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A study on clinical features and pathophysiology of drug-induced carnitine deficiency

Research Project

Project/Area Number 25461552
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Pediatrics
Research InstitutionShimane University

Principal Investigator

Kobayashi Hironori  島根大学, 医学部, 助教 (70397868)

Project Period (FY) 2013-04-01 – 2016-03-31
Project Status Completed (Fiscal Year 2015)
Budget Amount *help
¥4,940,000 (Direct Cost: ¥3,800,000、Indirect Cost: ¥1,140,000)
Fiscal Year 2015: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2014: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2013: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
Keywordsカルニチン欠乏症 / タンデムマス / ピボキシル基 / シベレスタット / 遊離カルニチン / 全身性カルニチン欠乏症 / 急性脳症 / 低血糖 / in vitro probe assay / ピバロイルカルニチン / カルニチン / アシルカルニチン分析 / 薬剤性カルニチン欠乏症 / ピバリン酸 / バルプロ酸
Outline of Final Research Achievements

In this study, we mainly focused on secondary carnitine deficiency due to pivalete-conjugated drugs. We retrospectively investigated the age, clinical manifestations, PCA administration period, and background of 25 patients who showed a decrease in free carnitine (C0, nmol/mL) concomitant with an increase in pivaloyl carnitine (C5) on acylcarnitine analysis with tandem mass spectrometry. Carnitine deficiency resulting from PCA treatment was most frequently observed in 1-year-old infants. Most patients manifested acute encephalopathy and/or hypoglycemia. Some patients developed carnitine deficiency after PCA administration for <14 days. We reported two patients who showed increased value of C5 by sivelestat sodium.
In the present study, we developed an in vitro probe assay for analyzing intra-and extra-cellular ACs.

Report

(4 results)
  • 2015 Annual Research Report   Final Research Report ( PDF )
  • 2014 Research-status Report
  • 2013 Research-status Report
  • Research Products

    (7 results)

All 2016 2015 2014

All Journal Article (3 results) (of which Int'l Joint Research: 1 results,  Peer Reviewed: 3 results,  Open Access: 1 results,  Acknowledgement Compliant: 2 results) Presentation (4 results)

  • [Journal Article] Clinical Features of Carnitine Deficiency Secondary to Pivalate-Conjugated Antibiotic Therapy2016

    • Author(s)
      Kobayashi H, Fukuda S, Yamada K, Hasegawa Y, Takahashi T, Purevsuren J, Yamaguchi S
    • Journal Title

      J Pediatr.

      Volume: April Pages: 183-187

    • DOI

      10.1016/j.jpeds.2016.02.080

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Int'l Joint Research / Acknowledgement Compliant
  • [Journal Article] 新生児マススクリーニングを契機に診断された母の全身性カルニチン欠乏症2016

    • Author(s)
      中澤 枝里子,菊池 信行 ,小林 弘典,長谷川 有紀,窪田 満,山口 清次
    • Journal Title

      日本マススクリーニング学会雑誌

      Volume: 26

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Acknowledgement Compliant
  • [Journal Article] Elevation of pivaloylcarnitine by sivelestat sodium in two children2015

    • Author(s)
      Yamada K, Kobayashi H, Bo R, Takahashi T, Hasegawa T, Nakamura M, Ishige N, Yamaguchi S
    • Journal Title

      Molecular Genetics and Metabolism

      Volume: 116 Issue: 3 Pages: 192-194

    • DOI

      10.1016/j.ymgme.2015.09.009

    • Related Report
      2015 Annual Research Report
    • Peer Reviewed / Open Access
  • [Presentation] 黄疸を契機に診断に至った全身性カルニチン欠乏症の一例2014

    • Author(s)
      李知子, 鶴田悟, 山田健治, 小林弘典, 長谷川有紀, 山口清次, 飯島一誠, 竹島泰弘
    • Organizer
      第56回日本先天代謝異常学会
    • Place of Presentation
      仙台
    • Year and Date
      2014-11-13 – 2014-11-15
    • Related Report
      2014 Research-status Report
  • [Presentation] L-カルニチン内服によるアシルカルニチンプロファイルの変化2014

    • Author(s)
      山田健治, 高橋知男, 小林弘典, 坊亮輔, 長谷川有紀, 山口清次
    • Organizer
      第41回日本マス・スクリーニング学会
    • Place of Presentation
      広島
    • Year and Date
      2014-08-22 – 2014-08-23
    • Related Report
      2014 Research-status Report
  • [Presentation] ピボキシル基を含む抗菌薬内服後の二次性カルニチン欠乏症22例の検討2014

    • Author(s)
      小林弘典, 山田健治, 高橋知男, 長谷川有紀, 山口清次
    • Organizer
      第117回日本小児科学会
    • Place of Presentation
      名古屋
    • Year and Date
      2014-04-11 – 2014-04-13
    • Related Report
      2014 Research-status Report
  • [Presentation] ピボキシル基を含む抗菌薬内服後の二次性カルニチン欠乏症22例の検討2014

    • Author(s)
      小林弘典、山田健治、高橋知男、長谷川有紀、山口清次
    • Organizer
      117回日本小児科学会学術集会
    • Place of Presentation
      名古屋国際会議場(名古屋市)
    • Related Report
      2013 Research-status Report

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Published: 2014-07-25   Modified: 2019-07-29  

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