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

Jens Sandahl Christiansen; Philippe F Backeljauw; Martin Bidlingmaier; Beverly M K Biller; Margaret C S Boguszewski; Felipe F Casanueva; Philippe Chanson; Pierre Chatelain; Catherine S Choong; David R Clemmons; Laurie E Cohen; Pinchas Cohen; Jan Frystyk; Adda Grimberg; Yukihiro Hasegawa; Morey W Haymond; Ken Ho; Andrew R Hoffman; Jeff M P Holly; Reiko Horikawa; Charlotte Höybye; Jens Otto Lunde Jorgensen; Gudmundur Johannsson; Anders Juul; Laurence Katznelson; John J. Kopchick; K O Lee; Kuk-Wha Lee; Xiaoping Luo; Shlomo Melmed; Bradley S Miller; Madhusmita Misra; Vera Popovic; Ron G Rosenfeld; Judith Ross; Richard J Ross; Paul Saenger; Christian J Strasburger; Michael O Thorner; Haim Werner; Kevin C J Yuen

doi:10.1530/EJE-16-0111

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

Jens Sandahl Christiansen, Philippe F Backeljauw, Martin Bidlingmaier, Beverly M K Biller, Margaret C S Boguszewski, Felipe F Casanueva, Philippe Chanson, Pierre Chatelain, Catherine S Choong, David R Clemmons, Laurie E Cohen, Pinchas Cohen, Jan Frystyk, Adda Grimberg, Yukihiro Hasegawa, Morey W Haymond, Ken Ho, Andrew R Hoffman, Jeff M P Holly, Reiko HorikawaCharlotte Höybye, Jens Otto Lunde Jorgensen, Gudmundur Johannsson, Anders Juul, Laurence Katznelson, John J. Kopchick, K O Lee, Kuk-Wha Lee, Xiaoping Luo, Shlomo Melmed, Bradley S Miller, Madhusmita Misra, Vera Popovic, Ron G Rosenfeld, Judith Ross, Richard J Ross, Paul Saenger, Christian J Strasburger, Michael O Thorner, Haim Werner, Kevin C J Yuen

Institut for Klinisk Medicin

58 Citationer (Scopus)

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.

Originalsprog	Engelsk
Tidsskrift	European Journal of Endocrinology
Vol/bind	174
Udgave nummer	6
Sider (fra-til)	C1-8
Antal sider	8
ISSN	0804-4643
DOI	https://doi.org/10.1530/EJE-16-0111
Status	Udgivet - jun. 2016

Adgang til dokumentet

10.1530/EJE-16-0111

Citationsformater

Christiansen, J. S., Backeljauw, P. F., Bidlingmaier, M., Biller, B. M. K., Boguszewski, M. C. S., Casanueva, F. F., Chanson, P., Chatelain, P., Choong, C. S., Clemmons, D. R., Cohen, L. E., Cohen, P., Frystyk, J., Grimberg, A., Hasegawa, Y., Haymond, M. W., Ho, K., Hoffman, A. R., Holly, J. M. P., ... Yuen, K. C. J. (2016). Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations. European Journal of Endocrinology, 174(6), C1-8. https://doi.org/10.1530/EJE-16-0111

Christiansen, JS, Backeljauw, PF, Bidlingmaier, M, Biller, BMK, Boguszewski, MCS, Casanueva, FF, Chanson, P, Chatelain, P, Choong, CS, Clemmons, DR, Cohen, LE, Cohen, P, Frystyk, J, Grimberg, A, Hasegawa, Y, Haymond, MW, Ho, K, Hoffman, AR, Holly, JMP, Horikawa, R, Höybye, C, Jorgensen, JOL, Johannsson, G, Juul, A, Katznelson, L, Kopchick, JJ, Lee, KO, Lee, K-W, Luo, X, Melmed, S, Miller, BS, Misra, M, Popovic, V, Rosenfeld, RG, Ross, J, Ross, RJ, Saenger, P, Strasburger, CJ, Thorner, MO, Werner, H & Yuen, KCJ 2016, 'Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations', European Journal of Endocrinology, bind 174, nr. 6, s. C1-8. https://doi.org/10.1530/EJE-16-0111

@article{6e4d7c795f694ce2bfef1d246afa0738,

title = "Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations",

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.",

keywords = "Clinical Trials as Topic, Consensus, Dwarfism, Pituitary, Hormone Replacement Therapy, Human Growth Hormone, Humans, Research Design, Journal Article",

author = "Christiansen, {Jens Sandahl} and Backeljauw, {Philippe F} and Martin Bidlingmaier and Biller, {Beverly M K} and Boguszewski, {Margaret C S} and Casanueva, {Felipe F} and Philippe Chanson and Pierre Chatelain and Choong, {Catherine S} and Clemmons, {David R} and Cohen, {Laurie E} and Pinchas Cohen and Jan Frystyk and Adda Grimberg and Yukihiro Hasegawa and Haymond, {Morey W} and Ken Ho and Hoffman, {Andrew R} and Holly, {Jeff M P} and Reiko Horikawa and Charlotte H{\"o}ybye and Jorgensen, {Jens Otto Lunde} and Gudmundur Johannsson and Anders Juul and Laurence Katznelson and Kopchick, {John J.} and Lee, {K O} and Kuk-Wha Lee and Xiaoping Luo and Shlomo Melmed and Miller, {Bradley S} and Madhusmita Misra and Vera Popovic and Rosenfeld, {Ron G} and Judith Ross and Ross, {Richard J} and Paul Saenger and Strasburger, {Christian J} and Thorner, {Michael O} and Haim Werner and Yuen, {Kevin C J}",

note = "{\textcopyright} 2016 The authors.",

year = "2016",

month = jun,

doi = "10.1530/EJE-16-0111",

language = "English",

volume = "174",

pages = "C1--8",

journal = "European Journal of Endocrinology",

issn = "0804-4643",

publisher = "BioScientifica Ltd.",

number = "6",

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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

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

SN - 0804-4643

VL - 174

SP - C1-8

JO - European Journal of Endocrinology

JF - European Journal of Endocrinology

IS - 6

ER -

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

Abstract

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