Study on the safety and effect of novel therapeutic agents for mucopolysaccharidosis

2016 Fiscal Year Final Research Report

• Project/Area Number: 26461523
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Gifu University
• Principal Investigator: Orii Kenji 岐阜大学, 医学部附属病院, 准教授 (30332688)
• Co-Investigator(Renkei-kenkyūsha): TOMATSU SHUNJI 岐阜大学, 大学院医学系研究科, 非常勤講師 (70237105)
• Research Collaborator: SUZUKI YASUYUKI 岐阜大学, 医学部, 教授 (90154559)
• Project Period (FY): 2014-04-01 – 2017-03-31
• Keywords: 先天代謝異常症 / ムコ多糖症 / 新規治療薬 / 骨関節症状

Outline of Final Research Achievements

In this study, we investigated the efficacy and safety of pentosan polysulfate in patients with mucopolysaccharidosis about joint symptoms. For adult patients with mucopolysaccharidosis, pentosan polysulfate was injected subcutaneously and safety and therapeutic effects were examined. For 3 adult patients with mucopolysaccharidosis type II, subcutaneous injection of pentosan polysulfate was performed once a week for 12 weeks. After administration of pentosan polysulfate, one patient improved joint pain, and three patients showed mild improvement in the range of joint movement of the shoulder joint. Side reactions were not observed except for mild internal bleeding at the site of subcutaneous injection. Urinary uronate excretion after administration of polysulfate pentosan decreased with one who stopped enzyme replacement therapy. It was considered to act on subcutaneous injection of polysulfate pentosan.

Free Research Field

小児科