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 2
DOI:
https://doi.org/10.30978/UJPE2024-4-27Ключові слова:
гормон росту; інсуліноподібний фактор росту-1; діти; лікування; настанова.Посилання
Bell J, Parker KL, Swinford RD, Hoffman AR, Maneatis T, Lippe B. Long-term safety of recombinant human growth hormone in children. J Clin Endocrinol Metab. 2010;95:167-177.
Growth Hormone Research Society. Consensus guidelines for the diagnosis and treatment of growth hormone (GH) deficiency in childhood and adolescence: summary statement of the GH Research Society. GH Research Society. J Clin Endocrinol Metab. 2000;85:3990-3993.
Darendeliler F, Karagiannis G, Wilton P. Headache, idiopathic intracranial hypertension and slipped capital femoral epiphysis during growth hormone treatment: a safety update from the KIGS database. Horm Res. 2007;68(suppl 5):41-47.
Malozowski S, Tanner LA, Wysowski DK, Fleming GA, Stadel BV. Benign intracranial hypertension in children with growth hormone deficiency treated with growth hormone. J Pediatr. 1995;126:996-999.
Mostoufi-Moab S, Isaacoff EJ, Spiegel D, Gruccio D, Ginsberg JP, Hobbie W, Shults J, Leonard MB. Childhood cancer survivors exposed to total body irradiation are at significant risk for slipped capital femoral epiphysis during recombinant growth hormone therapy. Pediatr Blood Cancer. 2013;60:1766-1771.
Vedi A, Neville K, Johnston K, Saravanja D, Cohn RJ. Slipped capital femoral epiphyses after total body irradiation. Pediatr Blood Cancer. 2014;61:1140.
Kim JY, Rosenfeld SR, Keyak JH. Increased prevalence of scoliosis in Turner syndrome. J Pediatr Orthop. 2001;21:765-766.
Mills JL, Schonberger LB, Wysowski DK, Brown P, Durako SJ, Cox C, Kong F, Fradkin JE. Long-term mortality in the United States cohort of pituitary-derived growth hormone recipients. J Pediatr. 2004;144:430-436.
Laurberg P, Jakobsen PE, Hoeck HC, Vestergaard P. Growth hormone and thyroid function: is secondary thyroid failure underdiagnosed in growth hormone deficient patients? Thyroidology. 1994;6:73-79.
Lesage C, Walker J, Landier F, Chatelain P, Chaussain JL, Bougneres PF. Near normalization of adolescent height with growthhormone therapy in very short children without growth-hormone deficiency. J Pediatr. 1991;119:29-34.
Heptulla RA, Boulware SD, Caprio S, Silver D, Sherwin RS, Tamborlane WV. Decreased insulin sensitivity and compensatory hyperinsulinemia after hormone treatment in children with short stature. J Clin Endocrinol Metab. 1997;82:3234-3238.
Saenger P, Attie KM, DiMartino-Nardi J, Hintz R, Frahm L, Frane JW. Metabolic consequences of 5-year growth hormone (GH) therapy in children treated with GH for idiopathic short stature. Genentech Collaborative Study Group. J Clin Endocrinol Metab. 1998;83:3115-3120.
Bareille P, Azcona C, Matthews DR, Conway GS, Stanhope R. Lipid profile, glucose tolerance and insulin sensitivity after more than four years of growth hormone therapy in non-growth hormone deficient adolescents. Clin Endocrinol (Oxf). 1999;51:347-353.
Cutfield WS, Wilton P, Bennmarker H, Albertsson-Wikland K, Chatelain P, Ranke MB, Price DA. Incidence of diabetes mellitus and impaired glucose tolerance in children and adolescents receiving growth-hormone treatment. Lancet. 2000;355:610-613.
Dahlgren J. Metabolic benefits of growth hormone therapy in idiopathic short stature. Horm Res Paediatr. 2011;76(suppl 3):56-58.
Aguiar-Oliveira M, Oliveira FT, Pereira RM, Oliveira CR, Blackford A, Valenca EH, Santos EG, Gois-Junior M, Meneguz-Moreno R, Araujo VP, Oliveira-Neto L, Almeida RP, Santos MA, Farias NT, Silveira DC, Cabral GW, Calazans FR, Seabra JD, Lopes TF, Rodrigues EO, Porto LA, Oliveira IP, Melo EV, Martari M, Salvatori R. Longevity in untreated congenital growth hormone deficiency due to a homozygous mutation in the GHRH receptor gene. J Clin Endocrinol Metab. 2010;95:714-721.
Orme SM, McNally RJQ, Cartwright RA, Belchetz PE; for the United Kingdom Acromegaly Study Group. Mortality and cancer incidence in acromegaly: a retrospective cohort study. J Clin Endocrinol Metab. 1998;83:2730-2734.
Speakman JR, Mitchell SE. Caloric restriction. Mol Aspects Med. 2011;32:159-221.
Cohen P, Clemmons DR, Rosenfeld RG. Does the GH-IGF axis play a role in cancer pathogenesis? Growth Horm IGF Res. 2000;10:297-305.
Swerdlow AJ, Higgins CD, Adlard P, Preece MA. Risk of cancer in patients treated with human pituitary growth hormone in the UK, 1959-1985: a cohort study. Lancet. 2002;360:273-277.
Gallagher EJ, LeRoith D. Minireview. IGF, insulin, and cancer. Endocrinology. 2011;152:2546-2551.
