Abstract | OBJECTIVES: METHODS: This was a postmarketing study of Japanese MPS II patients treated with 0.5 mg/kg intravenous idursulfase weekly, conducted over a period of 8 years after initial administration. Assessments included the safety profile, survival rate, degree of clinical improvement, change in urinary uronic acid (UA) concentration, and 6-minute walk test (6MWT). RESULTS: The safety and efficacy analysis populations included 145 and 143 patients, respectively. The incidence of serious adverse events was 42.8% and the incidence of adverse drug reactions was 48.3%. The 7-year survival rate was 82.7%. Improvements in the clinical features of hepatosplenomegaly, skin, joint, and respiratory disorders were reported (per investigator's assessment). The mean change in urinary UA concentration was -128.39 mg/g creatinine, and that of 6MWT walking distance was +31.8 m. CONCLUSION: Long-term idursulfase treatment was well tolerated, and effective in improving clinical features, reducing urinary UA, and slowing disease progression in Japanese MPS II patients.
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Authors | Kazuo Ueda, Jiro Hokugo |
Journal | Expert opinion on drug safety
(Expert Opin Drug Saf)
Vol. 19
Issue 7
Pg. 891-901
(Jul 2020)
ISSN: 1744-764X [Electronic] England |
PMID | 32342708
(Publication Type: Journal Article)
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Chemical References |
- Uronic Acids
- Iduronate Sulfatase
- idursulfase
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Topics |
- Administration, Intravenous
- Adolescent
- Adult
- Child
- Child, Preschool
- Disease Progression
- Enzyme Replacement Therapy
(adverse effects, methods)
- Female
- Humans
- Iduronate Sulfatase
(administration & dosage, adverse effects)
- Infant
- Japan
- Male
- Middle Aged
- Mucopolysaccharidosis II
(drug therapy)
- Product Surveillance, Postmarketing
- Survival Rate
- Uronic Acids
(urine)
- Walk Test
- Young Adult
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