Catherine Droitcourt1, Michel Rybojad2, Raphaël Porcher3, Caroline Juillard2, Anne Cosnes4, Pascal Joly5, Jean-Philippe Lacour6, Michel D'Incan7, Nicolas Dupin8, Bruno Sassolas9, Laurent Misery10, Jacqueline Chevrant-Breton11, Bénédicte Lebrun-Vignes12, Kristell Desseaux3, Dominique Valeyre13, Jean Revuz14, Abdellatif Tazi15, Olivier Chosidow16, Alain Dupuy17. 1. Université de Rennes 1, Inserm CIC 1414, Rennes; Pharmacoepidemiology Unit, Inserm CIC 1414, Rennes. 2. The Department of Dermatology, Hôpital Saint-Louis, AP-HP, Paris. 3. The Department of Biostatistics and Medical Informatics, Hôpital Saint-Louis, AP-HP, Paris; Université de Paris Diderot-Sorbonne Paris Cité, Paris; Inserm U717, Paris. 4. UFR Medicine, Université de Brest, Brest. 5. 11 chaussée de la Muette, Paris; Clinique Dermatologique. 6. Inserm U905, Institute for Research and Innovation in Biomedicine (IRIB), Université de Rouen, Rouen; The Department of Dermatology, Hôpital Archet-2, CHU Nice, Nice. 7. Université de Nice, Sophia Antipolis, Nice; The Department of Dermatology, Clermont-Ferrand. 8. The Department of Dermatology, Hôpital Cochin, AP-HP, Paris; Université René Descartes, Paris. 9. CHU Estaing, and Université d'Auvergne Clermont-Ferrand. 10. CHU Estaing, and Université d'Auvergne Clermont-Ferrand; The Department of Dermatology, CHRU Brest, Brest. 11. The Department of Dermatology, CHU Rennes; Université de Rennes 1, Inserm CIC 1414, Rennes. 12. Pharmacovigilance Center, Groupe Hospitalier Pitié-Salpêtrière - Charles Foix, AP-HP, Paris. 13. The Department of Pneumology, Hôpital Avicenne, AP-HP, Bobigny. 14. Université Paris-Nord Bobigny, Paris. 15. The Department of Pneumology, Hôpital Saint-Louis, AP-HP, Paris; Université de Paris Diderot-Sorbonne Paris Cité, Paris. 16. UFR Medicine, Université de Brest, Brest; The Department of Dermatology, Hôpital Henri-Mondor, AP-HP, Créteil; UPEC Université de Paris Est-Créteil Val-de-Marne, Créteil; French satellite of the Cochrane Skin Group and Centre d'Investigation Clinique 006-Inserm, Créteil, France. 17. The Department of Dermatology, CHU Rennes; Université de Rennes 1, Inserm CIC 1414, Rennes; Pharmacoepidemiology Unit, Inserm CIC 1414, Rennes. Electronic address: alain.dupuy@chu-rennes.fr.
Abstract
BACKGROUND:Thalidomide use in cutaneous sarcoidosis is based on data from small case series or case reports. The objective of this study was to evaluate the efficacy and safety of thalidomide in severe cutaneous sarcoidosis. METHODS: This study consisted of a randomized, double-bind, parallel, placebo-controlled, investigator-masked, multicenter trial lasting 3 months and an open-label study from month 3 to month 6. Adults with a clinical and histologic diagnosis of cutaneous sarcoidosis were included in nine hospital centers in France. Patients were randomized 1:1 to oral thalidomide (100 mg once daily) or to a matching oral placebo for 3 months. In the course of an open-label follow-up from month 3 to month 6, all patients received thalidomide, 100 mg to 200 mg daily. The proportions of patients with a partial or complete cutaneous response at month 3, based on at least a 50% improvement in three target lesions scored for area and infiltration, were compared across randomization groups. RESULTS: The intent-to-treat population included 39 patients. None of them had a complete cutaneous response. Four out of 20 patients in the thalidomide group (20%) vs four out of 19 patients in the placebo group (21%) had a partial cutaneous response at month 3 (difference in proportion of -1% [95% CI, -26% to +24%] for thalidomide vs placebo, P = 1.0). Eight patients with side effects were recorded in the thalidomide group vs three in the placebo group. We observed a large number of adverse event-related discontinuations in patients taking thalidomide in the first 3 months (four patients with thalidomide, zero with placebo) and in the 3 following months (five patients). CONCLUSIONS: At a dose of 100 mg daily for 3 months, our results do not encourage thalidomide use in cutaneous sarcoidosis. TRIAL REGISTRY: ClinicalTrials.gov; No.: NCT0030552; URL: www.clinicaltrials.gov.
RCT Entities:
BACKGROUND:Thalidomide use in cutaneous sarcoidosis is based on data from small case series or case reports. The objective of this study was to evaluate the efficacy and safety of thalidomide in severe cutaneous sarcoidosis. METHODS: This study consisted of a randomized, double-bind, parallel, placebo-controlled, investigator-masked, multicenter trial lasting 3 months and an open-label study from month 3 to month 6. Adults with a clinical and histologic diagnosis of cutaneous sarcoidosis were included in nine hospital centers in France. Patients were randomized 1:1 to oral thalidomide (100 mg once daily) or to a matching oral placebo for 3 months. In the course of an open-label follow-up from month 3 to month 6, all patients received thalidomide, 100 mg to 200 mg daily. The proportions of patients with a partial or complete cutaneous response at month 3, based on at least a 50% improvement in three target lesions scored for area and infiltration, were compared across randomization groups. RESULTS: The intent-to-treat population included 39 patients. None of them had a complete cutaneous response. Four out of 20 patients in the thalidomide group (20%) vs four out of 19 patients in the placebo group (21%) had a partial cutaneous response at month 3 (difference in proportion of -1% [95% CI, -26% to +24%] for thalidomide vs placebo, P = 1.0). Eight patients with side effects were recorded in the thalidomide group vs three in the placebo group. We observed a large number of adverse event-related discontinuations in patients taking thalidomide in the first 3 months (four patients with thalidomide, zero with placebo) and in the 3 following months (five patients). CONCLUSIONS: At a dose of 100 mg daily for 3 months, our results do not encourage thalidomide use in cutaneous sarcoidosis. TRIAL REGISTRY: ClinicalTrials.gov; No.: NCT0030552; URL: www.clinicaltrials.gov.
Authors: Sara A Berg; Howa Yeung; Joseph C English; Emily L Keimig; Ellen J Kim; Robert G Micheletti; Karolyn A Wanat; Marc A Judson; Robert P Baughman; Misha Rosenbach Journal: Sarcoidosis Vasc Diffuse Lung Dis Date: 2017-04-28 Impact factor: 0.670
Authors: Khalid Al-Kofahi; Peter Korsten; Christian Ascoli; Shanti Virupannavar; Mehdi Mirsaeidi; Ian Chang; Naim Qaqish; Lesley A Saketkoo; Robert P Baughman; Nadera J Sweiss Journal: Ther Clin Risk Manag Date: 2016-11-07 Impact factor: 2.423