Beneath the surface: a narrative review of multidimensional burdens and pathways to patient-centered care in PAH

深入剖析:肺动脉高压多维负担及以患者为中心的护理路径的叙述性综述

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Abstract

Pulmonary arterial hypertension (PAH) is a progressive, life-altering disease that imposes profound physical, psychological, and economic burdens on patients and caregivers. Therapeutic advances over the past three decades, most recently the introduction of sotatercept, have improved survival and functional outcomes. Despite this, many of the most pressing needs remain unseen, particularly those related to health-related quality of life (HRQoL). Patients often face years of diagnostic delays, and once diagnosed, experience fragmented care delivered across subspecialties. Rural, minority, and low-income populations encounter disproportionate barriers to specialty access, underscoring the role of social determinants of health (SDoH) in shaping outcomes. Alongside these systemic barriers, patients report high rates of depression, anxiety, fatigue, and social isolation-burdens that are frequently underestimated in clinical practice. To better capture the lived experience of PAH, patient-reported outcome measures (PROMs) provide insights beyond hemodynamic and survival metrics. HRQoL instruments such as the SF-36, CAMPHOR, PAH-SYMPACT, and EmPHasis-10 demonstrate validity in identifying high-risk patients, measuring treatment effects, and predicting outcomes. Despite their promise, challenges such as unclear minimal clinically important differences (MCIDs), workflow barriers, and limited uptake have hindered widespread integration. In parallel, supportive and palliative care services remain underutilized, despite strong evidence that early adoption improves symptom control, communication, and quality of life. This state-of-the-art narrative review proposes a two-tier framework for understanding PAH-related burdens. Patient-level burdens-including psychological distress, functional limitations, and treatment burden-can be addressed through integrating PROMs, HRQoL instruments, palliative, and mental health care. System-level burdens-driven by SDoH, racial and ethnic inequities, geographic barriers, and insurance gaps-require policy reforms, telehealth expansion, community partnerships, and greater diversity in research and clinical trials. Future progress in PAH depends on aligning therapeutic advances with what matters most to patients. Embedding specific PROMs into risk stratification, establishing MCIDs, leveraging digital tools for longitudinal monitoring, and integrating multidisciplinary and palliative services into pulmonary hypertension (PH) clinics will advance patient-centered care. Simultaneously, addressing system-level inequities through policy, community, and research initiatives will ensure equitable access and outcomes. By moving beyond survival to encompass the full spectrum of lived experience, PAH care can evolve into a holistic, patient-centered model.

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