单位:[1]Aarhus Univ, Aarhus, Denmark[2]Cincinnati Childrens Hosp, Cincinnati, OH USA[3]Klinikum Univ Munchen, Munich, Germany[4]Massachusetts Gen Hosp, Boston, MA 02114 USA[5]Univ Fed Parana, BR-80060000 Curitiba, Parana, Brazil[6]Univ Santiago de Compostela, Santiago De Compostela, Spain[7]Univ Paris Saclay, Le Kremlin Bicetre, France[8]Univ Lyon 1, F-69365 Lyon, France[9]Univ Western Australia, Subiaco, WA, Australia[10]Univ N Carolina, Chapel Hill, NC USA[11]Boston Childrens Hosp, Boston, MA USA[12]Univ So Calif, Los Angeles, CA USA[13]Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA[14]Tokyo Metropolitan Childrens Med Ctr, Tokyo, Japan[15]Baylor Coll Med, Houston, TX 77030 USA[16]Univ Queensland, Brisbane, Qld, Australia[17]VA Palo Alto Hlth Care Syst, Palo Alto, CA USA[18]Stanford Univ, Palo Alto, CA 94304 USA[19]Univ Bristol, Bristol, Avon, England[20]Natl Ctr Child Hlth & Dev, Tokyo, Japan[21]Karolinska Univ Hosp, Stockholm, Sweden[22]Univ Gothenburg, Gothenburg, Sweden[23]Univ Copenhagen, Rigshosp, DK-2100 Copenhagen, Denmark[24]Stanford Univ, Stanford, CA 94305 USA[25]Ohio Univ, Athens, OH 45701 USA[26]Natl Univ Singapore, Singapore 117548, Singapore[27]Univ Calif Los Angeles, Sch Med, Los Angeles, CA USA[28]Tongji Hosp, Wuhan, Peoples R China华中科技大学同济医学院附属同济医院[29]Cedars Sinai Med Ctr, Los Angeles, CA 90048 USA[30]Masonic Childrens Hosp, Minneapolis, MN USA[31]Univ Belgrade, Belgrade, Serbia[32]Stat5 Consulting, Los Altos, CA USA[33]Thomas Jefferson Univ, Jefferson Med Coll, Philadelphia, PA 19107 USA[34]Univ Sheffield, Sheffield, S Yorkshire, England[35]SUNY Stony Brook, Stony Brook, NY 11794 USA[36]Charite, D-13353 Berlin, Germany[37]Univ Virginia, Charlottesville, VA USA[38]Tel Aviv Univ, IL-69978 Tel Aviv, Israel[39]Swedish Neurosci Inst, Seattle, WA USA
Objective: The Growth Hormone (GH) Research Society (GRS) convened a workshop to address important issues regarding trial design, efficacy, and safety of long-acting growth hormone preparations (LAGH). Participants: A closed meeting of 55 international scientists with expertise in GH, including pediatric and adult endocrinologists, basic scientists, regulatory scientists, and participants from the pharmaceutical industry. Evidence: Current literature was reviewed for gaps in knowledge. Expert opinion was used to suggest studies required to address potential safety and efficacy issues. Consensus process: Following plenary presentations summarizing the literature, breakout groups discussed questions framed by the planning committee. Attendees reconvened after each breakout session to share group reports. A writing team compiled the breakout session reports into a draft document that was discussed and revised in an open forum on the concluding day. This was edited further and then circulated to attendees from academic institutions for review after the meeting. Participants from pharmaceutical companies did not participate in the planning, writing, or in the discussions and text revision on the final day of the workshop. Scientists from industry and regulatory agencies reviewed the manuscript to identify any factual errors. Conclusions: LAGH compounds may represent an advance over daily GH injections because of increased convenience and differing phamacodynamic properties, providing the potential for improved adherence and outcomes. Better methods to assess adherence must be developed and validated. Long-term surveillance registries that include assessment of efficacy, cost-benefit, disease burden, quality of life, and safety are essential for understanding the impact of sustained exposure to LAGH preparations.
通讯机构:[17]VA Palo Alto Hlth Care Syst, Palo Alto, CA USA[18]Stanford Univ, Palo Alto, CA 94304 USA
推荐引用方式(GB/T 7714):
Christiansen Jens Sandahl,Backeljauw Philippe F.,Bidlingmaier Martin,et al.Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations[J].EUROPEAN JOURNAL OF ENDOCRINOLOGY.2016,174(6):C1-C8.doi:10.1530/EJE-16-0111.
APA:
Christiansen, Jens Sandahl,Backeljauw, Philippe F.,Bidlingmaier, Martin,Biller, Beverly M. K.,Boguszewski, Margaret C. S....&Yuen, Kevin.(2016).Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations.EUROPEAN JOURNAL OF ENDOCRINOLOGY,174,(6)
MLA:
Christiansen, Jens Sandahl,et al."Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations".EUROPEAN JOURNAL OF ENDOCRINOLOGY 174..6(2016):C1-C8