Carel JC, Ecosse E, Landier F, MeguellatiHakkas D, Kaguelidou F, Rey G, Coste J. Long-term mortality after recombinant growth hormone treatment for isolated growth hormone deficiency or childhood short stature: preliminary report of the French SAGhE study. J Clin Endocrinol Metab. 2012;97:416-425.
Savendahl L, Maes M, Albertsson-Wikland K, Borgstrom B, Carel JC, Henrard S, Speybroeck N, Thomas M, Zandwijken G, Hokken-Koelega A. Long-term mortality and causes of death in isolated GHD, ISS, and SGA patients treated with recombinant growth hormone during childhood in Belgium, The Netherlands, and Sweden: preliminary report of 3 countries participating in the EU SAGhE study. J Clin Endocrinol Metab. 2012;97:E213–E217.
Wilton P, Mattsson AF, Darendeliler F. Growth hormone treatment in children is not associated with an increase in the incidence of cancer: experience from KIGS (Pfizer International Growth Database). J Pediatr. 2010;157:265-270.
Albertsson-Wikland K, Martensson A, Savendahl L, Niklasson A, Bang P, Dahlgren J, Gustafsson J, Kristrom B, Norgren S, Pehrsson NG, Oden A. Mortality is not increased in recombinant human growth hormonetreated patients when adjusting for birth characteristics. J Clin Endocrinol Metab. 2016;101:2149-2159.
Nishi Y, Tanaka T, Takano K, Fujieda K, Igarashi Y, Hanew K, Hirano T, Yokoya S, Tachibana K, Saito T, Watanabe S. Recent status in the occurrence of leukemia in growth hormone-treated patients in Japan. GH Treatment Study Committee of the Foundation for Growth Science, Japan. J Clin Endocrinol Metab. 1999;84:1961-1965.
Sklar CA, Mertens AC, Mitby P, Occhiogrosso G, Qin J, Heller G, Yasui Y, Robison LL. Risk of disease recurrence and second neoplasms in survivors of childhood cancer treated with growth hormone: a report from the Childhood Cancer Survivor Study. J Clin Endocrinol Metab. 2002;87:3136-3141.
Karavitaki N, Warner JT, Marland A, Shine B, Ryan F, Arnold J, Turner HE, Wass JA. GH replacement does not increase the risk of recurrence in patients with craniopharyngioma. Clin Endocrinol (Oxf). 2006;64:556-560.
Ergun-Longmire B, Mertens AC, Mitby P, Qin J, Heller G, Shi W, Yasui Y, Robison LL, Sklar CA. Growth hormone treatment and risk of second neoplasms in the childhood cancer survivor. J Clin Endocrinol Metab. 2006;91:3494-3498.
Essig S, Li Q, Chen Y, Hitzler J, Leisenring W, Greenberg M, Sklar C, Hudson MM, Armstrong GT, Krull KR, Neglia JP, Oeffinger KC, Robison LL, Kuehni CE, Yasui Y, Nathan PC. Risk of late effects of treatment in children newly diagnosed with standardrisk acute lymphoblastic leukaemia: a report from the Childhood Cancer Survivor Study cohort. Lancet Oncol. 2014;15:841-851.
Leung W, Rose SR, Zhou Y, Hancock ML, Burstein S, Schriock EA, Lustig R, Danish RK, Evans WE, Hudson MM, Pui CH. Outcomes of growth hormone replacement therapy in survivors of childhood acute lymphoblastic leukemia. J Clin Oncol. 2002;20: 2959-2964.
Packer RJ, Boyett JM, Janss AJ, Stavrou T, Kun L, Wisoff J, Russo C, Geyer R, Phillips P, Kieran M, Greenberg M, Goldman S, Hyder D, Heideman R, Jones-Wallace D, August GP, Smith SH, Moshang T. Growth hormone replacement therapy in children with medulloblastoma: use and effect on tumor control. J Clin Oncol. 2001;19:480-487.
Mackenzie S, Craven T, Gattamaneni HR, Swindell R, Shalet SM, Brabant G. Longterm safety of growth hormone replacement after CNS irradiation. J Clin Endocrinol Metab. 2011;96:2756-2761.
Howell SJ, Wilton P, Lindberg A, Shalet SM. Growth hormone replacement and the risk of malignancy in children with neurofibromatosis. J Pediatr. 1998;133:201-205.
Anneren G, Tuvemo T, Gustafsson J. Growth hormone therapy in young children with Down syndrome and a clinical comparison of Down and Prader—Willi syndromes. Growth Horm IGF Res. 2000;10(suppl B):S87–S91.
Howell JC, Joshi SA, Hornung L, Khoury J, Harris RE, Rose SR. Growth hormone improves short stature in children with Diamond-Blackfan anemia. Pediatr Blood Cancer. 2015;62:402-408.
Forlenza GP, Polgreen LE, Miller BS, Mac-Millan ML, Wagner JE, Petryk A. Growth hormone treatment of patients with Fanconi anemia after hematopoietic cell transplantation. Pediatr Blood Cancer. 2014;61:1142-1143.
Brock PR, de Zegher F, Casteels–Van Daele M, Vanderschueren–Lodeweyckx M. Malignant disease in Bloom’s syndrome children treated with growth hormone. Lancet. 1991;337:1345-1346.
