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Volumn 2, Issue 3, 2017, Pages 451-460

A Drug Development Tool for Trial Enrichment in Patients With Autosomal Dominant Polycystic Kidney Disease

Author keywords

end stage renal disease; renal function decline; total kidney volume; trial enrichment

Indexed keywords


EID: 85024852781     PISSN: 24680249     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ekir.2017.02.011     Document Type: Article
Times cited : (22)

References (24)
  • 1
    • 33845989853 scopus 로고    scopus 로고
    • Polycystic kidney disease: genes, proteins, animal models, disease mechanisms and therapeutic opportunities
    • Torres, V.E., Harris, P.C., Polycystic kidney disease: genes, proteins, animal models, disease mechanisms and therapeutic opportunities. J Intern Med 261 (2007), 17–31.
    • (2007) J Intern Med , vol.261 , pp. 17-31
    • Torres, V.E.1    Harris, P.C.2
  • 2
    • 0347357836 scopus 로고    scopus 로고
    • Polycystic kidney disease
    • Wilson, P.D., Polycystic kidney disease. N Engl J Med 350 (2004), 151–164.
    • (2004) N Engl J Med , vol.350 , pp. 151-164
    • Wilson, P.D.1
  • 3
    • 84863230673 scopus 로고    scopus 로고
    • Kidney volume and functional outcomes in autosomal dominant polycystic kidney disease
    • Chapman, A.B., Bost, J.E., Torres, V.E., et al. Kidney volume and functional outcomes in autosomal dominant polycystic kidney disease. Clin J Am Soc Nephrol 7 (2012), 479–486.
    • (2012) Clin J Am Soc Nephrol , vol.7 , pp. 479-486
    • Chapman, A.B.1    Bost, J.E.2    Torres, V.E.3
  • 4
    • 84924196321 scopus 로고    scopus 로고
    • Imaging classification of autosomal dominant polycystic kidney disease: a simple model for selecting patients for clinical trials
    • Irazabal, M.V., Rangel, L.J., Bergstralh, E.J., et al. Imaging classification of autosomal dominant polycystic kidney disease: a simple model for selecting patients for clinical trials. J Am Soc Nephrol 26 (2015), 160–172.
    • (2015) J Am Soc Nephrol , vol.26 , pp. 160-172
    • Irazabal, M.V.1    Rangel, L.J.2    Bergstralh, E.J.3
  • 5
    • 37249043883 scopus 로고    scopus 로고
    • Determinants of renal volume in autosomal-dominant polycystic kidney disease
    • Grantham, J.J., Cook, L.T., Torres, V.E., et al. Determinants of renal volume in autosomal-dominant polycystic kidney disease. Kidney Int 73 (2008), 108–116.
    • (2008) Kidney Int , vol.73 , pp. 108-116
    • Grantham, J.J.1    Cook, L.T.2    Torres, V.E.3
  • 6
    • 84942294959 scopus 로고    scopus 로고
    • Therapeutic area data standards for autosomal dominant polycystic kidney disease: a report from the Polycystic Kidney Disease Outcomes Consortium (PKDOC)
    • Perrone, R.D., Neville, J., Chapman, A.B., et al. Therapeutic area data standards for autosomal dominant polycystic kidney disease: a report from the Polycystic Kidney Disease Outcomes Consortium (PKDOC). Am J Kidney Dis 66 (2015), 583–590.
    • (2015) Am J Kidney Dis , vol.66 , pp. 583-590
    • Perrone, R.D.1    Neville, J.2    Chapman, A.B.3
  • 7
    • 8644246036 scopus 로고    scopus 로고
    • Joint modeling of longitudinal and time-to-event data: an overview
    • Tsiatis, A.A., Davidian, M., Joint modeling of longitudinal and time-to-event data: an overview. Statistica Sinica 14 (2004), 809–834.
    • (2004) Statistica Sinica , vol.14 , pp. 809-834
    • Tsiatis, A.A.1    Davidian, M.2
  • 8
    • 80052418167 scopus 로고    scopus 로고
    • Joint modelling of longitudinal and time-to-event data with application to predicting abdominal aortic aneurysm growth and rupture
