Abstract
Background
Patients’ experiences of living with pulmonary hypertension are likely to vary by geography and aetiology. The Pulmonary Hypertension Global Patient Survey (PH GPS) aims to understand patients’ perspectives and experiences, to drive healthcare improvements.
Methods
PH GPS was collaboratively designed through a multidisciplinary, iterative, consensus-building process and beta-tested by patients. Themes included healthcare provision, self-monitoring and research. It was predominantly disseminated by pulmonary hypertension organisations to patients or their proxies across all pulmonary hypertension groups. Descriptive statistics provided a narrative summary of key results.
Results
Data regarding 3329 adult (mean age 52.2 years; 81.7% female) and 135 paediatric patients were analysed, with this article focusing on the adult cohort. Most pulmonary arterial hypertension (PAH) patients reported diagnosis within 12 months of symptom onset. Variability was seen in survey response, pulmonary hypertension management and research participation, favouring the Global North and pulmonary hypertension groups 1 and 4. Genetic testing was reported by 33.9% (401 out of 1183) of patients, where eligible. 58% were experienced in self-monitoring their health. 20.6% of patients with pulmonary hypertension have completed patient-reported outcome measures (PROMs), with 7% feeling they changed management. 19.1% of adult patients have participated in research; however, 72.2% who have not participated would be willing to do so if invited.
Discussion
PH GPS is the largest survey of its kind in the pulmonary hypertension field, representing a globally collaborative endeavour. It highlights achievements such as efficient PAH diagnostic time frames. Shortcomings include underappreciating quality of life reported via PROMs. The Global South and pulmonary hypertension groups 2 and 3 were relatively underrepresented. Encouragingly, patients’ experiences of navigating healthcare were often positive, as demonstrated by a willingness to participate in research and being empowered to take charge of their health: findings which cross geographical divides.
Shareable abstract
3770 patients or their proxies from 90 countries contributed to this first Pulmonary Hypertension Global Patient Survey. The findings highlight the achievements, shortcomings and regional disparities of PH clinical care and research globally. https://bit.ly/3TdkkPu
Background
Pulmonary hypertension, characterised by elevated mean pulmonary arterial pressure, may lead to progressive right heart failure if not treated [1, 2]. Pulmonary hypertension arises from a spectrum of diseases which affect patients of all ages with differing management approaches [3–6]. Pulmonary hypertension group 1 (pulmonary arterial hypertension (PAH)) and pulmonary hypertension group 4 (predominantly chronic thromboembolic pulmonary hypertension (CTEPH)) are rare diseases which continue to cause substantial morbidity and mortality despite significant progress in management over the past two decades [7–8]. Pulmonary hypertension group 2 (pulmonary hypertension associated with left heart disease) and pulmonary hypertension group 3 (pulmonary hypertension associated with lung diseases and/or hypoxia) are common and have significant prognostic implications with limited pulmonary hypertension therapeutic options. Pulmonary hypertension group 5 consists of unclear and/or multifactorial mechanisms.
Pulmonary hypertension is known to impact patients’ health-related quality of life (HRQoL) negatively and significantly [9]. Patients’ experiences of living with pulmonary hypertension, clinical management and involvement in research are likely to show considerable geographical variation, but the extent is unknown [10]. The global perspectives of patients with pulmonary hypertension on healthcare, HRQoL, self-monitoring, health technology and research are lacking. Individual patient perspectives highlight the importance of personalised care and shared decision-making including prioritising HRQoL [10–12]. However, when global perspectives are considered, patients’ voices are empowered in steering international guidelines and driving innovation through clinical research [10, 13, 14].
A previous mixed-methods study of patients’ perspectives and experiences of living with PAH across five European countries harnessed cross-border collaboration, but the findings are now over a decade old [15]. Recent studies exploring the perspectives of patients with pulmonary hypertension including the James Lind Alliance priority setting partnership in Canada and the Living with PH Today survey in the United Kingdom can help identify areas for improvement in healthcare [16, 17]. However, noncontemporaneous national surveys with varying sets of questions and methodologies are challenging to compare and extrapolate at a global level. Large sample sizes are difficult to achieve with previous studies focusing on PAH, excluding paediatric cohorts and were Global North-centric. Therefore, there is a need for a global patient survey, designed with patients, delivered at scale and exploring a range of themes in depth. The Pulmonary Hypertension Global Patient Survey (PH GPS), an international patient-centred survey, required patients and multistakeholder co-design and collaboration to achieve the necessary global reach with meaningful outcomes. The aim of PH GPS, the first study of its kind, was to generate deeper comprehension of the impacts of pulmonary hypertension on patients and their experiences of healthcare across different regions in current times.
Methods
Survey development
The Pulmonary Vascular Research Institute (PVRI) Innovative Drug Development Initiative patient engagement and empowerment group conceived, designed and delivered PH GPS. The group consists of pulmonary hypertension healthcare professionals, academics, patients and multiple patient pulmonary hypertension organisations worldwide (appendix S8 in the supplementary material).
