Pulmonary hypertension in infants with bronchopulmonary dysplasia: risk factors, mortality and duration of hospitalisation.

OBJECTIVES: Pulmonary hypertension (PH) is a complication of bronchopulmonary dysplasia (BPD) and associated with increased mortality and morbidity. Our aim was to identify, in infants with BPD, the effect of PH on health-care utilisation and health related cost of care. METHODS: An electronic data recording system was used to identify infants ≤32 weeks of gestation who developed BPD. PH was classified as early (≤28 days after birth) or late (>28 days after birth). RESULTS: In the study period, 182 infants developed BPD; 22 (12.1%) developed late PH. Development of late PH was associated with a lower gestational age [24.6 (23.9-26.9) weeks, p=0.001] and a greater need for positive pressure ventilation on day 28 after birth (100%) compared to infants without late PH (51.9%) (odds ratio (OR) 19.5, 95% CI: 2.6-148), p<0.001. Late PH was associated with increased mortality (36.4%) compared those who did not develop late PH (1.9%) after adjusting for gestational age and ventilation duration (OR: 26.9, 95% CI: 3.8-189.4), p<0.001. In infants who survived to discharge, late PH development was associated with a prolonged duration of stay [147 (118-189) days] compared to the infants that did not develop late PH [109 (85-149) days] (p=0.03 after adjusting for gestational age). Infants who had late PH had a higher cost of stay compared to infants with BPD who did not develop late PH (median £113,494 vs. £78,677, p=0.016 after adjusting for gestational age). CONCLUSIONS: Development of late PH was associated with increased mortality, a prolonged duration of stay and higher healthcare cost.

Utility of Endoscopy in Hospitalized Patients with Gastrointestinal Hemorrhage and Pulmonary Hypertension.

BACKGROUND: Gastrointestinal hemorrhage (GIH) has been reported as one of the most common GI complications in patients with pulmonary hypertension (PH). There is paucity of data on the national burden of GIH in patients with PH. We aimed to assess the prevalence, trends and outcomes of endoscopic interventions in patients with PH who were admitted with GIH. METHOD: We queried National Inpatient Sample (NIS) database from 2005 to 2014 and identified the patients hospitalized with primary or secondary discharge diagnosis of PH (ICD 9 CM Code: 416.0, 416.8, and 416.9). Using Clinical Classification Software Coding system (153) patients with concurrent diagnosis of GIH were then identified. We studied the prevalence and trends of GIH in PH, factors associated with GIH, use of endoscopy, factors associated with utilization of endoscopic interventions, endoscopy outcomes including mortality, and overall healthcare burden. RESULTS: Out of 7,586,973 PH hospitalizations 3.2% (N = 246,358) had concurrent GIH, with a rising prevalence of GIH in PH patients during the last decade. Clinical predictors for GIH in PH included older age, congestive heart failure, anticoagulation therapy and concurrent alcohol abuse. Mean length of stay (LOS) in PH patients hospitalized with GIH was significantly higher than without GIH (8.6 vs. 6.4 days, p < 0.01) along with a significant increase in hospitalization cost ($20,189 vs. $14,807, p < 0.01). Similarly, odds of in-hospital mortality increase by ~ 1.5 times in PH patients with GIH than those without it (adjusted odds ratio [aOR: 1.45, 95%CI: 1.43-1.47]). Endoscopic interventions were performed in 48.6% of patients with PH and GIH during their hospitalization. Older patients were more likely to undergo endoscopy, as well as the patients who received blood transfusion, and those with hypovolemic shock. Patients with acute respiratory failure and acute renal failure were less likely to get endoscopy. Mean LOS in patients undergoing endoscopic intervention was significantly higher than those who did not receive any intervention (8.7 vs. 8.4 days, p < 0.01), without a substantial increase in hospitalization cost ($20,344 vs. $20,041, p < 0.01). Also, there was a significant decrease in in-hospital mortality in patients undergoing endoscopic interventions. CONCLUSION: Concurrent GIH in patients with PH increases length of stay; healthcare costs and increases in-hospital mortality. Use of endoscopic interventions in these patients is associated with reduced length of stay, in-hospital mortality without significantly increasing the overall health care burden and should be considered in hospitalized patients with PH who are admitted with GIH. Future studies comparing GIH patients with and without PH should be done to assess if PH is a risk factor for worse outcomes. CLINICAL TRIAL REGISTRATION NUMBER: No IRB required due to use of national de-identified data.

