Systemic sclerosis-associated pulmonary hypertension: why disease-specific composite endpoints are needed

Arthritis Res Ther. 2011 Jun 20;13(3):114. doi: 10.1186/ar3346.

Abstract

Pulmonary arterial hypertension (PAH) is a serious complication of systemic sclerosis (SSc). In clinical trials PAH-SSc has been grouped with other forms, including idiopathic PAH. The primary endpoint for most pivotal studies was improvement in exercise capacity. However, composite clinical endpoints that better reflect long-term outcome may be more meaningful. We discuss potential endpoints and consider why the same measures may not be appropriate for both idiopathic PAH and PAH-SSc due to inherent differences in clinical outcome and management strategies of these two forms of PAH. Failure to take this into account may compromise progress in managing PAH in SSc.

MeSH terms

  • Clinical Trials as Topic / methods*
  • Exercise Tolerance / drug effects
  • Heart Function Tests
  • Humans
  • Hypertension, Pulmonary* / drug therapy
  • Hypertension, Pulmonary* / etiology
  • Hypertension, Pulmonary* / physiopathology
  • Respiratory Function Tests
  • Scleroderma, Systemic* / complications
  • Scleroderma, Systemic* / drug therapy
  • Scleroderma, Systemic* / physiopathology
  • Severity of Illness Index*
  • Treatment Outcome