Advertisement
Advances in Pediatrics

Clinical Indications for Growth Hormone Therapy

  • Melinda Danowitz
    Affiliations
    CHOP Division of Pediatric Endocrinology, Abramson Building, Office 804F, 3401 Civic Center Boulevard, Philadelphia, PA 19104, USA
    Search for articles by this author
  • Adda Grimberg
    Correspondence
    Corresponding author. CHOP Div Pediatric Endocrinology, The Hub for Clinical Collaboration, 7th floor, 3500 Civic Center Boulevard, Philadelphia, PA 19104.
    Affiliations
    Children’s Hospital of Philadelphia, The Hub for Clinical Collaboration, Division of Endocrinology, 7th floor, 3500 Civic Center Boulevard, Philadelphia, PA 19104, USA

    Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
    Search for articles by this author

      Keywords

      To read this article in full you will need to make a payment

      Purchase one-time access:

      Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online access
      One-time access price info
      • For academic or personal research use, select 'Academic and Personal'
      • For corporate R&D use, select 'Corporate R&D Professionals'

      Subscribe:

      Subscribe to Advances in Pediatrics
      Already a print subscriber? Claim online access
      Already an online subscriber? Sign in
      Institutional Access: Sign in to ScienceDirect

      References

        • Grimberg A.
        • DiVall S.A.
        • Polychronakos C.
        • et al.
        Guidelines for growth hormone and insulin-like growth factor-1 treatment in children children and adolescents: growth hormone deficiency, idiopathic short stature, and primary insulin-like growth factor-1 deficiency.
        Horm Res Paediatr. 2016; 86: 361-397
        • Collett-Solberg P.F.
        • Ambler G.
        • Backeljauw P.F.
        • et al.
        Diagnosis, Genetics, and Therapy of Short Stature in Children: A Growth Hormone Research Society International Perspective.
        Horm Res Paediatr. 2019; 92: 1-14
        • Bosch L.
        • Katugampola H.
        • Dattani M.T.
        Congenital Hypopituitarism During the Neonatal Period: Epidemiology, Pathogenesis, Therapeutic Options, and Outcome.
        Front Pediatr. 2021; 8: 1-17
        • Halas J.G.
        • Grimberg A.
        Dilemmas of Growth Hormone Treatment for GH Deficiency and Idiopathic Short Stature: Defining, Distinguishing, and Deciding.
        Minerva Pediatr. 2020; 72: 206-225
        • Lanes R.
        • Soros A.
        • Gunczler P.
        • et al.
        Growth hormone deficiency, low levels of adiponectin, and unfavorable plasma lipid and lipoproteins.
        J Pediatr. 2006; 149: 324-329
        • Polak M.
        • Blair J.
        • Kotnik P.
        • et al.
        Early growth hormone treatment start in childhood growth hormone deficiency improves near adult height: analysis from NordiNet® International Outcome Study.
        Eur J Endocrinol. 2017; 177: 421-429
        • Hawkes C.P.
        • Grimberg A.
        Measuring Growth Hormone and Insulin-like Growth Factor-1 in Infants: What is Normal?.
        Pediatr Endocrinol Rev. 2013; 11: 126-146
        • Hawkes C.P.
        • Grimberg A.
        Insulin-like growth factor-1 is a marker for the nutritional state.
        Ped Endocrinol Rev. 2015; 13: 499-511
        • Kamoun C.
        • Hawkes C.P.
        • Grimberg A.
        Provocative growth hormone testing in children: how did we get here and where do we go now?.
        J Pediatr Endocrinol Metab. 2021; 34: 679-696
        • Miller J.
        • Lynn C.H.
        • Driscoll D.C.
        • et al.
        Nutritional phases in prader-willi syndrome.
        Am J Med Genet A. 2011; 155a: 1040-1049
        • Goldstone A.P.
        • Holland A.J.
        • Hauffa B.P.
        • et al.
        Recommendations for the diagnosis and management of Prader-Willi Syndrome.
        J Clin Endocrinol Metab. 2008; 93: 4183-4197
        • Cassidy S.B.
        • Driscoll D.J.
        Prader-Willi syndrome.
        Eur J Hum Genet. 2009; 17: 3-13
        • Cassidy S.B.
        • Schwartz S.
        • Miller J.L.
        • et al.
        Prader-Willi syndrome.
        Gen Med. 2012; 14: 10-26
        • Burman P.
