Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration:
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(2020) 15:248
RESEARCH
Open Access
Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration: a single-arm, open-label study Xuting Chang1, Jie Zhang1, Yuwu Jiang1, Bufan Yao2, Jingmin Wang1 and Ye Wu1*
Abstract Objective: This study aimed to explore the efficacy and safety of pantethine in children with pantothenate kinaseassociated 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 quality of life of family members improved after pantethine treatment, evaluated by PedsQL TM 2.0 FIM scores, whereas the quality of life 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 may delay the progression of motor dysfunction in our study. Pantethine was well-tolerated at 60 mg/kg per day. Trial registration: Clinical trial registration number at www.chictr.org.cn:ChiCTR1900021076, Registered 27 January2019, the first participant was enrolled 30 September 2018, and other 14 participants were enrolled after the trial was registered. Keywords: Pantethine, Pantothenate kinase-associated neurodIegeneration, Efficacy, Safety
* Correspondence: [email protected] 1 Department of Pediatrics, Peking University First Hospital, No.1, Xi’an Men Street, West District, Beijing 100034, China Full list of author information is available at the end of the article © The Author(s). 2020 Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article'
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