Catherine M. Avitabile, Usha S. Krishnan, Delphine Yung, Stephanie S. Handler, Nidhy Varghese, Angela Bates, Jeff Fineman, Rachel Sullivan, Grace Friere, Eric Austin, Mary P. Mullen, Carol Pereira, Eric J. Christensen, Gayane Yenokyan, Joseph M. Collaco, Steven H. Abman, Lew Romer, D. Dunbar Ivy, Erika B. Rosenzweig
University of Pennsylvania and Children’s Hospital of Philadelphia. Morgan Stanley Children’s Hospital, New York Presbyterian Vagelos College of Physicians and Surgeons and Columbia University. University of Washington School of Medicine. Medical College of Wisconsin. Baylor College of Medicine and Texas Children’s Hospital. Stollery Children’s Hospital and University of Alberta. University of California, San Francisco. Monroe Carell Jr. Children’s Hospital and Vanderbilt University Medical Center. Johns Hopkins All Children’s Hospital. Boston Children’s Hospital. Duke Clinical Research Institute. Johns Hopkins University School of Medicine. Children’s Hospital Colorado.
United States and Canada
Pulmonary Circulation
Pulm Circ 2024;
DOI: 10.1002/pul2.12339
Abstract
Pulmonary vasodilator treatment can improve hemodynamics, right ventricular function, symptoms, and survival in pediatric pulmonary hypertension (PH). However, clinical trial data are lacking due to many constraints. One major limitation is the lack of relevant trial endpoints reflective of hemodynamics or functional status in patients in whom standard exercise testing is impractical, unreliable, or not reproducible. The Kids Mod PAH trial (Mono- vs. Duo Therapy for Pediatric Pulmonary Arterial Hypertension) is an ongoing multicenter, Phase III, randomized, open-label, pragmatic trial to compare the safety and efficacy of first-line combination therapy (sildenafil and bosentan) to first-line monotherapy (sildenafil alone) in 100 pediatric patients with PH across North America. Investigators will measure participants’ physical activity with a research-grade, wrist-worn actigraphy device at multiple time points as an exploratory secondary outcome. Vector magnitude counts per minute and activity intensity will be compared between the treatment arms. By directly and noninvasively measuring physical activity in the ambulatory setting, we aim to identify a novel, simple, inexpensive, and highly reproducible approach for quantitative assessment of exercise tolerance in pediatric PH. These data will increase the field’s understanding of the effect of pulmonary vasodilator treatment on daily activity – a quantitative measure of functional status and wellbeing in pediatric PH and a potential primary outcome for future clinical trials in children with cardiopulmonary disorders.
Category
Diagnostic Testing for Pulmonary Vascular Disease. Non-invasive Testing
Age Focus: Pediatric Pulmonary Vascular Disease
Fresh or Filed Publication: Fresh (PHresh). Less than 1-2 years since publication
Article Access
Free PDF File or Full Text Article Available Through PubMed or DOI: Yes