The need for home enzyme replacement therapy for patients with lysosomal disease in Japan

[Introduction and Aim] In Europe, Australia, and North and South America, enzyme replacement therapy (ERT) at home is a common practice for patients with lysosomal diseases. In Japan, on the other hand, patients need to visit a specialized hospital every one to two weeks for regular ERT. For the past 10 years, we have been proposing to the government, authorities, and academic societies to realize ERT at home, and under the circumstances of the spread of the COVID-19 in 2020, the need for ERT at home has increased. The purpose of this study was to investigate the patient burden of ERT in Japan for patients with lysosomal diseases (Fabry disease, Gaucher disease, Pompe disease, and MPS) who belong to four major lysosomal disease patient groups in Japan, and to clarify the need for enzyme replacement therapy at home. [Results] In January 2021, we conducted a questionnaire survey of lysosome disease patients and their families via four lysosome disease patient organizations (194 patients in total). Among the patients with lysosomal disease, 57% of them needed to be accompanied by their families. In addition, it took about 40 min each way to visit a specialized hospital and 246 min to stay in the hospital. In Japan, 67% of the patients preferred to receive ERT at a place other than a specialized hospital (home, clinic, school, office, etc.). [Conclusions] It is clear that lysosomal disease patients and their families in Japan are burdened by ERT. As a result of our work to date, in March 2021, 11 enzymes approved in Japan for lysosomal disease will be available for use by home physicians. Were approved to be administered by nurses under the direction of home physicians.

A preliminary study on university students' attitudes towards COVID-19

This research compared university student-athletes and other university students to analyze differences in attitudes towards COVID-19. The objective was to examine the impact of student-athletes' attitudes on their future athletic activities and perform verification that will contribute to the deliberation and creation of university support measures for student-athletes. Google Forms were used to administer an online questionnaire to university students nationwide. The responses to questions on the questionnaire were recorded on either 5 or 8 point scales, as well as open-ended questions without word limitations at the end of the questionnaire. The results were analyzed by separating results into two cohorts;student-athletes and other university students, and then categorizing responses to each question as positive or negative. Cross-tabulation was used and then Fischer's exact test was performed. To analyze the open-ended questions, KH-Coder3 software, which excels at Japanese analysis and is used in a great deal of research, was used. The analysis found a significant difference for some questions. In particular, a greater percentage of student-athletes than other university students felt unease regarding infection at university facilities, the impact of infection on university life, and the impact on activities from April 2020 onwards. Some of the reasons for the higher percentage of responses indicating unease were the lack of transparency in student life, with universities not defining clear approaches.