Growth hormone treatment in children with prader-willi syndrome: Three years of longitudinal data in prepubertal children and adult height data from the KIGS database

Journal of Clinical Endocrinology and Metabolism

Volumn 102, Issue 5, 2017, Pages 1702-1711

  Bakker, Nienke E ^a,b   Lindberg, Anders ^c   Heissler, Joseph ^c   Wollmann, Hartmut A ^c   Camacho Hü Bner, Cecilia ^c   Hokken Koelega, Anita C ^a,b  

^a Dutch Growth Research Foundation   (Netherlands)

^b SOPHIA CHILDREN S HOSPITAL   (Netherlands)

^c PFIZER INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GROWTH HORMONE; HUMAN GROWTH HORMONE; RECOMBINANT PROTEIN; SOMATOMEDIN C;

ABDOMINAL PAIN; ADOLESCENT; ADVERSE OUTCOME; ARTHRALGIA; ARTICLE; BENIGN NEOPLASM; BODY HEIGHT; BODY MASS; BONE AGE; BRAIN PSEUDOTUMOR; CEREBROVASCULAR ACCIDENT; CHILD; CLINICAL EVALUATION; COHORT ANALYSIS; CONTROLLED STUDY; CONVULSION; CRYPTORCHISM; DRUG EFFICACY; DRUG SAFETY; ENURESIS; EPIPHYSIOLYSIS; FEMALE; FRACTURE; GESTATIONAL AGE; GYNECOMASTIA; HEADACHE; HUMAN; HYPERGLYCEMIA; HYPERSENSITIVITY; HYPERTROPHY; IMPAIRED GLUCOSE TOLERANCE; INFECTION; INSULIN DEPENDENT DIABETES MELLITUS; INSULIN RESISTANCE; INTUSSUSCEPTION; JOINT DISLOCATION; KYPHOSIS; LEUKEMIA; LONGITUDINAL STUDY; MAJOR CLINICAL STUDY; MALE; MENTAL DISEASE; MORTALITY RISK; NON INSULIN DEPENDENT DIABETES MELLITUS; OBSTRUCTIVE AIRWAY DISEASE; OUTCOME ASSESSMENT; PAPILLEDEMA; PERIPHERAL EDEMA; PNEUMONIA; PRADER WILLI SYNDROME; PRECOCIOUS PUBERTY; PREPUBERTY; PRIORITY JOURNAL; RETROSPECTIVE STUDY; SCOLIOSIS; SCORING SYSTEM; SHOCK; SLEEP DISORDERED BREATHING; SNORING; COMPLICATION; DEFICIENCY; DWARFISM, PITUITARY; FACTUAL DATABASE; GROWTH DISORDERS; INFANT; METABOLISM; PRADER-WILLI SYNDROME; PRESCHOOL CHILD;

BODY HEIGHT; CHILD; CHILD, PRESCHOOL; COHORT STUDIES; DATABASES, FACTUAL; DWARFISM, PITUITARY; FEMALE; GROWTH DISORDERS; HUMAN GROWTH HORMONE; HUMANS; INFANT; INSULIN-LIKE GROWTH FACTOR I; LONGITUDINAL STUDIES; MALE; PRADER-WILLI SYNDROME; RECOMBINANT PROTEINS; RETROSPECTIVE STUDIES;

EID: 85019097934     PISSN: 0021972X     EISSN: 19457197     Source Type: Journal    
DOI: 10.1210/jc.2016-2962     Document Type: Article

References (53)

