Incremental burden of disease in patients diagnosed with pulmonary arterial hypertension receiving monotherapy and combination vasodilator therapy.
BACKGROUND
Pulmonary 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.


METHODS
This 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).


RESULTS
Data 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.


CONCLUSIONS
Combination 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.
Adelphi Real World, Adelphi Mill, Bollington, Macclesfield, SK10 5JB, UK, mark.small@adelphigroup.com.
Mark
M
Small
James
J
Piercy
James
J
Pike
Annamaria
A
Cerulli
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000959:Antihypertensive Agents / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D004417:Dyspnea / Q000209:etiology / Q000503:physiopathology; D011464:Epoprostenol / Q000627:therapeutic use*; D005080:Exercise Test; D065627:Familial Primary Pulmonary Hypertension; D005260:Female; D006296:Health Services / Q000706:statistics & numerical data; D006760:Hospitalization / Q000706:statistics & numerical data; D006801:Humans; D006976:Hypertension, Pulmonary / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D058986:Phosphodiesterase 5 Inhibitors / Q000627:therapeutic use*; D011788:Quality of Life; D012189:Retrospective Studies; D012720:Severity of Illness Index; D016896:Treatment Outcome; D014655:Vascular Resistance; D014665:Vasodilator Agents / Q000627:therapeutic use*; D055815:Young Adult
D000959:Antihypertensive Agents; D058986:Phosphodiesterase 5 Inhibitors; D014665:Vasodilator Agents; D011464:Epoprostenol