        • Ritzen E.M.
        • Lindgren A.C.
        Endocrine Dysfunction in Prader-Willi Syndrome: A Review with Special Reference to GH.
        Endocr Rev. 2001; 22: 787-799
        • Whitman B.
        • Carrel A.
        • Bekx T.
        • et al.
        Growth Hormone Improves Body Composition and Motor Development in Infants with Prader-Willi Syndrome After Six Months.
        J Pediatr Endocrinol Met. 2004; 17: 591-600
        • Colmenares A.
        • Pinto G.
        • Taupin P.
        • et al.
        Effects on Growth and Metabolism of Growth Hormone Treatment for 3 years in 36 Children with Prader-Willi Syndrome.
        Horm Res Paediatr. 2011; 75: 123-130
        • Carrel A.L.
        • Myers S.E.
        • Whitman B.Y.
        • et al.
        Long-term growth hormone therapy changes the natural history of body composition and motor function in children with Prader-Willi Syndrome.
        J Clin Endocrinol Metab. 2010; 95: 1131-1136
        • de Lind van Winjgaarden R.F.A.
        • Otten B.J.
        • Festen D.A.M.
        • et al.
        High Prevalence of Central Adrenal Insufficiency in Patients with Prader-Willi Syndrome.
        J Clin Endocrinol Metab. 2008; 93: 1649-1654
        • Angulo M.A.
        • Butler M.G.
        • Cataletto M.E.
        Prader-Willi syndrome: a review of clinical, genetic, and endocrine findings.
        J Endocrinol Investig. 2015; 38: 1249-1263
        • Clausson B.
        • Gardosi J.
        • Francis A.
        • et al.
        Perinatal outcome in SGA births defined by customized versus population-based birthweight standards.
        BJOG. 2001; 108: 830-834
        • McCowan L.
        • Horgan R.P.
        Risk factors for small for gestational age infants.
        Best Pract Res Clin Obstet Gynaecol. 2009; 23: 779-793
        • Zeve D.
        • Regelmann M.O.
        • Holzman I.R.
        • et al.
        Small at Birth, but How Small? The Definition of SGA Revisited.
        Horm Res Paediatr. 2016; 86: 357-360
        • Albertsson-Wikland K.
        • Karlberg J.
        Natural growth in children born small for gestational age with and without catch-up growth.
        Acta Paediatr. 1994; 399: 64-70
        • Lem A.J.
        • van der Kaay D.C.M.
        • de Ridder M.A.J.
        • et al.
        Adult height in short children born SGA treated with growth hormone and gonadotropin releasing hormone analog: results of a randomized, dose-response GH trial.
        JCEM. 2012; 97: 4096-4105
        • Arends N.J.T.
        • Boonstra V.H.
        • Duivenvoorden J.
        • et al.
        Reduced insulin sensitivity and the presence of cardiovascular risk factors in short prepubertal children born small for gestational age (SGA).
        Clin Endocrinol. 2005; 62: 44-50
        • van Dijk M.
        • Bannink E.M.N.
        • van Pareren Y.K.
        • et al.
        Risk Factors for Diabetes Mellitus Type 2 and Metabolic Syndrome are Comparable for Previously Growth Hormone-Treated Young Adults Born Small for Gestational Age (SGA) and Untreated Short SGA Controls.
        JCEM. 2007; 92: 160-165
        • Cutfiled W.S.
        • Albert B.
        Growth Hormone Treatment for Idiopathic Short Stature.
        Pediatr Endocrinol Rev. 2018; 16: 113-122
        • Albertsson-Wikland K.
        • Aronson A.S.
        • Gutafsson J.
        • et al.
        Dose-dependent effect of growth hormone o final height in children with short stature without growth hormone deficiency.
        JCEM. 2008; 93: 4342-4350
        • Quigley C.A.
        • Crowe B.J.
        • Anglin G.
        • et al.
        Growth Hormone and Low Dose Estrogen in Turner Syndrome: Results of a United States Multi-Center Trial to Near-Final Height.
        JCEM. 2002; 87: 2033-2041
        • Gravholt C.H.
        • Andersen N.H.
        • Conway G.S.
        • et al.
        Clinical practice guidelines for the care of girls and women with Turner syndrome: proceedings from the 2016 Cincinnati International Turner Syndrome Meeting.
        Eur J Endocrinol. 2017; 177: 1-70
        • Gravholt C.H.
        • Viuff M.H.
        • Brun S.
        • et al.
        Turner syndrome: mechanisms and management.
        Nat Rev Endocrinol. 2019; 15: 601-614