The survey content was drafted via an iterative consensus-building modified-Delphi process involving all group members based on clinical expertise, existing literature and patients’ experiences to identify priority areas of uncertainty [18]. Key themes that emerged included time to diagnosis, treatments, HRQoL, patient-reported outcome measures (PROMs), access to specialist healthcare, research participation, digital health and telemedicine.
There were three sections to the online survey broadly relating to 1) experience of diagnosis/treatment; 2) hospital visits, research participation and digital health; and 3) impact on HRQoL, capturing up to 100 data points per participant. Patients in pulmonary hypertension groups 1 and 4 were led to a management specific subset of questions (supplementary figure S1). Most questions were tick-box or multiple choice in nature. Thorough beta-testing of the original English draft survey was performed by patients and patient advocates. The initial English language version of PH GPS was translated and validated via beta-testing in 23 other languages (appendix S6 in the supplementary material).
Survey dissemination
The survey was launched in two phases for different languages, October 2023 and February 2024 (supplementary table S1). For this analysis, we used data collected within the first two months from each launch date, after which point there was a significant drop off in the number of responses. Adult patients with a diagnosis of any pulmonary hypertension group or their proxies, and parents/guardians completing the survey on behalf of children (<16 years) were eligible to participate. PH GPS was primarily disseminated by patient pulmonary hypertension organisations using their routine media resources including both traditional and social media. The dedicated PVRI landing webpage hosted media resources along with further information and links to the survey available in 24 different languages.
This study complied with the Declaration of Helsinki. All data collected under this survey were processed and stored in line with applicable laws, including, but not limited to General Data Protection Regulation (EU 2016/679) (appendix S7 in the supplementary material); however, no personally identifiable data were requested.
Analysis
Responders were only included for analysis if all baseline information was captured (including country, sex and diagnosis). Responses were reported by 10 geographic regions as a compromise between reporting at a national or continental level. The European Union EuroVoc (https://op.europa.eu/en/web/eu-vocabularies) subregion definitions were used. Pulmonary hypertension classification and other clinical interpretations were based upon the latest international guidelines [14].
The current analysis focuses on providing a high-level overview at a global level from all pulmonary hypertension groups where responses to each question were presented by descriptive statistics. Continuous variables were expressed as mean±sd or median (interquartile range (IQR), 25–75%). Categorical variables were expressed as absolute frequencies (n) and percentage (%). Coding and statistical analysis were performed using Python and the open-source data frame library Polars. Unless otherwise specified, we focused on adult responders in the current analysis.
Further description of the methods is included in the supplementary material (appendix S1).
Results
Overview of patients and geographic representation
3770 participants responded to the survey; the final adult dataset was 3329 responders (2890 patients and 439 proxies) with mean±sd age of 52.2±15.9 years and 81.7% female. 59.6% of responses represent pulmonary hypertension group 1 (figure 1), with a relative underrepresentation of pulmonary hypertension groups 2 and 3 (355 (10.7%) out of 3329 and 240 (7.2%) out of 3329, respectively). The final paediatric dataset was 135 guardians of children with pulmonary hypertension, but is not described beyond demographics in this article. Table 1 shows a summary of patient demographics.
FIGURE 1.
Flow diagram of responders within the study.
TABLE 1.
Summary demographics of adult and paediatric patients
| Total | North America | Central America and Caribbean | South America | Central and Eastern Europe | Northern Europe | Southern Europe | Western Europe | Asia | Africa | Oceania | |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Adult patients | |||||||||||
