Open-Label Clinical Trials of Oral Pulse Dexamethasone for Adults with Idiopathic Nephrotic Syndrome

Monique E. Cho, Mary H. Branton, David A. Smith, Linda Bartlett, Lilian Howard, James C. Reynolds, Donald Rosenstein, Sanjeev M Sethi, M. Berenice Nava, Laura Barisoni, Fernando Custodio Fervenza, Jeffrey B. Kopp

Research output: Contribution to journalArticle

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Abstract

Background: In adults with primary focal segmental glomerulosclerosis (FSGS), daily prednisone may induce complete remissions (CR) and partial remissions (PR), but relapses are frequent and adverse events are common. Methods: We carried out 2 open-label, uncontrolled trials to explore the efficacy and tolerability of pulse oral dexamethasone as an alternative to daily prednisone. We enrolled adult patients with proteinuria > 3.5 g/day despite the use of renin-angiotensin-aldosterone blockade. In the first trial, we enrolled 14 subjects with FSGS and administered 4 dexamethasone doses (25 mg/m 2 ) daily for 4 days, repeated every 28 days over 32 weeks. The second trial involved a more intensive regimen. Eight subjects received 4 dexamethasone doses of 50 mg/ 2 every 4 weeks for 12 weeks, followed by 4 doses of 25 mg/m 2 every 4 weeks for 36 weeks; subjects were randomized to 2 doses every 2 weeks or 4 doses every 4 weeks. Results: In the first trial, we enrolled 13 subjects with FSGS and 1 with minimal change disease and found a combined CR and PR rate of 36%. In the second trial, we enrolled 8 subjects. The combined CR and PR rate was 29%. Analysis combining both trials showed a combined CR and PR rate of 33%. Adverse events were observed in 32% of subjects, with mood symptoms being most common. There were no serious adverse events related to the study. Conclusion: We conclude that high dose oral dexamethasone is well tolerated by adults with idiopathic nephrotic syndrome and may have some efficacy.

Original languageEnglish (US)
Pages (from-to)377-385
Number of pages9
JournalAmerican Journal of Nephrology
DOIs
StatePublished - Jan 1 2019

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Dexamethasone
Clinical Trials
Focal Segmental Glomerulosclerosis
Prednisone
Lipoid Nephrosis
Angiotensins
Aldosterone
Proteinuria
Renin
Recurrence
Congenital Nephrosis

ASJC Scopus subject areas

  • Nephrology

Cite this

Cho, M. E., Branton, M. H., Smith, D. A., Bartlett, L., Howard, L., Reynolds, J. C., ... Kopp, J. B. (2019). Open-Label Clinical Trials of Oral Pulse Dexamethasone for Adults with Idiopathic Nephrotic Syndrome. American Journal of Nephrology, 377-385. https://doi.org/10.1159/000497064

Open-Label Clinical Trials of Oral Pulse Dexamethasone for Adults with Idiopathic Nephrotic Syndrome. / Cho, Monique E.; Branton, Mary H.; Smith, David A.; Bartlett, Linda; Howard, Lilian; Reynolds, James C.; Rosenstein, Donald; Sethi, Sanjeev M; Nava, M. Berenice; Barisoni, Laura; Fervenza, Fernando Custodio; Kopp, Jeffrey B.

In: American Journal of Nephrology, 01.01.2019, p. 377-385.

Research output: Contribution to journalArticle

Cho, ME, Branton, MH, Smith, DA, Bartlett, L, Howard, L, Reynolds, JC, Rosenstein, D, Sethi, SM, Nava, MB, Barisoni, L, Fervenza, FC & Kopp, JB 2019, 'Open-Label Clinical Trials of Oral Pulse Dexamethasone for Adults with Idiopathic Nephrotic Syndrome', American Journal of Nephrology, pp. 377-385. https://doi.org/10.1159/000497064
Cho, Monique E. ; Branton, Mary H. ; Smith, David A. ; Bartlett, Linda ; Howard, Lilian ; Reynolds, James C. ; Rosenstein, Donald ; Sethi, Sanjeev M ; Nava, M. Berenice ; Barisoni, Laura ; Fervenza, Fernando Custodio ; Kopp, Jeffrey B. / Open-Label Clinical Trials of Oral Pulse Dexamethasone for Adults with Idiopathic Nephrotic Syndrome. In: American Journal of Nephrology. 2019 ; pp. 377-385.
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AU - Reynolds, James C.

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