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著作: Kubota Takuo/[福本 誠二]/Cheong Il Hae/Michigami Toshimi/Namba Noriyuki/[伊東 伸朗]/Tokunaga Shin/Gibbs Yoshimi/Ozono Keiichi/Long-term outcomes for Asian patients with X-linked hypophosphataemia: rationale and design of the SUNFLOWER longitudinal, observational cohort study./[BMJ Open]

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EID
368522
EOID
985908
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LastModified
2020年8月14日(金) 15:03:34
Operator
福本 誠二
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福本 誠二
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種別 必須 学術論文(審査論文)
言語 必須 英語
招待 推奨
審査 推奨 Peer Review
カテゴリ 推奨
共著種別 推奨
学究種別 推奨
組織 推奨
著者 必須
  1. (英) Kubota Takuo
    役割 任意
    貢献度 任意
    学籍番号 推奨
  2. 福本 誠二([徳島大学.先端酵素学研究所.基幹研究部門])
    役割 任意
    貢献度 任意
    学籍番号 推奨
  3. (英) Cheong Il Hae
    役割 任意
    貢献度 任意
    学籍番号 推奨
  4. (英) Michigami Toshimi
    役割 任意
    貢献度 任意
    学籍番号 推奨
  5. (英) Namba Noriyuki
    役割 任意
    貢献度 任意
    学籍番号 推奨
  6. 伊東 伸朗([東京大学医学部附属病院腎臓・内分泌内科])
    役割 任意
    貢献度 任意
    学籍番号 推奨
  7. (英) Tokunaga Shin
    役割 任意
    貢献度 任意
    学籍番号 推奨
  8. (英) Gibbs Yoshimi
    役割 任意
    貢献度 任意
    学籍番号 推奨
  9. (英) Ozono Keiichi
    役割 任意
    貢献度 任意
    学籍番号 推奨
題名 必須

(英) Long-term outcomes for Asian patients with X-linked hypophosphataemia: rationale and design of the SUNFLOWER longitudinal, observational cohort study.

副題 任意
要約 任意

(英) X-linked hypophosphataemic rickets/osteomalacia (XLH) is a chronic, debilitating genetic disease characterised by skeletal abnormalities and growth disorder. The burden of XLH begins in childhood and continues throughout life. Conventional medical therapy with phosphate, active vitamin D and surgery do not address the underlying pathophysiology of the disease. While treatment during childhood may improve bone deformity and growth retardation, a large proportion of adult patients still fail to reach normal stature. Furthermore, adult patients with XLH report comorbidities associated with unresolved childhood disease, as well as newly developed disease-related complications and significantly impaired quality of life (QOL). Despite the multiple negative aspects of XLH, Asian consensus statements for diagnosis and management are lacking. The Study of longitUdinal observatioN For patients with X-Linked hypOphosphataemic rickets/osteomalacia in collaboration With Asian partnERs study is a longitudinal observational cohort study of patients with XLH, designed to determine the medical characteristics and burdens (physical, emotional and financial) of this progressive disease and to evaluate the impact of treatment (including the use of burosumab) on clinical outcomes. The study was initiated in April 2018, and registration will remain open until 30 April 2022. The sample size planned for analyses is 160 patients, consisting of 100 patients in Japan and 60 patients in Korea. Up to 5 years of observation are planned per patient, from enrolment through to April 2023. Prospective and retrospective data will be collected to evaluate variables, including height/growth, rickets severity score, QOL, motor function and biomarkers for phosphate metabolism and bone turnover. Ethics approval was obtained from the Ethics Committee of Osaka University, the Ethics Committee of Kyowa Kirin Co and by the Ethics Committee of each participating medical institution. Two interim analyses and associated publications are planned using retrospective and enrolment data at year 1 and results at year 3. NCT03745521; UMIN000031605.

キーワード 推奨
発行所 推奨
誌名 必須 BMJ Open(BMJ Publishing Group Ltd)
(eISSN: 2044-6055)
ISSN 任意 2044-6055
ISSN: 2044-6055 (eISSN: 2044-6055)
Title: BMJ open
Title(ISO): BMJ Open
Publisher: BMJ Group
 (NLM Catalog  (Scopus  (CrossRef (Scopus information is found. [need login])
必須 10
必須 6
必須 e036367 e036367
都市 任意
年月日 必須 2020年 6月 29日
URL 任意
DOI 任意 10.1136/bmjopen-2019-036367    (→Scopusで検索)
PMID 任意 32601114    (→Scopusで検索)
NAID 任意
WOS 任意
Scopus 任意
評価値 任意
被引用数 任意
指導教員 推奨
備考 任意
  1. (英) Article.ELocationID: 10.1136/bmjopen-2019-036367

  2. (英) Article.DataBankList.DataBank.DataBankName: ClinicalTrials.gov

  3. (英) Article.DataBankList.DataBank.AccessionNumberList.AccessionNumber: NCT03745521

  4. (英) Article.PublicationTypeList.PublicationType: Journal Article

  5. (英) KeywordList.Keyword: bone diseases

  6. (英) KeywordList.Keyword: clinical trials

  7. (英) KeywordList.Keyword: health economics

  8. (英) KeywordList.Keyword: musculoskeletal disorders

  9. (英) CoiStatement: Competing interests: TK has received personal fees from Kyowa Kirin Co for the submitted work and grants from Kyowa Kirin Co outside the submitted work. SF has received grants from Teijin Pharma and Astellas Pharma; and held an endowed chair position with Chugai Pharmaceutical Co, Ono Pharmaceutical Co, Taisho Pharmaceutical Co and Kyowa Kirin Co outside the submitted work. TM has received personal fees (honorarium) from Kyowa Kirin Co for serving as a member of the advisory board during the conduct of this study. NN has received personal fees from Kyowa Kirin Co for the submitted work; and grants from Kyowa Kirin Co outside the submitted work. NI has received research grants from Kyowa Kirin Co outside the submitted work. ST and YG are the employees of Kyowa Kirin Co. KO has received lecture fees from Kyowa Kirin Co, Alexion Pharmaceuticals and Novo Nordisk Pharma outside the submitted work.