How Is Pulmonary Hypertension Characterised and Treated in Children With Trisomy 21? Observations From the TOPP Registry (Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension).

Pulmonary hypertension is common in children with Trisomy 21, frequently with multifactorial aetiologies. Registry data provide better understanding of disease development, diagnostic workup and treatment patterns in children with Trisomy 21. TOPP (Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension) is a centre-based, comprehensive registry.

Colorectal cancer chemoprevention: Exploring the path from molecular mechanisms to available drugs.

Colorectal cancer (CRC) remains a significant global health challenge despite advances in screening and diagnostic modalities that have contributed to reduced incidence and mortality. A substantial proportion of cases, however, continue to be diagnosed at advanced stages. Chemoprevention - the use of natural or synthetic substances to prevent cancer initiation or recurrence - has emerged as a promising strategy, particularly for individuals at elevated risk.

Enhancing Drug Development for Paediatric Pulmonary Hypertension-An Integrative Perspective.

As with adult pulmonary hypertension (PH), high morbidity and mortality persist with diverse types of paediatric PH. Despite major advances in pharmacologic therapies based on extensive studies in adult PH, few drugs have been comprehensively studied in neonates, infants, and children, leaving current paediatric PH care largely dependent on small observational studies and extrapolation of evidence from adult clinical trials. Challenges in developing successful clinical trials in children include the need to define distinct disease phenotypes with well-characterised natural history and outcomes, the lack of established age- and disease-specific study endpoints, small and heterogeneous paediatric populations, and the common off-label use of PH-targeted drug therapies without regulatory approval.

Procedural Variation May Contribute to 6-Minute Walk Distance Variability in Real-World Pediatric Pulmonary Arterial Hypertension Study.

The six-minute walk test (6MWT) is a common method to assess submaximal exercise capacity in children and adults with pulmonary arterial hypertension (PAH) and other chronic diseases. There is no guideline specifically for 6MWT in children. In this observational pilot study, we evaluated the impact of procedural variations on the outcome of the 6MWT in the real-world clinical setting at pediatric PAH programs.

Echocardiographic Prediction of Hemodynamics During Acute Vasoreactivity Testing in Pediatric Pulmonary Arterial Hypertension.

Purpose: In pediatric pulmonary arterial hypertension (PAH), echocardiography is frequently used to assess response to acute vasodilator therapy. We sought to evaluate whether echocardiography is sensitive enough to detect significant changes in hemodynamics using two commonly used thresholds (Barst and Sitbon criteria) as benchmarks to establish a positive response.

Methods: Simultaneous echocardiography and invasive hemodynamics were obtained in 71 children with PAH at their baseline and maximal vasodilatory conditions.