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Progression of chronic renal failure in a historical group of patients with nephropathic cystinosis

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Abstract

In a historical group of 205 patients with infantile or adolescent cystinosis treated without cysteamine, the rate of deterioration of renal function was analysed retrospectively. Patient survival curves and renal survival data are presented. Longitudinal data of serum creatinine values (n=3280) in 157 patients were plotted for each patient, smoothed by the method of the running medians and grouped into 12 serum creatinine classes. In every patient the age at the last smoothed serum creatinine value observed in each serum creatinine class was determined. These virtual age values were then summarized per serum creatinine class, expressed as median and centiles and plotted, thus describing the “natural” course of the disease. In 9 pairs of affected siblings the rate of progression showed a median difference of about 12 months. Our data describe the “natural” course of nephropathic cystinosis and can be used as a prognostic aid for recently detected patients. The data can also be applied for the assessment of the influence of new therapeutic strategies on the rate of progression of renal failure in cystinotic patients.

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Authors and Affiliations

  1. Research Institute of Child Nutrition, Heinstück 11, D-44225, Dortmund, Germany

    Friedrich Manz

  2. Clinic of Nephrology, Klinikum Mannheim, University of Heidelberg, D-68167, Mannheim, Germany

    Norbert Gretz

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  1. Friedrich Manz
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  2. Norbert Gretz
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European Collaborative Study on Cystinosis

Additional information

European Collaborative Study with the participation of the following centres of six European countries:France: Paris (M. Broyer), Strasbourg (J. Geisert);Germany: Bocholt (K. Grote), Bonn (H. P. Weber), Coburg (H. Hennemann), Düsseldorf (H. J. Bremer), Erlangen (D. Michalk), Essen (C. Feldhoff, K. Pistor), Freiburg (B. Geschöll-Bauer, F. Schindera), Giessen (H. Wolf), Hamburg (C. Bender-Götze, R. Grüttner), Hannover (J. Brodehl, J. H. H. Ehrich), Heidelberg (E. Harms, F. Manz, K. Schärer, H. Schmidt), Köln (M. Bulla), Mainz (H. Schulte-Wissermann), Marburg (M. Brandis, U. Willenbockel), München (W. Endres, B. Klare), Münster (L. Diekmann), Reutlingen (J. Rau), Stuttgart (R. Augustin, W. Hagge), Ulm (D. Leupold), Wolfsburg (H. Löhr), Würzburg (D. Gekle, F. Ott);Great Britain: Cardiff (K. Verrier-Jones), London (T. M. Barratt, C. Chantler, M. J. Dillon, G. B. Haycock);Portugal: Porto (A. Oliveira);Spain: Barcelona (L. Callis);Switzerland: Basel (F. Egli), Winterthur (A. Fanconi)

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Manz, F., Gretz, N. & European Collaborative Study on Cystinosis. Progression of chronic renal failure in a historical group of patients with nephropathic cystinosis. Pediatr Nephrol 8, 466–471 (1994). https://doi.org/10.1007/BF00856532

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  • DOI: https://doi.org/10.1007/BF00856532

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