title: A randomized, investigator-masked, double-blind, placebo-controlled trial on thalidomide in severe cutaneous sarcoidosis.
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.
  
  
  BACKGROUND
  ClinicalTrials.gov; No.: NCT0030552; URL: www.clinicaltrials.gov.
authors.affiliation: Université de Rennes 1, Inserm CIC 1414, Rennes; Pharmacoepidemiology Unit, Inserm CIC 1414, Rennes.
authors.forename: Catherine
authors.initials: C
authors.lastname: Droitcourt
authors.affiliation: The Department of Dermatology, Hôpital Saint-Louis, AP-HP, Paris.
authors.forename: Michel
authors.initials: M
authors.lastname: Rybojad
authors.affiliation: The Department of Biostatistics and Medical Informatics, Hôpital Saint-Louis, AP-HP, Paris; Université de Paris Diderot-Sorbonne Paris Cité, Paris; Inserm U717, Paris.
authors.forename: Raphaël
authors.initials: R
authors.lastname: Porcher
authors.affiliation: The Department of Dermatology, Hôpital Saint-Louis, AP-HP, Paris.
authors.forename: Caroline
authors.initials: C
authors.lastname: Juillard
authors.affiliation: UFR Medicine, Université de Brest, Brest.
authors.forename: Anne
authors.initials: A
authors.lastname: Cosnes
authors.affiliation: 11 chaussée de la Muette, Paris; Clinique Dermatologique.
authors.forename: Pascal
authors.initials: P
authors.lastname: Joly
authors.affiliation: Inserm U905, Institute for Research and Innovation in Biomedicine (IRIB), Université de Rouen, Rouen; The Department of Dermatology, Hôpital Archet-2, CHU Nice, Nice.
authors.forename: Jean-Philippe
authors.initials: JP
authors.lastname: Lacour
authors.affiliation: Université de Nice, Sophia Antipolis, Nice; The Department of Dermatology, Clermont-Ferrand.
authors.forename: Michel
authors.initials: M
authors.lastname: D'Incan
authors.affiliation: The Department of Dermatology, Hôpital Cochin, AP-HP, Paris; Université René Descartes, Paris.
authors.forename: Nicolas
authors.initials: N
authors.lastname: Dupin
authors.affiliation: CHU Estaing, and Université d'Auvergne Clermont-Ferrand.
authors.forename: Bruno
authors.initials: B
authors.lastname: Sassolas
authors.affiliation: CHU Estaing, and Université d'Auvergne Clermont-Ferrand; The Department of Dermatology, CHRU Brest, Brest.
authors.forename: Laurent
authors.initials: L
authors.lastname: Misery
authors.affiliation: The Department of Dermatology, CHU Rennes; Université de Rennes 1, Inserm CIC 1414, Rennes.
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Chevrant-Breton
authors.affiliation: Pharmacovigilance Center, Groupe Hospitalier Pitié-Salpêtrière - Charles Foix, AP-HP, Paris.
authors.forename: Bénédicte
authors.initials: B
authors.lastname: Lebrun-Vignes
authors.affiliation: The Department of Biostatistics and Medical Informatics, Hôpital Saint-Louis, AP-HP, Paris; Université de Paris Diderot-Sorbonne Paris Cité, Paris; Inserm U717, Paris.
authors.forename: Kristell
authors.initials: K
authors.lastname: Desseaux
authors.affiliation: The Department of Pneumology, Hôpital Avicenne, AP-HP, Bobigny.
authors.forename: Dominique
authors.initials: D
authors.lastname: Valeyre
authors.affiliation: Université Paris-Nord Bobigny, Paris.
authors.forename: Jean
authors.initials: J
authors.lastname: Revuz
authors.affiliation: The Department of Pneumology, Hôpital Saint-Louis, AP-HP, Paris; Université de Paris Diderot-Sorbonne Paris Cité, Paris.
authors.forename: Abdellatif
authors.initials: A
authors.lastname: Tazi
authors.affiliation: 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.
authors.forename: Olivier
authors.initials: O
authors.lastname: Chosidow
authors.affiliation: 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.
authors.forename: Alain
authors.initials: A
authors.lastname: Dupuy
mesh_terms: D000328:Adult; D000368:Aged; D004311:Double-Blind Method; D005260:Female; D005602:France; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D012074:Remission Induction; D012507:Sarcoidosis / Q000188:drug therapy*; D012871:Skin Diseases / Q000188:drug therapy*; D013792:Thalidomide / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D013792:Thalidomide