Supervised pulmonary hypertension exercise rehabilitation (SPHERe): study protocol for a multi-centre randomised controlled trial.

BACKGROUND: Supervised cardio-pulmonary rehabilitation may be safe and beneficial for people with pulmonary hypertension (PH) in groups 1 (pulmonary arterial hypertension) and 4 (chronic thromboembolic disease), particularly as a hospital in-patient. It has not been tested in the most common PH groups; 2 (left heart disease), 3 (lung disease), or 5 (other disorders). Further it has not been evaluated in the UK National Health Service (NHS) out-patient setting, or with long-term follow-up. The aim of this randomised controlled trial (RCT) is to test the clinical and cost-effectiveness of a supervised exercise rehabilitation intervention with psychosocial support compared to best practice usual care for people with PH in the community/outpatient setting. METHODS: This multi-centre, pragmatic, two-arm RCT with embedded process evaluation aims to recruit 352 clinically stable adults with PH (groups 1-5) and WHO functional class II-IV. Participants will be randomised to either the Supervised Pulmonary Hypertension Exercise Rehabilitation (SPHERe) intervention or control. The SPHERe intervention consists of 1) individual assessment and familiarisation sessions; 2) 8-week, twice-weekly, supervised out-patient exercise training; 3) psychosocial/motivational support and education; 4) guided home exercise plan. The control intervention consists of best practice usual care with a single one-to-one practitioner appointment, and general advice on physical activity. Outcomes will be measured at baseline, 4 months (post-intervention) and 12 months by researchers blinded to treatment allocation. The primary outcome is the incremental shuttle walk test at 4 months. Secondary outcomes include health-related quality of life (HRQoL), time to clinical worsening and health and social care use. A purposive sample of participants (n = 20 intervention and n = 20 control) and practitioners (n = 20) will be interviewed to explore experiences of the trial, outcomes and interventions. DISCUSSION: The SPHERe study is the first multi-centre clinical RCT to assess the clinical and cost effectiveness of a supervised exercise rehabilitation intervention compared to usual care, delivered in the UK NHS, for people in all PH groups. Results will inform clinicians and commissioners as to whether or not supervised exercise rehabilitation is effective and should be routinely provided for people with PH. TRIAL REGISTRATION: ISRCTN no. 10608766, prospectively registered on 18th March 2019.

Challenges and Special Aspects of Pulmonary Hypertension in Middle- to Low-Income Regions: JACC State-of-the-Art Review.

Challenges and special aspects related to the management and prognosis of pulmonary hypertension (PH) in middle- to low-income regions (MLIRs) range from late presentation to comorbidities, lack of resources and expertise, cost, and rare options of lung transplantation. Expert consensus recommendations addressing the specific challenges for prevention and therapy of PH in MLIRs with limited resources have been lacking. To date, 6 MLIR-PH registries containing mostly adult patients with PH exist. Importantly, the global prevalence of PH is much higher in MLIRs compared with high-income regions: group 2 PH (left heart disease), pulmonary arterial hypertension associated with unrepaired congenital heart disease, human immunodeficiency virus, or schistosomiasis are highly prevalent. This consensus statement provides selective, tailored modifications to the current PH guidelines to address the specific challenges faced in MLIRs, resulting in the first pragmatic and cost-effective consensus recommendations for PH care providers, patients, and their families.

Healthcare utilization, medical expenditure, and mortality in Korean patients with pulmonary hypertension.

