Clinical characteristics, treatment patterns and healthcare resource utilisation for portopulmonary hypertension patients in the USA: a real-world study
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) Diseas...
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BMJ Publishing Group
2025-06-01
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| Series: | BMJ Open |
| Online Access: | https://bmjopen.bmj.com/content/15/6/e089223.full |
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| author | Hilary Dubrock Julia Harley Mark Small Megan Scott Peter Agron Yuen Tsang Sumeet Panjabi |
| author_facet | Hilary Dubrock Julia Harley Mark Small Megan Scott Peter Agron Yuen Tsang Sumeet Panjabi |
| author_sort | Hilary Dubrock |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-309bc73a4cd64505974a57d2b07efb94 |
| institution | OA Journals |
| issn | 2044-6055 |
| language | English |
| publishDate | 2025-06-01 |
| publisher | BMJ Publishing Group |
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| series | BMJ Open |
| spelling | doaj-art-309bc73a4cd64505974a57d2b07efb942025-08-20T02:24:13ZengBMJ Publishing GroupBMJ Open2044-60552025-06-0115610.1136/bmjopen-2024-089223Clinical characteristics, treatment patterns and healthcare resource utilisation for portopulmonary hypertension patients in the USA: a real-world studyHilary Dubrock0Julia Harley1Mark Small2Megan Scott3Peter Agron4Yuen Tsang5Sumeet Panjabi6Mayo Clinic, Rochester, Minnesota, USAAdelphi Real World, Bollington, UKAdelphi Real World, Bollington, UKAdelphi Real World, Bollington, UKJanssen Pharmaceutical Companies of Johnson and Johnson, Titusville, New Jersey, USAJanssen Pharmaceutical Companies of Johnson and Johnson, Titusville, New Jersey, USAJanssen Pharmaceutical Companies of Johnson and Johnson, Titusville, New Jersey, USAObjectives 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.https://bmjopen.bmj.com/content/15/6/e089223.full |
| spellingShingle | Hilary Dubrock Julia Harley Mark Small Megan Scott Peter Agron Yuen Tsang Sumeet Panjabi Clinical characteristics, treatment patterns and healthcare resource utilisation for portopulmonary hypertension patients in the USA: a real-world study BMJ Open |
| title | Clinical characteristics, treatment patterns and healthcare resource utilisation for portopulmonary hypertension patients in the USA: a real-world study |
| title_full | Clinical characteristics, treatment patterns and healthcare resource utilisation for portopulmonary hypertension patients in the USA: a real-world study |
| title_fullStr | Clinical characteristics, treatment patterns and healthcare resource utilisation for portopulmonary hypertension patients in the USA: a real-world study |
| title_full_unstemmed | Clinical characteristics, treatment patterns and healthcare resource utilisation for portopulmonary hypertension patients in the USA: a real-world study |
| title_short | Clinical characteristics, treatment patterns and healthcare resource utilisation for portopulmonary hypertension patients in the USA: a real-world study |
| title_sort | clinical characteristics treatment patterns and healthcare resource utilisation for portopulmonary hypertension patients in the usa a real world study |
| url | https://bmjopen.bmj.com/content/15/6/e089223.full |
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