Growth retardation in children with cystinosis

Minerva Pediatrica

Volumn 62, Issue 3, 2010, Pages 307-314

  Besouw, M ^a   Levtchenko, E ^a  

^a UNIVERSITY HOSPITALS LEUVEN   (Belgium)

Author keywords

Cysteamine; Cystinosis; Growth disorders; Human growth hormone

Indexed keywords

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

AUTOSOMAL RECESSIVE DISORDER; CHILDHOOD DISEASE; CLINICAL FEATURE; CREATININE BLOOD LEVEL; CYSTINOSIS; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG EFFECT; DRUG MECHANISM; DRUG MEGADOSE; DRUG SAFETY; FANCONI RENOTUBULAR SYNDROME; GASTROINTESTINAL SYMPTOM; GENE MUTATION; GLUCOSE BLOOD LEVEL; GROWTH RETARDATION; HALITOSIS; HUMAN; INSULIN BLOOD LEVEL; NONHUMAN; PATHOGENESIS; REVIEW; SIDE EFFECT; CHILD; COMPLICATION; DISORDERS OF AMINO ACID AND PROTEIN METABOLISM; FORECASTING; GROWTH DISORDERS; LYSOSOME; METABOLISM;

AMINO ACID METABOLISM, INBORN ERRORS; CHILD; CYSTEAMINE; CYSTINE; FORECASTING; GROWTH DISORDERS; HUMAN GROWTH HORMONE; HUMANS; LYSOSOMES; RECOMBINANT PROTEINS;

EID: 77955388673     PISSN: 00264946     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (40)

