TY - JOUR
T1 - Growth Hormone Research Society perspective on biomarkers of GH action in children and adults
AU - Johannsson, Gudmundur
AU - Bidlingmaier, Martin
AU - Biller, Beverly
AU - Boguszewski, Margaret
AU - Casanueva, Felipe F
AU - Chanson, Philippe
AU - Choong, Catherine S
AU - Clayton, Peter E
AU - Clemmons, David
AU - Dattani, Mehul
AU - Frystyk, Jan
AU - Ho, Ken K Y
AU - Hoffman, Andrew
AU - Horikawa, Reiko
AU - Juul, Anders
AU - Kopchick, John
AU - Luo, Xiaoping
AU - Neggers, Sebastian
AU - Netchine, Irène
AU - Olsson, Daniel S
AU - Radovick, Sally
AU - Rosenfeld, Ron G
AU - Ross, Richard J
AU - Schilbach, Katharina
AU - Collett-Solberg, Paulo Ferrez
AU - Strasburger, Christian J
AU - Trainer, Peter J
AU - Wickstrom, Kerstin
AU - Yuen, Kevin Cj
AU - Jørgensen, Jens Otto Lunde
PY - 2018
Y1 - 2018
N2 - 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 pediatric 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 co-morbidities 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.
AB - 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 pediatric 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 co-morbidities 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.
KW - Journal Article
U2 - 10.1530/EC-18-0047
DO - 10.1530/EC-18-0047
M3 - Journal article
C2 - 29483159
SN - 2049-3614
VL - 7
SP - R126-R134
JO - Endocrine Connections
JF - Endocrine Connections
IS - 3
ER -