Characteristics and prospective 2-year follow-up of children with pulmonary arterial hypertension in France.
BACKGROUND
Limited data are available describing paediatric pulmonary arterial hypertension.


OBJECTIVE
To characterize the epidemiology, management and impact on quality of life and outcome of paediatric pulmonary arterial hypertension, excluding persistent pulmonary hypertension of the newborn and pulmonary arterial hypertension caused by congenital heart disease.


METHODS
In this multicentre study, children with pulmonary arterial hypertension were included and followed prospectively for two years at 21 referral centres in France. WHO functional class, 6-minute walk distance and quality of life (CHQ-PF50 questionnaire) were evaluated.


RESULTS
Fifty children were included with a mean age of 8.9 +/- 5.4 years from May 2005 until June 2006. The estimated prevalence for pulmonary arterial hypertension was 3.7 cases/million. Patients had idiopathic pulmonary arterial hypertension (60%), familial pulmonary arterial hypertension (10%), pulmonary arterial hypertension associated with, but not caused by, congenital heart disease (24%), pulmonary arterial hypertension associated with connective tissue disease (4%) or portal hypertension (2%). During follow-up, the combination of pulmonary arterial hypertension-specific therapies was increasingly prescribed (44% patients versus 22% at inclusion). Patients remained stable regarding clinical status, 6-minute walk distance and quality of life. Survival estimates after one and two years were 86% (95% CI 76, 96) and 82% (95% CI 71, 93), respectively.


CONCLUSIONS
In children, idiopathic/familial pulmonary arterial hypertension accounts for the majority of cases. A specific pulmonary arterial hypertension group in conjunction with congenital heart disease can be identified that resembles patients with idiopathic pulmonary arterial hypertension. Combined pulmonary arterial hypertension-specific therapies may have contributed to disease stability and favourable survival.
Fraisse
Alain
A
Hôpital de la Timone, 13000 Marseille, France.
Jais
Xavier
X
Schleich
Jean-Marc
JM
di Filippo
Sylvie
S
Maragnès
Pascale
P
Beghetti
Maurice
M
Gressin
Virginie
V
Voisin
Michel
M
Dauphin
Claire
C
Clerson
Pierre
P
Godart
François
F
Bonnet
Damien
D
D000293:Adolescent; D000959:Antihypertensive Agents / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D004522:Educational Status; D005080:Exercise Test; D005260:Female; D005500:Follow-Up Studies; D005602:France / Q000453:epidemiology; D020022:Genetic Predisposition to Disease; D006330:Heart Defects, Congenital / Q000150:complications / Q000453:epidemiology; D006439:Hemodynamics; D006801:Humans; D006976:Hypertension, Pulmonary / Q000175:diagnosis / Q000188:drug therapy / Q000453:epidemiology* / Q000209:etiology / Q000401:mortality; D007223:Infant; D053208:Kaplan-Meier Estimate; D008297:Male; D015995:Prevalence; D011446:Prospective Studies; D011788:Quality of Life; D012307:Risk Factors; D011795:Surveys and Questionnaires; D013997:Time Factors; D016896:Treatment Outcome
D000959:Antihypertensive Agents