Настанови щодо лікування гормоном росту та інсуліноподібним фактором росту-1 дітей та підлітків: дефіцит гормона росту, ідіопатична низькорослість і первинний дефіцит інсуліноподібного фактора росту-1
Частина 1
DOI:
https://doi.org/10.30978/UJPE2024-2-3-30Ключові слова:
гормон росту; інсуліноподібний фактор росту-1; діти; лікування; настанова.Анотація
Довідкова інформація/цілі: від імені Комітету з лікарських засобів, терапії та етики Педіатричного ендокринного товариства (Pediatric Endocrine Society (PES)) ми прагнули оновити рекомендації, опубліковані в 2003 р., щодо використання гормона росту (ГР). Оскільки ідіопатична низькорослість (ІН) має суперечливі діагностичні критерії, що часто стирає відмінності між нею, дефіцитом ГР (ДГР) і первинним дефіцитом інсуліноподібного фактора росту-1 (ПДІФР), ми зосередилися на цих трьох діагнозах, уперше додавши рекомбінантний інсуліноподібний фактор росту-1 (ІФР-1) до рекомендацій щодо ГР.
Методи. Ця настанова розроблена з використанням принципу градації рекомендацій, оцінки та експертизи ступеня їхньої обґрунтованості (Grading of Recommendations, Assessment, Development, and Evaluation (GRADE)).
Результати. Ця настанова містить рекомендації щодо клінічного ведення дітей та підлітків із затримкою росту внаслідок ДГР, ІН або ПДІФР, використовуючи найкращі наявні докази.
Висновки. Робоча група пропонує застосовувати рекомендації в клінічній практиці з урахуванням появи нових даних літератури та ризиків і переваг для пацієнта. У багатьох випадках уважний огляд визначає проблеми, які потребують подальшого дослідження.
Посилання
Wilson TA, Rose SR, Cohen P, Rogol AD, Backeljauw P, Brown R, Hardin DS, Kemp SF, Lawson M, Radovick S, Rosenthal SM, Silverman L, Speiser P. Update of guidelines for the use of growth hormone in children: The Lawson Wilkins Pediatric Endocrinology Society Drug and Therapeutics Committee. J Pediatr. 2003;143:415-421.
GRADE Working Group. Grading quality of evidence and strength of recommendations. BMJ. http://doi.org/10.1136/bmj.328.7454.1490.
National Center for Health Statistics. Centers for Disease Control Growth Charts for Children Aged 2 and Older in the United States. 2010. http://www.cdc.gov/growthcharts.
International Classification of Pediatric Endocrine Diagnoses Consortium. International Classification of Pediatric Endocrine Diagnoses. 2016. http://www.icped.org.
Guyatt GH, Schunemann HJ, Djulbegovic B, Akl EA. Guideline panels should not GRADE good practice statements. J Clin Epidemiol. 2014;68:597-600.
Topor LS, Feldman HA, Bauchner H, Cohen LE. Variation in methods of predicting adult height for children with idiopathic short stature. Pediatrics. 2010;126:938-944.
Wit JM, Ranke MB, Albertsson-Wikland K, Carrascosa A, Rosenfeld RG, Van Buuren S, Kristrom B, Schoenau E, Audi L, Hokken-Koelega AC, Bang P, Jung H, Blum WF, Silverman LA, Cohen P, Cianfarani S, Deal C, Clayton PE, de Graaff L, Dahlgren J, Kleintjens J, Roelants M. Personalized approach to growth hormone treatment: clinical use of growth prediction models. Horm Res Paediatr. 2013;79:257-270.
Root AW, Dana K, Lippe B. Treatment of growth hormone-deficient infants with recombinant human growth hormone to near-adult height: patterns of growth. Horm Res Paediatr. 2011;75:276-283.
Reiter EO, Price DA, Wilton P, Albertsson-Wikland K, Ranke MB. Effect of growth hormone (GH) treatment on the near-final height of 1,258 patients with idiopathic GH deficiency: analysis of a large international database. J Clin Endocrinol Metab. 2006;91:2047-2054.