Romano AA, Dana K, Bakker B, Davis DA, Hunold JJ, Jacobs J, Lippe B. Growth response, near-adult height, and patterns of growth and puberty in patients with noonan syndrome treated with growth hormone. J Clin Endocrinol Metab. 2009;94:2338-2344.
Raman S, Grimberg A, Waguespack SG, Miller BS, Sklar CA, Meacham LR, Patterson BC. Risk of neoplasia in pediatric patients receiving growth hormone therapy – a report from the Pediatric Endocrine Society Drug and Therapeutics Committee. J Clin Endocrinol Metab. 2015;100:2192-2203.
Poidvin A, Touze E, Ecosse E, Landier F, Bejot Y, Giroud M, Rothwell PM, Carel JC, Coste J. Growth hormone treatment for childhood short stature and risk of stroke in early adulthood. Neurology. 2014;83:780–786.
Allen DB. Growth hormone post-marketing surveillance: safety, sales, and the unfinished task ahead. J Clin Endocrinol Metab. 2010;95:52-55.
Allen DB. Growth hormone therapy for short stature: is the benefit worth the burden? Pediatrics. 2006;118:343-348.
Toogood AA, Shalet SM. Diagnosis of severe growth hormone (GH) deficiency in young adults who received GH replacement therapy during childhood. Acta Paediatr Suppl. 1997;423:117-120.
Juul A, Kastrup KW, Pedersen SA, Skakkebaek NE. Growth hormone (GH) provocative retesting of 108 young adults with childhoodonset GH deficiency and the diagnostic value of insulin-like growth factor I (IGF-I) and IGF-binding protein-3. J Clin Endocrinol Metab. 1997;82:1195-1201.
Quigley C, Zagar A, Liu CC, Brown D, Huseman C, Levitsky L, Repaske D, Tsalikian E, Chipman J. United States multicenter study of factors predicting the persistence of GH deficiency during the transition period between childhood and adulthood. Int J Pediatr Endocrinol. 2013;2013:6.
Attanasio AF, Lamberts SW, Matranga AM, Birkett MA, Bates PC, Valk NK, Hilsted J, Bengtsson BA, Strasburger CJ. Adult growth hormone (GH)-deficient patients demonstrate heterogeneity between childhood onset and adult onset before and during human GH treatment, Adult Growth Hormone Deficiency Study Group. J Clin Endocrinol Metab. 1997;82:82-88.
Attanasio AF, Howell S, Bates PC, Frewer P, Chipman J, Blum WF, Shalet SM. Body composition, IGF-I and IGFBP-3 concentrations as outcome measures in severely GH-deficient (GHD) patients after childhood GH treatment: a comparison with adult onset GHD patients. J Clin Endocrinol Metab. 2002;87:3368-3372.
Banerjee I, Tudorancea A, Scanlon MF, Gregory JW. Are factors at diagnosis of growth hormone deficiency in childhood associated with persistence of growth hormone deficiency into adult life? J Pediatr Endocrinol Metab. 2005;18:943-947.
Maghnie M, Strigazzi C, Tinelli C, Autelli M, Cisternino M, Loche S, Severi F. Growth hormone (GH) deficiency (GH deficiency) of childhood onset: reassessment of GH status and evaluation of the predictive criteria for permanent GH deficiency in young adults. J Clin Endocrinol Metab. 1999;84: 1324-1328.
Donaubauer J, Kiess W, Kratzsch J, Nowak T, Steinkamp H, Willgerodt H, Keller E. Reassessment of growth hormone secretion in young adult patients with childhood-onset growth hormone deficiency. Clin Endocrinol (Oxf). 2003;58:456-463.
Nicolson A, Toogood AA, Rahim A, Shalet SM. The prevalence of severe growth hormone deficiency in adults who received growth hormone replacement in childhood. Clin Endocrinol. 1996;44:311-316.
Hartman ML, Crowe BJ, Biller B, Ho K, Clemmons DR, Chipman JJ. Which patients do not require a GH stimulation test for the diagnosis of adult GH deficiency? J Clin Endocrinol Metab. 2002;87:477-485.
Biller B, Samuels MH, Zagar A, Cook DM, Arafah BM, Bonert V, Stavrou S, Kleinberg DL, Chipman JJ, Hartman ML. Sensitivity and specificity of six tests for the diagnosis of adult GH deficiency. J Clin Endocrinol Metab. 2002;87:2067-2079.
Maghnie M, Salati B, Bianchi S, Rallo M, Tinelli C, Autelli M, Aimaretti G, Ghigo E. Relationship between the morphological evaluation of the pituitary and the growth hormone (GH) response to GH-releasing hormone plus arginine in children and adults with congenital hypopituitarism. J Clin Endocrinol Metab. 2001;86:1574-1579.
Chen S, Leger J, Garel C, Hassan M, Czernichow P. Growth hormone deficiency with ectopic neurohypophysis. Anatomical variations and relationship between the visibility of the pituitary stalk asserted by magnetic resonance imaging and anterior pituitary function. J Clin Endocrinol Metab. 1999;84:2408-2413.
Leger J, Danner S, Simon D, Garel C, Czernichow P. Do all patients with childhoodonset growth hormone deficiency (GHD) and ectopic neurohypophysis have persistent GHD in adulthood? J Clin Endocrinol Metab. 2005;90:650-656.
