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 language | English |
|---|---|
| Pages (from-to) | 2485-2491 |
| Number of pages | 7 |
| Journal | Clinical Journal of the American Society of Nephrology |
| Volume | 6 |
| Issue number | 10 |
| DOIs | |
| Publication status | Published - Oct 1 2011 |
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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 journal › Article
}
TY - JOUR
T1 - Improvement in the renal prognosis in nephropathic cystinosis
AU - van Stralen, Karlijn J.
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.
PY - 2011/10/1
Y1 - 2011/10/1
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.
UR - http://www.scopus.com/inward/record.url?scp=80053968071&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=80053968071&partnerID=8YFLogxK
U2 - 10.2215/CJN.02000311
DO - 10.2215/CJN.02000311
M3 - Article
C2 - 21868618
AN - SCOPUS:80053968071
VL - 6
SP - 2485
EP - 2491
JO - Clinical journal of the American Society of Nephrology : CJASN
JF - Clinical journal of the American Society of Nephrology : CJASN
SN - 1555-9041
IS - 10
ER -