Cutfield W, Lindberg A, Albertsson Wikland K, Chatelain P, Ranke MB, Wilton P. Final height in idiopathic growth hormone deficiency: the KIGS experience. KIGS International Board. Acta Paediatr Suppl. 1999;88:72-75.
Blethen SL, Baptista J, Kuntze J, Foley T, LaFranchi S, Johanson A. Adult height in growth hormone (GH)-deficient children treated with biosynthetic GH. The Genentech Growth Study Group. J Clin Endocrinol Metab. 1997;82:418-420.
Carel JC, Ecosse E, Nicolino M, Tauber M, Leger J, Cabrol S, Bastie-Sigeac I, Chaussain JL, Coste J. Adult height after long term treatment with recombinant growth hormone for idiopathic isolated growth hormone deficiency: observational follow up study of the French population based registry. BMJ. 2002;325:70.
Brownstein CM, Mertens AC, Mitby PA, Stovall M, Qin J, Heller G, Robison LL, Sklar CA. Factors that affect final height and change in height standard deviation scores in survivors of childhood cancer treated with growth hormone: a report from the childhood cancer survivor study. J Clin Endocrinol Metab. 2004;89:4422-4427.
Radetti G, Buzi F, Paganini C, Pilotta A, Felappi B. Treatment of GH-deficient children with two different GH doses: effect on final height and cost-benefit implications. Eur J Endocrinol. 2003;148:515-518.
Rachmiel M, Rota V, Atenafu E, Daneman D, Hamilton J. Final height in children with idiopathic growth hormone deficiency treated with a fixed dose of recombinant growth hormone. Horm Res. 2007;68:236-243.
Cacciari E, Cicognani A, Pirazzoli P, Zucchini S, Salardi S, Balsamo A, Cassio A, Pasini A, Carla G, Tassinari D, Gualandi S. Final height of patients treated for isolated GH deficiency: examination of 83 patients. Eur J Endocrinol. 1997;137:53-60.
Thomas M, Massa G, Bourguignon JP, Craen M, De Schepper J, de Zegher F, Dooms L, Du Caju M, Francois I, Heinrichs C, Malvaux P, Rooman R, Thiry-Counson G, Vandeweghe M, Maes M. Final height in children with idiopathic growth hormone deficiency treated with recombinant human growth hormone: the Belgian experience. Horm Res. 2001;55:88-94.
Wit JM, Kamp GA, Rikken B. Spontaneous growth and response to growth hormone treatment in children with growth hormone deficiency and idiopathic short stature. Pediatr Res. 1996;39:295-302.
Carroll PV, Christ ER, Bengtsson BA, Carlsson L, Christiansen JS, Clemmons D, Hintz R, Ho K, Laron Z, Sizonenko P, Sonksen PH, Tanaka T, Thorne M. Growth hormone deficiency in adulthood and the effects of growth hormone replacement: a review. Growth Hormone Research Society Scientific Committee. J Clin Endocrinol Metab. 1998;83:382-395.
Molitch ME, Clemmons DR, Malozowski S, Merriam GR, Vance ML. Evaluation and treatment of adult growth hormone deficiency: an Endocrine Society clinical practice guideline. J Clin Endocrinol Metab. 2011;96:1587-1609.
Molitch ME, Clemmons DR, Malozowski S, Merriam GR, Shalet SM, Vance ML. Clinical practice guideline - evaluation and treatment of adult growth hormone deficiency: an endocrine society clinical practice guideline. J Clin Endocrinol Metab. 2006;91:1621-1634.
Capalbo D, Mattace Raso G, Esposito A, Di Mase R, Barbieri F, Meli R, Bruzzese D, Salerno M. Cluster of cardiometabolic risk factors in children with GH deficiency: a prospective, case-control study. Clin Endocrinol (Oxf). 2014;80:856-862.
Lanes R, Paoli M, Carrillo E, Villaroel O, Palacios A. Peripheral inflammatory and fibrinolytic markers in adolescents with growth hormone deficiency: relation to postprandial dyslipidemia. J Pediatr. 2004;145:657-661.
Shulman DI, Root AW, Diamond FB, Bercu BB, Martinez R, Boucek RJ Jr. Effects of one year of recombinant human growth hormone (GH) therapy on cardiac mass and function in children with classical GH deficiency. J Clin Endocrinol Metab. 2003;88:4095-4099.