BACKGROUND: Limited data exists regarding healthcare utilization, medical expenses, and prognosis of pulmonary hypertension (PH) according to the World Health Organization (WHO) classification. We aimed to investigate mortality risk, healthcare utilization and medical expenditure in patients with PH across the five diagnostic subgroups. METHODS: We identified 2185 patients with PH, defined as peak tricuspid regurgitation velocity > 3.4 m/sec, among the consecutive patients referred for echocardiography between 2009 and 2015. Using diagnostic codes, medical records, and echocardiographic findings, the enrolled patients were classified according to the five subgroups by WHO classification. Healthcare utilization, costs, and all-cause mortality were assessed. RESULTS: Diagnostic subgroups of PH demonstrated significantly different clinical features. During a median of 32.4 months (interquartile range, 16.2-57.8), 749 patients (34.3%) died. Mortality risk was the lowest in group II (left heart disease) and highest in group III (chronic lung disease). The etiologies of pulmonary arterial hypertension (PAH) had significant influence on the mortality risk in group I, showing the worst prognosis in PAH associated with connective tissue disease. Medical expenditure and healthcare utilization were different between the PH subgroups: groups II and V had more hospitalizations and medical expenses than other groups. Regardless of PH subgroups, the severity of PH was associated with higher mortality risk, more healthcare utilization and medical expenditure. CONCLUSIONS: Significant differences in clinical features and prognostic profiles between PH subgroups reflect the differences in pathophysiology and clinical consequences. Our findings highlight the importance of comprehensive understanding of PH according to the etiology and its severity.

Economic evaluation of sildenafil for the treatment of pulmonary arterial hypertension in Indonesia.

BACKGROUND: This study aims to assess the cost-effectiveness and budget impact of adopting sildenafil to the benefits package for the indication of pulmonary arterial hypertension (PAH), compared to beraprost. METHODS: Based on a societal perspective, a model-based economic evaluation was performed using local and international data to quantify the potential costs and health-related outcomes in terms of life years (LYs) and quality-adjusted life years (QALYs). RESULTS: The economic model calculated the incremental cost-effectiveness ratio (ICER) per QALY gained for using sildenafil as first-line therapy compared to beraprost for the patient in functional class (FC) II and III, i.e. USD 3098 and USD 2827, respectively. The results indicated that in spite of sildenafil being more expensive than beraprost, generic sildenafil could potentially be a good value for money since ICER per QALY is below one times gross domestic product (GDP) per capita in Indonesia. Furthermore, budget impact analysis estimated that the incremental budget needed within 5 years for including sildenafil compared to beraprost for PAH patients starting in FC II and FC III was USD 436,775 and USD 3.6 million, respectively. CONCLUSIONS: Compared to beraprost, sildenafil would be preferable for the treatment of PAH patients in FC II and FC III in Indonesia. The additional budget for adopting sildenafil compared to beraprost as the treatment of PAH in the benefits package was estimated at around USD 4.0 million.

Economic burden of incident interstitial lung disease (ILD) and the impact of comorbidity on costs of care.

INTRODUCTION: Evidence about the economic burden related to interstitial lung diseases (ILDs) and the cost-driving factors is sparse. In the knowledge that distinct comorbidities affect the clinical course of ILDs, our study investigates their impact on costs of care within first year after diagnosis. METHODS: Using claims data of individuals diagnosed with Idiopathic Interstitial Pneumonia (IIP) (n = 14 453) or sarcoidosis (n = 9106) between 2010 and 2013, we calculated total and ILD-associated mean annual per capita costs adjusted by age, sex and comorbidity burden via Generalized Linear Gamma models. Then, we assessed the cost impact of chronic obstructive pulmonary disease (COPD), diabetes, coronary artery disease, depression, gastro-esophageal reflux disease, pulmonary hypertension (PH), obstructive sleep apnoea syndrome (OSAS) and lung cancer using the model-based parameter estimates. RESULTS: Total mean annual per capita costs were €11 131 in the pooled cohort, €12 111 in IIP and €8793 in sarcoidosis, each with a 1/3 share of ILD-associated cost. Most comorbidities had a significant cost-driving effect, which was most pronounced for lung cancer in total (1.989 pooled, 2.491 sarcoidosis, 1.696 IIP) and for PH in ILD-associated costs (2.606 pooled, 2.347 IIP, 3.648 sarcoidosis). The lung-associated comorbidities COPD, PH, OSAS more strongly affected ILD-associated than total costs. CONCLUSION: Comorbidities increase the already substantial costs of care in ILDs. To support patient-centred ILD care, not only highly cost-driving conditions that are inherent with high mortality themselves require systematic management. Moreover, conditions that are more rather restricting the patient's activities of daily living should be addressed - despite a low-cost impact.