| Responders | 3329 (100) | 777 (23.3) | 257 (7.7) | 516 (15.5) | 446 (13.4) | 139 (4.2) | 257 (7.7) | 722 (21.7) | 140 (4.2) | 34 (1.0) | 41 (1.2) |
| Countries represented | 90 | 2 | 12 | 10 | 14 | 6 | 8 | 11 | 14 | 9 | 4 |
| Age years | 52.2±15.9 | 62.7±13.9 | 44.5±14.0 | 44.6±13.0 | 46.3±13.9 | 57.6±16.1 | 49.6±13.1 | 54.2±15.4 | 42.1±14.5 | 49.4±16.7 | 58.1±14.5 |
| Sex | |||||||||||
| Female | 2720 (81.7) | 651 (83.8) | 213 (82.9) | 446 (86.4) | 368 (82.5) | 119 (85.6) | 198 (77.0) | 557 (77.1) | 102 (72.9) | 30 (88.2) | 36 (87.8) |
| Prefer not to say | 7 (0.2) | ≤5 | ≤5 | ≤5 | ≤5 | ≤5 | |||||
| Pulmonary hypertension diagnosis | |||||||||||
| Group 1 | 1985 (59.6) | 515 (66.3) | 139 (54.1) | 309 (59.9) | 234 (52.5) | 98 (70.5) | 156 (60.7) | 387 (53.6) | 104 (74.3) | 18 (52.9) | 25 (61.0) |
| Group 2 | 355 (10.7) | 59 (7.6) | 27 (10.5) | 76 (14.7) | 88 (19.7) | ≤5 | 33 (12.8) | 53 (7.3) | 6 (4.3) | ≤5 | ≤5 |
| Group 3 | 240 (7.2) | 76 (9.8) | 16 (6.2) | 24 (4.7) | 24 (5.4) | ≤5 | 14 (5.4) | 60 (8.3) | 10 (7.1) | 6 (17.6) | ≤5 |
| Group 4 | 416 (12.5) | 57 (7.3) | 47 (18.3) | 44 (8.5) | 57 (12.8) | 19 (13.7) | 28 (10.9) | 154 (21.3) | ≤5 | ≤5 | ≤5 |
| Group 5 | 101 (3.0) | 21 (2.7) | 9 (3.5) | 9 (1.7) | 17 (3.8) | ≤5 | 6 (2.3) | 25 (3.5) | 6 (4.3) | ≤5 | ≤5 |
| Unsure | 232 (7.0) | 49 (6.3) | 19 (7.4) | 54 (10.5) | 26 (5.8) | 8 (5.8) | 20 (7.8) | 43 (6.0) | 9 (6.4) | ≤5 | ≤5 |
| Paediatric patients | |||||||||||
| Responders | 135 (100) | 11 (8.1) | 11 (8.1) | 32 (23.7) | 29 (21.5) | ≤5 | 10 (7.4) | 27 (20.0) | 11 (8.1) | ≤5 | ≤5 |
| Age years | 9.4±4.3 | 10.4±4.3 | 9.5±4.4 | 9.1±4.1 | 9.7±4.3 | 14.0±1.4 | 9.2±4.8 | 9.4±4.8 | 7.7±3.1 | 7 | 8 |
| Sex | |||||||||||
| Female | 74 (54.8) | ≤5 | ≤5 | 17 (53.1) | 17 (58.6) | ≤5 | ≤5 | 18 (66.7) | 6 (54.5) | ||
| Pulmonary hypertension diagnosis | |||||||||||
| Group 1 | 99 (73.3) | 10 (90.9) | 6 (54.5) | 19 (59.4) | 18 (62.1) | ≤5 | 8 (80) | 23 (85.2) | 11 (100) | ≤5 | ≤5 |
| Group 2 | 18 (13.3) | ≤5 | ≤5 | ≤5 | ≤5 | ≤5 | |||||
| Group 3 | ≤5 | ≤5 | ≤5 | ||||||||
| Group 4 | ≤5 | ≤5 | |||||||||
| Group 5 | ≤5 | ≤5 | ≤5 | ||||||||
| Unsure | 12 (8.9) | ≤5 | 8 (25.0) | ≤5 | ≤5 | ||||||
Data are presented as n (%), n or mean±sd.
Responses were received from 90 countries across six continents (figure 2). Highest responses were from the United States of America (n=733, 21.2%), Argentina (n=267, 7.7%), Mexico (n=231, 6.7%), Germany (n=169, 4.9%), the Netherlands (n=160, 4.6%), Ukraine (n=141, 4.1%), Spain (n=128, 3.7%), Poland (n=122, 3.5%), the United Kingdom (n=122, 3.5%) and India (n=102, 2.9%) (supplementary table S2). There were no large differences in the rates of full survey completion (defined by mandatory data completion) by region (supplementary table S3). Of the 24 languages available, responses were received in 22 languages; predominantly English 33.1% (1148 out of 3464) and Spanish 25.4% (881 out of 3464) (supplementary table S4). By language, the rate of mandatory data completion varied from 100% in Serbian to 0% in Chinese, with the overall majority rate of completion between 75% and 93% (supplementary table S5).
FIGURE 2.
Graphical representation of the number of responses and countries per geographical region. European regions as defined by EuroVoc (https://op.europa.eu/en/web/eu-vocabularies). North America: Canada, United States of America; Central America and Caribbean: Aruba, the Bahamas, Belize, Costa Rica, Dominica, Dominican Republic, Guatemala, Honduras, Mexico, Nicaragua, Panama, Puerto Rico; South America: Argentina, Bolivia, Brazil, Chile, Colombia, Ecuador, Paraguay, Peru, Uruguay, Venezuela; Central and Eastern Europe: Bosnia and Herzegovina, Bulgaria, Croatia, Czech Republic, Georgia, Hungary, Montenegro, North Macedonia, Poland, Romania, Serbia, Slovak Republic, Slovenia, Ukraine; Northern Europe: Denmark, Estonia, Finland, Latvia, Norway, Sweden; Southern Europe: Cyprus, Gibraltar, Greece, Italy, Malta, Portugal, Spain, Turkey; Western Europe: Andorra, Austria, Belgium, the Channel Islands, France, Germany, Ireland, Luxembourg, the Netherlands, Switzerland, United Kingdom; Asia: Bangladesh, Bhutan, Cambodia, China, India, Indonesia, Iran, Israel, Malaysia, Pakistan, the Philippines, Saudi Arabia, Thailand, United Arab Emirates; Africa: Algeria, Cabo Verde, Chad, Ghana, Kenya, Morocco, Mozambique, Nigeria, South Africa; Oceania: American Samoa, Australia, New Zealand, Samoa.
