Pulmonary Arterial Hypertension (PAH)
Conditions
Keywords
Pulmonary Arterial Hypertension, Prostacyclin Pathway, Patient-Reported Outcomes, Quality of Life, Treatment Adherence, Real-World Evidence, Iloprost, Selexipag, Observational Study
Brief summary
Pulmonary arterial hypertension (PAH) is a rare and serious condition that affects the blood vessels of the lungs and can significantly limit daily activities and quality of life. Some patients with PAH use inhaled iloprost, a medication that requires several inhalations per day, which can be difficult to maintain over time. Oral selexipag is an alternative treatment that may reduce treatment burden and improve adherence. The PROMISE study aims to evaluate how switching from inhaled iloprost to oral selexipag affects patients' quality of life, satisfaction with treatment, and adherence in real-world clinical practice. Patient-reported outcome questionnaires will be used to understand patients' perceptions of symptoms, daily functioning, and overall improvement after the transition. Adult patients with PAH who are receiving inhaled iloprost and whose physicians decide to switch treatment to oral selexipag will be followed over time. A comparison group of patients who continue using inhaled iloprost will also be observed. The study does not involve any experimental treatment or changes to routine clinical care. All medications are prescribed as part of standard medical practice. The results of this study may help improve understanding of the patient experience during treatment transitions in PAH and support more patient-centered treatment decisions.
Detailed description
Pulmonary arterial hypertension (PAH) is a progressive and life-threatening disease that requires long-term treatment with targeted therapies. Medications acting on the prostacyclin pathway are an important component of PAH management. Inhaled iloprost has demonstrated clinical and hemodynamic benefits but requires multiple daily inhalations, which may increase treatment burden and negatively affect adherence and daily functioning. Oral selexipag, a selective IP receptor agonist administered twice daily, offers a less complex dosing regimen and may improve treatment persistence and patient experience. In Brazil, the incorporation of oral selexipag into national treatment guidelines has led to an increasing number of patients transitioning from inhaled iloprost to oral therapy as part of routine clinical care. However, data on patient-reported outcomes, satisfaction, and real-world adherence following this transition are limited, particularly in Latin American populations. The PROMISE study is a prospective, observational, single-center study designed to assess the patient experience associated with the transition from inhaled iloprost to oral selexipag in adults with PAH. The study focuses on changes in health-related quality of life, treatment satisfaction, global perception of improvement, and objective and self-reported measures of adherence over time. A comparison group of patients who continue treatment with inhaled iloprost will be followed in parallel to provide contextual interpretation of observed changes. All treatment decisions, including initiation, continuation, titration, or discontinuation of medications, are made exclusively by the treating physician according to standard clinical practice. Participation in the study does not influence therapeutic choices, and no additional interventions beyond routine care are performed. Data collection consists of validated patient-reported outcome measures, structured questionnaires, and review of clinical information obtained during usual follow-up visits. By capturing patient-centered outcomes in a real-world setting, the PROMISE study aims to improve understanding of the impact of treatment transitions on daily life and adherence in PAH, supporting more informed, patient-focused management strategies.
Interventions
DRUGSelexipag
Exposure to oral selexipag following transition from inhaled iloprost as part of routine clinical care in adults with pulmonary arterial hypertension. The decision to initiate, titrate, or discontinue selexipag is made exclusively by the treating physician, independent of study participation. No study-mandated intervention or treatment assignment is performed. The study observes patient-reported outcomes, treatment satisfaction, and adherence associated with this treatment transition.
Sponsors
University of Sao Paulo General Hospital
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adults aged 18 years or older. * Confirmed diagnosis of pulmonary arterial hypertension (PAH, Group 1) by right heart catheterization. * Current treatment with inhaled iloprost as part of routine clinical care. * Clinical decision by the treating physician to transition from inhaled iloprost to oral selexipag. * Ability to understand and complete patient-reported outcome questionnaires. * Provision of written informed consent.
Exclusion criteria
* Significant cognitive impairment or sensory deficits that prevent completion of study questionnaires. * Unstable or decompensated comorbid conditions that may interfere with treatment adherence or study participation. * Refusal or inability to provide informed consent.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Health-Related Quality of Life (EMPHASIS-10) | From baseline (prior to transition) to 16-24 weeks of follow-up | Change in health-related quality of life assessed by the emPHasis-10 questionnaire (Pulmonary Hypertension-Specific Quality of Life Instrument), measured as the difference in total score between baseline (prior to transition from inhaled iloprost to oral selexipag) and follow-up. The emPHasis-10 is a patient-reported outcome measure ranging from 0 to 50, with lower scores indicating better health-related quality of life. The outcome evaluates within-patient change following treatment transition in routine clinical practice. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Treatment Satisfaction (TSQM-9) | From baseline (prior to transition) to 3-4 weeks and 16-24 weeks after transition | Change in treatment satisfaction assessed using the Treatment Satisfaction Questionnaire for Medication (TSQM-9), measured before the transition from inhaled iloprost and after transition to oral selexipag. The TSQM-9 is a patient-reported outcome measure evaluating treatment effectiveness, convenience, and global satisfaction domains. Domain scores range from 0 to 100, with higher scores indicating greater treatment satisfaction. The outcome allows within-patient comparison of treatment satisfaction before and after the treatment switch in routine clinical practice. |
| Patient Global Impression of Change (PGIC) | 3-4 weeks and 16-24 weeks after transition | Patient-reported global impression of change assessed using the Patient Global Impression of Change (PGIC) scale, reflecting the patient's overall perception of improvement or worsening following the transition from inhaled iloprost to oral selexipag. The PGIC is a patient-reported outcome measure consisting of a 7-point Likert scale ranging from "very much worse" to "very much improved," with higher scores indicating greater perceived improvement. This outcome also explores the correlation between PGIC ratings and changes in other patient-reported outcomes. |
| Objective Adherence to Selexipag | From transition to 16-24 weeks of follow-up | Objective treatment adherence assessed by the percentage of days with twice-daily selexipag use, treatment interruptions, and the highest titrated dose achieved during follow-up, as documented in routine clinical practice. |
| Perceived Treatment Burden | From baseline (prior to transition) to 16-24 weeks after transition | Perceived treatment burden evaluated before the transition, based on the number of daily inhalations with iloprost, and after the transition, based on patient-reported convenience of the oral selexipag regimen. |
| Adverse Events and Health Care Utilization | From transition to 16-24 weeks of follow-up | Occurrence of adverse events and health care utilization, including hospitalizations and unplanned outpatient visits, recorded during routine clinical follow-up after transition to oral selexipag. |
| Responder Status Based on EMPHASIS-10 | From baseline to 16-24 weeks of follow-up | Proportion of patients achieving a clinically meaningful improvement in health-related quality of life, defined as a decrease of at least 6 points (minimal clinically important difference) in the EMPHASIS-10 score between baseline and follow-up. |
Contacts
CONTACTCaio Fernandes Principal Investigator, MD
PRINCIPAL_INVESTIGATORCaio Fernandes, MD
UNIVERSIDADE SAO PAULO
Outcome results
None listed