    • Sweeting, M.J., Thompson, S.G., Joint modelling of longitudinal and time-to-event data with application to predicting abdominal aortic aneurysm growth and rupture. Biom J 53 (2011), 750–763.
    • (2011) Biom J , vol.53 , pp. 750-763
    • Sweeting, M.J.1    Thompson, S.G.2
  • 9
    • 33645562937 scopus 로고    scopus 로고
    • The sensitivity and specificity of markers for event times
    • Cai, T., Pepe, M.S., Zheng, Y., et al. The sensitivity and specificity of markers for event times. Biostatistics 7 (2006), 182–197.
    • (2006) Biostatistics , vol.7 , pp. 182-197
    • Cai, T.1    Pepe, M.S.2    Zheng, Y.3
  • 10
    • 0036176161 scopus 로고    scopus 로고
    • K/DOQI clinical practice guidelines for chronic kidney disease: evaluation, classification, and stratification
    • National Kidney Foundation. K/DOQI clinical practice guidelines for chronic kidney disease: evaluation, classification, and stratification. Am J Kidney Dis 39:Suppl 1 (2002), S1–S266.
    • (2002) Am J Kidney Dis , vol.39 , pp. S1-S266
  • 11
    • 70349505844 scopus 로고    scopus 로고
    • Estimating glomerular filtration rate
    • Miller, W.G., Estimating glomerular filtration rate. Clin Chem Lab Med 47 (2009), 1017–1019.
    • (2009) Clin Chem Lab Med , vol.47 , pp. 1017-1019
    • Miller, W.G.1
  • 12
    • 84902826499 scopus 로고    scopus 로고
    • Decline in estimated glomerular filtration rate and subsequent risk of end-stage renal disease and mortality
    • Coresh, J., Turin, T.C., Matsushita, K., et al., CKD Prognosis Consortium. Decline in estimated glomerular filtration rate and subsequent risk of end-stage renal disease and mortality. JAMA 311 (2014), 2518–2531.
    • (2014) JAMA , vol.311 , pp. 2518-2531
    • Coresh, J.1    Turin, T.C.2    Matsushita, K.3
  • 13
    • 84941354694 scopus 로고    scopus 로고
    • Predictors and the subsequent risk of end-stage renal disease—usefulness of 30% decline in estimated GFR over 2 years
    • e0132927
    • Chang, W.X., Asakawa, S., Toyoki, D., et al. Predictors and the subsequent risk of end-stage renal disease—usefulness of 30% decline in estimated GFR over 2 years. PLoS One, 10, 2015 e0132927.
    • (2015) PLoS One , vol.10
    • Chang, W.X.1    Asakawa, S.2    Toyoki, D.3
  • 14
    • 0000343716 scopus 로고
    • Submodel selection and evaluation in regression. The X-random case
    • Breiman, L., Spector, P., Submodel selection and evaluation in regression. The X-random case. Int Stat Rev 60 (1992), 291–319.
    • (1992) Int Stat Rev , vol.60 , pp. 291-319
    • Breiman, L.1    Spector, P.2
  • 15
    • 84918528189 scopus 로고    scopus 로고
    • Blood pressure in early autosomal dominant polycystic kidney disease
    • Schrier, R.W., Abebe, K.Z., Perrone, R.D., et al., for the HALT-PKD Trial Investigators. Blood pressure in early autosomal dominant polycystic kidney disease. New Engl J Med 371 (2014), 2255–2266.
    • (2014) New Engl J Med , vol.371 , pp. 2255-2266
    • Schrier, R.W.1    Abebe, K.Z.2    Perrone, R.D.3
  • 16
    • 77956029702 scopus 로고    scopus 로고
    • Sirolimus and kidney growth in autosomal dominant polycystic kidney disease
    • Serra, A.L., Poster, D., Kistler, A.D., et al. Sirolimus and kidney growth in autosomal dominant polycystic kidney disease. N Engl J Med 363 (2010), 820–829.
    • (2010) N Engl J Med , vol.363 , pp. 820-829
    • Serra, A.L.1    Poster, D.2    Kistler, A.D.3
  • 17
    • 85024925269 scopus 로고    scopus 로고
    • Qualification Process for Drug Development Tools, 2014. Available at: Accessed December 30
    • Food and Drug Administration. Qualification Process for Drug Development Tools, 2014. Available at: http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm230597.pdf. Accessed December 30, 2016.