There were variable percentages of responders to different sections, which gradually declined towards the end of the survey questions (supplementary figure S2). Percentage of responders to the key themes by order of questions within the survey were 100% for treatments given (pulmonary hypertension groups 1 and 4 only); 100% regarding perspectives on research; 89.7% (2987 out of 3329) on self-monitoring; 87.7% (2921 out of 3329) on PROMs; 86.1% (2867 out of 3329) on access to specialist healthcare; and 79.7% (2653 out of 3329) on HRQoL.
Experiences of pulmonary hypertension healthcare with variations between regions and pulmonary hypertension groups
Patients’ experiences with pulmonary hypertension healthcare provision varied by geography and with pulmonary hypertension groups (table 2). 83.1% (2382 out of 2867) of adult responders reported being under the care of a pulmonary hypertension specialist centre and this ranged from 69.2% (155 out of 223) in Central America and the Caribbean to 94.6% (35 out of 37) in Oceania. 16.4%, 37.2% and 46.4% of patients have been under the care of a pulmonary hypertension centre for <1 year, 1–5 years and >5 years, respectively. A higher proportion of patients with groups 1 and 4 pulmonary hypertension are under specialist care, compared to other pulmonary hypertension groups.
TABLE 2.
Variation in pulmonary hypertension healthcare provision by geographical regions and pulmonary hypertension group
| Total | Variation by geographical region | Variation by pulmonary hypertension group | |||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| North America | Central America and Caribbean | South America | Central and Eastern Europe | Northern Europe | Southern Europe | Western Europe | Asia | Africa | Oceania | Group 1 | Group 2 | Group 3 | Group 4 | Group 5 | Unsure | ||
| Care under pulmonary hypertension centre | |||||||||||||||||
| Responders | 2867 (100) | 683 (23.8) | 223 (7.8) | 438 (15.3) | 392 (13.7) | 120 (4.2) | 213 (7.4) | 614 (21.4) | 114 (3.9) | 33 (1.2) | 37 (1.3) | 1745 (60.9) | 309 (10.8) | 199 (6.9) | 359 (12.5) | 88 (3.1) | 167 (5.8) |
| Yes | 2382 (83.1) | 595 (87.1) | 155 (69.2) | 323 (73.7) | 326 (83.2) | 94 (78.3) | 183 (85.9) | 557 (90.7) | 89 (78.1) | 25 (75.8) | 35 (94.6) | 1505 (86.2) | 233 (75.4) | 154 (77.4) | 312 (86.9) | 73 (83.0) | 105 (62.9) |
| Healthcare cost reimbursed/covered by provider | |||||||||||||||||
| Responders | 2367 (100) | 595 (25.1) | 154 (6.5) | 320 (13.5) | 325 (13.7) | 91 (3.8) | 183 (7.7) | 553 (23.4) | 87 (3.7) | 24 (1) | 35 (1.5) | 1495 (63.2) | 233 (9.8) | 152 (6.4) | 310 (13.1) | 73 (3.1) | 104 (4.4) |
| Fully covered | 1325 (56.0) | 275 (46.2) | 38 (24.7) | 119 (37.2) | 220 (67.7) | 52 (57.1) | 138 (75.4) | 440 (79.6) | 15 (17.2) | ≤5 | 24 (68.6) | 841 (56.3) | 99 (42.5) | 98 (64.5) | 195 (62.9) | 45 (61.6) | 47 (45.2) |
| Partially covered | 434 (18.3) | 201 (33.8) | 16 (10.4) | 50 (15.6) | 34 (10.5) | 23 (25.3) | 10 (5.5) | 69 (12.5) | 15 (17.2) | 9 (37.5) | 7 (20.0) | 295 (19.7) | 40 (17.2) | 26 (17.1) | 43 (13.9) | 14 (19.2) | 16 (15.4) |
| Not covered | 397 (16.8) | 69 (11.6) | 77 (50.0) | 101 (31.6) | 41 (12.6 | ≤5 | 24 (13.1) | 20 (3.6) | 52 (59.8) | 7 (29.2) | ≤5 | 236 (15.8) | 60 (25.8) | 18 (11.8) | 48 (15.5) | 11 (15.1) | 24 (23.1) |
| Unsure | 211 (8.9) | 50 (8.4) | 23 (14.9) | 50 (15.6) | 30 (9.2) | 12 (13.2) | 11 (6.0) | 24 (4.3) | ≤5 | ≤5 | ≤5 | 123 (8.2) | 34 (14.6) | 10 (6.6) | 24 (7.7) | ≤5 | 17 (16.3) |
Data are presented as n (%) or n.
