title: Systemic sclerosis-associated pulmonary hypertension: why disease-specific composite endpoints are needed.
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.
authors.affiliation: Centre for Rheumatology, Royal Free Campus, University College Medical School, London, NW3 2PF, UK. c.denton@medsch.ucl.ac.uk
authors.forename: Christopher P
authors.initials: CP
authors.lastname: Denton
authors.forename: Jerome
authors.initials: J
authors.lastname: Avouac
authors.forename: Frank
authors.initials: F
authors.lastname: Behrens
authors.forename: Daniel E
authors.initials: DE
authors.lastname: Furst
authors.forename: Ivan
authors.initials: I
authors.lastname: Foeldvari
authors.forename: Marc
authors.initials: M
authors.lastname: Humbert
authors.forename: Doerte
authors.initials: D
authors.lastname: Huscher
authors.forename: Otylia
authors.initials: O
authors.lastname: Kowal-Bielecka
authors.forename: Marco
authors.initials: M
authors.lastname: Matucci-Cerinic
authors.forename: Peter
authors.initials: P
authors.lastname: Nash
authors.forename: Christian F
authors.initials: CF
authors.lastname: Opitz
authors.forename: David
authors.initials: D
authors.lastname: Pittrow
authors.forename: Lewis J
authors.initials: LJ
authors.lastname: Rubin
authors.forename: James R
authors.initials: JR
authors.lastname: Seibold
authors.forename: Oliver
authors.initials: O
authors.lastname: Distler
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D017079:Exercise Tolerance / Q000187:drug effects; D006334:Heart Function Tests; D006801:Humans; D006976:Hypertension, Pulmonary / Q000188:drug therapy / Q000209:etiology / Q000503:physiopathology; D012129:Respiratory Function Tests; D012595:Scleroderma, Systemic / Q000150:complications / Q000188:drug therapy / Q000503:physiopathology; D012720:Severity of Illness Index; D016896:Treatment Outcome