دورية أكاديمية
Randomised controlled trial for emphysema with a selective agonist of the γ-type retinoic acid receptor.
| العنوان: | Randomised controlled trial for emphysema with a selective agonist of the γ-type retinoic acid receptor. |
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| المؤلفون: | Stolk J; Dept of Pulmonology, Leiden University Medical Center, Leiden, The Netherlands. j.stolk.long@lumc.nl, Stockley RA, Stoel BC, Cooper BG, Piitulainen E, Seersholm N, Chapman KR, Burdon JG, Decramer M, Abboud RT, Mannes GP, Wouters EF, Garrett JE, Barros-Tizon JC, Russi EW, Lomas DA, MacNee WA, Rames A |
| المصدر: | The European respiratory journal [Eur Respir J] 2012 Aug; Vol. 40 (2), pp. 306-12. Date of Electronic Publication: 2012 Jan 26. |
| نوع المنشور: | Clinical Trial, Phase II; Clinical Trial, Phase III; Journal Article; Randomized Controlled Trial |
| اللغة: | English |
| بيانات الدورية: | Publisher: European Respiratory Society Country of Publication: England NLM ID: 8803460 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1399-3003 (Electronic) Linking ISSN: 09031936 NLM ISO Abbreviation: Eur Respir J Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: Sheffield, United Kingdom : European Respiratory Society Original Publication: Copenhagen : Published jointly by the Society and Munksgaard, 1988- |
| مواضيع طبية MeSH: | Emphysema/*drug therapy , Receptors, Retinoic Acid/*agonists, Adult ; Aged ; Animals ; Double-Blind Method ; Emphysema/metabolism ; Female ; Forced Expiratory Volume ; Gases ; Genotype ; Humans ; Inflammation ; Least-Squares Analysis ; Male ; Middle Aged ; Placebos ; Pyrazoles/therapeutic use ; Smoking ; Stilbenes/therapeutic use ; Tomography, X-Ray Computed ; Retinoic Acid Receptor gamma |
| مستخلص: | Palovarotene is an oral γ-selective retinoid agonist. In animal emphysema models, palovarotene reduced inflammation, promoted structural repair and functional improvement. REPAIR (Retinoid treatment of Emphysema in Patients on the α(1)-antitrypsin International Registry), was an investigator-initiated, double-blind, placebo-controlled randomised study to assess the safety and efficacy of 5 mg·day(-1) palovarotene given for 1 year to 262 patients with severe α(1)-antitrypsin deficiency and emphysema confirmed by computed tomography. Change in volume-adjusted 15th percentile point lung density from baseline in 1 year was the primary end-point; functional end-points were also regularly assessed. We randomly assigned 133 and 129 patients to placebo or palovarotene, respectively. Both groups were well matched for all baseline characteristics, including respiratory medications. 88% and 85% of patients completed 1 year of treatment with placebo and palovarotene, respectively. Palovarotene was generally well tolerated. In the study completers population, the placebo-corrected difference of lung density was -0.45 HU at week 28 (p=0.64) and -0.25 HU at week 52 (p=0.94). A nonsignificant treatment difference in most functional parameters of the lung in favour of the drug was observed over time suggesting potential pharmacological effects of palovarotene. Palovarotene 5 mg·day(-1) over 1 yr failed to show a significant benefit on lung density in moderate-to-severe emphysema secondary to severe α(1)-antitrypsin deficiency. |
| معلومات مُعتمدة: | G0901786 United Kingdom MRC_ Medical Research Council |
| المشرفين على المادة: | 0 (Gases) 0 (Placebos) 0 (Pyrazoles) 0 (Receptors, Retinoic Acid) 0 (Stilbenes) 28K6I5M16G (Palovarotene) |
| تواريخ الأحداث: | Date Created: 20120128 Date Completed: 20130110 Latest Revision: 20231213 |
| رمز التحديث: | 20231215 |
| DOI: | 10.1183/09031936.00161911 |
| PMID: | 22282548 |
| قاعدة البيانات: | MEDLINE |
| تدمد: | 1399-3003 |
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| DOI: | 10.1183/09031936.00161911 |