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Original Research: Diffuse Lung Disease |

A Randomized, Investigator-Masked, Double-Blind, Placebo-Controlled Trial on Thalidomide in Severe Cutaneous SarcoidosisThalidomide in Severe Cutaneous Sarcoidosis

Catherine Droitcourt, MD; Michel Rybojad, MD; Raphaël Porcher, PhD; Caroline Juillard, MD; Anne Cosnes, MD; Pascal Joly, MD, PhD; Jean-Philippe Lacour, MD, PhD; Michel D’Incan, MD, PhD; Nicolas Dupin, MD, PhD; Bruno Sassolas, MD; Laurent Misery, MD, PhD; Jacqueline Chevrant-Breton, MD; Bénédicte Lebrun-Vignes, MD; Kristell Desseaux, MSc; Dominique Valeyre, MD, PhD; Jean Revuz, MD; Abdellatif Tazi, MD, PhD; Olivier Chosidow, MD, PhD; Alain Dupuy, MD, PhD
Author and Funding Information

From the Department of Dermatology (Drs Droitcourt, Dupuy, and Chevrant-Breton), CHU Rennes; Université de Rennes 1 (Drs Droitcourt, Dupuy, and Chevrant-Breton), and Pharmacoepidemiology Unit (Drs Droitcourt and Dupuy), Inserm CIC 1414, Rennes; the Department of Dermatology (Drs Rybojad and Juillard), the Department of Biostatistics and Medical Informatics (Dr Porcher and Ms Desseaux), and the Department of Pneumology (Dr Tazi), Hôpital Saint-Louis, AP-HP, Paris; Université de Paris Diderot-Sorbonne Paris Cité (Drs Porcher and Tazi and Ms Desseaux), Paris; Inserm U717 (Dr Porcher and Ms Desseaux), Paris; the Department of Dermatology (Dr Dupin), Hôpital Cochin, AP-HP, Paris; Université René Descartes (Dr Dupin), Paris; Pharmacovigilance Center (Dr Lebrun-Vignes), Groupe Hospitalier Pitié-Salpêtrière - Charles Foix, AP-HP, Paris; the Department of Pneumology (Dr Valeyre), Hôpital Avicenne, AP-HP, Bobigny; Université Paris-Nord (Dr Valeyre) Bobigny; 11 chaussée de la Muette (Dr Revuz), Paris; Clinique Dermatologique (Dr Joly), CHU Rouen, Rouen; Inserm U905, Institute for Research and Innovation in Biomedicine (IRIB) (Dr Joly), Université de Rouen, Rouen; the Department of Dermatology (Dr Lacour), Hôpital Archet-2, CHU Nice, Nice; Université de Nice (Dr Lacour), Sophia Antipolis, Nice; the Department of Dermatology (Dr D’Incan), CHU Estaing, and Université d’Auvergne (Dr D’Incan) Clermont-Ferrand; the Department of Dermatology (Drs Sassolas and Misery), CHRU Brest, Brest; UFR Medicine (Dr Misery), Université de Brest, Brest; the Department of Dermatology (Drs Cosnes and Chosidow), Hôpital Henri-Mondor, AP-HP, Créteil; UPEC Université de Paris Est-Créteil Val-de-Marne (Dr Chosidow), Créteil; and French satellite of the Cochrane Skin Group and Centre d’Investigation Clinique 006-Inserm (Dr Chosidow), Créteil, France.

CORRESPONDENCE TO: Alain Dupuy, MD, PhD, Department of Dermatology, Pontchaillou Hospital, 2 rue Henri le Guilloux, 35000 Rennes, France; e-mail: alain.dupuy@chu-rennes.fr


Dr Sassolas is currently at Cavala Blanche Hospital (Brest, France).

FUNDING/SUPPORT: Assistance-Publique Hôpitaux-de-Paris provided funding for this trial [Grant CRC03156]. Laphal Industries provided thalidomide and placebo.

Reproduction of this article is prohibited without written permission from the American College of Chest Physicians. See online for more details.


Chest. 2014;146(4):1046-1054. doi:10.1378/chest.14-0015
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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

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