Metwalley KA, Farghaly HS, Abd El-Hafeez HA. Evaluation of left ventricular mass and function, lipid profile, and insulin resistance in Egyptian children with growth hormone deficiency: a single-center prospective case-control study. Indian J Endocrinol Metab. 2013;17:876-882.
Salerno M, Esposito V, Farina V, Radetti G, Umbaldo A, Capalbo D, Spinelli L, Muzzica S, Lombardi G, Colao A. Improvement of cardiac performance and cardiovascular risk factors in children with GH deficiency after two years of GH replacement therapy: an observational, open, prospective, case-control study. J Clin Endocrinol Metab. 2006;91:1288-1295.
Salerno M, Esposito V, Spinelli L, Di Somma C, Farina V, Muzzica S, et al. Left ventricular mass and function in children with GH deficiency before and during 12 months GH replacement therapy. Clin Endocrinol (Oxf). 2004;60:630-636.
Capalbo D, Lo Vecchio A, Farina V, Spinelli L, Palladino A, Tiano C, Lettiero T, Lombardi G, Colao A, Salerno M. Subtle alterations of cardiac performance in children with growth hormone deficiency: results of a two-year prospective, case-control study. J Clin Endocrinol Metab. 2009;94:3347-3355.
Boot AM, Engels MA, Boerma GJ, Krenning EP, De Muinck Keizer-Schrama SM. Changes in bone mineral density, body composition, and lipid metabolism during growth hormone (GH) treatment in children with GH deficiency. J Clin Endocrinol Metab. 1997;82:2423-2428.
Kuromaru R, Kohno H, Ueyama N, Hassan HM, Honda S, Hara T. Long-term prospective study of body composition and lipid profiles during and after growth hormone (GH) treatment in children with GH deficiency: gender-specific metabolic effects. J Clin Endocrinol Metab. 1998;83:3890-3896.
van der Sluis IM, Boot AM, Hop WC, De Rijke YB, Krenning EP, de Muinck Keizer-Schrama SM. Long-term effects of growth hormone therapy on bone mineral density, body composition, and serum lipid levels in growth hormone deficient children: a 6-year follow-up study. Horm Res. 2002;58:207-214.
Gonc EN, Kandemir N. Long-term effects of growth hormone (GH) on bone mineral status and bone turnover markers in patients with isolated GH deficiency and multiple pituitary hormone deficiency. Clin Endocrinol (Oxf). 2007;66:672-677.
Lanes R, Soros A, Flores K, Gunczler P, Carrillo E, Bandel J. Endothelial function, carotid artery intima-media thickness, epicardial adipose tissue, and left ventricular mass and function in growth hormone-deficient adolescents: apparent effects of growth hormone treatment on these parameters. J Clin Endocrinol Metab. 2005;90:3978-3982.
Ciresi A, Amato MC, Criscimanna A, Mattina A, Vetro C, Galluzzo A, D’Acquisto G, Giordano C. Metabolic parameters and adipokine profile during GH replacement therapy in children with GH deficiency. Eur J Endocrinol. 2007;156:353-360.
Lanes R, Soros A, Gunczler P, Paoli M, Carrillo E, Villaroel O, Palacios A. Growth hormone deficiency, low levels of adiponectin, and unfavorable plasma lipid and lipoproteins. J Pediatr. 2006;149:324-329.
Foster C, Burton A, Scholl J, Scott ML, Gunter V, McCormick K. Lipid patterns in treated growth hormone deficient children vs. short stature controls. J Pediatr Endocrinol Metab. 2014;27:909-914.
Martin M, Wilkins L. Pituitary dwarfism: diagnosis and treatment. J Clin Endocrinol Metab. 1958;18:679-693.
Coutant R, Rouleau S, Despert F, Magontier N, Loisel D, Limal JM. Growth and adult height in GH-treated children with nonacquired GH deficiency and idiopathic short stature: the influence of pituitary magnetic resonance imaging findings. J Clin Endocrinol Metab. 2001;86:4649-4654.