Maghnie M, Aimaretti G, Bellone S, Bona G, Bellone J, Baldelli R, de Sanctis C, Gargantini L, Gastaldi R, Ghizzoni L, Secco A, Tinelli C. Diagnosis of GH deficiency in the transition period: accuracy of insulin tolerance test and insulin-like growth factor-I measurement. Eur J Endocrinol. 2005;152:589-596.
Toogood AA, Beardwell CG, Shalet SM. The severity of growth hormone deficiency in adults with pituitary disease is related to the degree of hypopituitarism. Clin Endocrinol. 1994;41:511-516.
Attanasio AF, Howell S, Bates PC, Blum WF, Frewer P, Quigley C, Shalet SM. Confirmation of severe GH deficiency after final height in patients diagnosed as GH deficient during childhood. Clin Endocrinol (Oxf). 2002;56:503-507.
di Iorgi N, Secco A, Napoli F, Tinelli C, Calcagno A, Fratangeli N, Ambrosini L, Rossi A, Lorini R, Maghnie M. Deterioration of growth hormone (GH) response and anterior pituitary function in young adults with childhood-onset GH deficiency and ectopic posterior pituitary: a two-year prospective follow-up study. J Clin Endocrinol Metab. 2007;92:3875-3884.
Toogood AA, Ryder WD, Beardwell CG, Shalet SM. The evolution of radiation-induced growth hormone deficiency in adults is determined by the baseline growth hormone status. Clin Endocrinol (Oxf). 1995;43:97-103.
Clayton PE, Shalet SM. Dose dependency of time of onset of radiation-induced growth hormone deficiency. J Pediatr. 1991;118:226-228.
Gleeson HK, Gattamaneni HR, Smethurst L, Brennan BM, Shalet SM. Reassessment of growth hormone status is required at final height in children treated with growth hormone replacement after radiation therapy. J Clin Endocrinol Metab. 2004;89:662-666.
Secco A, di Iorgi N, Napoli F, Calandra E, Calcagno A, Ghezzi M, Frassinetti C, Fratangeli N, Parodi S, Benassai M, Leitner Y, Gastaldi R, Lorini R, Maghnie M, Radetti G. Reassessment of the growth hormone status in young adults with childhood-onset growth hormone deficiency: reappraisal of insulin tolerance testing. J Clin Endocrinol Metab. 2009;94:4195-4204.
Aimaretti G, Baffoni C, Bellone S, Di Vito L, Corneli G, Arvat E, Benso L, Camanni F, Ghigo E. Retesting young adults with childhood-onset growth hormone (GH) deficiency with GH-releasing-hormone-plus-arginine test. J Clin Endocrinol Metab. 2000;85: 3693-3699.
Mauras N, Pescovitz OH, Allada V, Messig M, Wajnrajch MP, Lippe B; on behalf of the Transition Study Group. Limited efficacy of growth hormone (GH) during transition of GH-deficient patients from adolescence to adulthood: a phase III multicenter, doubleblind, randomized two-year trial. J Clin Endocrinol Metab. 2005;90:3946-3955.
Tauber M, Jouret B, Cartault A, Lounis N, Gayrard M, Marcouyeux C, Pienkowski C, Oliver I, Moulin P, Otal P, Joffre F, Arnaud C, Rochiccioli P. Adolescents with partial growth hormone (GH) deficiency develop alterations of body composition after GH discontinuation and require follow-up. J Clin Endocrinol Metab. 2003;88:5101-5106.
Conceicao FL, da Costa e Silva A, Leal Costa AJ, Vaisman M. Glucagon stimulation test for the diagnosis of GH deficiency in adults. J Endocrinol Invest. 2003;26:1065-1070.
Yuen KC, Biller BM, Katznelson L, Rhoads SA, Gurel MH, Chu O, Corazzini V, Spiller K, Gordon MB, Salvatori R, Coo DM. Clinical characteristics, timing of peak responses and safety aspects of two dosing regimens of the glucagon stimulation test in evaluating growth hormone and cortisol secretion in adults. Pituitary. 2013;16:220-230.
Drake WM, Carroll PV, Maher KT, Metcalfe KA, Camacho-Hubner C, Shaw NJ, Dunger DB, Cheetham TD, Savage MO, Monson JP. The effect of cessation of growth hormone (GH) therapy on bone mineral accretion in GH-deficient adolescents at the completion of linear growth. J Clin Endocrinol Metab. 2003;88:1658-1663.
Carroll PV, Drake WM, Maher KT, Metcalfe K, Shaw NJ, Dunger DB, Cheetham TD, Camacho-Hubner C, Savage MO, Monson JP. Comparison of continuation or cessation of growth hormone (GH) therapy on body composition and metabolic status in adolescents with severe GH deficiency at completion of linear growth. J Clin Endocrinol Metab. 2004;89:3890-3895.
Johannsson G, Albertsson-Wikland K, Bengtsson BA. Discontinuation of growth hormone (GH) treatment: metabolic effects in GH-deficient and GH-sufficient adolescent patients compared with control subjects. Swedish Study Group for Growth Hormone Treatment in Children. J Clin Endocrinol Metab. 1999;84:4516-4524.
Attanasio AF, Shavrikova E, Blum WF, Cromer M, Child CJ, Paskova M, Lebl J, Chipman JJ, Shalet SM. Continued growth hormone (GH) treatment after final height is necessary to complete somatic development in childhood-onset GH-deficient patients. J Clin Endocrinol Metab. 2004;89:4857-4862.
