Disease targeting therapies in patients with Eisenmenger syndrome: Response to treatment and long-term efficiency

ObjectivesTo examine long-term efficacy of disease targeting therapies (DTT) in patients with Eisenmenger syndrome.MethodsAll adult patients with Eisenmenger syndrome treated with DTT at our institution were included. Functional class (FC), oxygen saturation and 6-minute walk test distance (6MWTd) were analysed retrospectively.ResultsBetween 2002 and 2010, 79 Eisenmenger patients (21 males, 16 with Down syndrome) aged 34 ± 10 years (range 17-68 years) were included. Median follow-up was 3.3 years (range 0.2 to 8.9 years). 6MWTd increased early after initiation of DTT, with a plateau after approximately 3 years and no obvious trend towards a deterioration on average during longer-term follow-up. Two patients died during follow-up and escalation of treatment was required in 18 patients after a median period of 2.5 years. Escalation of therapy was also associated with an increase in 6MWTd. In addition, FC improved on DTT and oxygen saturation, increased, both at rest and peak exercise. This effect was more pronounced in the patients with the lowest baseline oxygen saturation at rest.ConclusionsLong-term DTT is safe and improves objective exercise capacity and subjective symptoms. Response to DTT was typically observed early after initiation of DTT and was, on average, maintained longer-term. However, 1 in 5 patients required escalation of DTT, with time, due to symptomatic deterioration and this was associated with an afresh improvement in 6MWTd.