70.8% (1576 out of 2225) of patients reported travelling by car to their pulmonary hypertension centre, with a median (IQR) distance of 56.3 (118.0) km and 51.4% travelling more than an hour each way. Distance travelled varied by region which was furthest in Asia (mean±sd 401.3±1193.8 km) (supplementary table S6). 69.5% (1547 out of 2225) and 96.2% (2141 out of 2225) of patients visit their pulmonary hypertension centre at least every 6 or 12 months, respectively.
Geographical differences were seen with financial coverage of pulmonary hypertension healthcare cost, with highest partial or full cover in Western Europe at 92.1% and lowest in Asia at 34.4%. Pulmonary hypertension group 2 patients reported receiving the least (59.7%, 139 out of 233) partial or full cover of their healthcare cost.
Perceptions of pulmonary hypertension healthcare
67.8% (1509 out of 2225) patients perceive some degree of difficulty or stressfulness with attending their appointments. 71.6% (1594 out of 2225) reported that all their appointments occur only in-person, but 81.0% (1803 out of 2225) were agreeable to some aspects of their healthcare being delivered remotely where possible. 81.8% (1820 out of 2225) of patients feel that that they fully understand the information provided; however, 45.3% (1007 out of 2225) perceive some degree of incongruity with how they feel compared to the results of their medical consultations.
HRQoL and PROMs
Patients experienced significant negative effects on their wellbeing (figure 3). 40.7% (1195 out of 2939) perceived themselves as physically active and 74.0% (1513 out of 2045) had their employment or education negatively impacted to some degree. In the preceding 6 months to the survey, 46.9% (1461 out of 3116) of patients reported side-effects due to pulmonary hypertension medications; nausea was most common (46.3%; 677 out of 1461) (supplementary table S7). Only 31.8% of responders who experienced side-effects discussed with their healthcare provider the possibility of changing pulmonary hypertension medication. Negative impacts on wellbeing are most likely to be discussed with patients’ pulmonary hypertension specialist (79.8%), family/friends (47.5%) or life partner (40.2%).
FIGURE 3.
Impact of pulmonary hypertension on a) self-reported emotions, cognition and wellbeing (n=2650) and b) activities of daily living.
Patients (n=2203) on average noted slight improvement in their symptoms since diagnosis (mean±sd 3.12±1.02 on Likert scale, where 1=significantly worse, 2=slightly worse, 3=no change, 4=slightly better, 5=significantly better) (table 3; supplementary table S8). Patients in Africa scored the lowest with a mean±sd of 2.8±1.2 (n=23), and the highest scores were seen in Southern Europe with a mean±sd of 3.3±1.0 (n=166). 20.6% (601 out of 2921) of patients reported having ever completed a PROM, with significant geographical variability (table 3). Of those who have completed PROMs, only 26.3% received feedback from their pulmonary hypertension specialist team and only 7% perceived that their management had been altered based on PROM responses.
TABLE 3.
Patients’ perception of and experience with aspects of wellbeing
| Total | North America | Central America and Caribbean | South America | Central and Eastern Europe | Northern Europe | Southern Europe | Western Europe | Asia | Africa | Oceania | |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Change in symptoms since diagnosis on a scale of 1–5 (with 1 being significantly worse and 5 being fully recovered) | |||||||||||
| Responders | 2203 (100) | 561 (25.5) | 137 (6.2) | 282 (12.8) | 306 (13.9) | 86 (3.9) | 166 (7.5) | 531 (24.1) | 79 (3.6) | 23 (1) | 32 (1.5) |
| Score | 3.12±1.02 | 3.1±1.0 | 3.2±1.0 | 3.2±1.1 | 3.0±1.1 | 3.1±1.0 | 3.3±1.0 | 3.1±1.0 | 2.9±1.0 | 2.8±1.2 | 3.1±1.0 |
| Filled PROM questionnaire | |||||||||||
| Responders | 2921 (100) | 695 (23.8) | 226 (7.7) | 448 (15.4) | 403 (13.8) | 122 (4.2) | 220 (7.5) | 617 (21.1) | 120 (4.1) | 33 (1.1) | 37 (1.3) |
| Yes | 601 (20.6) | 117 (16.8) | 32 (14.2) | 53 (11.8) | 100 (24.8) | 47 (38.5) | 47 (21.4) | 168 (27.2) | 25 (20.8) | ≤5 | 10 (27.0) |
| Self-monitor | |||||||||||
| Responders | 2987 (100) | 708 (23.7) | 232 (7.8) | 464 (15.5) | 408 (13.7) | 125 (4.2) | 225 (7.5) | 632 (21.2) | 122 (4.1) | 33 (1.1) | 38 (1.2) |
| Yes | 1428 (47.8) | 379 (53.5) | 117 (50.4) | 232 (50.0) | 171 (41.9) | 44 (35.2) | 104 (46.2) | 285 (45.1) | 66 (54.1) | 15 (45.5) | 15 (39.5) |
| Used to | 245 (8.2) | 52 (7.3) | 22 (9.5) | 23 (5.0) | 63 (15.4) | 8 (6.4) | 15 (6.7) | 41 (6.5) | 12 (9.8) | ≤5 | ≤5 |
| No | 1144 (38.3) | 235 (33.2) | 78 (33.6) | 177 (38.1) | 162 (39.7) | 65 (52.2) | 90 (40.0) | 273 (43.2) | 37 (30.3) | 12 (36.4) | 15 (39.5) |
| Other | 170 (5.7) | 42 (5.9) | 15 (6.5) | 32 (6.9) | 12 (2.9) | 8 (6.4) | 16 (7.1) | 33 (5.2) | 7 (5.7) | ≤5 | ≤5 |
| Registered as disabled | |||||||||||
| Responders | 3095 (100) | 737 (23.8) | 240 (7.8) | 484 (15.7) | 417 (13.5) | 125 (4) | 235 (7.6) | 652 (21.1) | 129 (4.2) | 34 (1.1) | 38 (1.2) |
| Yes | 1683 (54.4) | 500 (67.8) | 50 (20.8) | 266 (55.0) | 262 (62.8) | 52 (41.6) | 127 (54.0) | 379 (58.1) | 14 (10.9) | 10 (29.4) | 22 (57.9) |
Data are presented as n (%), mean±sd or n. PROM: patient-reported outcome measure.