1. Cassidy SB, Driscoll DJ. Prader-Willi syndrome. Eur J Hum Genet. 2008;17(1):3-13.
2. Vogels A, Van Den Ende J, Keymolen K, Mortier G, Devriendt K, Legius E, Fryns JP. Minimum prevalence, birth incidence and causeof death for Prader-Willi syndrome in Flanders. Eur J Hum Genet. 2003;12(3):238-240.
3. A° kefeldt A, Gillberg C, Larsson C. Prader-Willi syndrome in aSwedish rural county: Epidemiological aspects. Dev Med Child Neurol. 2008;33(8):715-721.
4. Holm VA, Cassidy SB, Butler MG, Hanchett JM, Greenswag LR, Whitman BY, Greenberg F. Prader-Willi syndrome: Consensusdiagnostic criteria. Pediatrics. 1993;91(2):398-402.
5. Goldstone AP, Holland AJ, Hauffa BP, Hokken-Koelega AC, Tauber M; Speakers Contributors at the Second Expert Meeting ofthe Comprehensive Care of Patients With PWS. Recommendationsfor the diagnosis and management of Prader-Willi syndrome. J Clin Endocrinol Metab. 2008;93(11):4183-4197.
6. Swaab DF. Prader-Willi syndrome and the hypothalamus. Acta Paediatr Suppl. 2012;86:50-54.
7. Driscoll DJ, Miller JL, Schwartz S, Cassidy SB. Prader-Willi Syndrome. In: Pagon RA, Bird TD, Dolan CR, Stephens K, Adam MP, eds. Gene Reviews® [Internet]. Seattle, WA: University of Washington, Seattle; 1993-2017. Available at: Https://www. ncbi. nlm. nih. gov/books/NBK1330/. Accessed 28 January 2015.
8. Grugni G, Crin ò A, Pagani S, Meazza C, Buzi F, De Toni T, Gargantini L, Pilotta A, Pozzan GB, Radetti G, Ragusa L, Salvatoni A, Sartorio A, Bozzola M; Genetic Obesity Study Group, Italian Society of Pediatric Endocrinology and Diabetology. Growthhormone secretory pattern in non-obese children and adolescentswith Prader-Willi syndrome. J Pediatr Endocrinol Metab. 2011;24(7-8):477-481.
9. Carrel AL, Myers SE, Whitman BY, Eickhoff J, Allen DB. Longtermgrowth hormone therapy changes the natural history of bodycomposition and motor function in children with Prader-Willisyndrome. J Clin Endocrinol Metab. 2010;95(3):1131-1136.
10. Festen DA, de Lind van Wijngaarden R, van Eekelen M, Otten BJ, Wit JM, Duivenvoorden HJ, Hokken-Koelega AC. Randomizedcontrolled GH trial: Effects on anthropometry, body compositionand body proportions in a large group of children with Prader-Willisyndrome. Clin Endocrinol (Oxf). 2008;69(3):443-451.
11. de Lind van Wijngaarden RF, Siemensma EP, Festen DA, Otten BJ, van Mil EG, Rotteveel J, Odink RJ, Bindels-de Heus GC, vanLeeuwen M, Haring DA, Bocca G, Houdijk EC, Hoorweg-Nijman JJ, Vreuls RC, Jira PE, van Trotsenburg AS, Bakker B, Schroor EJ, Pilon JW, Wit JM, Drop SL, Hokken-Koelega AC. Efficacy andsafety of long-term continuous growth hormone treatment inchildren with Prader-Willi syndrome. J Clin Endocrinol Metab. 2009;94(11):4205-4215.
12. Tauber M, Hokken-Koelega AC, Hauffa BP, Goldstone AP. Aboutthe benefits of growth hormone treatment in children with Prader-Willi syndrome. J Pediatr. 2009;154(5):778, author reply 779.
13. Lindgren AC, Lindberg A. Growth hormone treatment completelynormalizes adult height and improves body composition in Prader-Willi syndrome: Experience from KIGS (Pfizer International Growth Database). Horm Res. 2008;70(3):182-187.
14. Festen DA, Wevers M, Lindgren AC, Bö hm B, Otten BJ, Wit JM, Duivenvoorden HJ, Hokken-Koelega AC. Mental and motor developmentbefore and during growth hormone treatment in infantsand toddlers with Prader-Willi syndrome. Clin Endocrinol (Oxf). 2008;68(6):919-925.
15. Siemensma EP, Tummers-de Lind van Wijngaarden RF, Festen DA, Troeman ZC, van Alfen-van der Velden AA, Otten BJ, Rotteveel J, Odink RJ, Bindels-de Heus GC, van Leeuwen M, Haring DA, Oostdijk W, Bocca G, Mieke Houdijk EC, van Trotsenburg AS, Hoorweg-Nijman JJ, van Wieringen H, Vreuls RC, Jira PE, Schroor EJ, van Pinxteren-Nagler E, Willem Pilon J, Lunshof LB, Hokken-Koelega AC. Beneficial effects of growth hormone treatment oncognition in children with Prader-Willi syndrome: A randomizedcontrolled trial and longitudinal study. J Clin Endocrinol Metab. 2012;97(7):2307-2314.
16. Gondoni LA, Vismara L, Marzullo P, Vettor R, Liuzzi A, Grugni G. Growth hormone therapy improves exercise capacity in adult patientswith Prader-Willi syndrome. J Endocrinol Invest. 2014;31(9):765-772.
