BACKGROUND: For invalidating symptoms in primary Sj  gren's syndrome (pSS), there is still a need for easy-to-administer, cost-effective and well-tolerated systemic treatment. @entity6963 (LEF) is structurally unrelated to other immunomodulatory drugs and might be efficacious in pSS, given its characteristic immunoregulatory modes of action. OBJECTIVE: To investigate the safety and efficacy of LEF in pSS in a phase II open-label pilot study. METHODS: 15 @entity1 with pSS with early and active disease received LEF 20 mg once daily for 24 weeks. Tolerability, safety and efficacy of LEF were evaluated every 8 weeks. Additional safety visits were performed every fortnight. RESULTS: Mild gastrointestinal discomfort (including @entity294 ) and hair loss were mainly reported. Five @entity1 developed @entity1252 -like @entity272 on the face, arms or trunk, responding well to topical corticosteroids, nevertheless causing the withdrawal of one @entity1 . Two @entity1 with pre-existing @entity101 had to increase dosages of anti- @entity101 drugs. Increased levels of @entity493 aminotransferase normalised after dose reduction in two @entity1 . A decrease in general fatigue and an increase in physical functioning were observed after 24 weeks. Serum IgG levels decreased from 8 weeks onwards. Schirmer test values increased, not reaching statistical significance, whereas sialometry values did not change. In four of five repeated biopsies, the lymphocytic focus score decreased at the rate of 1 focus/4 mm(2). A remarkable amelioration of @entity1333 was observed in three @entity1 . CONCLUSIONS: Although the safety profile seems fairly acceptable, the observed indications for efficacy were modest and may be doubtful in justifying a randomised controlled trial of LEF in pSS.

Safety and efficacy of XXXX in primary Sj  gren's syndrome: a phase II pilot study.

@entity1 :: ('9606', 'Species') :: ['patients', 'patient'] @entity6963 :: ('MESH:C045463', 'Chemical') :: ['Leflunomide'] @entity101 :: ('MESH:D006973', 'Disease') :: ['hypertension', 'hypertensive'] @entity1333 :: ('MESH:D014657', 'Disease') :: ['leucocytoclastic vasculitis'] @entity294 :: ('MESH:D003967', 'Disease') :: ['diarrhoea'] @entity1252 :: ('MESH:D008180', 'Disease') :: ['lupus'] @entity272 :: ('MESH:D012871', 'Disease') :: ['skin lesions'] @entity493 :: ('CHEBI:16449', 'Chemical') :: ['alanine']
@entity6963 :: (MESH:C045463,Chemical) :: ['leflunomide']
