Abstract | BACKGROUND: Spontaneous remission is recognized in mycosis fungoides (MF) and Sézary syndrome (SS). OBJECTIVE: We analyzed the outcome of 44 patients with previously treated CD25-positive (CD25+), recurrent/persistent MF/SS randomly assigned to receive placebo as part of a phase III trial. METHODS: This trial investigated the efficacy and safety of two doses of denileukin diftitox in patients with MF/SS who had received up to 3 prior therapies. The primary end point was overall response rate. Multivariate regression analyses were used to assess the relationship between baseline covariates and clinical outcomes. RESULTS: The overall response rate was 15.9% for placebo recipients (complete response: 2.3%; partial response: 13.6%), reflecting the baseline rate of disease remission that can be expected in a clinical trial. The median progression-free survival (PFS) in the placebo arm was moderately short at 4.4 months compared with the active-agent arm but important to consider in the context of recent single-arm phase II studies of other therapies for MF/SS that report PFS of approximately 6 months. Multivariate analyses identified no significant effects of any baseline factors on either overall response rate or PFS, although there was a trend toward poorer PFS with advanced age. Because sepsis occurred significantly more often in the placebo arm versus the active-treatment arm, the role of antibiotics in causing remission cannot be discounted (6.8% vs 0%; P < .05). LIMITATIONS: This study had a relatively small sample size, yielding a wide 95% confidence interval. CONCLUSION: The results may serve as a useful comparator for other active-treatment studies of MF/SS that lack a placebo-control arm.
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Authors | H Miles Prince, Madeleine Duvic, Ann Martin, Wolfram Sterry, Chalid Assaf, David J Straus |
Journal | Journal of the American Academy of Dermatology
(J Am Acad Dermatol)
Vol. 67
Issue 5
Pg. 867-75
(Nov 2012)
ISSN: 1097-6787 [Electronic] United States |
PMID | 22285675
(Publication Type: Clinical Trial, Phase III, Journal Article, Research Support, Non-U.S. Gov't)
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Copyright | Copyright © 2011 American Academy of Dermatology, Inc. Published by Mosby, Inc. All rights reserved. |
Chemical References |
- Antineoplastic Agents
- Diphtheria Toxin
- Interleukin-2
- Interleukin-2 Receptor alpha Subunit
- Placebos
- Recombinant Fusion Proteins
- denileukin diftitox
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Topics |
- Antineoplastic Agents
(therapeutic use)
- Diphtheria Toxin
(therapeutic use)
- Humans
- Intention to Treat Analysis
- Interleukin-2
(therapeutic use)
- Interleukin-2 Receptor alpha Subunit
(metabolism)
- Multivariate Analysis
- Mycosis Fungoides
(metabolism, pathology, therapy)
- Neoplasm Regression, Spontaneous
- Placebos
- Quality of Life
- Recombinant Fusion Proteins
(therapeutic use)
- Sezary Syndrome
(metabolism, pathology, therapy)
- Skin Neoplasms
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