17. Katz-Salamon M, Lindgren AC, Cohen G. The effect of growthhormone on sleep-related cardio-respiratory control in Prader-Willisyndrome. Acta Paediatr. 2012;101(6):643-648.
18. Nakamura Y, Nagai T, Iida T, Ozeki S, Nohara Y. Growth hormonesupplement treatment reduces the surgical risk for Prader-Willi syndrome patients. Eur Spine J. 2012;21(Suppl 4):S483-S491.
19. Tauber M, Diene G, Molinas C, Hébert M. Review of 64 cases ofdeath in children with Prader-Willi syndrome (PWS). Am J Med Genet A. 2008;146A(7):881-887.
20. Eiholzer U. Deaths in children with Prader-Willi syndrome:a contribution to the debate about the safety of growth hormonetreatment in children with PWS. Horm Res. 2005;63(1):33-39.
21. de Lind van Wijngaarden RF, Otten BJ, Festen DA, Joosten KF, deJong FH, Sweep FC, Hokken-Koelega AC. High prevalence ofcentral adrenal insufficiency in patients with Prader-Willi syndrome. J Clin Endocrinol Metab. 2008;93(5):1649-1654.
22. de Lind van Wijngaarden RF, Joosten KF, van den Berg S, Otten BJ, de Jong FH, Sweep CG, de Weerd AW, Hokken-Koelega AC. Therelationship between central adrenal insufficiency and sleep-relatedbreathing disorders in children with Prader-Willi syndrome. J Clin Endocrinol Metab. 2009;94(7):2387-2393.
23. Eiholzer U, l'Allemand D, van der Sluis I, Steinert H, Gasser T, Ellis K. Body composition abnormalities in children with Prader-Willisyndrome and long-termeffects of growth hormone therapy. Horm Res. 2000;53(4):200-206.
24. Carrel AL, Myers SE, Whitman BY, Allen DB. Benefits of long-termGH therapy in Prader-Willi syndrome: A 4-year study. J Clin Endocrinol Metab. 2002;87(4):1581-1585.
25. Bakker NE, Kuppens RJ, Siemensma EP, Tummers-de Lind vanWijngaarden RF, Festen DA, Bindels-de Heus GC, Bocca G, Haring DA, Hoorweg-Nijman JJ, Houdijk EC, Jira PE, Lunshof L, Odink RJ, Oostdijk W, Rotteveel J, Schroor EJ, Van Alfen AA, Van Leeuwen M, Van Pinxteren-Nagler E, Van Wieringen H, Vreuls RC, Zwaveling-Soonawala N, de Ridder MA, Hokken-Koelega AC. Eight years of growth hormone treatment in children withPrader-Willi syndrome: Maintaining the positive effects. J Clin Endocrinol Metab. 2013;98(10):4013-4022.
26. Whittington JE, Holland AJ, Webb T, Butler J, Clarke D, Boer H. Population prevalence and estimated birth incidence and mortalityrate for people with Prader-Willi syndrome in one UK Health Region. J Med Genet. 2001;38(11):792-798.
27. Marshall WA, Tanner JM. Variations in the pattern of pubertalchanges in boys. Arch Dis Child. 1970;45(239):13-23.
28. Marshall WA, Tanner JM. Variations in pattern of pubertalchanges in girls. Arch Dis Child. 1969;44(235):291-303.
29. Prader A, Largo RH, Molinari L, Issler C. Physical growth of Swisschildren from birth to 20 years of age: First Zurich longitudinalstudy of growth and development. Helv Paediatr Acta Suppl. 1989;52:1-125.
30. Freeman JV, Cole TJ, Chinn S, Jones PR, White EM, Preece MA. Cross sectional stature and weight reference curves for the UK 1990. Arch Dis Child. 1995;73(1):17-24.
31. Hauffa BP, Schlippe G, Roos M, Gillessen-Kaesbach G, Gasser T. Spontaneous growth in German children and adolescents withgenetically confirmed Prader-Willi syndrome. Acta Paediatr. 2007;89(11):1302-1311.
32. Greulich WW, Pyle SI. Radiographic Atlas of Skeletal Developmentof Hand and Wrist. 2nd ed. Palo Alto, CA: Stanford University Press.
33. Riis P. Thirty years of bioethics: The Helsinki Declaration1964-2003. New Rev Bioeth. 2003;1(1):15-25.
34. Niklasson A, Ericson A, Fryer JG, Karlberg J, Lawrence C, Karlberg P. An update of the Swedish reference standards for weight, lengthand head circumference at birth for given gestational age(1977-1981). Acta Paediatr Scand. 1991;80(8-9):756-762.
35. Siemensma EP, de Lind van Wijngaarden RF, Otten BJ, de Jong FH, Hokken-Koelega AC. Testicular failure in boys with Prader-Willisyndrome: Longitudinal studies of reproductive hormones. J Clin Endocrinol Metab. 2012;97(3):E452-E459.
36. Siemensma EP, van Alfen-van der Velden AA, Otten BJ, Laven JS, Hokken-Koelega AC. Ovarian function and reproductive hormonelevels in girls with Prader-Willi syndrome: A longitudinal study. J Clin Endocrinol Metab. 2012;97(9):E1766-E1773.
