Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia
This is a multicenter observational case-control analysis lasting 12 months aimed at determining the prevalence of pulmonary hypertension (PAH) in patients with Thalassemia Major and Intermedia. The patients will be followed, treated and examined according to the best standard clinical practice dictated by the Italian Society for the study of Hemoglobinopathies (SITE), Thalassemia International Federation (TIF)and the Task Force for Diagnosis and Treatment of Pulmonary Hypertension of European Society of Cardiology (ESC); European Respiratory Society (ERS); International Society of Heart and Lung Transplantation (ISHLT) guidelines. ...
Pulmonary Vascular Disease Phenomics Program PVDOMICS
It is recognized that patients with various forms of heart and lung disease exhibit varying degrees of pulmonary hypertension, pulmonary vascular remodeling, and right ventricular dysfunction. The genetic, molecular, and cellular processes driving these phenomena are not well understood. Rapid advances in high throughput omic methodology, combined with powerful bioinformatics and network biology capability, have created the opportunity to conduct studies that broadly search for homologies and differences across the spectrum of disease states associated with pulmonary hypertension, and determinants of the spectrum of right ventricular...
Riociguat in Children With Pulmonary Arterial Hypertension (PAH)
This study was designed to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of riociguat at age-, sex- and body-weight-adjusted doses of 0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5 mg TID in children from ≥6 to less than 18 years with pulmonary arterial hypertension (PAH) group 1. The study design consisted of a main study part followed by an optional long-term extension part. The main treatment period consisted of two phases: titration phase up to 8 weeks and a maintenance phase up to 16 weeks.
Side Effects of Pulmonary Hypertension Medications
The study seeks to analyze the patient reported effects of pulmonary hypertension medications and compare these with the side effects described on the package inserts. Side effects for these PH medications have been described in the adult population, but have never been described in the pediatric population. This information can better improve patient care and be used to characterize the side effects resultant from these medications.
Thrombosis Outcomes in Pediatric Venous Thromboembolism
'The TOP Study' is a prospective cohort study whose main objective is to develop better prognostic biomarkers and identify children at risk of adverse thrombotic outcomes very early in the course after an initial venous thromboembolic events (VTE). The study will compare biomarkers in children that develop poor VTE outcomes (such as recurrence, postthrombotic syndrome and post PE impairment ) after an initial VTE with those that do not develop such outcomes.
Trans-pulmonary Biomarkers in Pulmonary Hypertension
The investigators are testing whether the addition of Pulmonary Hypertension-related biomarkers, measured across the pulmonary circulation, to the standard hemodynamic evaluation for Pulmonary Hypertension will lead to more informed choices of Pulmonary Hypertension therapy and improved patient outcomes.
TReatment Of Pulmonary HYpertension 1 Study
The objective of this study is to assess the safety, performance and initial effectiveness of the TIVUS™ System when used for pulmonary artery denervation through subjective and objective change in clinical parameters and haemodynamic evaluation. This is a prospective, multi-center, non-randomized, open-label clinical trail. The study will be conducted in up to 5 centers and will recruit up to 15 patients diagnosed with PAH, functional class III who have stable PAH on a stable drug regimen of two pulmonary arterial hypertension specific medications.
TReatment Of Pulmonary HYpertension 1-US Study
The objective of this study is to assess the safety, performance and initial effectiveness of the TIVUS™ System when used for pulmonary artery denervation through subjective and objective change in clinical parameters and haemodynamic evaluation. This is a prospective, multi-center, non-randomized, open-label clinical trail. The study will be conducted in up to 4 centers and will recruit up to 15 patients diagnosed with PAH, functional class III who have stable PAH on a stable drug regimen of two pulmonary arterial hypertension specific medications.
Infusion of Allogeneic Mesenchymal Stem Cells in Patients With Diffuse Cutaneous Systemic Sclerosis With Refractory Pulmonary Involvement
Progressive SSc is an entity with limited therapeutic alternatives and with asurvival rate of less than 45% in the first 3 to 5 years. The disease causessevere limitation in quality of life ranging from functional limitation to depression. Up to 20% of patients will be refractory to conventional treatment with diseasemodifying anti-rheumatic drugs (DMARDs) and cyclophosphamide therapy.This favors the progression to visceral involvement including gastrointestinal,lung and pulmonary hypertension. The latter being a poor prognostic factor,increases mortality in this group of patients and drastically affects their qualityof life. For...
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