Profiles and treatment patterns of patients with pulmonary arterial hypertension on monotherapy at experienced centres

Abstract

Aims Guideline recommendations highlight the critical role of combination therapy for the treatment of pulmonary arterial hypertension (PAH). Conversely, registry data demonstrate that a considerable number of PAH patients remain on monotherapy. The reasons for this discrepancy remain elusive. The aim of this study was to assess the patient profiles, treatment patterns, and disease characteristics of patients diagnosed with PAH who were kept on monotherapy at experienced pulmonary hypertension (PH) centres and to capture potential reasons for monotherapy. Methods and results We analysed the patient profiles of 182 patients on monotherapy with PAH‐targeted drugs, managed at experienced PH expert centres (Cologne, Giessen, Heidelberg, and Dresden). Patients were identified based on their latest follow‐up visit and analysed retrospectively from the time of PAH diagnosis to last follow‐up. Patients were dichotomized by age, and patient characteristics, treatment patterns, response to therapy, change in risk status, and drug tolerability were recorded during the course of their disease. Patients' mean age was 69.1 ± 13.1 years at the most recent follow‐up (Key Time Point 1) and 64.5 ± 14.9 years at the time of diagnosis (Key Time Point 2). The mean time on monotherapy was 60.7 ± 53.8 months; 35.7/64.3% of patients were male/female. The majority (66.5%) had idiopathic PAH, followed by PAH associated with connective tissue disease (17.0%) and portopulmonary PH (8.2%). Among patients on monotherapy, there were five main clusters: (i) patients with failed escalation attempts mostly because of intolerability (26.9%); (ii) low risk on monotherapy, favourable response, and no reason for escalation (24.2%); (iii) patients with mild PAH (36.3%); (iv) elderly patients with PAH and multiple co‐morbidities (38.5%); and (v) patients with associated forms of PAH where the level of evidence for combination therapies is considered low (16.5%). There were substantial differences between patients above or below the median age (68 years). The most frequently used monotherapy for PAH was phosphodiesterase type 5 inhibitors (75.3%). Conclusions A considerable number of PAH patients are on monotherapy at large PH expert centres, characterized by specific reasons that justify this kind of treatment. Nevertheless, as comprehensive treatment strategies have shown improved long‐term outcomes even in mildly symptomatic patients, each case of monotherapy should be justified.

Adherence and treatment patterns of disease-specific drugs among patients with pulmonary arterial hypertension: A nationwide, new-user cohort study

“…However, differences in initial treatment for patients with PAH were observed between the guideline recommendations and the findings of this study and other studies. Wissmüller et al (2022) reported five main reasons for taking a single medication among 182 patients managed at experienced PH care centers, including 1) failure to escalate due to intolerability, 2) being at low risk and having a favorable response to monotherapy, 3) having mild PAH syndrome, 4) being older and having multiple comorbidities, and 5) lack of satisfactory evidence of combination therapy for some subgroups of PAH (HIV, portal hypertension, and CHD) (Wissmüller et al, 2022). In this study, the mean (SD) age of patients with PAH was 57.5 (17.8) years, and only one-fifth of them were aged <40 years.…”

Phenotypes and treatment outcomes in idiopathic pulmonary arterial hypertension patients with comorbidities

“…However, such data are difficult to interpret, as the treatment regimens differed between phenotypes, with combination therapies prescribed much less in patients with comorbidities. The latter is partly explained by poorer drug tolerability and a higher rate of discontinuations (particularly ERA) in such phenotypes [ 7 , 32 ], as well as reluctancy to prescribe combination therapies in IPAH patients with multiple comorbidities [ 37 ]. Thus, it is not entirely clear whether lower treatment efficacy is due to comorbidities or because patients receive combination therapies less frequently.…”

“… Individual treatment decision in patients with comorbidities [ 37 ]: up to now only data on treatment of pulmonary arterial hypertension (PAH) patients with comorbidities is available for cases with left heart phenotype or patients with heart failure with preserved ejection fraction (HFpEF) risk factors and elderly patients (>65 years). IPAH: idiopathic PAH; PVR: pulmonary vascular resistance; ESC/ERS: European Society of Cardiology/European Respiratory Society.…”

“…The latter is partly explained by poorer drug tolerability and a higher rate of discontinuations (particularly ERA) in such phenotypes [7,32], as well as reluctancy to prescribe combination therapies in IPAH patients with multiple comorbidities. [38] Thus, it is not entirely clear whether lower treatment efficacy is due to comorbidities or because patients receive combination therapies less frequently. Furthermore, it is currently unknown to what extent these experiences of an overall positive benefit vs risk profile of (combination) therapy in IPAH patients with comorbidities can also be transferred to patients with associated PAH such as CTD-PAH, as most analyses from studies and registries were only performed for patients with IPAH and therefore the data for other forms of PAH are even rarer.…”

Real-World Treatment Patterns Among Patients with Connective Tissue Disorder-Related Pulmonary Arterial Hypertension in the United States: A Retrospective Claims-Based Analysis

“…Regarding treatment patterns in PAH, evidence suggests that monotherapy with PDE5i remains the most commonly used strategy [ 27 , 28 ] despite meta-analyses reporting that combination therapy significantly reduces the risk of progression by approximately 35% [ 19 , 20 ]. In contrast to PAH, there is currently limited information on treatment patterns among patients with CTD-related PAH [ 15 ].…”