Clinical characteristics, treatment patterns and healthcare resource utilisation for portopulmonary hypertension patients in the USA: a real-world study

Abstract

Objectives: To investigate demographic, clinical characteristics, treatment patterns and healthcare resource utilisation (HCRU) among patients with portopulmonary hypertension (PoPH).

Design: Retrospective analysis of a real-world cross-sectional survey, the pulmonary arterial hypertension (PAH) Disease Specific Programme.

Setting: Secondary care settings in the USA.

Participants: Pulmonologists (n=22), cardiologists (n=17) and internists (n=7) who saw ≥2 patients with PAH/month provided data on 90 PoPH patients. Mean age of patients was 56.8 years, 51.1% were male, 76.7% were white/Caucasian.

Primary and secondary outcome measures: Demographic characteristics, initial presentation/diagnosis, disease trajectory, symptoms, comorbidities, treatment patterns and HCRU.

Results: The most common diagnostic tests were echocardiogram (62.2%), right heart catheterisation (56.7%) and electrocardiogram (48.9%). Dyspnea on exertion (64.4%), dyspnea following exertion (51.1%) and fatigue (48.9%) were the most common symptoms prior to PAH diagnosis as well as the most bothersome. Overall, 13.3% patients had mild disease before treatment vs 42.2% at time of survey. Most (60.0%) had stable disease (21.1% were improving and 18.8% were deteriorating at the time of the survey). Most common recent symptoms were dyspnea on exertion (51.1%), fatigue (37.8%) and dyspnea following exertion (32.2%). Patients had a mean of 1.8 comorbidities. In total, 80.0% of PoPH patients were receiving PAH-specific treatment; 41.7%, 22.2% and 12.5% received monotherapy, dual therapy and triple therapy, respectively. Patients had seen their physician 4.1 times in the past 12 months (3.5 visits specifically for PAH), while 31.1% had been hospitalised for PAH. Most (70.0%) patients were managed in a multidisciplinary team, and 8.9% of patients relied on a caregiver.

Conclusions: This study highlights the high symptomatic burden coupled with mostly moderate-to-severe disease status and a high hospitalisation rate despite the majority of PoPH patients being on therapy. Therefore, new treatment options in PoPH are in great need.

Keywords: Patient Care Management; Patients; Pulmonary Disease.

Figure 1. Bar chart of the symptoms present prior to the diagnosis of PAH (A) and of the conditions suspected or investigated before a diagnosis of PAH was confirmed (B). Bars represent the percentage of patients. COPD, chronic obstructive pulmonary disease; GERD, gastro-oesophageal reflux disease; PAH, pulmonary arterial hypertension.

Figure 2. Bar chart of physician-perceived disease severity prior to treatment initiation and at the time of survey (A); Bar chart of physician-perceived NYHA FC prior to treatment initiation and at the time of the survey (B). FC, functional classification; NYHA, New York Heart Association; (FC I, no limitation of physical activity; FC II, mild limitation of physical activity; FC III, marked limitation of physical activity; FC IV unable to perform any physical activity).

Figure 3. Bar chart of physician-reported PAH symptoms experienced by patients in the 4 weeks prior to survey date and the most troublesome patient symptoms. PAH, pulmonary arterial hypertension.

Figure 4. Bar chart of top 15 most frequent physician-reported diagnosed patient comorbidities experienced. *Includes all physician-reported comorbidities other than concomitant liver disease.