Diagnosis, Genetics, and Therapy of Short Stature in Children: A Growth Hormone Research Society International Perspective

Paulo F. Collett-Solberg, Geoffrey Ambler, Philippe F. Backeljauw, Martin Bidlingmaier, Beverly M.K. Biller, Margaret C.S. Boguszewski, Pik To Cheung, Catherine Seut Yhoke Choong, Laurie E. Cohen, Pinchas Cohen, Andrew Dauber, Cheri L. Deal, Chunxiu Gong, Yukihiro Hasegawa, Andrew R. Hoffman, Paul L. Hofman, Reiko Horikawa, Alexander A.L. Jorge, Anders Juul, Peter KamenickýVaman Khadilkar, John J. Kopchick, Berit Kriström, Maria De Lurdes A. Lopes, Xiaoping Luo, Bradley S. Miller, Madhusmita Misra, Irene Netchine, Sally Radovick, Michael B. Ranke, Alan D. Rogol, Ron G. Rosenfeld, Paul Saenger, Jan M. Wit, Joachim Woelfle

Research output: Contribution to journalArticlepeer-review

19 Scopus citations

Abstract

The Growth Hormone Research Society (GRS) convened a Workshop in March 2019 to evaluate the diagnosis and therapy of short stature in children. Forty-six international experts participated at the invitation of GRS including clinicians, basic scientists, and representatives from regulatory agencies and the pharmaceutical industry. Following plenary presentations addressing the current diagnosis and therapy of short stature in children, breakout groups discussed questions produced in advance by the planning committee and reconvened to share the group reports. A writing team assembled one document that was subsequently discussed and revised by participants. Participants from regulatory agencies and pharmaceutical companies were not part of the writing process. Short stature is the most common reason for referral to the pediatric endocrinologist. History, physical examination, and auxology remain the most important methods for understanding the reasons for the short stature. While some long-standing topics of controversy continue to generate debate, including in whom, and how, to perform and interpret growth hormone stimulation tests, new research areas are changing the clinical landscape, such as the genetics of short stature, selection of patients for genetic testing, and interpretation of genetic tests in the clinical setting. What dose of growth hormone to start, how to adjust the dose, and how to identify and manage a suboptimal response are still topics to debate. Additional areas that are expected to transform the growth field include the development of long-acting growth hormone preparations and other new therapeutics and diagnostics that may increase adult height or aid in the diagnosis of growth hormone deficiency.

Original languageEnglish (US)
Pages (from-to)1-14
Number of pages14
JournalHormone Research in Paediatrics
Volume92
Issue number1
DOIs
StatePublished - Dec 1 2019

Bibliographical note

Funding Information:
G.A., M.B., P.T.C., C.G., Y.H., P.L.H., R.H., V.K., and X.L. have no conflicts of interest to declare; P.F.B. was a consultant for Novo Nordisk and received research funding from Novo Nordisk, Ip-sen, and Opko; B.M.K.B. was a principal investigator of research grants to the Massachusetts General Hospital from Novo Nordisk and Opko, and a recipient of consulting honoraria from Merck Serono, Novo Nordisk, Pfizer, and Strongbridge; M.C.S.B. received speaker fees from Pfizer; C.S.Y.C. is an investigator for studies funded by Opko and Merck Serono; L.E.C. is an investigator for studies funded by Ascendis and Opko; P.C. was a consultant for Ascendis, Genexine, GenSci, and Opko; P.F.C.-S. received travel grants and speaker fees from Pfizer, Merck-Serono, Novo Nordisk, and Sandoz; A.D. was a consultant for Opko Biologics, Novo Nordisk, Pfizer, and Ipsen, and received research funding from Novo Nordisk and Ipsen and holds a patent for the use of recombinant PAPPA2 as a growth promoting therapy; C.L.D. served on the GeNeSIS International Advisory Board (Lilly) and as an ad hoc consultant for Lilly, EMD Serono/Merck, Hoffman LaRoche, Sandoz, Pfizer, Versartis, Opko, and Lumos, received speaker fees from Lilly, EMD Serono/Merck, Hoffman LaRoche, Sandoz, Pfizer, and Novo Nordisk, and research support from Lilly, EMD Serono, Hoffman LaRoche, Pfizer, Sandoz, Versartis, and Opko. A.R.H. acts as consultant for Ascendis, Genexine, and Novo Nordisk; A.A.L.J. received speaker fees from Sandoz; A.J. is the principal investigator of a multicenter study on effects of GH in short SGA children (North European Small for Gestational Age Study, NESGAS) which received unrestricted financial support from Novo Nordisk, received speaker fees from Sandoz, Ipsen, Novo Nordisk, and Merck; P.K. received a grant from Novartis and was a consultant for Ipsen; J.J.K. was a consultant for Sandoz and Merck KGaA and received speaker fees from Pfizer; B.K. received speaker fees from Merck Darmstadt, Novo Nordisk, Pfizer, Sandoz, and GeneScience Pharmaceuticals, was an investigator for the PATRO observational study (Sandoz), and participated in the Pfizer iGRO board; M.L.A.L. received travel grants from several Pharmaceutical Companies; B.S.M. was a consultant for Abb-vie, Ascendis, BioMarin, Bluebird Bio, Novo Nordisk, Pfizer, Sandoz, Sanofi Genzyme, and Tolmar, and has received research support from Alexion, Abbvie, Amgen, Ascendis, BioMarin, Novo Nordisk, Opko, Protalix, Sandoz, Sangamo, Sanofi Genzyme, Tol-mar, and Takeda; M.M. was a consultant for Sanofi; co-investigator on an investigator-initiated grant from Novo Nordisk; and received grant funding from NICHD, NIDDK, and NIMH; I.N. received speaker fees from Sandoz and Merck Serono, and research support from Merck Serono and Pfizer; S.R. was a consultant for Ascendis Pharma and CVS-Caremark; M.B.R. received speaker fees from Sandoz, Merck, Mediagnost, and Pfizer; A.D.R. was a consultant to Acerus Pharma, AYTU BioScience, Clarus Pharmaceuticals, Inc, United States Anti-Doping Agency (USADA), and Ultragenyx Pharmaceutics; R.G.R. was a consultant for BioMarin, Opko, Genexine, Ascendis, Lumos, and Æterna Zentaris; P.S. was an investigator for Ascendis, Opko, and Novo Nordisk, and a consultant for Genexine; J.M.W. is member of advisory boards of Opko, Merck, Ammonett, Æterna Zentaris, Agios, and BioMarin, and received speaker fees from Pfizer, Versartis, Sandoz, Lilly, Novo Nordisk, JCR, Merck, and Ipsen; J.W. received research support from Pfizer and Ipsen, speaker fees from Ipsen, Novo Nordisk, Merck, Hexal, and Pfizer, and has attended scientific advisory boards for Ipsen, Novo Nordisk, and Ferring.

Funding Information:
The workshop was supported, in part, by unrestricted educational grants from Æterna Zentaris, BioMarin, Ascendis, Chiasma, Ferring, GeneScience, IDS, Ipsen, Lumos, Merck, Novo Nordisk, Pfizer, Sandoz, and Strongbridge.

Keywords

  • Growth
  • Growth hormone
  • Guideline
  • Pediatrics
  • Treatment

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