Title

Growth hormone research society perspective on biomarkers of gh action in children and adults

Authors

Gudmundur Johannsson, Göteborg University, Sahlgrenska Academy
Martin Bidlingmaier, Klinikum der Universität München
Beverly M.K. Biller, Massachusetts General Hospital
Margaret Boguszewski, Universidade Federal do Parana
Felipe F. Casanueva, Universidad de Santiago de Compostela, Facultad de Medicina
Philippe Chanson, AP-HP Assistance Publique - Hopitaux de Paris
Peter E. Clayton, The University of Manchester
Catherine S. Choong, University of Western Australia
David Clemmons, The University of North Carolina at Chapel Hill
Mehul Dattani, UCL Great Ormond Street Institute of Child Health
Jan Frystyk, Odense Universitetshospital
Ken Ho, The University of Queensland
Andrew R. Hoffman, Stanford University
Reiko Horikawa, National Center for Child Health and Development
Anders Juul, Københavns Universitet
John J. Kopchick, Ohio University Heritage College of Osteopathic Medicine
Xiaoping Luo, Tongji Medical College
Sebastian Neggers, Erasmus MC
Irene Netchine, Sorbonne Universite
Daniel S. Olsson, Sahlgrenska Universitetssjukhuset
Sally Radovick, Rutgers Robert Wood Johnson Medical School
Ron Rosenfeld, Oregon Health & Science University
Richard J. Ross, The University of Sheffield
Katharina Schilbach, Klinikum der Universität München
Paulo Solberg, Universidade do Estado do Rio de Janeiro
Christian Strasburger, Charité – Universitätsmedizin Berlin
Peter Trainer, The University of Manchester
Kevin C.J. Yuen, University of Arizona College of Medicine – Phoenix
Kerstin Wickstrom, Medical Products Agency
Jens O.L. Jorgensen, Aarhus Universitetshospital

Document Type

Article

Publication Date

3-1-2018

Abstract

© 2018 Growth Hormone Research Society. 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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