CP-141 Bosentan treatment for autoimmune diseases

Abstract

BackgroundBosentan, an orphan drug, is a dual endothelin receptor antagonist indicated in pulmonary hypertension and in the prevention of new digital ulcers (DU) in patients with systemic sclerosis and ongoing digital ulcer disease.PurposeTo evaluate the effectiveness and safety of bosentan in the treatment of clinical manifestations associated with underlying autoimmune disease (Raynaud’s phenomenon (RP), DU and other location skin ulcers (SU)), all of them considered rare diseases.Material and methodsRetrospective observational study including patients treated with bosentan from January 2012 to September 2014, on compassionate use.Variables collected were: sex, age, underlying disease, indication, previous treatments, dose and follow-up time.Effectiveness was evaluated as: absence of new ulcers, improvement of the basal ulcers and decrease in the number of RP episodes.The safety profile was determined according to the adverse reactions.Results14 patients were included; follow up (median, range): 22 (2–55) months; sex: 10 (71%) female; age: 52 (25–81) years.All of them had previously been treated with first-line treatment until resistance or intolerance had developed.In 11 cases bosentan was indicated to treat RP and prevent/treat DU (4 systemic sclerosis, 3 pre-systemic sclerosis, 2 systemic lupus erythematosus (SLE), 1 dermatomyositis and 1 Buerger´s disease. Getting the next results: 63.6% effective (7), 18.2% ineffective (2) and 18.2% could not be evaluated.In the other 3 cases, bosentan was used to treat other location SUs (1 polyarteritis nodosa,1 SLE and 1 necrobiosis lipoidica) with 100% effectiveness.The treatment was discontinued in two cases due to digestive intolerance. In another two cases, the dose was adjusted as a consequence of an initial increase in the hepatic enzymes.ConclusionBosentan may be considered an appropriate alternative in these diseases which have been refractory to conventional treatment. The number of patients is limited due to the low prevalence of these diseases and to the off-label use of the drug. Therefore, it could be said that value of research lies in the possibility of opening new therapeutic perspectives with this drug.References and/or AcknowledgementsNo conflict of interest.