본문 바로가기
KERD
논문KCI 등재

Growth hormone treatment in non–growth hormonedeficient children

2

조회

0

다운로드

1

인용

0

참고문헌

키워드

주제분류소아과학

초록

Until 1985 growth hormone (GH) was obtained from pituitary extracts, and wasavailable in limited amounts only to treat severe growth hormone deficiency (GHD). With the availability of unlimited quantities of GH obtained from recombinant DNAtechnology, researchers started to explore new modalities to treat GHD children,as well as to treat a number of other non-GHD conditions. Although with somedifferences between different countries, GH treatment is indicated in children withTurner syndrome, chronic renal insufficiency, Prader-Willi syndrome, deletions/mutations of the SHOX gene, as well as in short children born small for gestationalage and with idiopathic short stature. Available data from controlled trials indicatethat GH treatment increases adult height in patients with Turner syndrome, inpatients with chronic renal insufficiency, and in short children born small forgestational age. Patients with SHOX deficiency seem to respond to treatmentsimilarly to Turner syndrome. GH treatment in children with idiopathic short statureproduces a modest mean increase in adult height but the response in the individualpatient is unpredictable. Uncontrolled studies indicate that GH treatment may bebeneficial also in children with Noonan syndrome. In patients with Prader-Willisyndrome GH treatment normalizes growth and improves body composition andcognitive function. In any indication the response to GH seems correlated to thedose and the duration of treatment. GH treatment is generally safe with no majoradverse effects being recorded in any condition.

Abstract

Until 1985 growth hormone (GH) was obtained from pituitary extracts, and wasavailable in limited amounts only to treat severe growth hormone deficiency (GHD). With the availability of unlimited quantities of GH obtained from recombinant DNAtechnology, researchers started to explore new modalities to treat GHD children,as well as to treat a number of other non-GHD conditions. Although with somedifferences between different countries, GH treatment is indicated in children withTurner syndrome, chronic renal insufficiency, Prader-Willi syndrome, deletions/mutations of the SHOX gene, as well as in short children born small for gestationalage and with idiopathic short stature. Available data from controlled trials indicatethat GH treatment increases adult height in patients with Turner syndrome, inpatients with chronic renal insufficiency, and in short children born small forgestational age. Patients with SHOX deficiency seem to respond to treatmentsimilarly to Turner syndrome. GH treatment in children with idiopathic short statureproduces a modest mean increase in adult height but the response in the individualpatient is unpredictable. Uncontrolled studies indicate that GH treatment may bebeneficial also in children with Noonan syndrome. In patients with Prader-Willisyndrome GH treatment normalizes growth and improves body composition andcognitive function. In any indication the response to GH seems correlated to thedose and the duration of treatment. GH treatment is generally safe with no majoradverse effects being recorded in any condition.

참고문헌 (67)

