title: Disease targeting therapies in patients with Eisenmenger syndrome: response to treatment and long-term efficiency.
abstract: OBJECTIVE
  To examine long-term efficacy of disease targeting therapies (DTT) in patients with Eisenmenger syndrome.
  
  
  METHODS
  All adult patients with Eisenmenger syndrome treated with DTT at our institution were included. Functional class (FC), oxygen saturation and 6-minute walk test distance (6 MWTd) were analysed retrospectively.
  
  
  RESULTS
  Between 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). 6 MWTd 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 6 MWTd. 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.
  
  
  CONCLUSIONS
  Long-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 6 MWTd.
authors.affiliation: Adult Congenital Heart Disease Centre and Centre for Pulmonary Hypertension, Royal Brompton Hospital, London, UK.
authors.forename: Gerhard-Paul
authors.initials: GP
authors.lastname: Diller
authors.forename: Rafael
authors.initials: R
authors.lastname: Alonso-Gonzalez
authors.forename: Konstantinos
authors.initials: K
authors.lastname: Dimopoulos
authors.forename: Maria
authors.initials: M
authors.lastname: Alvarez-Barredo
authors.forename: Chiehyang
authors.initials: C
authors.lastname: Koo
authors.forename: Aleksander
authors.initials: A
authors.lastname: Kempny
authors.forename: Carl
authors.initials: C
authors.lastname: Harries
authors.forename: Lisa
authors.initials: L
authors.lastname: Parfitt
authors.forename: Anselm S
authors.initials: AS
authors.lastname: Uebing
authors.forename: Lorna
authors.initials: L
authors.lastname: Swan
authors.forename: Philip S
authors.initials: PS
authors.lastname: Marino
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Wort
authors.forename: Michael A
authors.initials: MA
authors.lastname: Gatzoulis
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000806:Angiotensin-Converting Enzyme Inhibitors / Q000008:administration & dosage; D000959:Antihypertensive Agents / Q000008:administration & dosage; D004311:Double-Blind Method; D016503:Drug Delivery Systems / Q000379:methods*; D004541:Eisenmenger Complex / Q000188:drug therapy* / Q000503:physiopathology*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D013997:Time Factors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000806:Angiotensin-Converting Enzyme Inhibitors; D000959:Antihypertensive Agents