Long-term efficacy of bosentan in treatment of pulmonary arterial hypertension in children.

The aim of the present study was to evaluate a 5-yr experience of bosentan in children with pulmonary arterial hypertension (PAH). A retrospective, observational study was made of children in the UK Pulmonary Hypertension Service for Children (Great Ormond Street Hospital for Children, London, UK) who were given bosentan as monotherapy or in combination, from February 2002 to May 2008 and followed up for ≥ 6 months. Detailed studies were made of 101 children with idiopathic PAH (IPAH) (n = 42) and PAH associated with congenital heart disease (n = 59). Before treatment, World Health Organization (WHO) functional class, 6-min walk distance (6MWD), height, weight and haemodynamic data were determined. Evaluations were analysed after 6 months and annually to a maximum of 5 yrs. Median duration of treatment was 31.5 months. Initial improvement in WHO functional class and 6MWD was maintained for up to 3 yrs. Height and weight increased but the z-scores did not improve. After 3 yrs, bosentan was continued as monotherapy in only 21% of children with IPAH, but in 69% of repaired cases and 56% of those with Eisenmenger syndrome. The Kaplan-Meier survival estimates for the 101 patients were 96, 89, 83 and 60% at 1, 2, 3 and 5 yrs, respectively. A treatment regime that includes bosentan is safe and appears to be effective in slowing disease progression in children with PAH.

Childhood idiopathic pulmonary arterial hypertension: a national cohort study.

OBJECTIVE: To clarify the clinical characteristics and epidemiology of idiopathic pulmonary arterial hypertension (IPAH) in childhood, a rare condition with a bad prognosis, poorly documented in children. Also, to describe the long-term outcome. DESIGN: A retrospective study of 7 years' experience. SETTING: UK Service for Pulmonary Hypertension in Children based at a tertiary referral centre. PATIENTS: 64 children. INTERVENTIONS: Patients were initially treated with prostanoids (n=15), bosentan (n=23), sildenafil (n=9), combination therapy (n=11) or calcium channel antagonists (n=6). MAIN OUTCOME MEASURES: WHO functional class, distance walked in 6 minutes, escalation of therapy, survival, transplant-free survival. RESULTS: Incidence of IPAH was 0.48 cases per million children per year and the prevalence was 2.1 cases per million. 31% presented with syncope. Oedema was rare. During the first year of follow-up WHO functional class and 6-minute walk distance improved significantly. Survival at 1, 3 and 5 years was 89%, 84% and 75%, respectively; while transplant-free survival was 89% 76% and 57%, respectively. Factors predicting worse survival were WHO functional class (HR 2.4, p=0.04) and poor height and weight z-score (p<0.05 for both) at presentation. CONCLUSIONS: We showed, for the first time, that the incidence of IPAH is lower in children than adults and that the clinical features can be different. Most children present with clinical evidence of advanced disease and clinical status at presentation is predictive of outcome. This 7-year experience confirms the significant improvement in survival over historical controls.

The relationship between parental perceptions of diabetes and glycaemic control.

AIM: To measure the relationship between perceived child competence, parental self-efficacy, and children's glycaemic control. METHODS: Cross-sectional outpatient based questionnaire survey of 78 parents of children aged 6-12 years with insulin dependent diabetes mellitus, diagnosed for at least one year. Parental perceptions of their child's competence were assessed, together with parental perceptions of their own self-efficacy in managing their child's diabetes. Glycaemic control was assessed by the average annual HbA1C level. RESULTS: The response rate was 64.5% (51 parents); 82% were mothers and the socioeconomic class and ethnicity spread was representative of the general population. The mean age of the children was 10 years and duration of diabetes 4.4 years. Poorer glycaemic control was associated with higher perceived child competence, together with lower perceived age of responsibility, lower perceived seriousness, and less frequent blood tests. Higher parental self-efficacy and higher perceived child competence predicted a higher level of normalisation, as did lower perceived seriousness, a lower perceived parental responsibility for management, and a less protective style of parenting. CONCLUSION: Parents' perceptions of their children's diabetes are significantly related to glycaemic control; however, those who appear more competent at managing diabetes may overestimate their child's capabilities, leading to poorer glycaemic control.