Deal C, Hasselmann C, Pfaffle RW, Zimmermann AG, Quigley CA, Child CJ, Shavrikova EP, Cutler GB Jr, Blum WF. Associations between pituitary imaging abnormalities and clinical and biochemical phenotypes in children with congenital growth hormone deficiency: data from an international observational study. Horm Res Paediatr. 2013;79:283-292.
Maghnie M, Lindberg A, Koltowska-Häggström M, Ranke MB. Magnetic resonance imaging of CNS in 15 043 children with GH deficiency in KIGS (Pfizer International Growth Database). Eur J Endocrinol. 2013;168:211-217.
MacGillivray M, Clopper R, Sandberg D, Buchlis J, Irizarry L. Final height outcomes of hypopituitary children after growth hormone (GH) treatment during the pituitary GH and recombinant GH eras. Pediatr Res. 1997;41:69.
Frisch H, Birnbacher R. Final height and pubertal development in children with growth hormone deficiency after long-term treatment. Horm Res. 1995;43:132-134.
Dean HJ, McTaggart TL, Fish DG, Friesen HG. The educational, vocational, and marital status of growth hormone-deficient adults treated with growth hormone during childhood. Am J Dis Child. 1985;139:1105-1110.
Binder G, Weidenkeller M, Blumenstock G, Langkamp M, Weber K, Franz AR. Rational approach to the diagnosis of severe growth hormone deficiency in the newborn. J Clin Endocrinol Metab. 2010;95:2219-2226.
Ogilvy-Stuart AL, Hands SJ, Adcock CJ, Holly JM, Matthews DR, Mohamed-Ali V, Yudkin JS, Wilkinson AR, Dunger DB. Insulin, insulin-like growth factor I (IGF-I), IGF-binding protein-1, growth hormone, and feeding in the newborn. J Clin Endocrinol Metab. 1998;83:3550-3557.
Cornblath M, Parker ML, Reisner SH, Forbes AE, Daughaday WH. Secretion and metabolism of growth hormone in premature and full-term infants. J Clin Endocrinol Metab. 1965;25:209-218.
Mehta A, Hindmarsh PC, Stanhope RG, Turton JP, Cole TJ, Preece MA, Dattani MT. The role of growth hormone in determining birth size and early postnatal growth, using congenital growth hormone deficiency (GHD) as a model. Clin Endocrinol (Oxf). 2005;63:223-231.
Binder G, Martin DD, Kanther I, Schwarze CP, Ranke MB. The course of neonatal cholestasis in congenital combined pituitary hormone deficiency. J Pediatr Endocrinol Metab. 2007;20:695-702.
Kelly A, Tang R, Becker S, Stanley CA. Poor specificity of low growth hormone and cortisol levels during fasting hypoglycemia for the diagnoses of growth hormone deficiency and adrenal insufficiency. Pediatrics. 2008;122: e522-e528.
Hawkes CP, Grimberg A. Measuring growth hormone and insulin-like growth factor-I in infants: what is normal? Pediatr Endocrinol Rev. 2013;11:126-146.
Cole TJ, Hindmarsh PC, Dunger DB. Growth hormone (GH) provocation tests and the response to GH treatment in GH deficiency. Arch Dis Child. 2004;89:1024-1027.
Wilson DM, Frane J. A brief review of the use and utility of growth hormone stimulation testing in the NCGS: do we need to do provocative GH testing? Growth Horm IGF Res. 2005;15(suppl A):S21-S25.
Rogol AD, Blethen SL, Sy JP, Veldhuis JD. Do growth hormone (GH) serial sampling, insulin-like growth factor-I (IGF-I) or auxological measurements have an advantage over GH stimulation testing in predicting the linear growth response to GH therapy? Clin Endocrinol (Oxf). 2003;58:229-237.
Zadik Z, Chalew SA, Kowarski A. Assessment of growth hormone secretion in normal stature children using 24-hour integrated concentration of GH and pharmacological stimulation. J Clin Endocrinol Metab. 1990;71:932-936.
Ghigo E, Bellone J, Aimaretti G, Bellone S, Loche S, Cappa M, Bartolotta E, Dammacco F, Camanni F. Reliability of provocative tests to assess growth hormone secretory status. Study in 472 normally growing children. J Clin Endocrinol Metab. 1996;81: 3323-3327.