  1. Bondy CA, "Care of girls and women with Turner syndrome: a guideline of the Turner Syndrome Study Group", J Clin Endocrinol Metab, 92, pp.10~25 (2007)
  2. Davenport ML, "Approach to the patient with Turner syndrome", J Clin Endocrinol Metab, 95, pp.1487~1495 (2010)
  3. Rao E, "Pseudoautosomal deletions encompassing a novel homeobox gene cause growth failure in idiopathic short stature and Turner syndrome", Nat Genet, 16, pp.54~63 (1997)
  4. Soucek O, "Prepubertal girls with Turner syndrome and children with isolated SHOX deficiency have similar bone geometry at the radius", J Clin Endocrinol Metab, 98, pp.E1241~E1247 (2013)
  5. Baxter L, "Recombinant growth hormone for children and adolescents with Turner syndrome", Cochrane Dat abas e Syst Rev, (1), pp.CD003887~ (2007)
  6. Stephure DK, "Impact of growth hormone supplementation on adult height in turner syndrome: results of the Canadian randomized controlled trial", J Clin Endocrinol Metab, 90, pp.3360~3366 (2005)
  7. Ross JL, "Growth hormone plus childhood low-dose estrogen in Turner's syndrome", N Engl J Med, 364, pp.1230~1242 (2011)
  8. Davenport ML, "Growth hormone treatment of early growth failure in toddlers with Turner syndrome: a randomized, controlled, multicenter trial", J Clin Endocrinol Metab, 92, pp.3406~3416 (2007)
  9. Linglart A, "Growth hormone treatment before the age of 4 years prevents short stature in young girls with Turner syndrome", Eur J Endocrinol, 164, pp.891~897 (2011)
  10. Menke LA, "Efficacy and safety of oxandrolone in growth hormone-treated girls with turner syndrome", J Clin Endocrinol Metab, 95, pp.1151~1160 (2010)
  11. Gault EJ, "Effect of oxandrolone and timing of pubertal induction on final height in Turner's syndrome:randomised, double blind, placebo controlled trial", BMJ, 342, pp.d1980~ (2011)
  12. Quigley CA, "Growth hormone and low dose estrogen in Turner syndrome:results of a United States multi-center trial to near-final height", J Clin Endocrinol Metab, 87, pp.2033~2041 (2002)
  13. van Pareren YK, "Final height in girls with turner syndrome after long-term growth hormone treatment in three dosages and low dose estrogens", J Clin Endocrinol Metab, 88, pp.1119~1125 (2003)
  14. Mehls O, "Growth hormone treatment in short children with chronic kidney disease", Acta Paediatr, 97, pp.1159~1164 (2008)
  15. Hodson EM, "Growth hormone for children with chronic kidney disease", Cochrane Database Syst Rev, 2, pp.CD003264~ (2012)
  16. Haffner D, "Effect of growth hormone treatment on the adult height of children with chronic renal failure. German Study Group for Growth Hormone Treatment in Chronic Renal Failure", N Engl J Med, 343, pp.923~930 (2000)
  17. Nissel R, "Factors predicting the near-final height in growth hormone-treated children and adolescents with chronic kidney disease", J Clin Endocrinol Metab, 93, pp.1359~1365 (2008)
  18. Mehls O, "Predicting the response to growth hormone treatment in short children with chronic kidney disease", J Clin Endocrinol Metab, 95, pp.686~692 (2010)
  19. Cassidy SB, "Prader-Willi syndrome", Genet Med, 14, pp.10~26 (2012)
  20. Butler MG, "Prader-Willi Syndrome: Obesity due to Genomic Imprinting", Curr Genomics, 12, pp.204~215 (2011)
  21. Cappa M, "Growth hormone (GH) response to combined pyridostigmine and GH-releasing hormone administration in patients with Prader-Labhard-Willi syndrome", Horm Res, 39, pp.51~55 (1993)
  22. Tauber M, "Auxological and endocrine evolution of 28children with Prader-Willi syndrome: effect of GH therapy in 14 children", Horm Res, 53, pp.279~287 (2000)
  23. Corrias A, "GH", IGF-I axis in Prader-Willi syndrome: evaluation of IGF-I levels and of the somatotroph responsiveness to various provocative stimuli. Genetic Obesity Study Group of Italian Society of Pediatric Endocrinology and Diabetology, 2000, pp.23, 84~89
  24. Grugni G, "Growth hormone secretory pattern in non-obese children and adolescents with Prader-Willi syndrome", J Pediatr Endocrinol Metab, 24, pp.477~481 (2011)
  25. Diene G, "Endocrine disorders in children with Prader-Willi syndrome: data from 142 children of the French database", Horm Res Paediatr, 74, pp.121~128 (2010)
  26. Eiholzer U, "Low insulin, IGF-I and IGFBP-3 levels in children with Prader-Labhart-Willi syndrome", Eur J Pediatr, 157, pp.890~893 (1998)
  27. Bakker NE, "Eight years of growth hormone treatment in children with Prader-Willi syndrome: maintaining the positive effects", J Clin Endocrinol Metab, 98, pp.4013~4022 (2013)
  28. Carrel AL, "Growth hormone improves body composition, fat utilization, physical strength and agility, and growth in Prader-Willi syndrome: A controlled study", J Pediatr, 134, pp.215~221 (1999)
  29. Siemensma EP, "Beneficial effects of growth hormone treatment on cognition in children with Prader-Willi syndrome: a randomized controlled trial and longitudinal study", J Clin Endocrinol Metab, 97, pp.2307~2314 (2012)
  30. Deal CL, "GrowthHormone Research Society workshop summary: consensus guidelines for recombinant human growth hormone therapy in Prader-Willi syndrome", J Clin Endocrinol Metab, 98, pp.E1072~E1087 (2013)
  31. Festen DA, "Mental and motor development before and during growth hormone treatment in infants and toddlers with Prader-Willi syndrome", Clin Endocrinol, 68, pp.919~925 (2008)
  32. Carrel AL, "Growth hormone improves mobility and body composition in infants and toddlers with Prader-Willi syndrome", J Pediatr, 145, pp.744~749 (2004)
  33. Tartaglia M, "Mutations in PTPN11, encoding the protein tyrosine phosphatase SHP-2, cause Noonan syndrome", Nat Genet, 29, pp.465~468 (2001)
