• Mark Small, James Piercy, James Pike, and Annamaria Cerulli.
• Adelphi Real World, Adelphi Mill, Bollington, Macclesfield, SK10 5JB, UK, mark.small@adelphigroup.com.
• Adv Ther. 2014 Feb 1;31(2):168-79.

IntroductionPulmonary arterial hypertension (PAH) is a rare, severely debilitating disease with high mortality. There are limited data available on treatment patterns and burden of disease from conditions of actual care.MethodsThis analysis assesses the burden of disease for patients with PAH treated with monotherapy and combination therapies excluding and including intravenous (IV) prostacyclin analogues (PGI2). Data were drawn from the Adelphi PAH Disease Specific Programme, a cross sectional survey of consulting patients undertaken in the US, Germany, Italy and the UK in 2010. Outcomes included demographics, clinical characteristics, health-care resource utilization, and quality of life measured by the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR).ResultsData were analyzed from 446 patients receiving ≥1 of 3 PAH-specific treatment classes. Physicians comprised mainly pulmonologists and cardiologists. The symptoms, functioning and quality of life scales of the CAMPHOR instrument were completed by 218, 229, and 214 patients, respectively. Although 46.2% of patients were classified as World Health Organization (WHO) functional class III or IV, only 24.4% of the population received combination therapy. Combination therapy including IV PGI2 was used in 4.7% of all patients. Patients on monotherapy had the lowest pulmonary vascular resistance values, the highest recorded 6-min walk distance and the lowest recorded levels of dyspnea. Patients on combination therapy including IV PGI2 scored worse on all three variables and had more hospitalizations than patients on less aggressive combination therapy. With increasing therapeutic regimens, the CAMPHOR scores were higher, indicating worse states of health.ConclusionsCombination treatment and particularly the use of prostacyclins remain underused in an unselected population of PAH patients surveyed under conditions of actual care. The disease burden is substantial and increases with greater severity of disease and more aggressive treatments. This necessitates improvement in optimizing current therapy, as well as novel and innovative combination options.

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