Ranke MB, Lindberg A, Chatelain P, Wilton P, Cutfield W, Albertsson-Wikland K, Price DA. Derivation and validation of a mathematical model for predicting the response to exogenous recombinant human growth hormone (GH) in prepubertal children with idiopathic GH deficiency. J Clin Endocrinol Metab. 1999;84:1174-1183.
Bright GM, Julius JR, Lima J, Blethen SL. Growth hormone stimulation test results as predictors of recombinant human growth hormone treatment outcomes: preliminary analysis of the National Cooperative Growth Study database. Pediatrics. 1999;104: 1028-1031.
Fujieda K, Hanew K, Hirano T, Igarashi Y, Nishi Y, Tachibana K, Takano K, Tanaka T, Yokoya S. Growth response to growth hormone therapy in patients with different degrees of growth hormone deficiency. Endocr J. 1996;43:S19-S25.
Carel JC, Tresca JP, Letrait M, Chaussain JL, Lebouc Y, Job JC, Coste J. Growth hormone testing for the diagnosis of growth hormone deficiency in childhood: a population register-based study. J Clin Endocrinol Metab. 1997;82:2117-2121.
Stanley TL, Levitsky LL, Grinspoon SK, Misra M. Effect of body mass index on peak growth hormone response to provocative testing in children with short stature. J Clin Endocrinol Metab. 2009;94:4875-4881.
Loche S, Guzzetti C, Pilia S, Ibba A, Civolani P, Porcu M, Minerba L, Casini MR. Effect of body mass index on the growth hormone response to clonidine stimulation testing in children with short stature. Clin Endocrinol (Oxf). 2011;74:726-731.
Argente J, Caballo N, Barrios V, Pozo J, Munoz MT, Chowen JA, Hernandez M. Multiple endocrine abnormalities of the growth hormone and insulin-like growth factor axis in prepubertal children with exogenous obesity: effect of short- and long-term weight reduction. J Clin Endocrinol Metab. 1997;82:2076-2083.
Dichtel LE, Yuen KC, Bredella MA, Gerweck AV, Russell BM, Riccio AD, Gurel MH, Sluss PM, Biller BM, Miller KK. Overweight/Obese adults with pituitary disorders require lower peak growth hormone cutoff values on glucagon stimulation testing to avoid overdiagnosis of growth hormone deficiency. J Clin Endocrinol Metab. 2014;99:4712-4719.
Clemmons DR. Consensus statement on the standardization and evaluation of growth hormone and insulin-like growth factor assays. Clin Chem. 2011;57:555-559.
Ross HA, Lentjes EWGM, Menheere PMM, Sweep CGJ. Harmonization of growth hormone measurement results: the empirical approach. Clin Chim Acta. 2014;432:72-76.
Hauffa BP, Lehmann N, Bettendorf M, Mehls O, Dorr HG, Partsch CJ, Schwarz HP, Stahnke N, Steinkamp H, Said E, Sander S, Ranke MB. Central reassessment of GH concentrations measured at local treatment centers in children with impaired growth: consequences for patient management. Eur J Endocrinol. 2004;150:291-297.
Rakover Y, Lavi I, Masalah R, Issam T, Weiner E, Ben-Shlomo I. Comparison between four immunoassays for growth hormone (GH) measurement as guides to clinical decisions following GH provocative tests. J Pediatr Endocrinol Metab. 2000;13:637-643.
Morsky P, Tiikkainen U, Ruokonen A, Markkanen H. Problematic determination of serum growth hormone: experience from external quality assurance surveys. 1998-2003. Scand J Clin Lab Invest. 2005;65:377-386.
Muller A, Scholz M, Blankenstein O, Binder G, Pfaffle R, Korner A, Kiess W, Heider A, Bidlingmaier M, Thiery J, Kratzsch J. Harmonization of growth hormone measurements with different immunoassays by data adjustment. Clin Chem Lab Med. 2011;49:1135-1142.
