title: Ambrisentan for sarcoidosis associated pulmonary hypertension.
abstract: BACKGROUND
  Sarcoidosis associated pulmonary hypertension (SAPH) is associated with significant morbidity and mortality. There is a paucity of information concerning therapy for this condition.
  
  
  METHODS
  We performed a prospective, open-label, proof of concept trial of ambrisentan for SAPH. 21 subjects with SAPH received 5 mg/day of ambrisentan for 4 weeks and then 10/mg day for 20 subsequent weeks.
  
  
  RESULTS
  No significant change was noted in the 6-minute walk distance over the course of the study (mean change between week 0 and 24: 9.8 +/- 54.6 meters, p: NS). There were also no significant differences between weeks 0 and 24 in terms of dyspnea as measured by the modified Borg scale, serum brain naturetic peptide, diffusing capacity, and quality of life as measured by the Short Form-36. There was a high dropout rate: overall: 11/21, 52%; social reasons: 3/21, 14%; medical reasons: 8/21, 38% because of dyspnea: 6/21, 29% and/or edema: 4/21, 19%. Of those who completed the 24 week study (10/21, 48%), there was an improvement in their WHO functional class and a marked improvement in their health related quality of life as measured by the St. George Respiratory questionnaire (-15.3 +/- 25.0). However both these improvments did not reach statistical significance possibly because of the small sample size.
  
  
  CONCLUSIONS
  Although ambrisentan was not well tolerated by many of these subjects with SAPH, in those who remained in this 24-week trial, improvements in WHO functional class and in health related quality of life suggested a possible benefit of this drug in selected patients.
authors: Division of Pulmonary and Critical Care Medicine, Medical University of South Carolina, USA. judsonm@mail.amc.edu
authors: M A
authors: MA
authors: Judson
authors: K B
authors: KB
authors: Highland
authors: S
authors: S
authors: Kwon
authors: J F
authors: JF
authors: Donohue
authors: R
authors: R
authors: Aris
authors: N
authors: N
authors: Craft
authors: S
authors: S
authors: Burt
authors: H J
authors: HJ
authors: Ford
mesh_terms: D000328:Adult; D000959:Antihypertensive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D005080:Exercise Test; D017079:Exercise Tolerance / Q000187:drug effects; D005260:Female; D006801:Humans; D006976:Hypertension, Pulmonary / Q000188:drug therapy* / Q000209:etiology / Q000503:physiopathology; D008168:Lung / Q000187:drug effects / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D009657:North Carolina; D010666:Phenylpropionates / Q000009:adverse effects / Q000627:therapeutic use*; D011446:Prospective Studies; D011724:Pyridazines / Q000009:adverse effects / Q000627:therapeutic use*; D011788:Quality of Life; D020127:Recovery of Function; D012129:Respiratory Function Tests; D012507:Sarcoidosis / Q000150:complications*; D013022:South Carolina; D011795:Surveys and Questionnaires; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D000959:Antihypertensive Agents; D010666:Phenylpropionates; D011724:Pyridazines; C467894:ambrisentan