Improvement in the renal prognosis in nephropathic cystinosis

Karlijn J. van Stralen, Francesco Emma, Kitty J. Jager, Enrico Verrina, Franz Schaefer, Guido F. Laube, Malcolm A. Lewis, Elena N. Levtchenko

Research output: Contribution to journalArticle

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Abstract

Background and objectives: Nephropathic cystinosis (NC) is an autosomal recessive disorder occurring in one to two per 100,000 newborns. Because of the rarity of NC, long-term outcome data are scarce. Design, setting, participants, & measurements: 245 NC patients from 18 countries provided data to the ESPN/ERA-EDTA registry. We matched NC patients on renal replacement therapy (RRT) to non-NC children on RRT. Results: Between 1979 and 2008, mean age at the start of RRT among NC children increased by 0.15 year per calendar year (95% confidence interval, 0.10 to 0.21) from 8.8 to 12.7 years, whereas we did not observe this in non-NC children. Five-year survival after the start of RRT improved in NC patients from 86.1% (before 1990) to 100% (since 2000) as compared with the control population (89.6% and 94.0%). NC patients received a renal allograft more often (relative risk, 1.09; 95% confidence interval, 1.00 to 1.17) as compared with matched RRT children, and 5-year graft survival was better (94.0% versus 84.0%). NC dialysis patients were less often hypertensive than non-NC children matched for age, country, and dialysis modality (42.7% versus 51.7%) and had lower parathyroid hormone levels (median, 56 versus 140 pg/ml). Although height at start of RRT slightly improved during the past decade, children with NC remained significantly shorter than non-NC children at the start of RRT. Conclusions: We demonstrated improved survival of the renal function as well as better patient and graft survival after the start of RRT in a large European cohort of NC patients over the last two decades.

Original languageEnglish
Pages (from-to)2485-2491
Number of pages7
JournalClinical Journal of the American Society of Nephrology
Volume6
Issue number10
DOIs
Publication statusPublished - Oct 1 2011

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Cystinosis
Renal Replacement Therapy
Kidney
Graft Survival
Dialysis
Confidence Intervals

ASJC Scopus subject areas

  • Nephrology
  • Transplantation
  • Epidemiology
  • Critical Care and Intensive Care Medicine

Cite this

Improvement in the renal prognosis in nephropathic cystinosis. / van Stralen, Karlijn J.; Emma, Francesco; Jager, Kitty J.; Verrina, Enrico; Schaefer, Franz; Laube, Guido F.; Lewis, Malcolm A.; Levtchenko, Elena N.

In: Clinical Journal of the American Society of Nephrology, Vol. 6, No. 10, 01.10.2011, p. 2485-2491.

Research output: Contribution to journalArticle

van Stralen, KJ, Emma, F, Jager, KJ, Verrina, E, Schaefer, F, Laube, GF, Lewis, MA & Levtchenko, EN 2011, 'Improvement in the renal prognosis in nephropathic cystinosis', Clinical Journal of the American Society of Nephrology, vol. 6, no. 10, pp. 2485-2491. https://doi.org/10.2215/CJN.02000311
van Stralen, Karlijn J. ; Emma, Francesco ; Jager, Kitty J. ; Verrina, Enrico ; Schaefer, Franz ; Laube, Guido F. ; Lewis, Malcolm A. ; Levtchenko, Elena N. / Improvement in the renal prognosis in nephropathic cystinosis. In: Clinical Journal of the American Society of Nephrology. 2011 ; Vol. 6, No. 10. pp. 2485-2491.
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abstract = "Background and objectives: Nephropathic cystinosis (NC) is an autosomal recessive disorder occurring in one to two per 100,000 newborns. Because of the rarity of NC, long-term outcome data are scarce. Design, setting, participants, & measurements: 245 NC patients from 18 countries provided data to the ESPN/ERA-EDTA registry. We matched NC patients on renal replacement therapy (RRT) to non-NC children on RRT. Results: Between 1979 and 2008, mean age at the start of RRT among NC children increased by 0.15 year per calendar year (95{\%} confidence interval, 0.10 to 0.21) from 8.8 to 12.7 years, whereas we did not observe this in non-NC children. Five-year survival after the start of RRT improved in NC patients from 86.1{\%} (before 1990) to 100{\%} (since 2000) as compared with the control population (89.6{\%} and 94.0{\%}). NC patients received a renal allograft more often (relative risk, 1.09; 95{\%} confidence interval, 1.00 to 1.17) as compared with matched RRT children, and 5-year graft survival was better (94.0{\%} versus 84.0{\%}). NC dialysis patients were less often hypertensive than non-NC children matched for age, country, and dialysis modality (42.7{\%} versus 51.7{\%}) and had lower parathyroid hormone levels (median, 56 versus 140 pg/ml). Although height at start of RRT slightly improved during the past decade, children with NC remained significantly shorter than non-NC children at the start of RRT. Conclusions: We demonstrated improved survival of the renal function as well as better patient and graft survival after the start of RRT in a large European cohort of NC patients over the last two decades.",
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AU - Emma, Francesco

