the estimated slopes before and after the treatment were -0.047 and -0.007, respectively: difference in the slope, 0.04; P<0.001). Growth in response to ERT could be an important treatment outcome or an endpoint for future studies.

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Background

Hyperglycaemia is common in critically ill adult patients. Many studies have identified the content, methods, and effects of glycaemic control but have not explored the effects of knowledge, attitudes, and practices (KAP) on glycaemic control in critically ill adults. Various factors also influence the KAP of intensive care unit (ICU) staff.

Aims

To assess KAP regarding glucose management for critically ill adults among nurses and medical professionals and identify the factors that influence their KAP in ICUs.

Methods

A multicentre cross-sectional survey.

Results

In total, 403/459 (response rate: 87.8%) participants from ICUs in nine tertiary hospitals in China participated in this study, 82.4% of whom were female and 93.4% of whom were nurses. The mean work experience was 8.88 years, and the mean critical care experience was 6.59 years. The scoring rate for the three dimensions of knowledge, attitudes, and practices were 82.35%, 87.69%, and 76%, respectively. We did not find any other factors affecting the KAP scores except for the level of knowledge awareness (p < 0.001), awareness of the importance (p < 0.001), and training for glucose control (p = 0.004).

Conclusion

ICU staff KAP regarding glycaemic control in critically ill adults among ICU professionals were acceptable in China. However, ICU professionals' current knowledge regarding nutrition, glucose variability, and skills related to glucose management could be improved.

Relevance to Clinical Practice

ICU educators should provide more skills-related training for healthcare professionals in the glycaemic management of critically ill adults. Moreover, the process of managing blood glucose in adult ICU patients is a collaborative, multidisciplinary team effort, with monitoring and feedback required during implementation.