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

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/m2) 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/m2 every 4 weeks for 12 weeks, followed by 4 doses of 25 mg/m2 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.

• Authors: Monique E Cho, Mary H Branton, David A Smith, Linda Bartlett, Lilian Howard, James C Reynolds, Donald Rosenstein, Sanjeev Sethi, M Berenice Nava, Laura Barisoni, Fernando C Fervenza, Jeffrey B Kopp
• Journal: American journal of nephrology (Am J Nephrol) Vol. 49 Issue 5 Pg. 377-385 ( 2019) ISSN: 1421-9670 [Electronic] Switzerland
• PMID: 30965344 (Publication Type: Clinical Trial, Phase II, Journal Article, Randomized Controlled Trial, Research Support, N.I.H., Intramural)
• Copyright: Published by S. Karger AG, Basel.

Chemical References

• Immunosuppressive Agents
• Dexamethasone

Topics

• Administration, Oral
• Adult
• Aged
• Dexamethasone (administration & dosage, adverse effects)
• Female
• Follow-Up Studies
• Glomerulosclerosis, Focal Segmental (complications, drug therapy, immunology)
• Humans
• Immunosuppressive Agents (administration & dosage, adverse effects)
• Male
• Middle Aged
• Nephrotic Syndrome (drug therapy, immunology)
• Pulse Therapy, Drug
• Remission Induction (methods)
• Young Adult