Shalet SM, Shavrikova E, Cromer M, Child CJ, Keller E, Zapletalova J, Moshang T, Blum WF, Chipman JJ, Quigley CA, Attanasio AF. Effect of growth hormone (GH) treatment on bone in postpubertal GH-deficient patients: a 2-year randomized, controlled, dose-ranging study. J Clin Endocrinol Metab. 2003;88:4124-4129.
Underwood LE, Attie KM, Baptista J. Growth hormone (GH) dose-response in young adults with childhood-onset GH deficiency: a two-year, multicenter, multipledose, placebo-controlled study. J Clin Endocrinol Metab. 2003;88:5273-5280.
Conway GS, Szarras-Czapnik M, Racz K, Keller A, Chanson P, Tauber M, Zacharin M. Treatment for 24 months with recombinant human GH has a beneficial effect on bone mineral density in young adults with childhood-onset GH deficiency. Eur J Endocrinol. 2009;160:899-907.
Elmlinger MW, Kuhnel W, Weber MM, Ranke MB. Reference ranges for two automated chemiluminescent assays for serum insulin-like growth factor I (IGF-I) and IGFbinding protein 3 (IGFBP-3). Clin Chem Lab Med. 2004;42:654-664.
Bidlingmaier M, Friedrich N, Emeny RT, Spranger J, Wolthers OD, Roswall J, Korner A, Obermayer-Pietsch B, Hubener C, Dahlgren J, Frystyk J, Pfeiffer AF, Doering A, Bielohuby M, Wallaschofski H, Arafat AM. Reference intervals for insulin-like growth factor-1 (IGF-I) from birth to senescence: results from a multicenter study using a new automated chemiluminescence IGF-I immunoassay conforming to recent international recommendations. J Clin Endocrinol Metab. 2014;99:1712-1721.
De Sanctis V, Tosetto I, Iughetti L, Antoniazzi F, Clementi M, Toffolutti T, Facchin P, Monti E, Pisanello L, Tonini G, Greggio NA. The SHOX gene and the short stature. Roundtable on diagnosis and treatment of short stature due to SHOX haploinsufficiency: how genetics, radiology and anthropometry can help the pediatrician in the diagnostic process Padova (April 20th, 2011). Pediatr Endocrinol Rev. 2012;9:727-733.
Wit JM, Oostdijk W, Losekoot M, van Duyvenvoorde HA, Ruivenkamp CA, Kant SG. Mechanisms in endocrinology: novel genetic causes of short stature. Eur J Endocrinol. 2016;174:R145–173.
McCaughey ES, Mulligan J, Voss LD, Betts PR. Randomised trial of growth hormone in short normal girls. Lancet. 1998;351:940-944.
Leschek EW, Rose SR, Yanovski JA, Troendle JF, Quigley CA, Chipman JJ, Crowe BJ, Ross JL, Cassorla FG, Blum WF, Cutler GB Jr, Baron J. Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature: a andomized, double-blind, placebo-controlled trial. J Clin Endocrinol Metab. 2004;89:3140-3148.
Albertsson-Wikland K, Aronson AS, Gustafsson J, Hagenas L, Ivarsson SA, Jonsson B, Kristrom B, Marcus C, Nilsson KO, Ritzen EM, Tuvemo T, Westphal O, Aman J. Dosedependent effect of growth hormone on final height in children with short stature without growth hormone deficiency. J Clin Endocrinol Metab. 2008;93:4342-4350.
Deodati A, Cianfarani S. Impact of growth hormone therapy on adult height of children with idiopathic short stature: systematic review. BMJ. 2011;342:c7157.
Finkelstein BS, Imperiale TF, Speroff T, Marrero U, Radcliffe D, Cuttler L. Effect of growth hormone therapy on height in children with idiopathic short stature: a metaanalysis. Arch Pediatr Adolesc Med. 2002;3:230-240.
Sotos JF, Tokar NJ. Growth hormone significantly increases the adult height of children with idiopathic short stature: comparison of subgroups and benefit. Int J Pediatr Endocrinol. 2014;2014:15.
Ranke MB, Lindberg A, Price DA, Darendeliler F, Albertsson-Wikland K, Wilton P, Reiter EO. Age at growth hormone therapy start and first-year responsiveness to growth hormone are major determinants of height outcome in idiopathic short stature. Horm Res. 2007;68:53-62.
Bakker B, Frane J, Anhalt H, Lippe B, Rosenfeld RG. Height velocity targets from the national cooperative growth study for firstyear growth hormone responses in short children. J Clin Endocrinol Metab. 2008;93:352-357.
Wit JM, Rekers-Mombarg L, Cutler GB, Crowe B, Beck TJ, Roberts K, Gill A, Chaussain JL, Frisch H, Yturriaga R, Attanasio AF. Growth hormone (GH) treatment to final height in children with idiopathic short stature: evidence for a dose effect. J Pediatr. 2005;146:45-53.
Loche S, Cambiaso P, Setzu S, Carta D, Marini R, Borrelli P, Cappa M. Final height after growth hormone therapy in non-growthhormone-deficient children with short stature. J Pediatr. 1994;125:196-200.
Cohen P, Rogol AD, Weng W, Kappelgaard A, Rosenfeld RG, Germak J, The American Norditropin Study Group. Efficacy of IGFbased growth hormone (GH) dosing in nonGH-deficient (nonGHD) short stature children with low IGF-I is not related to basal IGF-I levels. Clin Endocrinol (Oxf) 2013;78:405-414.
