Treatment with recombinant human growth hormone in short children with nephropathic cystinosis: No evidence for increased deterioration rate of renal function

Elke Wühl, Dieter Haffner, Norbert Gretz, Gisela Offner, William G. Van't Hoff, Michel Broyer, Otto Mehls, Gérard Champion, Sally Hulton, Klaus Eugen Bonzel, Joachim Kreuder, Dirk E. Müller-Wiefel, Gisela Offner, Norbert Albers, Dieter Haffner, Otto Mehls, Elke Wühl, William Van't Hoff, Susan Rigden, Hans Peter WeberMichel Foulard, Pierre Cochat, Gianluigi Ardissino, Klaus G. Pistor, Marie Jeanne Krier, Jean Luc André, Maria C J W De Jong, Michel Broyer, Chantal Loirat, Georges Deschenes, Albert Bensman, Friedrich K. Trefz, Marianne Wigger, Gilbert Landthaler, Michel Fischbach, François Bouissou, Hubert Nivet

Research output: Contribution to journalArticle

Abstract

To evaluate the effect of long-term treatment with recombinant human GH (rhGH) on renal function in short children with nephropathic cystinosis with and without concomitant cysteamine treatment, 36 growth-retarded children with nephropathic cystinosis (age 7.3 ± 2.7 y; creatinine clearance (C(CR)) 50 ± 27 mL (min X 1.73 m2)-1 were treated with 1 IU rhGH/kg/wk for up to 5 y. The rise in serum creatinine before and during rhGH treatment was compared with that in a historical control group of cystinotic patients. The effect of concomitant cysteamine treatment on the evolution of renal function before and after the start or rhGH was evaluated separately in patients without (group A) and with cysteamine treatment (group B). The decline of C(CR) was also compared with that in noncystinotic patients with chronic renal failure with and without rhGH treatment. At study entry, serum creatinine values in group A were similar to those in the historical controls, whereas group B had significantly lower serum creatinine values. Treatment with rhGH did not accelerate the rise in creatinine independently of cysteamine treatment. There were no significant differences in the mean decline of C(Cr) per year in cystinotic compared with noncystinotic patients with chronic renal failure with or without rhGH treatment. rhGH therapy for up to 5 y does not accelerate the deterioration of renal function. This justifies the continuation of controlled studies of rhGH treatment in these patients. The study also provides further evidence that cysteamine therapy reduces the progression of renal failure in children with cystinosis.

Original languageEnglish
Pages (from-to)484-488
Number of pages5
JournalPediatric Research
Volume43
Issue number4 I
Publication statusPublished - Apr 1998

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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    Wühl, E., Haffner, D., Gretz, N., Offner, G., Van't Hoff, W. G., Broyer, M., Mehls, O., Champion, G., Hulton, S., Bonzel, K. E., Kreuder, J., Müller-Wiefel, D. E., Offner, G., Albers, N., Haffner, D., Mehls, O., Wühl, E., Van't Hoff, W., Rigden, S., ... Nivet, H. (1998). Treatment with recombinant human growth hormone in short children with nephropathic cystinosis: No evidence for increased deterioration rate of renal function. Pediatric Research, 43(4 I), 484-488.