Ambrisentan for sarcoidosis associated pulmonary hypertension

Sarcoidosis Vasc Diffuse Lung Dis. 2011 Oct;28(2):139-45.


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.

Conclusion: 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.

Publication types

  • Clinical Trial
  • Multicenter Study

MeSH terms

  • Adult
  • Antihypertensive Agents / adverse effects
  • Antihypertensive Agents / therapeutic use*
  • Exercise Test
  • Exercise Tolerance / drug effects
  • Female
  • Humans
  • Hypertension, Pulmonary / drug therapy*
  • Hypertension, Pulmonary / etiology
  • Hypertension, Pulmonary / physiopathology
  • Lung / drug effects
  • Lung / physiopathology
  • Male
  • Middle Aged
  • North Carolina
  • Phenylpropionates / adverse effects
  • Phenylpropionates / therapeutic use*
  • Prospective Studies
  • Pyridazines / adverse effects
  • Pyridazines / therapeutic use*
  • Quality of Life
  • Recovery of Function
  • Respiratory Function Tests
  • Sarcoidosis / complications*
  • South Carolina
  • Surveys and Questionnaires
  • Time Factors
  • Treatment Outcome


  • Antihypertensive Agents
  • Phenylpropionates
  • Pyridazines
  • ambrisentan