Sadia Quyam, Sarah Drumm, Shiv Mungala, Katherine Bunclark, Gerald Fischer, Eva Otter, Hakim Ghani, Wendy Gin‐Sing, Luke Howard, Marcin Kurzyna, Pisana Ferrari, Lynsay Macdonald, Joseph Newman, Millicent Stone, Mark Toshner, Joanna Pepke Zaba, Maurice Beghetti, Shahin Moledina
Great Ormond Street Hospital for Children and University College London. Pulmonary Vascular Research Institute. Royal Statistical Society. University of Cambridge and Royal Papworth Hospital. Pulmonary Hypertension Association Europe. Imperial College Healthcare NHS Trust. European Heart Center. Alliance for Pulmonary Hypertension. Children’s University Hospital Geneva.
United Kingdom, Austria, Poland, Belgium and Switzerland
Pulmonary Circulation
Pulm Circ 2026; 16:
DOI: 10.1002/pul2.70269
Abstract
The Pulmonary Hypertension Global Patient Survey (PH GPS) provides the first international examination of experiences among children with pulmonary hypertension (PH) and their caregivers. Through an online survey distributed via PH associations across 32 countries, we collected responses from 136 caregivers about diagnostic journeys, treatment experiences, healthcare access and research participation. Qualitative analysis revealed challenges around the ‘invisible’ nature of PH, with caregivers describing how schools and communities sometimes doubted their child’s limitations in the absence of visible symptoms, adding to family burden. Quantitative findings demonstrated extensive caregiver burden with 40% providing constant care while facing significant employment consequences, including leaving work altogether, reduced hours, or career changes. Although 78.8% of children accessed specialist centres, significant barriers persisted: 41.2% encountered diagnostic delays exceeding 6 months, 34.8% travelled over 2 h for appointments and 27.7% received no healthcare cost reimbursement. Treatment experiences revealed 37.7% of children experienced side effects, yet only one-third discussed changes with their clinical teams, reflecting pragmatic decisions within limited therapeutic options. Research participation was low (19.2%), though families with trial experience showed greater willingness for future participation, compared to those without experience (70.8% vs. 49.5%). Barriers centred on awareness gaps (16.4%), safety concerns (14.5%), and age-related considerations (10.9%), distinct from adult PH populations where logistical challenges predominate. These findings demonstrate that paediatric care must extend beyond medical management to address family needs including social-work assistance for benefits navigation, flexible care delivery models incorporating telemedicine, pro-active communication about treatment tolerability and research protocols designed with families from inception.
Category
Quality of Life Associated with Pulmonary Vascular Disease
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