Tanaka T, Tachibana K, Shimatsu A, Katsumata N, Tsushima T, Hizuka N, Fujieda K, Yokoya S, Irie M. A nationwide attempt to standardize growth hormone assays. Horm Res. 2005;64(suppl 2):6-11.
Butenandt O, Kunze D. Growth velocity in constitutional delay of growth and development. J Pediatr Endocrinol Metab. 2010;23:19-25.
Saggese G, Cesaretti G, Giannessi N, Bracaloni C, Cinquanta L, Cioni C. Stimulated growth hormone (GH) secretion in children with delays in pubertal development before and after the onset of puberty: relationship with peripheral plasma GH-releasing hormone and somatostatin levels. J Clin Endocrinol Metab. 1992;74:272-278.
Martinez AS, Domene HM, Ropelato MG, Jasper HG, Pennisi PA, Escobar ME, Heinrich JJ. Estrogen priming effect on growth hormone (GH) provocative test: a useful tool for the diagnosis of GH deficiency. J Clin Endocrinol Metab. 2000;85:4168-4172.
Marin G, Domene HM, Barnes KM, Blackwell BJ, Cassorla FG, Cutler GB Jr: The effects of estrogen priming and puberty on the growth hormone response to standardized treadmill exercise and arginine-insulin in normal girls and boys. J Clin Endocrinol Metab. 1994;79:537-541.
Gonc EN, Kandemir N, Ozon A, Alikasifoglu A. Final heights of boys with normal growth hormone responses to provocative tests following priming. J Pediatr Endocrinol Metab. 2008;21:963-971.
Wetterau LA. The pros and cons of sex steroid priming in growth hormone stimulation testing. J Pediatr Endocrinol Metab. 2012;25:1049-1055.
Spiliotis BE, August GP, Hung W, Sonis W, Mendelson W, Bercu BB. Growth hormone neurosecretory dysfunction. A treatable cause of short stature. JAMA. 1984;251:2223-2230.
Kowarski A, Thompson RG, Migeon CJ, Blizzard RM. Determination of integrated plasma concentrations and true secretion rates of human growth hormone. J Clin Endocrinol Metab. 1971;32: 356-360.
Rochiccioli P, Dechaux E, Tauber MT, Pienkowski C, Tiberge M. Growth hormone treatment in patients with neurosecretory dysfunction. Horm Res. 1990;33(suppl 4):97-101.
Radetti G, Buzi F, Cassar W, Paganini C, Stacul E, Maghnie M. Growth hormone secretory pattern and response to treatment in children with short stature followed to adult height. Clin Endocrinol (Oxf). 2003;59:27-33.
Hernandez M, Nieto JA, Sobradillo B, Pombo M, Ferrandez A, Rejas J. Multicenter clinical trial to evaluate the therapeutic use of recombinant growth hormone from mammalian cells in the treatment of growth hormone neurosecretory dysfunction. Horm Res. 1991;35:13-18.
Lanes R. Diagnostic limitations of spontaneous growth hormone measurements in normally growing prepubertal children. Am J Dis Child. 1989;143:1284-1286.
Rose SR, Ross JL, Uriarte M, Barnes KM, Cassorla FG, Cutler GB Jr: The advantage of measuring stimulated as compared with spontaneous growth hormone levels in the diagnosis of growth hormone deficiency. N Engl J Med. 1988;319:201-207.
Blizzard R, Johanson A. Disorders of growth; in Kappy M, Blizzard R, Migeon C (eds): Wilkins - The Diagnosis and Treatment of Endocrine Disorders in Childhood and Adolescence. Springfield, Charles C Thomas. 1994, pp 383-455.
Mauras N, Attie KM, Reiter EO, Saenger P, Baptista J, Genentech I, Cooperative Study G. High dose recombinant human growth hormone (GH) treatment of GH-deficient patients in puberty increases near-final height: a randomized, multicenter trial. J Clin Endocrinol Metab. 2000;85:3653-3660.
Rikken B, Massa GG, Wit JM, Delamarre-van de Waal, HA, Drayer NM, de Muinck Keizer-Schrama SM, Oostdijk W, Otten BJ, Reeser HM, Jansen M, Vulsma T, Waelkens JJJ, Zelissen PMJ. Final height in a large cohort of dutch patients with growth hormone deficiency treated with growth hormone. Horm Res. 1995;43:135-137.