Baseline history of patients using selexipag for pulmonary arterial hypertension.

INTRODUCTION: Since its introduction to the market in 2016, selexipag has been an alternative oral therapy among both treatment-naïve patients and those with mono or dual therapy failure; however, limited information is available regarding the presentation and management of patients with pulmonary arterial hypertension (PAH) prior to selexipag initiation. This study examined treatment patterns, healthcare utilization, and costs in the 12 months prior to and the 6 months following selexipag initiation. METHODS: This was a retrospective study of adult commercial and Medicare Advantage with Part D (MAPD) health plan members with a medical or pharmacy claim for selexipag from 1 January 2016 through 31 May 2017, a diagnosis of pulmonary hypertension, and continuous health plan enrollment for 12 months prior to selexipag initiation (baseline period). Treatment patterns, healthcare utilization, and costs were measured over the baseline period and the 6 months following selexipag initiation (among patients with ⩾6 months of follow up). RESULTS: After inclusion and exclusion criteria were applied, 95 patients were included in the analysis. At study start, 57.9% of patients were prescribed combination therapy, increasing to 69.5% immediately prior to selexipag initiation. Approximately 60% of patients had one baseline regimen. Emergency visits and inpatient admissions during the baseline period occurred in 63.2% and 48.4% of patients, respectively. Baseline medical costs rose steadily, increasing 266.8% in commercial and 26.7% in MAPD enrollees from the beginning to the end of the 12-month baseline period. PAH-related healthcare costs accounted for more than 80% of total costs. Mean medical costs in the 6 months following selexipag initiation were US$17,215 in commercial and US$23,976 in MAPD enrollees. CONCLUSIONS: The majority of patients with PAH remained on the same therapy in the 12 months prior to selexipag initiation despite high rates of healthcare utilization and increasing costs. Mean medical costs appeared to decrease after adding or switching to selexipag.

The patient experience of pulmonary hypertension: a large cross-sectional study of UK patients.

BACKGROUND: Pulmonary Hypertension Association UK (PHA-UK) is the only charity in the UK especially for people affected by pulmonary hypertension (PH). To better understand the impact of PH on patients and carers beyond clinical symptoms, the PHA-UK carried out a cross-sectional survey on the effect of PH on daily living, along with a follow-up survey assessing the financial impact of PH. METHODS: This is a descriptive cross-sectional survey of adult patients with PH in the UK. A quantitative survey of four key topics (time to diagnosis, quality of life [QoL], financial impact and specialist treatment), was made available to PHA-UK members and patients on PH therapy, with a follow-up financial impact survey sent to those responders who agreed to be contacted further. Data collection was carried out in January and February 2017 for the main survey, and November and December 2017 for the financial impact survey. RESULTS: The main survey was completed by 567 individuals, and the financial follow-up survey by 171. Mean age of responders was 69 ± 17 years with 70% female. 60% of respondents said PH had a major impact on their QoL, with 45% reporting that treatment and management improves their QoL 'a lot'. The time between first experiencing symptoms and diagnosis was ≥1 year for 48% of patients, with 40% seeing 4+ doctors before diagnosis. 63% of patients reported financial worries. Patients in part-time and full-time work reported the greatest financial burden, with a 13 and 33% fall in monthly income respectively. Patients had positive experiences of treatment in specialist centres, with 62% rating their care 'excellent', and 92% saying they preferred travelling to a specialist centre rather than seeing a local non-specialist. CONCLUSIONS: This study reports the largest UK survey exploring issues affecting patients with PH. The study shows that despite the availability of new therapies, patients are still experiencing delays prior to diagnosis, and experiencing both emotional and financial impacts from the disease. By identifying the areas patients find most important in their treatment, this research can inform future care policies and long-term management to support patients living with PH and their families.