Self-monitoring and digital health
47.8% (1428 out of 2987) of patients across all pulmonary hypertension groups self-monitor their health status, which was similar across regions and age brackets (table 3; supplementary table S9). Of those, 51.5% (712 out of 1382) do so via analogue means (paper or calendar) and 28.4% (393 out of 1382) do so via a smartphone application. 93.4% (2742 out of 2935) of adult patients with pulmonary hypertension own a smartphone, 5.3% (155 out of 2935) own a mobile phone without internet access and 1.3% (38 out of 2935) do not own a mobile phone (supplementary tables S10 and S11). This is a consistent finding across geographic regions and across age brackets until the age of 70 years, after which smartphone ownership declines (80–89 years 77%, n=91) (supplementary tables S10 and S11). In terms of activity monitoring, 34.2% (1004 out of 2935) of patients use a smartwatch, 15.8% (464 out of 2935) use their mobile phone and 47.8% (1404 out of 2935) do not track their activity, with variation shown by geography and age (supplementary table S12). 69.1% (969 out of 1402) of patients already share the self-collected information with their clinical team and a further 25.5% (358 out of 1402) would be willing to do so.
Disability and support
54.4% (1683 out of 3095) of patients were officially registered as disabled due to pulmonary hypertension (table 3 and supplementary table S13) with benefits including parking (67.3%, 1133 out of 1683), financial support (41.2%, 693 out of 1683) and enhanced care support (15.7%, 265 out of 1683). Access to rehabilitation was available for 39.7% (944 out of 2377) of responders, but 41.6% were unsure. Most responders (54.8%, 1445 out of 2638) were part of a national patient pulmonary hypertension organisation, where the most useful perceived areas of support included information/education (84.8%), patient meetings/support groups (51.9%), informative website (46.4%) and awareness activities (30.7%) (supplementary table S14).
Research participation
73.4% (2449 out of 3329) of patients with pulmonary hypertension have not participated in clinical research trials (figure 4). Participation in research varied by geography from 5.9% (two out of 34) in Africa to 25.2% (112 out of 446) in Central and Eastern Europe. Similarly, participation varied by pulmonary hypertension group, with the lowest in group 3 (9.2%, 22 out of 240) and highest in pulmonary hypertension group 1 (23.6%, 466 out of 1986).
FIGURE 4.
Cascade diagram showing patients’ experiences of and perspectives on clinical trial participation.
Of those patients who had not participated, 72.2% would consider taking part if approached (figure 4), ranging from 64.8% (272 out of 420) in South America to 88.8% (95 out of 107) in Northern Europe, and from 67.4% (182 out of 268) in pulmonary hypertension group 2 to 74.4% (145 out of 195) in pulmonary hypertension group 3.
Most patients who have previously participated in research trials had good experiences (73.1%, 464 out of 635) and were willing to participate again particularly if their initial experience was positive (82.8%, 384 out of 464). These trends were consistent across geographic regions and pulmonary hypertension groups (supplementary table S15).
90.8% (2769 out of 3048) would be willing to allow researchers to access and use their anonymised healthcare data that had already been collected as part of routine clinical care. 48.7% (1483 out of 3048) of adult responders opted in to provide their email address to be contacted for future survey-based research.
Pulmonary hypertension group 1 and 4 specific results
Globally, 56.2% and 47.7% of diagnoses of idiopathic/heritable PAH or CTEPH, respectively, were made within 12 months of symptom onset, with regional variation (figure 5a and b). 33.9% (401 out of 1183) of responders with PAH, where eligible, had genetic testing which varied geographically (supplementary table S17). Extended results are in the supplementary material (appendix S2; supplementary tables S16–S18).
FIGURE 5.
Charts of time to diagnosis from point of symptom onset by geographical regions for a) idiopathic or heritable pulmonary arterial hypertension or b) chronic thromboembolic pulmonary hypertension.
