Patient-Reported Outcomes and Adherence After Transition From Inhaled Iloprost to Oral Selexipag in Pulmonary Arterial Hypertension

Official title

PROMISE: Evaluation of Patient-Reported Outcomes and Adherence After Transition From Inhaled Iloprost to Oral Selexipag in Pulmonary Arterial Hypertension

Overview

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

• Drug Selexipag
  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.

Primary outcome measures

• Change in Health-Related Quality of Life (EMPHASIS-10) [Time frame: From baseline (prior to transition) to 16-24 weeks of follow-up]

Secondary outcome measures (6)

• Change in Treatment Satisfaction (TSQM-9) [Time frame: From baseline (prior to transition) to 3-4 weeks and 16-24 weeks after transition]
• Patient Global Impression of Change (PGIC) [Time frame: 3-4 weeks and 16-24 weeks after transition]
• Objective Adherence to Selexipag [Time frame: From transition to 16-24 weeks of follow-up]
• Perceived Treatment Burden [Time frame: From baseline (prior to transition) to 16-24 weeks after transition]
• Adverse Events and Health Care Utilization [Time frame: From transition to 16-24 weeks of follow-up]
• Responder Status Based on EMPHASIS-10 [Time frame: From baseline to 16-24 weeks of follow-up]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

Center list to be confirmed — check the primary protocol.

Publications

• 1. Simonneau G, Montani D, Celermajer DS, Denton CP, Gatzoulis MA, Krowka M, et al. Haemodynamic definitions and updated clinical classification of pulmonary hypertension. Eur Respir J. janeiro de 2019;53(1):1801913. 2. Humbert M, Sitbon O, Simonneau G. Treatment of Pulmonary Arterial Hypertension. N Engl J Med. 30 de setembro de 2004;351(14):1425-36. 3. Calderaro D, Alves Junior JL, Fernandes CJC

Identifiers

NCT: NCT07356375 · SGP 27905

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