Demographics, treatment trends, and survival rate in incident pulmonary artery hypertension in Korea: A nationwide study based on the health insurance review and assessment service database.

Epidemiologic data regarding pulmonary arterial hypertension (PAH) have relied on registries from Western countries. We assessed the current status of PAH in the Korean population. The Health Insurance Review and Assessment Service (HIRA) claim database, which comprises nationwide medical insurance data of Koreans from 2008-2016, was assessed to determine the current status of PAH. Overall, 1,307 patients were newly diagnosed with PAH from 2008-2016 (0.0005%, annual incidence: 4.84 patients/1 million people/year). The mean age at diagnosis was 44±13 years (range 18-65) and patients were mostly women (n = 906, 69.3%). Cases of idiopathic PAH (51.6%) accounted for the largest proportion, followed by acquired PAH (APAH) associated with congenital heart disease (25.8%) and APAH with connective tissue disease (17.2%). Overall, 807 (61.7%) patients received a single PAH-specific treatment based on their last prescription, of which bosentan (50.6%) was the most frequently used. Only 240 (18.4%) patients received combination therapy, with the bosentan-beraprost combination (32.9%) being the most common. During the mean follow-up of 1.9 years, the 1-, 2-, 3-, and 5-year estimated survival rates were 85%, 62%, 54%, and 46%, respectively. The prevalence and incidence of PAH in the Korean population is currently comparable with that in previous registries. The 5-year survival rate was slightly higher in the Korean population than previously reported.

Prostacyclin Use Among Patients with Pulmonary Arterial Hypertension in the United States: A Retrospective Analysis of a Large Health Care Claims Database.

BACKGROUND: Prostacyclins play an important role in the management of pulmonary arterial hypertension (PAH). Intravenous prostacyclin was the first disease-specific treatment for patients with PAH. Subcutaneous and nonparenteral (oral or inhaled) formulations have subsequently become available. However, data are lacking on how these different prostacyclin formulations are being used in clinical practice. OBJECTIVES: To (a) conduct retrospective analyses of a large U.S. health care claims database to describe the characteristics of patients with PAH initiating prostacyclin therapy, and (b) evaluate their treatment patterns, health care resource use, and associated costs. METHODS: Truven Commercial and Medicare databases were used to define annual cohorts of adults with PAH between January 1, 2010, and October 31, 2015. These patients were identified based on claims with ICD-9-CM diagnoses indicative of PAH (codes 416.0 or 416.8) and claims for PAH-specific medications and PAH-related procedures. Patients with evidence of receiving a prostacyclin were identified, and prostacyclin use was categorized as parenteral versus nonparenteral. Health care costs were assessed alternatively employing an all-cause and PAH-related perspective. RESULTS: Of 13,633 adults with identified PAH, 3,006 (22.0%) received a prostacyclin during at least 1 year of the study period, and annual prevalence of prostacyclin use ranged from 19.9% to 22.6%. Across calendar years, the median age of prostacyclin users ranged from 56 to 58 years, and 71.9%-75.8% were female. Among prostacyclin users, parenteral prostacyclin use declined from 63.2% in 2010 to 46.5% in 2015, while use of nonparenteral prostacyclins increased from 39.7% to 56.2% over the same period (both P < 0.001). Few patients (2.7%-4.1%) received both parenteral and nonparenteral formulations in a given calendar year. Among patients using prostacyclins, receipt of other PAH-specific medications increased from 62.1% in 2010 to 79.2% in 2015. Comparing the 6 months preceding the first prostacyclin prescription (any formulation) to the 6 months subsequent, mean overall health care costs rose from $61,243 to $119,283, and PAH-related health care costs increased from $58,815 to $116,661, driven mainly by PAH-specific medications, spending on which increased from $15,053 to $73,705 (all P < 0.001). CONCLUSIONS: While overall use of prostacyclins was relatively constant from 2010 to 2015, our findings revealed a shift from parenteral to nonparenteral formulations, coupled with increased prescribing of PAH-related medications from other drug classes. Further research is needed to better understand how these changes in patterns of prostacyclin use affect levels of health care resource utilization and costs and patients' overall quality of life. DISCLOSURES: This research was funded by Actelion Pharmaceuticals US, a Janssen pharmaceutical company of Johnson & Johnson. Burger has received grant funding from Actelion, Gilead Sciences, and United Therapeutics; personal fees from Actelion and Gilead Sciences; and nonfinancial support from Actelion. Pruett, Lickert, and Drake are employees of Actelion. Pruett and Lickert own shares in Actelion. Berger and Murphy are employees of Evidera, a consultancy that received payment from Actelion to conduct this research. Pruett, Lickert, Berger, and Drake contributed to study conception and participated with Burger in study design. Lickert and Murphy performed the data analyses. Burger, Pruett, Lickert, Murphy, and Drake interpreted the data. All authors participated in manuscript drafting and/or critical revision, approved the final manuscript, and agree to be accountable for all aspects of the work.