  34. Romano AA, "Noonan syndrome: clinical features, diagnosis, and management guidelines", Pediatrics, 126, pp.746~759 (2010)
  35. Roberts AE, "Noonan syndrome", Lancet, 381, pp.333~342 (2013)
  36. Limal JM, "Noonan syndrome: relationships between genotype, growth, and growth factors", J Clin Endocrinol Metab, 91, pp.300~306 (2006)
  37. Binder G, "PTPN11mutations are associated with mild growth hormone resistance in individuals with Noonan syndrome", J Clin Endocrinol Metab, 90, pp.5377~5381 (2005)
  38. Noordam C, "Long-term GH treatment improves adult height in children with Noonan syndrome with and without mutations in protein tyrosine phosphatase, nonreceptor-type 11", Eur J Endocrinol, 159, pp.203~208 (2008)
  39. Chacko EM, "Short stature and its treatment in Turner and Noonan syndromes", Curr Opin Endocrinol Diabetes Obes, 19, pp.40~46 (2012)
  40. Dahlgren J, "GH therapy in Noonan syndrome : Review of final height data", Horm Res, 72(Suppl 2), pp.46~48 (2009)
  41. Romano AA, "Growth response, near-adult height, and patterns of growth and puberty in patients with noonan syndrome treated with growth hormone", J Clin Endocrinol Metab, 94, pp.2338~2344 (2009)
  42. Munns CJ, "Expression of SHOX in human fetal and childhood growth plate", J Clin Endocrinol Metab, 89, pp.4130~4135 (2004)
  43. Binder G, "Short stature due to SHOX deficiency: genotype, phenotype, and therapy", Horm Res Paediatr, 75, pp.81~89 (2011)
  44. Rosilio M, "Genotypes and phenotypes of children with SHOX deficiency in France", J Clin Endocrinol Metab, 97, pp.E1257~E1265 (2012)
  45. Blum WF, "Growth hormone is effective in treatment of short stature associated with short stature homeobox-containing gene deficiency: two-year results of a randomized, controlled, multicenter trial", J Clin Endocrinol Metab, 92, pp.219~228 (2007)
  46. Blum WF, "GH treatment to final height produces similar height gains in patients with SHOX deficiency and Turner syndrome: results of a multicenter trial", J Clin Endocrinol Metab, 98, pp.E1383~E1392 (2013)
  47. Saenger P, "Small for gestational age: short stature and beyond", Endocr Rev, 28, pp.219~251 (2007)
  48. Clayton PE, "Management of the child born small for gestational age through to adulthood: a consensus statement of the International Societies of Pediatric Endocrinology and the Growth Hormone Research Society", J Clin Endocrinol Metab, 92, pp.804~810 (2007)
  49. Maiorana A, "Impact of growth hormone therapy on adult height of children born small for gestational age", Pediatrics, 124, pp.e519~e531 (2009)
  50. Van Pareren Y, "Adult height after long-term, continuous growth hormone (GH) treatment in short children born small for gestational age: results of a randomized, double-blind, dose-response GH trial", J Clin Endocrinol Metab, 88, pp.3584~3590 (2003)
  51. Carel JC, "Improvement in adult height after growth hormone treatment in adolescents with short stature born small for gestational age: results of a randomized controlled study", J Clin Endocrinol Metab, 88, pp.1587~1593 (2003)
  52. Dahlgren J, "Final height in short children born small for gestational age treated with growth hormone", Pediatr Res, 57, pp.216~222 (2005)
  53. van Dijk M, "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", J Clin Endocrinol Metab, 92, pp.160~165 (2007)
  54. Dos Santos C, "A common polymorphism of the growth hormone receptor is associated with increased responsiveness to growth hormone", Nat Genet, 36, pp.720~724 (2004)
  55. Willemsen RH, "Long-term effects of growth hormone (GH) treatment on body composition and bone mineral density in short children born smallfor-gestational-age: six-year follow-up of a randomized controlled GH trial", Clin Endocrinol, 67, pp.485~492 (2007)
  56. Schweizer R, "Muscle function improves during growth hormone therapy in short children born small for gestational age: results of a peripheral quantitative computed tomography study on body composition", J Clin Endocrinol Metab, 93, pp.2978~2983 (2008)
  57. Lem AJ, "Adult height in short children born SGA treated with growth hormone and gonadotropin releasing hormone analog: results of a randomized, dose-response GH trial", J Clin Endocrinol Metab, 97, pp.4096~4105 (2012)
  58. Cohen P, "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, 93, pp.4210~4217 (2008)
  59. Loche S, "Growth hormone treatment in non-growth hormone-deficient short children", J Endocrinol Invest, 28, pp.193~198 (2005)
  60. Hindmarsh PC, "Effect of growth hormone on short normal children", Br Med J, 295, pp.573~577 (1987)
  61. Hopwood NJ, "Growth response of children with nongrowth-hormone deficiency and marked short stature during three years of growth hormone therapy", J Pediatr, 123, pp.215~222 (1993)
  62. Ranke MB, "Treatment of children and adolescents with idiopathic short stature", Nat Rev Endocrinol, 9, pp.325~334 (2013)
  63. McCaughey ES, "Randomised trial of growth hormone in short normal girls", Lancet, 351, pp.940~944 (1998)
  64. Leschek EW, "Effect of growth hormone treatment on adult height in peripubertal children with idiopathic short stature: a randomized, double-blind, placebo-controlled trial", J Clin Endocrinol Metab, 89, pp.3140~3148 (2004)
  65. Albertsson-Wikland K, "Dose-dependent effect of growth hormone on final height in children with short stature without growth hormone deficiency", J Clin Endocrinol Metab, 93, pp.4342~4350 (2008)
  66. Deodati A, "Impact of growth hormone therapy on adult height of children with idiopathic short stature: systematic review", BMJ, 342, pp.c7157~ (2011)
  67. Kemp SF, "Efficacy and safety results of long-term growth hormone treatment of idiopathic short stature", J Clin Endocrinol Metab, 90, pp.5247~5253 (2005)

