Long-term evaluation of chlorambucil plus prednisone in the idiopathic nephrotic syndrome of childhood

S. A. Williams, Sudesh P Makker, J. R. Ingelfinger, W. E. Grupe

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55 Scopus citations


The authors evaluated the long-term effects of a 5 to 15-wk course of chlorambucil and prednisone in 59 children with idiopathic nephrotic syndrome who had previously received prednisone alone and had had frequent relapses or steroid dependency or resistance. By actuarial analysis of 65 courses of dual therapy followed up for 1 to 12 yr (mean, 5.0), they found that 95% of patients were in remission at 1 yr and 85% at 4 yr. All but 2 had remissions lasting longer than those induced by steroids alone, and only 8 others had 1 or more relapses after therapy. Life-table analysis of 2 dosage schedules of chlorambucil at 4 yr showed that 91% of patients on low doses and 80% of those on high doses were still in remission. Although immediate complications were minimal, the potential for long-term toxicity still requires careful selection of patients who receive chlorambucil. Prolonged use of chlorambucil in daily doses above 0.3 mg/kg of body weight per day or cumulative doses above 14 mg/kg is no longer warranted. Measured in terms of both the immediate and long-term responses, chlorambucil appears to lower the frequency of relapses in idiopathic nephrotic syndrome.

Original languageEnglish (US)
Pages (from-to)929-933
Number of pages5
JournalNew England Journal of Medicine
Issue number17
StatePublished - 1980
Externally publishedYes

ASJC Scopus subject areas

  • Medicine(all)


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