Albertsson-Wikland K, Kristrom B, Jonsson B, Hochberg Z. Long-term response to GH therapy in short children with a delayed infancy-childhood transition (DICT). Pediatr Res. 2011;69:504-510.
Kristrom B, Lundberg E, Jonsson B, Albertsson-Wikland K; study group. IGF-1 and growth response to adult height in a randomized GH treatment trial in short nonGH-deficient children. J Clin Endocrinol Metab. 2014;99:2917-2924.
Bang P, Bjerknes R, Dahlgren J, Dunkel L, Gustafsson J, Juul A, Kristrom B, Tapanainen P, Aberg V. A comparison of different definitions of growth response in short prepubertal children treated with growth hormone. Horm Res Paediatr. 2011;75:335-345.
Karlberg J, Fryer JG, Engstrom I, Karlberg P. Analysis of linear growth using a mathematical model. II. From 3 to 21 years of age. Acta Paediatr Scand Suppl. 1987;337:12-29.
Cohen P, Rogol AD, Deal CL, Saenger P, Reiter EO, Ross JL, Chernausek SD, Savage MO, Wit JM. Consensus statement on the diagnosis and treatment of children with idiopathic short stature: a summary of the Growth Hormone Research Society, the Lawson Wilkins Pediatric Endocrine Society, and the European Society for Paediatric Endocrinology Workshop. J Clin Endocrinol Metab. 2008;93:4210-4217.
Laron Z. Laron syndrome (primary growth hormone resistance or insensitivity): the personal experience. 1958-2003. J Clin Endocrinol Metab. 2004;89:1031-1044.
Laron Z. Consequences of not treating children with Laron syndrome (primary growth hormone insensitivity). J Pediatr Endocrinol Metab. 2001;14(suppl 5):1243-1248; discussion. 1261-1262.
Laron Z, Lilos P, Klinger B. Growth curves for Laron syndrome. Arch Dis Child. 1993;68:768-770.
Rosenbloom AL, Guevara-Aguirre J, Rosenfeld RG, Francke U. Growth hormone receptor deficiency in Ecuador. J Clin Endocrinol Metab. 1999;84:4436-4443.
Backeljauw PF, Kuntze J, Frane J, Calikoglu AS, Chernausek SD. Adult and near-adult height in patients with severe insulin-like growth factor-I deficiency after long-term therapy with recombinant human insulinlike growth factor-I. Horm Res Paediatr. 2013;80:47-56.
Laron Z. The essential role of IGF-I: lessons from the long-term study and treatment of children and adults with Laron syndrome. J Clin Endocrinol Metab. 1999;84:4397-4404.
Shevah O, Laron Z. Genetic analysis of the pedigrees and molecular defects of the GHreceptor gene in the Israeli cohort of patients with Laron syndrome. Pediatr Endocrinol Rev. 2006;3(suppl 3):489-497.
Woods KA, Clark AJ, Amselem S, Savage MO. Relationship between phenotype and genotype in growth hormone insensitivity syndrome. Acta Paediatr Suppl. 1999;88:158-162; discussion 163.
Goddard AD, Covello R, Luoh SM, Clackson T, Attie KM, Gesundheit N, Rundle AC, Wells JA, Carlsson LM. Mutations of the growth hormone receptor in children with idiopathic short stature. The Growth Hormone Insensitivity Study Group. N Engl J Med. 1995;333:1093-1098.
Attie KM, Julius JR, Stoppani C, Rundle AC. National Cooperative Growth Study substudy VI: the clinical utility of growth-hormone-binding protein, insulin-like growth factor I, and insulin-like growth factorbinding protein 3 measurements. J Pediatr. 1997;131:S56–S60.
Blum WF, Cotterill AM, Postel-Vinay MC, Ranke MB, Savage MO, Wilton P. Improvement of diagnostic criteria in growth hormone insensitivity syndrome: solutions and pitfalls. Pharmacia Study Group on Insulinlike Growth Factor I Treatment in Growth Hormone Insensitivity Syndromes. Acta Paediatr Suppl. 1994;399:117-124.
Schwarze CP, Wollmann HA, Binder G, Ranke MB. Short-term increments of insulin-like growth factor I (IGF-I) and IGFbinding protein-3 predict the growth response to growth hormone (GH) therapy in GH-sensitive children. Acta Paediatr Suppl. 1999;88:200-208.
Jorge AA, Souza SC, Arnhold IJ, Mendonca BB. Poor reproducibility of IGF-I and IGF binding protein-3 generation test in children with short stature and normal coding region of the GH receptor gene. J Clin Endocrinol Metab. 2002;87:469-472.
Selva KA, Buckway CK, Sexton G, Pratt KL, Tjoeng E, Guevara-Aguirre J, Rosenfeld RG. Reproducibility in patterns of IGF generation with special reference to idiopathic short stature. Horm Res. 2003;60:237-246.
Buckway CK, Guevara-Aguirre J, Pratt KL, Burren CP, Rosenfeld RG. The IGF-I generation test revisited: a marker of GH sensitivity. J Clin Endocrinol Metab. 2001;86:5176-5183.
Savage MO. Phenotypes, investigation and treatment of primary IGF-1 deficiency. Endocr Dev. 2013;24:138-149.
Wit JM, Kiess W, Mullis P. Genetic evaluation of short stature. Best Pract Res Clin Endocrinol Metab. 2011;25:1-17.
