Contribues for pulmonary hypertension: from healthcare organization to a comprehensive patient clinical management

• Autor(a) principal: Reis, Abílio Óscar Silva
• Data de Publicação: 2018
• Tipo de documento: Artigo
• Idioma: eng
• Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
• Texto Completo: http://hdl.handle.net/10773/25877
• topic: Pulmonary hypertension; Pulmonary arterial hypertension; Healthcare organization; Pulmonary hypertension information technologies; Quality of Life; Health-related quality of life; Disability

As a background for this Thesis, the author presents the definition and implementation of a healthcare organization system for Pulmonary Hypertension (PH) in Portugal and the development of a dedicated information technology system for PH management and clinical research (PAHTool™), in both cases he was actively involved. Taking advantage of the clinical research potentialities created by the referral centers’ network that was implemented and the availability of PAHTool™, the author and his colleagues conducted a series of studies to characterize the Portuguese PH population in terms of epidemiology, clinical characteristics, treatment options, outcomes, and Quality of Life and Disability impairment. All these studies have been published or accepted for publication by peer-reviewed scientific journals. “Pulmonary Hypertension in Portugal: 1st Data from a Nationwide Registry” was a prospective, observational, multicenter registry from five PH centers in Portugal. Predominance of females (65.2%), relatively young patients (43.416.4 years) and idiopathic pulmonary arterial hypertension (IPAH) etiology (37.0%) were the most relevant findings in the pulmonary arterial hypertension (PAH) group. Most patients were treated with single (50%), double (28%) or triple (9%) combination therapy. The 1-year survival estimate was 93.5% and 93.9% for the PAH and chronic thromboembolic pulmonary hypertension (CTEPH) groups, respectively. The main findings of this registry were in line with that of other international registries. “Pulmonary Hypertension: Real-world Data from a Portuguese Expert Referral Center” was a retrospective analysis of a cohort of 101 patients with pre-capillary PH (pcPH) referenced to an expert tertiary care referral center in northern Portugal, from 2002 to 2013. This study provided long-term (3.82.7 years) real-world data of a predominantly incident population (80.2%), where the most relevant demographic findings were the female gender predominance (66.3%) and the relatively young age of the population (49.619.6 years). The high number (>60%) of patients in advanced stage of the disease at presentation suggested delayed diagnosis and highlighted the need to increase PH awareness among clinicians and explains why most patients were treated with combination therapy (53.2%). There was a significant improvement of World Health Organization functional class (WHO FC) (p<0.003), 6-minutes walking distance (6MWD) (p=0.003), mean pulmonary artery pressure (mPAP) (p=0.002), and pulmonary vascular resistance (PVR) (p=0.008), without significant changes in mean right atrial pressure (mRAP) and cardiac index (CI). The cumulative probability of survival at 1-, 3- and 5-years for the total cohort (respectively 86.6%, 76.7%, 64.1%), PAH (91.8%, 80.3%, 66.2%) and CTEPH (81.5%, 75.3%, 67.3%) and the substantial proportion of patients with idiopathic/heritable pulmonary arterial hypertension (I/HPAH) treated with triple combination therapy (61.1%) highlight the access to modern targeted therapies and compliance with international guidelines. “Long-term Survival in Pulmonary Arterial Hypertension and Chronic Thromboembolic Pulmonary Hypertension: Insights from a Referral Center in Portugal” intended to assess the long-term survival of a cohort of 142 patients followed up in a Portuguese referral center. This study showed that comparing the cohorts of patients treated in two periods, 2011-2016 and 2005-2010, a trend toward improved survival at 5 years was found for connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) (67% vs 33%) and CTEPH (84% vs 77%), but not for patients with I/HPAH (75% vs 84%). Earlier diagnosis, increasing use of parenteral prostanoids, and improvement of surgical treatments (pulmonary endarterectomy and lung transplantation) access were deemed necessary to further improve PAH and CTEPH patient’s outcomes. “Portuguese Validation of the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) Questionnaire” was a study aiming to translate and validate the CAMPHOR questionnaire for the European Portuguese-speaking population. Good internal consistency and reproducibility, as well as excellent convergent reliability and group validity was found for the Portuguese version of the CAMPHOR questionnaire. In “Health-Related Quality of Life in Pulmonary Hypertension and Its Clinical Correlates: A Cross-Sectional Study” it was demonstrated that HRQoL, measured by two different patient-reported outcomes measures (PROMs) (CAMPHOR, a PH-specific questionnaire, and Nottingham Health Profile, a general one), is impaired in Portuguese patients with PAH and other forms of pcPH, particularly in patients with increased disease severity. WHO FC, 6MWD, and Borg dyspnea index were highly correlated with health-related quality of life (HRQoL), measured by both instruments and quality of life (QoL), measured by CAMPHOR. In “Disability and Its Clinical Correlates in Pulmonary Hypertension Measured Through the World Health Organization Disability Assessment Schedule II (WHODAS 2.0): a prospective, observational study” the studied PH population showed mild disability, with higher degree of disability in the domains of Mobility and Life activities. WHODAS 2.0 scores at baseline were robustly predictive of 6MWD and WHO functional class evolution. This study was the first one to assess disability in PH patients, using WHODAS 2.0