AU - Jager, Kitty J.

AU - Verrina, Enrico

AU - Schaefer, Franz

AU - Laube, Guido F.

AU - Lewis, Malcolm A.

AU - Levtchenko, Elena N.

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N2 - Background and objectives: Nephropathic cystinosis (NC) is an autosomal recessive disorder occurring in one to two per 100,000 newborns. Because of the rarity of NC, long-term outcome data are scarce. Design, setting, participants, & measurements: 245 NC patients from 18 countries provided data to the ESPN/ERA-EDTA registry. We matched NC patients on renal replacement therapy (RRT) to non-NC children on RRT. Results: Between 1979 and 2008, mean age at the start of RRT among NC children increased by 0.15 year per calendar year (95% confidence interval, 0.10 to 0.21) from 8.8 to 12.7 years, whereas we did not observe this in non-NC children. Five-year survival after the start of RRT improved in NC patients from 86.1% (before 1990) to 100% (since 2000) as compared with the control population (89.6% and 94.0%). NC patients received a renal allograft more often (relative risk, 1.09; 95% confidence interval, 1.00 to 1.17) as compared with matched RRT children, and 5-year graft survival was better (94.0% versus 84.0%). NC dialysis patients were less often hypertensive than non-NC children matched for age, country, and dialysis modality (42.7% versus 51.7%) and had lower parathyroid hormone levels (median, 56 versus 140 pg/ml). Although height at start of RRT slightly improved during the past decade, children with NC remained significantly shorter than non-NC children at the start of RRT. Conclusions: We demonstrated improved survival of the renal function as well as better patient and graft survival after the start of RRT in a large European cohort of NC patients over the last two decades.

AB - Background and objectives: Nephropathic cystinosis (NC) is an autosomal recessive disorder occurring in one to two per 100,000 newborns. Because of the rarity of NC, long-term outcome data are scarce. Design, setting, participants, & measurements: 245 NC patients from 18 countries provided data to the ESPN/ERA-EDTA registry. We matched NC patients on renal replacement therapy (RRT) to non-NC children on RRT. Results: Between 1979 and 2008, mean age at the start of RRT among NC children increased by 0.15 year per calendar year (95% confidence interval, 0.10 to 0.21) from 8.8 to 12.7 years, whereas we did not observe this in non-NC children. Five-year survival after the start of RRT improved in NC patients from 86.1% (before 1990) to 100% (since 2000) as compared with the control population (89.6% and 94.0%). NC patients received a renal allograft more often (relative risk, 1.09; 95% confidence interval, 1.00 to 1.17) as compared with matched RRT children, and 5-year graft survival was better (94.0% versus 84.0%). NC dialysis patients were less often hypertensive than non-NC children matched for age, country, and dialysis modality (42.7% versus 51.7%) and had lower parathyroid hormone levels (median, 56 versus 140 pg/ml). Although height at start of RRT slightly improved during the past decade, children with NC remained significantly shorter than non-NC children at the start of RRT. Conclusions: We demonstrated improved survival of the renal function as well as better patient and graft survival after the start of RRT in a large European cohort of NC patients over the last two decades.

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