Chipman JJ, Hicks JR, Holcombe JH, Draper MW. Approaching final height in children treated for growth hormone deficiency. Horm Res. 1995;43:129-131.
De Luca F, Maghnie M, Arrigo T, Lombardo F, Messina MF, Bernasconi S. Final height outcome of growth hormone-deficient patients treated since less than five years of age. Acta Paediatr. 1996;85:1167-1171.
Juul A, Bernasconi S, Clayton PE, Kiess W, DeMuinck-Keizer Schrama S, Drugs and Therapeutics Committee of the European Society for Paediatric Endocrinology (ESPE): European audit of current practice in diagnosis and treatment of childhood growth hormone deficiency. Horm Res. 2002;58:233-241.
Hughes IP, Harris M, Cotterill A, Ambler G, Cowell CT, Cutfield S, Werther G, Choong CS. Comparison of weight versus body surface area based growth hormone dosing for children: implications for response. Clin Endocrinol (Oxf). 2013;80:384-394.
Cohen P, Germak J, Rogol AD, Weng W, Kappelgaard AM, Rosenfeld RG. Variable degree of growth hormone (GH) and insulin-like growth factor (IGF) sensitivity in children with idiopathic short stature compared with GH-deficient patients: evidence from an IGF-based dosing study of short children. J Clin Endocrinol Metab. 2010;95:2089-2098.
Cohen P, Weng W, Rogol AD, Rosenfeld RG, Kappelgaard AM, Germak J. Dose-sparing and safety-enhancing effects of an IGF-I-based dosing regimen in short children treated with growth hormone in a 2-year randomized controlled trial: therapeutic and pharmacoeconomic considerations. Clin Endocrinol (Oxf). 2014;81:71-76.
MacGillivray MH, Baptista J, Johanson A. Outcome of a four-year randomized study of daily versus three times weekly somatropin treatment in prepubertal naive growth hormone-deficient children. Genentech Study Group. J Clin Endocrinol Metab. 1996;81:1806-1809.
Rosenbloom AL, Knuth C, Shulman D. Growth hormone by daily injection in patients previously treated for growth hormone deficiency. South Med J. 1990;83:653-655.
Burns EC, Tanner JM, Preece MA, Cameron N. Final height and pubertal development in 55 children with idiopathic growth hormone deficiency, treated for between 2 and 15 years with human growth hormone. Eur J Pediatr. 1981;137:155-164.
Hibi I, Tanaka T. Final height of patients with idiopathic growth hormone deficiency after long-term growth hormone treatment. Committee for Treatment of Growth Hormone Deficient Children, Growth Science Foundation, Japan. Acta Endocrinol (Copenh). 1989;120:409-415.
de Muinck Keizer-Schrama S, Van den Broeck J, Sas T, Hokken-Koelega A. Final height of growth hormone-treated GH-deficient children and girls with Turner’s syndrome: the Dutch experience. The Dutch Advisory Group on Growth Hormone. Horm Res. 1999;51(suppl 3):127-131.
Sas TC, de Ridder MA, Wit JM, Rotteveel J, Oostdijk W, Reeser HM, Otten BJ, de Muinck Keizer-Schrama SM. Adult height in children with growth hormone deficiency: a randomized, controlled, growth hormone dose-response trial. Horm Res Paediatr. 2010;74:172-181.
Wikland KA, Kristrom B, Rosberg S, Svensson B, Nierop AF. Validated multivariate models predicting the growth response to GH treatment in individual short children with a broad range in GH secretion capacities. Pediatr Res. 2000;48:475-484.
Ranke MB, Lindberg A, Mullis PE, Geffner ME, Tanaka T, Cutfield WS, Tauber M, Dunger D. Towards optimal treatment with growth hormone in short children and adolescents: evidence and theses. Horm Res Paediatr. 2013;79:51-67.