Discussion
PH GPS is the first global survey to study the impact from all pulmonary hypertension groups on patients’ lives, which provides a unique insight into their experiences with and perspectives on healthcare and clinical research. This survey generated novel in-depth data across a broad range of themes from >3000 responders across six continents and 90 countries. Notably, this was achieved through a patient-centred approach and by forging new collaborations between leading patient pulmonary hypertension organisations. A large, diverse and inclusive patient participation was a key priority, realised through the availability of 24 languages developed through an innovative hybrid human and automated translation model which enabled the results to be more representative and robust. This optimised the meaningfulness and relevance of the survey for patients and heightened both the response rate and the transferability of the results to guide future patient care. Despite improvements in survival and most patients reporting being under the care of a specialist centre, pulmonary hypertension continues to have profound impacts on patients’ lives, including symptoms, daily functioning and employment [3, 5, 7, 8, 19–21].
There were encouraging key results from this survey including patients’ reporting that diagnoses of idiopathic and heritable PAH from symptom onset were generally <12 months, which was quicker than reported previously [17, 22–24]. Most patients reported comprehension of information provided by their pulmonary hypertension healthcare provider and were agreeable to some aspects of their healthcare being delivered remotely. As most patients had experience in self-monitoring their health, were willing to share self-collected information with their pulmonary hypertension team and reported some degree of difficulty and stress in visiting pulmonary hypertension centres, this illustrated the need for flexible and personalised delivery of healthcare. The fact that impaired mobility is the highest ranked impact of pulmonary hypertension on daily function gives credence to the importance and meaningfulness of the 6-min walk test in pulmonary hypertension as a prognostic marker and trial primary end-point [25]. Patients’ experience with the education, support and advocacy provided by patient pulmonary hypertension organisations cannot be overstated and their value was recently highlighted by the 7th World Symposium on Pulmonary Hypertension taskforce (7WSPH) [10].
PH GPS highlighted several important shortcomings in patient care. Across all pulmonary hypertension groups, only a fifth of patients reported having ever completed PROMs. Only a minority received feedback on completed PROMs from their pulmonary hypertension team and only rarely were PROMs perceived to be used to guide pulmonary hypertension management. The underutilisation of patient pulmonary hypertension organisation resources and PROMs persists despite the advocacy of previous survey findings and international guidelines [14, 15]. This is due to a range of possible factors including a lack of awareness or understanding of PROMs, uncertainty as to how best to incorporate the tools into patient assessment, or a lack of time during consultations. Despite the high rate of side-effects experienced from pulmonary hypertension management, patients seldom discussed alternative options with their pulmonary hypertension clinicians. However, most patients were ready to highlight detrimental impacts on wellbeing associated with pulmonary hypertension with their specialist. The need for holistic care was demonstrated by patients reporting uncertainty regarding accessibility of rehabilitation services and the value of the social support offered by patient organisations, also highlighted by the 7WSPH [10]. Despite progress in the understanding of genetics in PAH and its potential relevance to therapies and outcomes, only approximately a third of eligible pulmonary hypertension group 1 patients had genetic testing. The majority of those who did not have genetic testing were not offered by their healthcare provider despite only a minority reporting financial cost to be the limiting factor. There is a clear unmet need regarding pulmonary hypertension groups 2 and 3 as shown by the underrepresentation in this survey and lower research participation despite being far more common than PAH or CTEPH [2].
From a global perspective, this survey shed light on the expected geographical healthcare inequities with differences in availability to specialist diagnostics (e.g. genetic testing), therapies (e.g. parenteral prostacyclin analogues), interventions (e.g. pulmonary endarterectomy or balloon pulmonary angioplasty) or research participation, which all broadly favour the Global North. Variability of treatment provided globally is challenging to decipher, due to the differences in the number of responders by region. Of note is the relative underrepresentation of the Global South in this survey, which does not reflect the true prevalence of pulmonary hypertension worldwide [26]. In pulmonary hypertension group 1, parenteral pulmonary hypertension therapy was generally less prescribed in the Global South, which may reflect the geographical variation in experience and resources. Central and South America showed the highest proportion of patients not receiving mechanical intervention for CTEPH and marginal representation of patients with CTEPH from Asia, Africa and Oceania additionally highlights imbalances in global healthcare.
However, PH GPS does provide numerous reasons for optimism with trends showing that, irrespective of geography or diagnostic subgroup, there is willingness to engage in clinical research and digital health which are key areas of innovation. Perceived barriers to trial participation are generally remediable and positive research experience improves the likelihood of further participation. The scope of PH GPS itself is a marker of a globally connected and highly motivated pulmonary hypertension community.