The economic burden of chronic thromboembolic pulmonary hypertension in Russian Federation.

AIM: The aim of current study was to estimate the economic burden of the chronic thromboembolic pulmonary hypertension (CTEPH) in Russia based on patient registry. MATERIALS AND METHODS: Cost of illness study was based on data derived from CTEPH patient registry that was developed at the Division of hypertension of FSBI "National Medical Research Center of Cardiology". Demographic and clinical patient characteristics were analyzed with descriptive statistic methods. Cost of illness study was performed from the state perspective and with bottom-up approach. Bootstrapping was used for calculation of average costs per patient/year. Within the study direct costs (medical costs: outpatient, inpatient, emergency, PAH-specific therapy, concomitant therapy; non-medical costs: pension due to disability status, payments for patients on sick-leave) and indirect costs (loss in GDP) were estimated. RESULTS: Overall, 113 CTEPH patients (67 women and 46 men) from 33 Russian regions were included, mean age of patients with CTEPH was 54.6±13.95 years. Most of the patients (55%) were in able-bodied age. It was found that about half of patients with diagnosed CTEPH had a disability. Average duration of disease at the time of analysis was 6.88±11.41 years. Period from the first occurrence of symptoms to the confirmation of diagnosis of CTEPH was 2.58±5.21 years on average. More than 70% of patients had III and IV FC (WHO) at the time of diagnosis. Mean number of outpatient visits was 1.97±1.65 per patient/year, and inpatient visits were reported for 59% of patients. About 54% of patients used PAH-specific therapy, moreover 46% patients had interruptions of PAH-specific therapy (58.4±66.3 days). The total costs of CTEPH per patient/year were calculated as 805,901 RUB. The overall burden of CTEPH in Russia for total CTEPH population (470 patients) was 379 million RUB per year. CONCLUSION: CTEPH is the rare disease that is characterized with later diagnosis due to absence of disease-specific symptoms. Therefore economic burden of the CTEPH is significantly low in comparison to widespread cardiovascular diseases. Development of network of expert PH-centers and increase of the access for PAH-specific therapy will help to increase the quality of health care for patients with CTEPH.

All-cause Healthcare Costs and Mortality in Patients with Systemic Sclerosis with Lung Involvement.