David A, Hwa V, Metherell LA, Netchine I, Camacho-Hubner C, Clark AJ, Rosenfeld RG, Savage MO. Evidence for a continuum of genetic, phenotypic, and biochemical abnormalities in children with growth hormone insensitivity. Endocr Rev. 2011;32:472-497.
Rosenfeld RG, von Stein T. A database and website for molecular defects of the GH-IGF axis: www.growthgenetics.com. Horm Res Paediatr. 2013;80:443-448.
Dauber A, Rosenfeld RG, Hirschhorn JN. Genetic evaluation of short stature. J Clin Endocrinol Metab. 2014;99:3080-3092.
Johnston LB, Savage MO. Partial growth hormone insensitivity. J Pediatr Endocrinol Metab. 1999;12(suppl 1):251-257.
Goddard AD, Dowd P, Chernausek S, Geffner M, Gertner J, Hintz R, Hopwood N, Kaplan S, Plotnick L, Rogol A, Rosenfield R, Saenger P, Mauras N, Hershkopf R, Angulo M, Attie K. Partial growth-hormone insensitivity: the role of growth-hormone receptor mutations in idiopathic short stature. J Pediatr. 1997;131:S51-S55.
Guevara-Aguirre J, Rosenbloom AL, Vasconez O, Martinez V, Gargosky SE, Allen L, Rosenfeld RG. Two-year treatment of growth hormone (GH) receptor deficiency with recombinant insulin-like growth factor I in 22 children: comparison of two dosage levels and to GH-treated GH deficiency. J Clin Endocrinol Metab. 1997;82:629-633.
Laron Z. Insulin-like growth factor 1 (IGF1): a growth hormone. Mol Pathol. 2001;54:311-316.
Hunziker EB, Wagner J, Zapf J. Differential effects of insulin-like growth factor I and growth hormone on developmental stages of rat growth plate chondrocytes in vivo. J Clin Invest. 1994;93:1078-1086.
Ranke MB, Wolfle J, Schnabel D, Bettendorf M. Treatment of dwarfism with recombinant human insulin-like growth factor-1. Dtsch Arztebl Int. 2009;106:703-709.
Grimberg A. p53 and IGFBP-3: apoptosis and cancer protection. Mol Genet Metab. 2000;70:85-98.
Besson A, Salemi S, Deladoey J, Vuissoz JM, Eble A, Bidlingmaier M, Burgi S, Honegger U, Fluck C, Mullis PE. Short stature caused by a biologically inactive mutant growth hormone (GH-C53S). J Clin Endocrinol Metab. 2005;90:2493-2499.
Kowarski AA, Schneider J, Ben-Galim E, Weldon VV, Daughaday WH. Growth failure with normal serum RIA-GH and low somatomedin activity: somatomedin restoration and growth acceleration after exogenous GH. J Clin Endocrinol Metab. 1978;47:461-464.
Hogler W, Martin DD, Crabtree N, Nightingale P, Tomlinson J, Metherell L, Rosenfeld R, Hwa V, Rose S, Walker J, Shaw N, Barrett T, Frystyk J. IGFALS gene dosage effects on serum IGF-I and glucose metabolism, body composition, bone growth in length and width, and the pharmacokinetics of recombinant human IGF-I administration. J Clin Endocrinol Metab. 2014;99:E703–E712.
Domene HM, Hwa V, Argente J, Wit JM, Camacho-Hubner C, Jasper HG, Pozo J, van Duyvenvoorde HA, Yakar S, Fofanova-Gambetti OV, Rosenfeld RG; International ALS Collaborative Group. Human acidlabile subunit deficiency: clinical, endocrine and metabolic consequences. Horm Res. 2009;72:129-141.
Laron Z. Insulin-like growth factor-I treatment of children with Laron syndrome (primary growth hormone insensitivity). Pediatr Endocrinol Rev. 2008;5:766-771.
Azcona C, Preece MA, Rose SJ, Fraser N, Rappaport R, Ranke MB, Savage MO. Growth response to rhIGF-I 80 microg/kg twice daily in children with growth hormone insensitivity syndrome: relationship to severity of clinical phenotype. Clin Endocrinol (Oxf). 1999;51:787-792.
Grahnen A, Kastrup K, Heinrich U, Gourmelen M, Preece MA, Vaccarello MA, Guevara-Aguirre J, Rosenfeld RG, Sietnieks A. Pharmacokinetics of recombinant human insulin-like growth factor I given subcutaneously to healthy volunteers and to patients with growth hormone receptor deficiency. Acta Paediatr Suppl. 1993;82(suppl 391):9-13; discussion 14.
Laron Z. If one daily injection of IGF-I has the same growth-promoting effect as 2 injections per day, why continue to give two injections? Pediatr Endocrinol Rev. 2013;10:277-279.
Guevara-Aguirre J, Rosenbloom AL, Guevara-Aguirre M, Saavedra J, Procel P. Recommended IGF-I dosage causes greater fat accumulation and osseous maturation than lower dosage and may compromise longterm growth effects. J Clin Endocrinol Metab. 2013;98:839-845.
Woods KA, Savage MO. Laron syndrome: typical and atypical forms. Baillieres Clin Endocrinol Metab. 1996;10:371-387.
