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Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis: results of a randomized, double-blind, multinational, placebo-controlled phase II/III trial
SUMMARY: BACKGROUND: Oral liarozole, a retinoic acid metabolism-blocking agent, may be an alternative to systemic retinoid therapy in patients with lamellar ichthyosis. OBJECTIVE: To demonstrate the efficacy and safety of once-daily oral liarozole in the treatment of moderate/severe lamellar ichthyo...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BlackWell Publishing Ltd
2014
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4232915/ https://www.ncbi.nlm.nih.gov/pubmed/24102348 http://dx.doi.org/10.1111/bjd.12626 |
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author | Vahlquist, A Blockhuys, S Steijlen, P van Rossem, K Didona, B Blanco, D Traupe, H |
author_facet | Vahlquist, A Blockhuys, S Steijlen, P van Rossem, K Didona, B Blanco, D Traupe, H |
author_sort | Vahlquist, A |
collection | PubMed |
description | SUMMARY: BACKGROUND: Oral liarozole, a retinoic acid metabolism-blocking agent, may be an alternative to systemic retinoid therapy in patients with lamellar ichthyosis. OBJECTIVE: To demonstrate the efficacy and safety of once-daily oral liarozole in the treatment of moderate/severe lamellar ichthyosis. METHODS: This was a double-blind, multinational, parallel phase II/III trial (NCT00282724). Patients aged ≥ 14 years with moderate/severe lamellar ichthyosis [Investigator's Global Assessment (IGA) score ≥ 3] were randomized 3 : 3 : 1 to receive oral liarozole (75 or 150 mg) or placebo once daily for 12 weeks. Assessments included: IGA; a five-point scale for erythema, scaling and pruritus severity; Short Form-36 health survey; Dermatology Life Quality Index (DLQI); and safety parameters. The primary efficacy variable was response rate at week 12 (responder: ≥ 2-point decrease in IGA from baseline). RESULTS: Sixty-four patients were enrolled. At week 12, 11/27 (41%; liarozole 75 mg), 14/28 (50%; liarozole 150 mg) and one out of nine (11%; placebo) patients were responders; the difference between groups (liarozole 150 mg vs. placebo) was not significant (P = 0·056). Mean IGA and scaling scores decreased from baseline in both liarozole groups at weeks 8 and 12 vs. placebo; erythema and pruritus scores were similar between treatment groups. Improvement in DLQI score was observed in both liarozole groups. Treatment with liarozole for 12 weeks was well tolerated. CONCLUSIONS: The primary efficacy variable did not reach statistical significance, possibly owing to the small sample size following premature termination. However, once-daily oral liarozole, 75 and 150 mg, improved scaling and DLQI and was well tolerated in patients with moderate/severe lamellar ichthyosis. |
format | Online Article Text |
id | pubmed-4232915 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2014 |
publisher | BlackWell Publishing Ltd |
record_format | MEDLINE/PubMed |
spelling | pubmed-42329152014-12-29 Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis: results of a randomized, double-blind, multinational, placebo-controlled phase II/III trial Vahlquist, A Blockhuys, S Steijlen, P van Rossem, K Didona, B Blanco, D Traupe, H Br J Dermatol Original Articles SUMMARY: BACKGROUND: Oral liarozole, a retinoic acid metabolism-blocking agent, may be an alternative to systemic retinoid therapy in patients with lamellar ichthyosis. OBJECTIVE: To demonstrate the efficacy and safety of once-daily oral liarozole in the treatment of moderate/severe lamellar ichthyosis. METHODS: This was a double-blind, multinational, parallel phase II/III trial (NCT00282724). Patients aged ≥ 14 years with moderate/severe lamellar ichthyosis [Investigator's Global Assessment (IGA) score ≥ 3] were randomized 3 : 3 : 1 to receive oral liarozole (75 or 150 mg) or placebo once daily for 12 weeks. Assessments included: IGA; a five-point scale for erythema, scaling and pruritus severity; Short Form-36 health survey; Dermatology Life Quality Index (DLQI); and safety parameters. The primary efficacy variable was response rate at week 12 (responder: ≥ 2-point decrease in IGA from baseline). RESULTS: Sixty-four patients were enrolled. At week 12, 11/27 (41%; liarozole 75 mg), 14/28 (50%; liarozole 150 mg) and one out of nine (11%; placebo) patients were responders; the difference between groups (liarozole 150 mg vs. placebo) was not significant (P = 0·056). Mean IGA and scaling scores decreased from baseline in both liarozole groups at weeks 8 and 12 vs. placebo; erythema and pruritus scores were similar between treatment groups. Improvement in DLQI score was observed in both liarozole groups. Treatment with liarozole for 12 weeks was well tolerated. CONCLUSIONS: The primary efficacy variable did not reach statistical significance, possibly owing to the small sample size following premature termination. However, once-daily oral liarozole, 75 and 150 mg, improved scaling and DLQI and was well tolerated in patients with moderate/severe lamellar ichthyosis. BlackWell Publishing Ltd 2014-01 2014-01-13 /pmc/articles/PMC4232915/ /pubmed/24102348 http://dx.doi.org/10.1111/bjd.12626 Text en © 2013 The Authors. British Journal of Dermatology published by John Wiley & Sons Ltd on behalf of British Association of Dermatologists. http://creativecommons.org/licenses/by-nc-nd/3.0/ This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made. |
spellingShingle | Original Articles Vahlquist, A Blockhuys, S Steijlen, P van Rossem, K Didona, B Blanco, D Traupe, H Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis: results of a randomized, double-blind, multinational, placebo-controlled phase II/III trial |
title | Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis:
results of a randomized, double-blind, multinational, placebo-controlled phase II/III
trial |
title_full | Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis:
results of a randomized, double-blind, multinational, placebo-controlled phase II/III
trial |
title_fullStr | Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis:
results of a randomized, double-blind, multinational, placebo-controlled phase II/III
trial |
title_full_unstemmed | Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis:
results of a randomized, double-blind, multinational, placebo-controlled phase II/III
trial |
title_short | Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis:
results of a randomized, double-blind, multinational, placebo-controlled phase II/III
trial |
title_sort | oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis:
results of a randomized, double-blind, multinational, placebo-controlled phase ii/iii
trial |
topic | Original Articles |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4232915/ https://www.ncbi.nlm.nih.gov/pubmed/24102348 http://dx.doi.org/10.1111/bjd.12626 |
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