37. de Lind van Wijngaarden RF, de Klerk LW, Festen DA, Hokken-Koelega AC. Scoliosis in Prader-Willi syndrome: Prevalence, effectsof age, gender, body mass index, lean body mass and genotype. Arch Dis Child. 2008;93(12):1012-1016.
38. Deal CL, Tony M, Hö ybyeC, Allen DB, Tauber M, Christiansen JS;2011 Growth Hormone in Prader-Willi Syndrome Clinical Care Guidelines Workshop Participants. Growth Hormone Research Society workshop summary: Consensus guidelines for recombinanthuman growth hormone therapy in Prader-Willi syndrome. J Clin Endocrinol Metab. 2013;98(6):E1072-E1087.
39. Myers SE, Whitman BY, Carrel AL, Moerchen V, Bekx MT, Allen DB. Two years of growth hormone therapy in young children withPrader-Willi syndrome: Physical and neurodevelopmental benefits. Am J Med Genet A. 2007;143A(5):443-448.
40. Siemensma EP, de Lind vanWijngaarden RF, Otten BJ, de Jong FH, Hokken-Koelega AC. Pubarche and serum dehydroepiandrosteronesulfate levels in childrenwith Prader-Willi syndrome. Clin Endocrinol(Oxf). 2011;75(1):83-89.
41. Unanue N, Bazaes R, I~niguez G, Cortés F, Avila A, Mericq V. Adrenarche in Prader-Willi syndrome appears not related to insulinsensitivity and serum adiponectin. Horm Res. 2007;67(3):152-158.
42. Angulo MA, Castro-Magana M, Lamerson M, Arguello R, Accacha S, Khan A. Final adult height in children with Prader-Willisyndrome with and without human growth hormone treatment. Am J Med Genet A. 2007;143A(13):1456-1461.
43. Critical evaluation of the safety of recombinant human growthhormone administration: Statement from the Growth Hormone Research Society. J Clin Endocrinol Metab. 2001;86(5):1868-1870.
44. Clayton PE, Cowell CT. Safety issues in children and adolescentsduring growth hormone therapy-a review. Growth Hormone IGFRes. 2000;10:306-317.
45. Festen DA, deWeerd AW, van den Bossche RA, Joosten K, Hoeve H, Hokken-Koelega AC. Sleep-related breathing disorders in prepubertalchildren with Prader-Willi syndrome and effects of growth hormonetreatment. J Clin Endocrinol Metab. 2006;91(12):4911-4915.
46. de Lind van Wijngaarden RF, deKlerk LW, Festen DA, Duivenvoorden HJ, Otten BJ, Hokken-Koelega AC. Randomized controlled trial toinvestigate the effects of growth hormone treatment on scoliosis inchildren with Prader-Willi syndrome. J Clin Endocrinol Metab. 2009;94(4):1274-1280.
47. Hoybye C. Growth hormone treatment of Prader-Willi syndromehas long-term, positive effects on body composition. Acta Paediatr. 2015;104(4):422-427.
48. Bakker NE, van Doorn J, Renes JS, Donker GH, Hokken-Koelega AC. IGF-1 levels, complex formation, IGF bioactivity in growthhormone-treated children with Prader-Willi syndrome. J Clin Endocrinol Metab. 2015;100(8):3041-3049.
49. Feigerlová E, Diene G, Oliver I, Gennero I, Salles JP, Arnaud C, Tauber M. Elevated insulin-like growth factor-I values in childrenwith Prader-Willi syndrome compared with growth hormone (GH)deficiency children over two years of GH treatment. J Clin Endocrinol Metab. 2010;95(10):4600-4608.
50. Davies HD, Leusink GL, Mc Connell A, Deyell M, Cassidy SB, Fick GH, Coppes MJ. Myeloid leukemia in Prader-Willi syndrome. J Pediatr. 2003;142(2):174-178.
51. Stevenson DA, Heinemann J, Angulo M, Butler MG, Loker J, Rupe N, Kendell P, Cassidy SB, Scheimann A. Gastric rupture and necrosisin Prader-Willi syndrome. J Pediatr Gastroenterol Nutr. 2007;45(2):272-274.
52. Wharton RH, Wang T, Graeme-Cook F, Briggs S, Cole RE. Acuteidiopathic gastric dilation with gastric necrosis in individuals withPrader-Willi syndrome. Am J Med Genet. 1997;73(4):437-441.
53. Corrias A, Grugni G, Crino A, Di Candia S, Chiabotto P, Cogliardi A, Chiumello G, De Medici C, Spera S, Gargantini L, Iughetti L, Luce A, Mariani B, Ragusa L, Salvatoni A, Andrulli S, Mussa A, Beccaria L; Study Group for Genetic Obesity of the Italian Societyof Pediatric Endocrinology and Diabetology (SIEDP/ISPED). Assessmentof central adrenal insufficiency in children and adolescentswith Prader-Willi syndrome. Horumon To Rinsho. 2012;76:843-850.