        • Shankar R.K.
        • Backejauw P.F.
        Current best practice in the management of Turner syndrome.
        Ther Adv Endocrinol Metab. 2018; 9: 33-40
        • Bolar K.
        • Hoffman A.R.
        • Maneatis T.
        • et al.
        Long-Term Safety of Recombinant Human Growth Hormone in Turner Syndrome.
        JCEM. 2008; 93: 344-351
        • Gawlik A.
        • Hankus M.
        • Such K.
        • et al.
        Hypogonadism and Sex Steroid Replacement Therapy in Girls with Turner Syndrome.
        J Pediatr Adol Gynec. 2016; 29: 542-550
        • O Klein K.
        • Rosenfield R.L.
        • Santen R.J.
        • et al.
        Estrogen Replacement in Turner Syndrome: Literature Review and Practical Considerations.
        JCEM. 2018; 103: 1790-1803
        • Mintz C.S.
        • Seaver L.H.
        • Irons M.
        • et al.
        ACMG Professional Practice and Guidelines Committee. Focused Revision: ACMG practice resource: Genetic evaluation of short stature.
        Genet Med. 2021; 23: 813-815
        • Rappold G.
        • Blum W.F.
        • Shavrikova E.P.
        • et al.
        Genotypes and phenotypes in children with short stature: clinical indicators of SHOX haploinufficiency.
        J Med Genet. 2007; 44: 306-313
        • Fukami M.
        • Seki A.
        • Ogata T.
        SHOX Haploinsufficiency as a Cause of Syndromic and Nonsyndromic Short Stature.
        Mol Syndromol. 2016; 7: 3-11
        • Vannelli S.
        • Baffico M.
        • Buganza R.
        • et al.
        SHOX deficiency in children with growth impairment: evaluation of known and new auxological and radiological indicators.
        Ital J Pediatr. 2020; 46: 163
        • Benabbad I.
        • Rosilio M.
        • Child C.J.
        • et al.
        Growth Hormone-Treated Children with SHOX Deficiency: Data from an Observational Study and a Clinical Trial.
        Horm Res Paediatr. 2017; 87: 42-50
        • Genoni G.
        • Monzani A.
        • Castagno M.
        • et al.
        Improving clinical diagnosis in SHOX deficiency: the importance of growth velocity.
        Pediatr Rese. 2018; 83: 438-444
        • Marstrand-Joergensen M.R.
        • Jensen R.B.
        • Aksglaede L.
        • et al.
        Prevalence of SHOX haploinsufficiency among short statured children.
        Pediatr Res. 2017; 81: 335-341
        • Babu D.
        • Vannelli S.
        • Fanelli A.
        • et al.
        Variants in the 5’UTR reduce SHOX expression and contribute to SHOX haploinsufficiency.
        Eur J Hum Genet. 2021; 29: 110-121
        • Roberts A.E.
        • Araki T.
        • Swanson K.
        • et al.
        Germline gain-of-function mutations in SOS1 cause Noonan syndrome.
        Nat Genet. 2007; 39: 70-74
        • Bhambhani V.
        • Muenke M.
        Noonan Syndrome.
        AFP. 2014; 89: 37-43
        • Tafazoli A.
        • Eshraghi P.
        • Koleti Z.K.
        • et al.
        Noonan syndrome- a new survey.
        Arch Med Sci. 2017; 13: 215-222
        • Kruszka P.
        • Porras A.R.
        • Addissie Y.A.
        • et al.
        Noonan syndrome in diverse populations.
        Am J Med Genet. 2017; 173: 2323-2334
        • Romano A.A.
        • Allanson J.E.
        • Dahlgren J.
        • et al.
        Noonan Syndrome: Clinical Features, Diagnosis, and Management Guidelines.
        Pediatr. 2010; 126: 746-759
        • Seo G.H.
        • Yoo H.-W.
        Growth hormone therapy in patients with Noonan syndrome.
        Ann Pediatr Endocrinol Met. 2018; 23: 176-181
        • Jeong I.
        • Kang E.
        • Cho J.H.
        • et al.
        Long-term efficacy of recombinant human growth hormone therapy in short-statured patients with Noonan syndrome.
        Ann Pediatr Endocrinol Met. 2016; 21: 26-30
        • Siklar Z.
        • Genens M.
        • Poyrazoglu S.
        • et al.
        The Growth Characteristics of Patients with Noonan Syndrome: Results of Three Years of Growth Hormone Treatment: A Nationwide Multicenter Study.
        JCRPE. 2016; 8: 305-312
        • Malaquias A.C.
        • Noronha R.M.
        • Souza T.T.O.
        • et al.