    • (2016)
    • Food and Drug Administration1
  • 18
    • 85024874943 scopus 로고    scopus 로고
    • Qualification of novel methodologies for drug development: guidance to applicants, 2014. Available at: Accessed December 30
    • European Medicines Agency. Qualification of novel methodologies for drug development: guidance to applicants, 2014. Available at: http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2009/10/WC500004201.pdf. Accessed December 30, 2016.
    • (2016)
    • European Medicines Agency1
  • 19
    • 28044456536 scopus 로고    scopus 로고
    • Sonographic assessment of the severity and progression of autosomal dominant polycystic kidney disease: the Consortium of Renal Imaging Studies in Polycystic Kidney Disease (CRISP)
    • O'Neill, W.C., Robbin, M.L., Bae, K.T., et al. Sonographic assessment of the severity and progression of autosomal dominant polycystic kidney disease: the Consortium of Renal Imaging Studies in Polycystic Kidney Disease (CRISP). Am J Kidney Dis 46 (2005), 1058–1064.
    • (2005) Am J Kidney Dis , vol.46 , pp. 1058-1064
    • O'Neill, W.C.1    Robbin, M.L.2    Bae, K.T.3
  • 20
    • 85024892275 scopus 로고    scopus 로고
    • Total kidney volume is a prognostic biomarker of renal function decline and progression to end-stage renal disease in patients with autosomal dominant polycystic kidney disease
    • Perrone, R.D., Mouksassi, M.-S., Romero, K., et al. Total kidney volume is a prognostic biomarker of renal function decline and progression to end-stage renal disease in patients with autosomal dominant polycystic kidney disease. Kidney Int. Rep 2 (2017), 442–450.
    • (2017) Kidney Int. Rep , vol.2 , pp. 442-450
    • Perrone, R.D.1    Mouksassi, M.-S.2    Romero, K.3
  • 21
    • 84999079279 scopus 로고    scopus 로고
    • Change in measured GFR versus eGFR and CKD outcomes
    • Ku, E., Xie, D., Shlipak, M., et al., CRIC Study Investigators. Change in measured GFR versus eGFR and CKD outcomes. J Am Soc Nephrol 27 (2016), 2196–2204.
    • (2016) J Am Soc Nephrol , vol.27 , pp. 2196-2204
    • Ku, E.1    Xie, D.2    Shlipak, M.3
  • 22
    • 85024926568 scopus 로고    scopus 로고
    • Biomarker Qualification Program. Available at: Accessed December 30
    • Food and Drug Administration. Biomarker Qualification Program. Available at: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm284076.htm. Accessed December 30, 2016.
    • (2016)
    • Food and Drug Administration1
  • 23
    • 85024862482 scopus 로고    scopus 로고
    • Guidance for Industry. Qualification of Biomarker —Total Kidney Volume in Studies for Treatment of Autosomal Dominant Polycystic Kidney Disease. Available at: Accessed December 30
    • Guidance for Industry. Qualification of Biomarker —Total Kidney Volume in Studies for Treatment of Autosomal Dominant Polycystic Kidney Disease. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM458483.pdf. Accessed December 30, 2016.
    • (2016)
  • 24
    • 70349459455 scopus 로고    scopus 로고
    • The coalition against major diseases: developing tools for an integrated drug development process for Alzheimer's and Parkinson's diseases
    • Romero, K., de Mars, M., Frank, D., et al. The coalition against major diseases: developing tools for an integrated drug development process for Alzheimer's and Parkinson's diseases. Clin Pharmacol Ther 86 (2009), 365–367.
    • (2009) Clin Pharmacol Ther , vol.86 , pp. 365-367
    • Romero, K.1    de Mars, M.2    Frank, D.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.