The Pulmonary Hypertension Global Patient Survey: Understanding the Invisible Burden of Paediatric Pulmonary Hypertension

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

https://doi.org/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.

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