TY - JOUR

T1 - Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations

AU - Christiansen, Jens Sandahl

AU - Backeljauw, Philippe F

AU - Bidlingmaier, Martin

AU - Biller, Beverly M K

AU - Boguszewski, Margaret C S

AU - Casanueva, Felipe F

AU - Chanson, Philippe

AU - Chatelain, Pierre

AU - Choong, Catherine S

AU - Clemmons, David R

AU - Cohen, Laurie E

AU - Cohen, Pinchas

AU - Frystyk, Jan

AU - Grimberg, Adda

AU - Hasegawa, Yukihiro

AU - Haymond, Morey W

AU - Ho, Ken

AU - Hoffman, Andrew R

AU - Holly, Jeff M P

AU - Horikawa, Reiko

AU - Höybye, Charlotte

AU - Jorgensen, Jens Otto Lunde

AU - Johannsson, Gudmundur

AU - Juul, Anders

AU - Katznelson, Laurence

AU - Kopchick, John J.

AU - Lee, K O

AU - Lee, Kuk-Wha

AU - Luo, Xiaoping

AU - Melmed, Shlomo

AU - Miller, Bradley S

AU - Misra, Madhusmita

AU - Popovic, Vera

AU - Rosenfeld, Ron G

AU - Ross, Judith

AU - Ross, Richard J

AU - Saenger, Paul

AU - Strasburger, Christian J

AU - Thorner, Michael O

AU - Werner, Haim

AU - Yuen, Kevin C J

N1 - © 2016 The authors.

PY - 2016/6

Y1 - 2016/6

N2 - 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.

AB - 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.

KW - Clinical Trials as Topic

KW - Consensus

KW - Dwarfism, Pituitary

KW - Hormone Replacement Therapy

KW - Human Growth Hormone

KW - Humans

KW - Research Design

KW - Journal Article

U2 - 10.1530/EJE-16-0111

DO - 10.1530/EJE-16-0111

M3 - Journal article

C2 - 27009113

VL - 174

SP - C1-8

JO - European Journal of Endocrinology

JF - European Journal of Endocrinology

SN - 0804-4643

IS - 6

ER -