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- W4249715293 abstract "Abstract Objective This study aimed to explore the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration (PKAN).Methods A single-arm, open-label study was conducted. All subjects received pantethine during the 24-week period of treatment. The primary endpoints were change of the Unified Parkinson’s Disease Rating Scale (UPDRS) I–III and Fahn–Marsden (FM) score from baseline to week 24 after treatment.Results Fifteen children with PKAN were enrolled, and all patients completed the study. After 24 weeks of treatment with pantethine at 60 mg/kg per day, there was no difference in either UPDRS I–III (t = 0.516, P = 0.614) or FM score (t = 0.353, P = 0.729) between the baseline and W24. Whereas the rates of increase in UPDRS I-III (Z = 2.614, p = 0.009) and FM scores (Z = 2.643, p = 0.008) were slowed. Four patients (26.7%) were evaluated as “slightly improved” by doctors through blinded video assessment. Patients with lower baseline UPDRS I–III or FM scores were more likely to be improved. The living quality of family members improved after pantethine treatment, evaluated by PedsQL TM 2.0 FIM scores, whereas the living quality of the patients was unchanged at W24, evaluated by PedsQL TM 4.0 and PedsQL TM 3.0 NMM. Serum level of CoA was comparable between baseline and W24. There was no drug related adverse event during the study.Conclusions Pantethine could not significantly improve motor function in children with PKAN after 24 weeks treatment, but it could probably delay the progression of motor dysfunction in our study. 26.7% of patients showed slightly improved. Pantethine was well-tolerated at 60 mg/kg per day." @default.
- W4249715293 created "2022-05-12" @default.
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- W4249715293 date "2020-04-25" @default.
- W4249715293 modified "2023-09-26" @default.
- W4249715293 title "Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration: A single-arm, open-label study." @default.
- W4249715293 doi "https://doi.org/10.21203/rs.3.rs-23934/v1" @default.
- W4249715293 hasPublicationYear "2020" @default.
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