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Growth Hormone Research Society perspective on biomarkers of GH action in children and adults

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单位: [1]Univ Gothenburg, Sahlgrenska Acad, Dept Internal Med & Clin Nutr, Gothenburg, Sweden [2]Klinikum Univ Munchen, Med Klin & Poliklin 4, Munich, Germany [3]Massachusetts Gen Hosp, Neuroendocrine Unit, Boston, MA 02114 USA [4]Univ Fed Parana, Curitiba, Parana, Brazil [5]Univ Santiago, Complejo Hosp, Dept Med, Santiago De Compostela, Spain [6]AP HP, Paris, France [7]Inserm, Paris, France [8]Univ Manchester, Fac Biol Med & Hlth, Dev Biol & Med, Manchester, Lancs, England [9]Univ Western Australia, Princess Margaret Hosp, Dept Endocrinol, Nedlands, WA, Australia [10]Univ Western Australia, Sch Med, Nedlands, WA, Australia [11]Univ N Carolina, Dept Med, Chapel Hill, NC USA [12]Great Ormond St Inst Child Hlth, London, England [13]Odense Univ Hosp, Dept Endocrinol, Odense, Denmark [14]Princess Alexandra Hosp, Brisbane, Qld, Australia [15]Univ Queensland, Brisbane, Qld, Australia [16]Stanford Univ, Dept Med, Palo Alto, CA 94304 USA [17]VA Palo Hlth Care Syst, Palo Alto, CA USA [18]Natl Ctr Child Hlth & Dev, Tokyo, Japan [19]Univ Copenhagen, Rigshosp, Dept Growth & Reprod, Copenhagen, Denmark [20]Ohio Univ, Edison Biotechnol Inst, Athens, OH 45701 USA [21]Ohio Univ, Heritage Coll Osteopath Med, Athens, OH 45701 USA [22]Huazhong Univ Sci & Technol, Tongji Hosp, Tongji Med Coll, Dept Pediat, Wuhan, Hubei, Peoples R China [23]Erasmus Univ, Med Ctr, Pituitary Ctr Rotterdam, Sect Endocrinol,Dept Med, Rotterdam, Netherlands [24]Sorbonne Univ, Hop Trousseau, AP HP, INSERM UMRs 938,Serv Explorat Fonct Endocrinienne, Paris, France [25]Univ Gothenburg, Sahlgrenska Acad, Inst Med, Dept Endocrinol, Gothenburg, Sweden [26]Sahlgrens Univ Hosp, Gothenburg, Sweden [27]Rutgers State Univ, Robert Wood Johnson Med Sch, New Brunswick, NJ USA [28]Oregon Hlth & Sci Univ, Dept Pediat, Portland, OR USA [29]Univ Sheffield, Sheffield, S Yorkshire, England [30]Univ Estado Rio de Janeiro, Rio De Janeiro, Brazil [31]Charite, Berlin, Germany [32]Univ Manchester, Christie NHS Fdn Trust, Manchester, Lancs, England [33]Univ Arizona, Coll Med, Dept Neuroendocrinol, Barrow Pituitary Ctr,Barrow Neurol Inst, Phoenix, AZ USA [34]Med Prod Agcy, Uppsala, Sweden [35]Aarhus Univ Hosp, Aarhus, Denmark
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关键词: GH IGF-I GH deficiency acromegaly

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Objective: The Growth Hormone Research Society (GRS) convened a Workshop in 2017 to evaluate clinical endpoints, surrogate endpoints and biomarkers during GH treatment of children and adults and in patients with acromegaly. Participants: GRS invited 34 international experts including clinicians, basic scientists, a regulatory scientist and physicians from the pharmaceutical industry. Evidence: Current literature was reviewed and expert opinion was utilized to establish the state of the art and identify current gaps and unmet needs. Consensus process: Following plenary presentations, breakout groups discussed questions framed by the planning committee. The attendees re-convened after each breakout session to share the group reports. A writing team compiled the breakout session reports into a document that was subsequently discussed and revised by participants. This was edited further and circulated for final review after the meeting. Participants from pharmaceutical companies were not part of the writing process. Conclusions: The clinical endpoint in paediatric GH treatment is adult height with height velocity as a surrogate endpoint. Increased life expectancy is the ideal but unfeasible clinical endpoint of GH treatment in adult GH-deficient patients (GHDA) and in patients with acromegaly. The pragmatic clinical endpoints in GHDA include normalization of body composition and quality of life, whereas symptom relief and reversal of comorbidities are used in acromegaly. Serum IGF-I is widely used as a biomarker, even though it correlates weakly with clinical endpoints in GH treatment, whereas in acromegaly, normalization of IGF-I may be related to improvement in mortality. There is an unmet need for novel biomarkers that capture the pleiotropic actions of GH in relation to GH treatment and in patients with acromegaly.

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出版当年[2017]版:
大类 | 3 区 医学
小类 | 4 区 内分泌学与代谢
最新[2025]版:
大类 | 3 区 医学
小类 | 3 区 内分泌学与代谢
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出版当年[2016]版:
Q3 ENDOCRINOLOGY & METABOLISM
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Q3 ENDOCRINOLOGY & METABOLISM

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第一作者单位: [1]Univ Gothenburg, Sahlgrenska Acad, Dept Internal Med & Clin Nutr, Gothenburg, Sweden
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