Assessment of measurement properties of peak VO2 in children with pulmonary arterial hypertension

Using data from the subpopulation of 106 patients who were developmentally and physically able to perform exercise testing, all of whom were World Health Organization Functional Class (WHO FC) I, II, or III, reliability was assessed using the intraclass correlation coefficient and Bland-Altman plot on screening and baseline data. Relationships between percentage change in peak VO2 from baseline to end of treatment and other endpoints were evaluated using correlation coefficients and regression analyses.The intraclass correlation was 0.79 between screening and baseline peak VO2, an agreement that was supported by the Bland-Altman plot. Percentage change in peak VO2 correlated well (r ≥0.40) and showed responsiveness to a physician global assessment of change and with change in WHO FC (for baseline classes I and III). Percentage change in peak VO2 did not correlate with change in the Family Cohesion of the Child Health Questionnaire (r？=？0.04) or with a subject global assessment of change (r？=？0.12). The latter may have been influenced by child and parental-proxy response and instrument administration.In pediatric PAH patients who are developmentally and physically able to perform exercise testing, peak VO2 measurements exhibited good reliability and improvements that were associated with improvements in certain other clinical endpoints, such as the WHO FC and a physician global assessment.ClinicalTrials.gov identifier NCT00159913.Pulmonary arterial hypertension (PAH) is a relatively rare condition associated with high mortality [1]. It is characterized by increased pulmonary vascular resistance and pulmonary arterial pressure leading to right ventricular failure and ultimately death [2]. It may be inherited (heritable PAH [HPAH], classified as familial or sporadic), develop spontaneously (idiopathic PAH [IPAH]), or occur in association with congenital heart defects, connective tissue disease, or other causes (associated PAH [APAH]) [3]. Oral sildenafil citrate (REVAT