관련 논문AI추천

저신장 소아를 위한 성장호르몬 치료박용훈영남대학교 의과대학 · Journal of Yeungnam Medical Science2005 · 제22권 제1호Response to three years of growth hormone therapy in girls with Turner syndrome박홍규, 황일태, Hae-Sang Lee, Jin Soon Hwang, Jung Hee Ko대한소아내분비학회 · Annals of Pediatric Endocrinology & Metabolism2013 · 제18권 제1호Effect of growth hormone treatment on children with idiopathic short stature and idiopathic growth hormone deficiency김용대, 임민지, 한헌석대한소아내분비학회 · Annals of Pediatric Endocrinology & Metabolism2017 · 제22권 제2호Comparison of the effectiveness of recombinant human growth hormone therapy in preterm and full-term children with short stature born small for gestational ageChoi Jin-Ho, Hwang Soojin, Kim Dohyung, Kim Ja Hye, Yoo Han-Wook, Yoon Ji-Hee대한소아내분비학회 · Annals of Pediatric Endocrinology & Metabolism2025 · 제30권 제3호Response of children with Turner syndrome with different types of karyotype abnormalities to growth hormone treatment김혜순, 유지숙, Choi Jung Eun, Park Mi Jung대한소아내분비학회 · Annals of Pediatric Endocrinology & Metabolism2024 · 제29권 제5호Response to growth hormone according to provocation test results in idiopathic short stature and idiopathic growth hormone deficiency이정현, 전종근, Chung Woo Yeong, Kim Hyun-Ji, Kim Jeongyun, Kim Ye Jin, Kwak Min Jung, Lee Jeong Eun, Yoo Jae-Ho, Yoon Ju Young대한소아내분비학회 · Annals of Pediatric Endocrinology & Metabolism2022 · 제27권 제1호

이 간행물의 다른 논문 (9건)