Limitations and the future
Responses to the survey are at risk of selection bias and likely to be overrepresented from patients who access patient pulmonary hypertension organisations as they were the main channel of survey dissemination, and may not fully represent the general population of patients with pulmonary hypertension. This also explains, in part, why pulmonary hypertension groups 2 and 3 are relatively underrepresented, as patient pulmonary hypertension organisations primarily advocate for patients in pulmonary hypertension groups 1 and 4. A further limitation possibly affecting the representativeness is the high female-to-male ratio of responders across all pulmonary hypertension groups, which is out of proportion to what is seen in clinical practice, but is a phenomenon reported in other survey-based research [26–29]. Furthermore, the majority of participants were prevalent patients, which may skew responses. The online nature of the survey may limit diversity by excluding those without internet access; however, this was expected and partly offset by allowing response via a proxy. Similarly, due to the open nature of routine media resources of patient pulmonary hypertension organisations, the denominator in terms of response rate is unknown and unquantifiable. Modest drop-off was observed during the survey leading to expected relative missingness for latter sections given the length and depth of the questionnaire [29, 30]. There was also a risk of recall bias or discrepancy between responders’ perceptions and pulmonary hypertension healthcare providers’ adjudication, but responders were instructed to refer to their clinic letters and drug prescriptions throughout to help minimise this.
We were cautious about drawing overarching conclusions from such a granular dataset from thousands of responders so varied by diagnosis, demographics and geography. This would necessitate further outputs from this study in due course including an in-depth look at HRQoL, qualitative survey responses, health technology assessment and the paediatric cohort. An international consensus will also be published from patients, patient pulmonary hypertension organisations and healthcare providers which will summarise the “call to action” from this study. The underrepresentation of the Global South and our approach at geographical division may skew the descriptive interpretation of patients’ experiences [31]. Therefore, the next round of PH GPS should consider approaches in targeting the Global South and perhaps a minimum number of responses to obtain a more in-depth regional insight. There is a compelling need to repeat and build on this global survey in the future to understand how patients’ experiences and perceptions change over time and evolve by geographic region and pulmonary hypertension group, while also looking to incorporate corresponding insights from pulmonary hypertension healthcare providers regarding clinical management.
Conclusion
The scale and depth of PH GPS helps to fill the knowledge gap about patients’ perspectives on and experiences of their healthcare. This survey highlights achievements in pulmonary hypertension healthcare, such as the reported efficient time to PAH diagnosis, patients’ comprehension of their condition and valuable support from patient pulmonary hypertension organisations. PH GPS also illuminates areas for improvement including underutilisation and underappreciating patient-reported health to guide clinical management. Despite the global reach, the Global South and pulmonary hypertension groups 2 and 3 continue to be relatively underrepresented, limiting insight into these cohorts. There is optimism for the future through an empowered global patient cohort primed to participate in research, engage with digital health and take charge of their wellbeing; trends which cross the geographical divide and diagnostic subgroups.
Acknowledgements:
We are grateful to the thousands of patients, carers and family members who responded to the survey; Michael Nio, Sarah Drumm and colleagues at the Pulmonary Vascular Research Institute for project management; iOWNA for assistance with early programming of the online survey; and PHA US, PHA Europe, ELF PH Patient Advisory Group and all other patient organisations involved in this study; please see the supplementary material for the organisations and individuals involved.
Footnotes
Data sharing statement: Requests for limited and fully anonymised data to answer specific research questions can be made to the PVRI via: https://pvrinstitute.org/sites/default/files/2024-05/Data%20Access%20Policy%20-%20Feb%202024.docx. This URL also provides further information on the project's Data Access Policy. A GitHub repository is accessible via https://github.com/ShivMunagala/PH-GPS for open-access code under the MIT License.
Provenance: Submitted article, peer reviewed.
Author contributions: Concept and design: All authors. Acquisition, analysis, or interpretation of data: All authors. Drafting of the manuscript: J. Newman, H. Ghani and J. Pepke-Zaba. Critical review of the manuscript for important intellectual content: All authors. Statistical analysis: J. Newman, H. Ghani and S. Munagala. Administrative, technical, or material support: M. Stone. Supervision: J. Pepke-Zaba. Access, responsibility and Analysis: J. Newman, H. Ghani, S. Munagala and J. Pepke-Zaba had full access to all the data in the study and take responsibility for the integrity of the data and the accuracy of the analyses.
This article has an editorial commentary: https://doi.org/10.1183/23120541.00963-2025
Conflicts of interest: The authors declare no potential conflicts of interest.
Support statement: J. Newman is supported by a British Heart Foundation Clinical Research Training Fellowship (FS/CRTF/22/24390). M. Stone is supported as Entrepreneur in Residence, Royal Society. L. MacDonald receives research funding from the Dinosaur Trust. The views expressed are those of the authors and not necessarily those of the affiliated organisations. The funders did not have a role in the design and conduct of the study; collection, management, analysis and interpretation of the data; preparation, review or approval of the manuscript; or decision to submit the manuscript for publication. The researchers acted independently and take responsibility for the integrity of the study.
Supplementary material
Please note: supplementary material is not edited by the Editorial Office, and is uploaded as it has been supplied by the author.
Supplementary material
00297-2025.SUPPLEMENT
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