1. Hogg RJ, Furth S, Lemley KV, Portman R, Schwartz GJ, Coresh J et al. National Kidney Foundation's Kidney Disease Outcomes Quality Initiative clinical practice guidelines for chronic kidney disease in children and adolescents: evaluation, classification, and stratification. Pediatrics 2003;111:1416-21.
2. Smith JM, Stablein DM, Munoz R, Hebert D, McDonald RA. Contributions of the Transplant Registry: The 2006 Annual Report of the North American Pediatric Renal Trials and Collaborative Studies (NAPRTCS). Pediatr Transplant 2007;11:366-73.
3. Town M, Jean G, Cherqui S, Attard M, Forestier L, Whitmore SA et al. A novel gene encoding an integral membrane protein is mutated in nephropathic cystinosis. Nat Genet 1998;18:319-24.
4. Gahl WA, Thoene JG, Schneider JA. Cystinosis. N Engl J Med 2002;347:111-21.
5. Chan AM, Lynch MJ, Bailey JD, Ezrin C, Fraser D. Hypothyroidism in cystinosis. A clinical, endocrinologic and histologic study involving sixteen patients with cystinosis. Am J Med 1970;48:678-92.
6. Fivush B, Green OC, Porter CC, Balfe JW, O'Regan S, Gahl WA. Pancreatic endocrine insufficiency in posttransplant cystinosis. Am J Dis Child 1987;141:1087-9.
7. Fivush B, Flick JA, Gahl WA. Pancreatic exocrine insufficiency in a patient with nephropathic cystinosis. J Pediatr 1988;112:49-51
8. Winkler L, Offner G, Krull F, Brodehl J. Growth and pubertal development in nephropathic cystinosis. Eur J Pediatr 1993;152:244-9.
9. Chik CL, Friedman A, Merriam GR, Gahl WA. Pituitary-testicular function in nephropathic cystinosis. Ann Intern Med 1993;119:568-75.
10. Besouw MT, Kremer JA, Janssen MC, Levtchenko EN. Fertility status in male cystinosis patients treated with cysteamine. Fertil Steril 2010;93:1880-831.
11. Gahl WA, Reed GF, Thoene JG, Shulman JD, Rizzo WB, Jonas AJ et al. Cysteamine therapy for children with nephropathic cystinosis. N Engl J Med 1987;316:971-7.
12. Abitbol C, Foreman JW, Strife CF, McEnery PT. Quantitation of growth deficits in children with renal diseases. Semin Nephrol 1989;9:31-6.
13. Elenberg E, Norling LL, Kleinman RE, Ingelfinger JR. Feeding problems in cystinosis. Pediatr Nephrol 1998;12:365-70.
14. Mahan JD, Warady BA. Assessment and treatment of short stature in pediatric patients with chronic kidney disease:a consensus statement. Pediatr Nephrol 2006;21:917-30.
15. Coleman JE, Watson AR. Gastrostomy buttons for nutritional support in children with cystinosis. Pediatr Nephrol 2000;14:833-6.
16. Norden AG, Lapsley M, Lee PJ, Pusey CD, Scheinman SJ, Tam FWK et al. Glomerular protein sieving and implications for renal failure in Fanconi syndrome. Kidney Int 2001;60:1885-92.
17. Wilmer MJ, Christensen EI, van den Heuvel LP, Monnens LA, Levtchenko EN. Urinary protein excretion pattern and renal expression of megalin and cubilin in nephropathic cystinosis. Am J Kidney Dis 2008;51:893-903.
18. Steinherz R, Chesney RW, Schulman JD, DeLuca HF, Phelps M. Circulating vitamin D metabolites in nephropathic cystinosis. J Pediatr 1983;102:592-4.
19. Etches P, Pickering D, Smith R. Cystinotic rickets treated with vitamin D metabolites. Arch Dis Child 1977;52:661-4.
20. Verge CF, Lam A, Simpson JM, Cowell CT, Howard NJ, Silink M. Effects of therapy in X-linked hypophosphatemic rickets. N Engl J Med 1991;325:1843-8.
21. Zimakas PJ, Sharma AK, Rodd CJ. Osteopenia and fractures in cystinotic children post renal transplantation. Pediatr Nephrol 2003;18:384-90.
22. Gahl WA, Balog JZ, Kleta R. Nephropathic cystinosis in adults.natural history and effects of oral cysteamine therapy. Ann Intern Med 2007;147:242-50.
23. Belldina EB, Huang MY, Schneider JA, Brundage RC, Tracy TS. Steady-state pharmacokinetics and pharmacodynamics of cysteamine bitartrate in paediatric nephropathic cystinosis patients. Br J Clin Pharmacol 2003;56:520-5.
24. Levtchenko EN, van Dael CM, de Graaf-Hess AC, Wilmer MJ, Van den Heuvel LP, Monnens LA et al. Strict cysteamine dose regimen is required to prevent nocturnal cystine accumulation in cystinosis. Pediatr Nephrol 2006;21:110-3.
25. Szabo S, Reichlin S. Somatostatin in rat tissues is depleted by cysteamine administration. Endocrinology 1981;109:2255-7.
26. Fukuhara S, Suzuki H, Masaoka T, Arakawa M, Hosoda H, Minegishi Y et al. Enhanced ghrelin secretion in rats with cysteamine-induced duodenal ulcers. Am J Physiol Gastrointest Liver Physiol 2005;289:G138-G145.
27. Yang CM, Chen AG, Hong QH, Liu JX, Liu JS. Effects of cysteamine on growth performance, digestive enzyme activities, and metabolic hormones in broilers. Poult Sci 2006;85:1912-6.
28. Xiao D, Lin HR. Cysteamine-a somatostatin-inhibiting agent-induced growth hormone secretion and growth acceleration in juvenile grass carp (Ctenopharyngodon idellus). Gen Comp Endocrinol 2003;134:285-95.
29. Tse MC, Cheng CH, Chan KM. Effects of chronic cysteamine treatment on growth enhancement and insulin-like growth factor I and II mRNA levels in common carp tissues. Br J Nutr 2006;96:650-9.
30. Besouw MT, Levtchenko EN, Willemsen MA, Noordam K. Growth hormone producing prolactinoma in juvenile cystinosis:a simple coincidence? Pediatr Nephrol 2008;23:307-10.
31. Wuhl E, Haffner D, Offner G, Broyer M, Van't HW, Mehls O. Long-term treatment with growth hormone in short children with nephropathic cystinosis. J Pediatr 2001;138.880-7.
32. Wuhl E, Haffner D, Gretz N, Offner G, Van't Hoff WG, Broyer M et al. Treatment with recombinant human growth hormone in short children with nephropathic cystinosis:no evidence for increased deterioration rate of renal function. The European Study Group on Growth Hormone Treatment in Short Children with Nephropathic Cystinosis. Pediatr Res 1998;43:484-8.
33. Andersson HC, Markello T, Schneider JA, Gahl WA. Effect of growth hormone treatment on serum creatinine concentration in patients with cystinosis and chronic renal disease. J Pediatr 1992;120:716-20.
34. Dohil R, Newbury RO, Sellers ZM, Deutsch R, Schneider JA. The evaluation and treatment of gastrointestinal disease in children with cystinosis receiving cysteamine. J Pediatr 2003;143:224-30.
35. Besouw M, Blom H, Tangerman A, de Graaf-Hess A, Levtchenko E. The origin of halitosis in cystinotic patients due to cysteamine treatment. Mol Genet Metab 2007;91:228-33.
36. Dohil R, Fidler M, Gangoiti JA, Kaskel F, Schneider JA, Barshop BA. Twice-daily cysteamine bitartrate therapy for children with cystinosis. J Pediatr 2010;156:71-5.
37. Edmondson SR, Baker NL, Oh J, Kovacs G, Werther GA, Mehls O. Growth hormone receptor abundance in tibial growth plates of uremic rats:GH/IGF-I treatment. Kidney Int 2000;58:62-70.
38. Hazel SJ, Gillespie CM, Moore RJ, Clark RG, Jureidini KF, Martin AA. Enhanced body growth in uremic rats treated with IGF-I and growth hormone in combination. Kidney Int 1994;46:58-68.
39. Cherqui S, Sevin C, Hamard G, Kalatzis V, Sich M, Pequignot MO et al. Intralysosomal cystine accumulation in mice lacking cystinosin, the protein defective in cystinosis. Mol Cell Biol 2002;22:7622-32.
40. Nevo N, Chol M, Bailleux A, Kalatzis V, Morisset L, Devuyst O et al. Renal phenotype of the cystinosis mouse model is dependent upon genetic background. Nephrol Dial Transplant 2009 [Epub ahead of print].