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

Jens Sandahl Christiansen¹, Philippe F Backeljauw², Martin Bidlingmaier³, Beverly M K Biller⁴, Margaret C S Boguszewski⁵, Felipe F Casanueva⁶, Philippe Chanson⁷, Pierre Chatelain⁸, Catherine S Choong⁹, David R Clemmons¹⁰, Laurie E Cohen¹¹, Pinchas Cohen¹², Jan Frystyk¹, Adda Grimberg¹³, Yukihiro Hasegawa¹⁴, Morey W Haymond¹⁵, Ken Ho¹⁶, Andrew R Hoffman¹⁷, Jeff M P Holly¹⁸, Reiko Horikawa¹⁹, Charlotte Höybye²⁰, Jens Otto L Jorgensen¹, Gudmundur Johannsson²¹, Anders Juul²², Laurence Katznelson²³, John J Kopchick²⁴, K O Lee²⁵, Kuk-Wha Lee²⁶, Xiaoping Luo²⁷, Shlomo Melmed²⁸, Bradley S Miller²⁹, Madhusmita Misra⁴, Vera Popovic³⁰, Ron G Rosenfeld³¹, Judith Ross³², Richard J Ross³³, Paul Saenger³⁴, Christian J Strasburger³⁵, Michael O Thorner³⁶, Haim Werner³⁷, Kevin Yuen³⁸

Affiliations

¹ Aarhus University Aarhus, Denmark.
² Cincinnati Children's Hospital Cincinnati, Ohio, USA.
³ Klinikum der Universität München Munich, Germany.
⁴ Massachusetts General Hospital Boston, Massachusetts, USA.
⁵ Federal University of Parana Curitiba, Brazil.
⁶ Santiago de Compostela University Santiago de Compostela, Spain.
⁷ Université Paris Saclay Le Kremlin-BicϪtre, France.
⁸ University Claude Bernard Lyon, France.
⁹ University of Western Australia Subiaco, Western Australia, Australia.
¹⁰ University of North Carolina Chapel Hill, North Carolina, USA.
¹¹ Boston Children's Hospital Boston, Massachusetts, USA.
¹² University of Southern California Los Angeles, California, USA.
¹³ Children's Hospital of Philadelphia Philadelphia, Pennsylvania, USA.
¹⁴ Tokyo Metropolitan Children's Medical Center Tokyo, Japan.
¹⁵ Baylor College of Medicine Houston, Texas, USA.
¹⁶ University of Queensland Brisbane, Queensland, Australia.
¹⁷ VA Palo Alto Health Care System and Stanford University Palo Alto, California, USA arhoffman@stanford.edu.
¹⁸ University of Bristol Bristol, UK.
¹⁹ National Center for Child Health and Development Tokyo, Japan.
²⁰ Karolinska University Hospital Stockholm, Sweden.
²¹ University of Gothenburg Gothenburg, Sweden.
²² Rigshospitalet University of Copenhagen, Copenhagen, Denmark.
²³ Stanford University Stanford, California, USA.
²⁴ Ohio University Athens, Ohio, USA.
²⁵ National University of Singapore Singapore, Singapore.
²⁶ UCLA School of Medicine Los Angeles, California, USA.
²⁷ Tongji Hospital Wuhan, China.
²⁸ Cedars-Sinai Medical Center Los Angeles, California, USA.
²⁹ Masonic Children's Hospital Minneapolis, Minnesota, USA.
³⁰ University of Belgrade Belgrade, Serbia.
³¹ Stat5 Consulting Los Altos, California, USA.
³² Jefferson Medical College Philadelphia, Pennsylvania, USA.
³³ University of Sheffield Sheffield, UK.
³⁴ SUNY Stony Brook, New York, USA.
³⁵ Charite-Universitätsmedizin Berlin, Germany.
³⁶ University of Virginia Charlottesville, Virginia, USA.
³⁷ Tel Aviv University Tel Aviv, Israel.
³⁸ Swedish Neuroscience Institute Seattle, Washington, USA.

PMID: 27009113
PMCID: PMC5081743
DOI: 10.1530/EJE-16-0111

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

Jens Sandahl Christiansen et al. Eur J Endocrinol. 2016 Jun.

. 2016 Jun;174(6):C1-8.

doi: 10.1530/EJE-16-0111. Epub 2016 Mar 23.

Authors

Affiliations

¹ Aarhus University Aarhus, Denmark.
² Cincinnati Children's Hospital Cincinnati, Ohio, USA.
³ Klinikum der Universität München Munich, Germany.
⁴ Massachusetts General Hospital Boston, Massachusetts, USA.
⁵ Federal University of Parana Curitiba, Brazil.
⁶ Santiago de Compostela University Santiago de Compostela, Spain.
⁷ Université Paris Saclay Le Kremlin-BicϪtre, France.
⁸ University Claude Bernard Lyon, France.
⁹ University of Western Australia Subiaco, Western Australia, Australia.
¹⁰ University of North Carolina Chapel Hill, North Carolina, USA.
¹¹ Boston Children's Hospital Boston, Massachusetts, USA.
¹² University of Southern California Los Angeles, California, USA.
¹³ Children's Hospital of Philadelphia Philadelphia, Pennsylvania, USA.
¹⁴ Tokyo Metropolitan Children's Medical Center Tokyo, Japan.
¹⁵ Baylor College of Medicine Houston, Texas, USA.
¹⁶ University of Queensland Brisbane, Queensland, Australia.
¹⁷ VA Palo Alto Health Care System and Stanford University Palo Alto, California, USA arhoffman@stanford.edu.
¹⁸ University of Bristol Bristol, UK.
¹⁹ National Center for Child Health and Development Tokyo, Japan.
²⁰ Karolinska University Hospital Stockholm, Sweden.
²¹ University of Gothenburg Gothenburg, Sweden.
²² Rigshospitalet University of Copenhagen, Copenhagen, Denmark.
²³ Stanford University Stanford, California, USA.
²⁴ Ohio University Athens, Ohio, USA.
²⁵ National University of Singapore Singapore, Singapore.
²⁶ UCLA School of Medicine Los Angeles, California, USA.
²⁷ Tongji Hospital Wuhan, China.
²⁸ Cedars-Sinai Medical Center Los Angeles, California, USA.
²⁹ Masonic Children's Hospital Minneapolis, Minnesota, USA.
³⁰ University of Belgrade Belgrade, Serbia.
³¹ Stat5 Consulting Los Altos, California, USA.
³² Jefferson Medical College Philadelphia, Pennsylvania, USA.
³³ University of Sheffield Sheffield, UK.
³⁴ SUNY Stony Brook, New York, USA.
³⁵ Charite-Universitätsmedizin Berlin, Germany.
³⁶ University of Virginia Charlottesville, Virginia, USA.
³⁷ Tel Aviv University Tel Aviv, Israel.
³⁸ Swedish Neuroscience Institute Seattle, Washington, USA.

PMID: 27009113
PMCID: PMC5081743
DOI: 10.1530/EJE-16-0111

Abstract

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.

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References

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

Affiliations

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

Authors

Affiliations

Abstract

References

MeSH terms

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LinkOut - more resources

Full Text Sources

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Medical