OBJECTIVE: Patients with systemic sclerosis (SSc) often develop interstitial lung disease (ILD) and/or pulmonary arterial hypertension (PAH). The effect of ILD and PAH on healthcare costs among patients with SSc is not well described. The objective of this analysis was to describe healthcare costs in patients with newly diagnosed SSc and SSc patients newly diagnosed with ILD and/or PAH in the United States. METHODS: This retrospective cohort analysis was conducted in the Truven Health MarketScan Commercial and Medicare Supplemental healthcare claims databases from 2003 to 2014. Based on International Classification of Diseases-9-Clinical Modification diagnosis codes on medical claims, patients were classified into 3 groups: incident SSc, SSc with incident ILD (SSc-ILD), and SSc with incident PAH (SSc-PAH). Patients were required to have continuous enrollment for 5 years to measure all-cause healthcare costs. Costs (adjusted to US$) were reported overall and by service type and year following diagnosis. Because of the overlap between groups, statistical comparisons were not conducted. RESULTS: There were 1957 patients with incident SSc, 219 with incident SSc-ILD, and 108 patients with incident SSc-PAH. Average (mean ± SD) all-cause healthcare costs over followup were higher for patients with incident SSc-ILD ($191,107 ± $322,193) or patients with incident SSc-PAH ($254,425 ± $240,497), compared to patients with incident SSc ($101,839 ± $167,155). Average annual costs over the 5-year period ranged from $18,513 to $23,268 for patients with incident SSc, from $31,285 to $55,446 for patients with incident SSc-ILD, and from $44,454 to $63,320 for patients with incident SSc-PAH. Costs tended to be the highest in the fifth year of followup. CONCLUSION: Among patients with SSc, ILD and PAH can result in substantial increases in healthcare costs.

Update on pulmonary arterial hypertension research: proceedings from a meeting of experts.

BACKGROUND: While pulmonary arterial hypertension (PAH) remains a progressive, symptomatic condition characterized by increased pulmonary vascular resistance, ultimately leading to right heart failure, great strides have been made in its understanding and treatment over the past two decades. REVIEW: Continued research in pre-clinical, clinical, and health economic areas of research, in addition to registry analyses and technology advances, is critical for understanding the pathophysiology of the disease and devising the best ways to monitor and manage patients. On December 3, 2016, the latest pre-clinical, clinical, health economic outcome, and registry data on PAH was presented in a symposium sponsored by Actelion. This paper reviews the published research and insight into upcoming research that was presented at this interactive meeting.

The impact and financial burden of pulmonary arterial hypertension on patients and caregivers: results from a national survey.

Pulmonary arterial hypertension (PAH) is a chronic progressive devastating disease. Symptom burden might impair health-related quality of life of patients. Furthermore, treatment on this disease brings significant financial burden to patients' families. Both physiological and psychological symptoms have been reported, but limited evidence regarding the impact of PAH on patients and caregivers exists, especially the emotional issues and their association with patients' health quality. The main purpose of this study was to describe the impact of PAH on patients and their caregivers in a Chinese population.This large-scale national survey enrolled 174 participants to complete questionnaires using face-to-face semistructured interviews.PAH influenced all aspects of patients' lives including daily activities, work, emotions, and personal relationships. Both patients and caregivers reported a major impact on family finances and on their work. The majority of patients had feelings of isolation. A lack of public understanding about PAH contributes to social isolation. Most patients and caregivers would like to get information regarding PAH doctors and patient organization contacts to obtain support.This survey-based report provides information regarding the way and extent to which PAH impacts both patients and their caregivers and provides some means for comparison with non-Chinese populations. It is important for physicians and the community to offer more support and information for PAH patients and their families.

Healthcare resource utilization and costs for patients with pulmonary arterial hypertension: real-world documentation of functional class.

BACKGROUND AND AIMS: Pulmonary arterial hypertension (PAH) is a rare medical disease in which patients experience increased pulmonary vascular resistance (PVR) and pulmonary arterial pressure that can result in remodeling of the pulmonary vasculature and heart, and eventually lead to right heart failure and death. As PAH progresses, patients become unable to perform even routine daily tasks without severe shortness of breath (dyspnea), fatigue, dizziness, and fainting (syncope). Treatment strategies largely depend on assessment of an individual patient's WHO Functional Class. The aim of the present study was to determine whether PAH functional decline, as described by the WHO Functional class (FC), is associated with increased healthcare costs for patients. METHODS: Patients with a prescription for a FDA-approved treatment for PAH and a medical claim indicating chronic pulmonary heart disease or right heart catheterization were identified from an administrative claims database. Provider-reported data from prior authorization forms required for advanced PAH therapies and medical charts were examined for reported FC. Healthcare resource utilization and costs were the primary outcomes of interest. Costs were accounted in 2014 US dollars ($) from a healthcare payer perspective. RESULTS: Patients with a reported FC-IV were observed to have the worst outcomes; averaging significantly more inpatient admissions, longer average lengths of stay, and more emergency department visits than the other FC sub-groups, resulting in higher medical costs. CONCLUSIONS: Using administrative data to document disease severity, this study replicates and expands on findings obtained from the registry study; disease severity was associated with higher healthcare resource utilization and costs. Stakeholders' implications for patient management are discussed.