        Impact of Growth Hormone Therapy on Adult Height in Patients with PTPN11 Mutations Related to Noonan Syndrome.
        Horm Res Paediatr. 2019; 91: 252-261
        • Ranke M.B.
        • Lindberg A.
        • Carlsson M.
        • et al.
        Treatment with Growth Hormone in Noonan Syndrome Observed during 25 years of KIGS: Near Adult Height and Outcome Prediction.
        Horm Res Paediatr. 2019; 91: 46-55
        • Raman S.
        • Grimberg A.
        • Waguespack S.G.
        • et al.
        Risk of Neoplasia in Pediatric Patients Receiving Growth Hormone Therapy- A Report From the Pediatric Endocrine Society Drug and Therapeutics Committee.
        JCEM. 2015; 100: 2192-2203
        • Noonan J.A.
        Noonan syndrome and related disorders: Alterations in growth and puberty.
        Rev Endocr Metabol Disord. 2006; 7: 251-255
        • Seikaly M.G.
        • Ho P.L.
        • Emmett L.
        • et al.
        Chronic renal insufficiency in children: The 2001 Annual Report of the NAPRTCS.
        Pediatr Nephrol. 2003; 18: 796-804
        • Groothoff J.W.
        • Offringa M.
        • Grootenhuis M.
        • et al.
        Long-term consequences of renal insufficiency in children: lessons learned from the Dutch LERIC study.
        Nephrol Dial Transplant. 2018; 33: 552-560
        • Mitsnefes M.
        • Ho P.-L.
        • McEnery P.T.
        Hypertension and Progression of Chronic Renal Insufficiency in Children: A Report of the North American Pediatric Renal Transplant Cooperative Study (NAPRTCS).
        J Am Soc Nephrol. 2003; 14: 2618-2622
        • Drube J.
        • Wan M.
        • Bonthius M.
        • et al.
        Clinical practice recommendations for growth hormone treatment in children with chronic kidney disease.
        Nat Rev Nephrol. 2019; 15: 577-589
        • Akchurin O.M.
        • Kogon A.J.
        • Kumar J.
        • et al.
        Approach to growth hormone therapy in children with chronic kidney disease varies across North America: the Midwest Pediatric Nephrology Consortium report.
        BMC Nephrol. 2017; 18: 181
        • Fine R.N.
        • Kohaut E.
        • Brown D.
        • et al.
        Long-term treatment of growth retarded children with chronic renal insufficiency, with recombinant human growth hormone.
        Kidney Int. 1996; 49: 781-785
        • Hokken-Koelega A.
        • Mulder P.
        • De Jong R.
        • et al.
        Long-term effects of growth hormone treatment on growth and puberty in patients with chronic renal insufficiency.
        Pediatr Nephrol. 2000; 14: 701-706
        • Norman L.J.
        • Macdonald I.A.
        • Watson A.R.
        Optimising nutrition in chronic renal insufficiency- growth.
        Pediatr Nephrol. 2004; 19: 1245-1252
      1. Committee on Practice and Ambulatory Medicine, Bright Futures Periodicity Schedule Workgroup. 2021 Recommendations for Preventive Pediatric Health Care.
        Pediatr. 2021; 147 (e2020049776)
        • Collett-Solberg P.F.
        • Jorge A.A.L.
        • Boguszewski M.C.S.
        • et al.
        Growth hormone therapy in children: research and practice- A review.
        Growth Horm IGF Res. 2019; 44: 20-32
        • Grimberg A.
        Cardiovascular Disease in Former Pediatric Recipients of Growth Hormone: Another Look at Growth Hormone Safety.
        JAMA Pediatr. 2021; 175: e205232
        • Souza F.M.
        • Collet-Solberg P.F.
        Adverse effects of growth hormone replacement therapy in children.
        Arq Bras Endocrinol Metab. 2011; 55: 559-565
        • Allen D.B.
        • Backeljauw P.
        • Bidlingmaier M.
        • et al.
        GH safety workshop position paper: a critical appraisal of recombinant human GH therapy in children and adults.
        Eur Soc Endocrinol. 2015; 174: 1-9
        • Grimberg A.
        • Kanter G.P.
        US Growth Hormone Use in the Idiopathic Short Stature Era: Trends in Insurer Payments and Patient Financial Burden.
        J Endocr Soc. 2019; 3: 2023-2031
        • Grimberg A.
        • Allen D.B.
        Growth Hormone Treatment for Growth Hormone Deficiency and Idiopathic Short Stature: New Guidelines Shaped by the Presence and Absence of Evidence.
        Curr Opin Pediatr. 2017; 29: 466-471