Kristrom B, Aronson AS, Dahlgren J, Gustafsson J, Halldin M, Ivarsson SA, Nilsson NO, Svensson J, Tuvemo T, Albertsson-Wikland K. Growth hormone (GH) dosing during catch-up growth guided by individual responsiveness decreases growth response variability in prepubertal children with GH deficiency or idiopathic short stature. J Clin Endocrinol Metab. 2009;94: 483-490.
Cohen P, Bright GM, Rogol AD, Kappelgaard AM, Rosenfeld RG. Effects of dose and gender on the growth and growth factor response to GH in GH-deficient children: implications for efficacy and safety. J Clin Endocrinol Metab. 2002;87:90-98.
Burgers AMG, Biermasz NR, Schoones JW, Pereira AM, Renehan AG, Zwahlen M, Egger M, Dekkers OM. Meta-analysis and dose-response metaregression: circulating insulin-like growth factor I (IGF-I) and mortality. J Clin Endocrinol Metab. 2011;96:2912-2920.
Chanson P, Arnoux A, Mavromati M, Brailly-Tabard S, Massart C, Young J, Piketty ML, Souberbielle JC; VARIETE Investigators: Reference values for insulin-like growth factor I (IGF-I) serum concentrations: comparison of six immunoassays. J Clin Endocrinol Metab. 2016;101:3450-3458.
Renehan AG, Zwahlen M, Minder C, O’Dwyer ST, Shalet SM, Egger M. Insulin-like growth factor (IGF)-I, IGF binding protein-3, and cancer risk: systematic review and meta-regression analysis. Lancet. 2004;363:1346-1353.
Schairer C, McCarty CA, Isaacs C, Sue LY, Pollak MN, Berg CD, Ziegler RG. Circulating insulin-like growth factor (IGF)-I and IGF binding protein (IGFBP)-3 levels and postmenopausal breast cancer risk in the prostate, lung, colorectal, and ovarian cancer screening trial (PLCO) cohort. Horm Cancer. 2010;1:100-111.
Douglas JB, Silverman DT, Pollak MN, Tao Y, Soliman AS, Stolzenberg-Solomon RZ. Serum IGF-I, IGF-II, IGFBP-3, and IGF-I/IGFBP-3 molar ratio and risk of pancreatic cancer in the prostate, lung, colorectal, and ovarian cancer screening trial. Cancer Epidemiol Biomarkers Prev. 2010;19:2298-2306.
Stanhope R, Uruena M, Hindmarsh P, Leiper AD, Brook CG. Management of growth hormone deficiency through puberty. Acta Paediatr Scand Suppl. 1991;372:47-52; discussion 53.
Albertsson Wikland K, Alm F, Aronsson S, Gustafsson J, Hagenas L, Hager A, et al. Effect of growth hormone (GH) during puberty in GH-deficient children: preliminary results from an ongoing randomized trial with different dose regimens. Acta Paediatr Suppl. 1999;88:80-84.
Coelho R, Brook CG, Preece MA, Stanhope RG, Dattani MT, Hindmarsh PC. A randomised study of two doses of biosynthetic human growth hormone on final height of pubertal children with growth hormone deficiency. Horm Res. 2008;70:85-88.
Ranke MB, Lindberg A. Observed and predicted total pubertal growth during treatment with growth hormone in adolescents with idiopathic growth hormone deficiency, Turner syndrome, short stature, born small for gestational age and idiopathic short stature: KIGS analysis and review. Horm Res Paediatr. 2011;75:423-432.
Tauber M, Moulin P, Pienkowski C, Jouret B, Rochiccioli P. Growth hormone (GH) retesting and auxological data in 131 GH-deficient patients after completion of treatment. J Clin Endocrinol Metab. 1997;82:352-356.
Loche S, Bizzarri C, Maghnie M, Faedda A, Tzialla C, Autelli M, Casini MR, Cappa M. Results of early reevaluation of growth hormone secretion in short children with apparent growth hormone deficiency. J Pediatr. 2002;140:445-449.
Zucchini S, Pirazzoli P, Baronio F, Gennari M, Bal MO, Balsamo A, Gualandi S, Cicognani A. Effect on adult height of pubertal growth hormone retesting and withdrawal of therapy in patients with previously diagnosed growth hormone deficiency. J Clin Endocrinol Metab. 2006;91:4271-4276.