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Clinical features of paediatric pulmonary hypertension: a registry study
Rolf M F Berger1, Maurice Beghetti, Tilman Humpl
1Centre for Congenital Heart Diseases-Paediatric Cardiology, Beatrix Children's Hospital, University Medical Centre Groningen, University of Groningen, Netherlands. r.m.f.berger@umcg.nl
Insights
The Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension (TOPP) registry reveals key features of pediatric pulmonary hypertension. Findings emphasize the need for child-specific data over adult study extrapolations.
Area of Science:
- Pediatric Cardiology
- Pulmonology
- Clinical Research
Background:
- Pediatric pulmonary hypertension (PH) significantly impacts child morbidity and mortality.
- Current understanding of pediatric PH is limited.
- The TOPP registry was established to address this knowledge gap.
Purpose of the Study:
- To gather comprehensive data on demographics, treatment, and outcomes in pediatric PH.
- To characterize the specific clinical features of PH in children.
- To inform clinical practice and research by providing child-specific data.
Main Methods:
- Prospective, global enrollment of pediatric patients (≤18 years) with confirmed PH.
- Data collection included patient and disease characteristics, medical history, and hemodynamic indices.
- Follow-up was determined by individual physician care plans.
Main Results:
- 362 patients had confirmed PH; 88% had pulmonary arterial hypertension (PAH), often associated with congenital heart disease.
- Idiopathic/familial PAH comprised 57% of PAH cases.
- Common symptoms included dyspnea and fatigue; syncope was noted in specific subgroups.
- A significant proportion (64%) presented with preserved functional class (I or II).
Conclusions:
- The TOPP registry highlights unique clinical characteristics of pediatric PH.
- Results underscore the necessity of dedicated pediatric data, rather than relying on adult study findings.
- This data is crucial for improving the diagnosis and management of PH in children.
Background:
Paediatric pulmonary hypertension, is an important cause of morbidity and mortality, and is insufficiently characterised in children. The Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension (TOPP) registry is a global, prospective study designed to provide information about demographics, treatment, and outcomes in paediatric pulmonary hypertension.
Methods:
Consecutive patients aged 18 years or younger at diagnosis with pulmonary hypertension and increased pulmonary vascular resistance were enrolled in TOPP at 31 centres in 19 countries from Jan 31, 2008, to Feb 15, 2010. Patient and disease characteristics, including age at diagnosis and at enrolment, sex, ethnicity, presenting symptoms, pulmonary hypertension classification, comorbid disorders, medical and family history, haemodynamic indices, and functional class were recorded. Follow-up was decided by the patients' physicians according to the individual's health-care needs.
Findings:
362 of 456 consecutive patients had confirmed pulmonary hypertension (defined as mean pulmonary artery pressure ≥25 mm Hg, pulmonary capillary wedge pressure ≤12 mm Hg, and pulmonary vascular resistance index ≥3 WU/m(-2)). 317 (88%) patients had pulmonary arterial hypertension (PAH), which was idiopathic [IPAH] or familial [FPAH] in 182 (57%), and associated with other disorders in 135 (43%), of which 115 (85%) cases were associated with congenital heart disease. 42 patients (12%) had pulmonary hypertension associated with respiratory disease or hypoxaemia, with bronchopulmonary dysplasia most frequent. Finally, only three patients had either chronic thromboembolic pulmonary hypertension or miscellaneous causes of pulmonary hypertension. Chromosomal anomalies, mainly trisomy 21, were reported in 47 (13%) of patients with confirmed disease. Median age at diagnosis was 7 years (IQR 3-12); 59% (268 of 456) were female. Although dyspnoea and fatigue were the most frequent symptoms, syncope occurred in 31% (57 of 182) of patients with IPAH or FPAH and in 18% (eight of 45) of those with repaired congenital heart disease; no children with unrepaired congenital systemic-to-pulmonary shunts had syncope. Despite severe pulmonary hypertension, functional class was I or II in 230 of 362 (64%) patients, which is consistent with preserved right-heart function.
Interpretation:
TOPP identifies important clinical features specific to the care of paediatric pulmonary hypertension, which draw attention to the need for paediatric data rather than extrapolation from adult studies.
Funding:
Actelion Pharmaceuticals.
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