Guevara-Aguirre J, Rosenbloom AL, Vaccarello MA, Fielder PJ, de lV, Diamond FB Jr, Rosenfeld RG. Growth hormone receptor deficiency (Laron syndrome): clinical and genetic characteristics. Acta Paediatr Scand Suppl. 1991;377:96-103.
Theunissen NC, Kamp GA, Koopman HM, Zwinderman KA, Vogels T, Wit JM. Quality of life and self-esteem in children treated for idiopathic short stature. J Pediatr. 2002;140:507-515.
Sandberg DE, Colsman M. Growth hormone treatment of short stature: status of the quality of life rationale. Horm Res. 2005;63: 275-283.
Voss LD, Sandberg DE. The psychological burden of short stature: evidence against. Eur J Endocrinol. 2004;151(suppl 1):S29– S33.
Chaplin JE, Kristrom B, Jonsson B, Hagglof B, Tuvemo T, Aronson AS, Dahlgren J, Albertsson-Wikland K. Improvements in behaviour and self-esteem following growth hormone treatment in short prepubertal children. Horm Res Paediatr. 2011;75:291-303.
Ross JL, Sandberg DE, Rose SR, Leschek EW, Baron J, Chipman JJ, Cassorla FG, Quigley CA, Crowe BJ, Roberts K, Cutler GB Jr. Psychological adaptation in children with idiopathic short stature treated with growth hormone or placebo. J Clin Endocrinol Metab. 2004;89:4873-4878.
Christensen TL, Djurhuus CB, Clayton P, Christiansen JS. An evaluation of the relationship between adult height and health-related quality of life in the general UK population. Clin Endocrinol. 2007;67:407-412.
Gardner M, Boshart ML, Yeguez CE, Desai KM, Sandberg DE. Coming up short: risks of bias in assessing psychological outcomes in growth hormone therapy for short stature. J Clin Endocrinol Metab. 2016;101:23-30.
Allen DB, Fost N: hGH for short stature: ethical issues raised by expanded access. J Pediatr. 2004;144:648-652.
Allen DB, Cuttler L. Clinical practice. Short stature in childhood-challenges and choices. N Engl J Med. 2013;368:1220-1228.
Gill DG: “Anything you can do, I can do bigger?”: the ethics and equity of growth hormone for small normal children. Arch Dis Child. 2006;91:270-272.
Rosenbloom AL. Is there a role for recombinant insulin-like growth factor-I in the treatment of idiopathic short stature? Lancet. 2006;368:612-616.
Bullinger M, Quitmann J, Silva N, Rohenkohl A, Chaplin JE, DeBusk K, Mimoun E, Feigerlova E, Herdman M, Sanz D, Wollmann H, Pleil A, Power M. Cross-cultural equivalence of the patientand parent-reported quality of life in short stature youth (QoLISSY) questionnaire. Horm Res Paediatr. 2014;82:18-30.
Quitmann J, Rohenkohl A, Bullinger M, Chaplin JE, Herdman M, Sanz D, Mimoun E, Feigerlova E, DeBusk K, Power M, Wollmann H, Pleil A. Parental perception of health-related quality of life in children and adolescents with short stature: literature review and introduction of the parent-reported QoLISSY instrument. Pediatr Endocrinol Rev. 2013;11:147-160.
Silvers JB, Marinova D, Mercer MB, Connors A, Cuttler L. A national study of physician recommendations to initiate and discontinue growth hormone for short stature. Pediatrics. 2010;126:468-476.
Allen DB, Backeljauw P, Bidlingmaier M, Biller BMK, Boguszewski M, Burman P, Butler G, Chihara K, Christiansen J, Cianfarani S, Clayton P, Clemmons D, Cohen P, Darendeliler F, Deal C, Dunger D, Erfurth EM, Fuqua JS, Grimberg A, Haymond M, Higham C, Ho K, Hoffman AR, Hokken-Koelega A, Johannsson G, Juul A, Kopchick J, Lee P, Pollak M, Radovick S, Robison L, Rosenfeld R, Ross RJ, Savendahl L, Saenger P, Toft Sorensen H, Stochholm K, Strasburger C, Swerdlow A, Thorner M. GH safety workshop position paper: a critical appraisal of recombinant human GH therapy in children and adults. Eur J Endocrinol. 2016;174:P1-P9.
Backeljauw PF, Miller BS, Dutailly P, Houchard A, Lawson E, Hale DE, Reiner B, Sperling MA. Recombinant human growth hormone plus recombinant human insulinlike growth factor-1 coadministration therapy in short children with low insulin-like growth factor-1 and growth hormone sufficiency: results from a randomized, multicenter, open-label, parallel-group, active treatment-controlled trial. Horm Res Paediatr. 2015;83:268-279.
Hoybye C, Cohen P, Hoffman AR, Ross R, Biller BM, Christiansen JS, Growth Hormone Research Society. Status of long-acting-growth hormone preparations – 2015. Growth Horm IGF Res. 2015;25:201-206.
Christiansen JS, Backeljauw PF, Bidlingmaier M, Biller BM, Boguszewski MC, 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 JM, Horikawa R, Hoybye C, Jorgensen JO, Johannsson G, Juul A, Katznelson L, Kopchick JJ, Lee KO, Lee KW, 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 K. Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations. Eur J Endocrinol. 2016;174:C1–C8.
Wit JM, Oostdijk W. Novel approaches to short stature therapy. Best Pract Res Clin Endocrinol Metab. 2015;29:353-366.