Considerations for optimal management of patients with pulmonary arterial hypertension: a multi-stakeholder roundtable discussion.

A roundtable panel of national and regional managed care decision makers and providers met to discuss pulmonary arterial hypertension (PAH) and strategies for management. As a rare, complex disease with high economic costs and potentially devastating outcomes, PAH necessitates that managed care providers balance optimal care with efficient use of healthcare resources. PAH specialists are recognized by health plans as knowledgeable experts and integral partners in managing patients and resources. The diagnosis of PAH must be confirmed by a right heart catheterization. Available therapies are indicated almost exclusively for patients with PAH (riociguat is also indicated in chronic thromboembolic pulmonary hypertension) and target 1 of 3 pathways: endothelin receptor antagonists for the endothelin pathway; phosphodiesterase type-5 inhibitors and soluble guanylate cyclase stimulators for the nitric oxide pathway; and prostanoids as well as a prostacyclin receptor agonist for the prostacyclin pathway, with combination therapy becoming more common. Even in the modern treatment era, as shown in the REVEAL and French registries, PAH is often diagnosed years after symptoms first appear, which leads to a poor prognosis and increased burden on the healthcare system. Facilitating treatment of patients with PAH through centers of excellence, and coordinating care management between health plans and providers with evidence-based approaches can lead to both better results for patients and lower healthcare costs. When PAH experts have access to the right treatments for the right patients at the right time, they can work with insurers to improve the health of patients with PAH while helping to reduce the impact on the healthcare system.

Nationwide trends in inpatient admissions of pulmonary hypertension in the United States from 2000 to 2013.

INTRODUCTION: Pulmonary hypertension (PH) is a disorder of the pulmonary vasculature with high mortality and bears a large economic burden on the healthcare system. We conducted a review of the largest inpatient database in the United States and analyzed the trends in hospitalizations due to PH from the turn of the century (2000) to 2013 to evaluate the rate of hospitalizations and determine the cost and mortality associated with PH. MATERIAL AND METHODS: We analyzed the National Inpatient Sample Database (NIS) for all patients in which PH (Primary or Secondary) or cor pulmonale was the primary discharge diagnosis (ICD-9: 416.0, 416.8 and 416.9) from 2000 to 2013. The NIS is the largest all-payer inpatient database in the United States and contains data from approximately 8 million hospital stays each year. The statistical significance of the difference in the number of hospital discharges, lengths of stays and associated hospital costs over the study period was calculated. RESULTS: In 2000, there were 12,066 hospital admissions with the principal discharge diagnosis of pulmonary hypertension, which increased to 13,605 admissions in 2013 (p < 0.001). The mean length of stay for PH increased from 5.89 days to 6.67 days during this period (p = 0.04). During the same period, the hospital charges increase by 174.5% from US$ 24,973 in 2000 to US$ 68,545 in 2013 (Adjusted for inflation). The aggregate cost of hospital visits of a patient increased by 209.5% from US$ 301,324,218 in 2000 to US$ 932,554,725 in 2013. CONCLUSIONS: The number of inpatient discharges related to PH has increased even though the number of inpatient discharges with PAH has been reported to be lower in literature. The mean length of stay has also shown a mild increase. This increase is associated with a significant increase in the mean and aggregate cost. These inpatient costs associated with PH contribute significantly to the total healthcare burden. Further research on cost-effective evaluation and management of PH is required.