Goal-oriented therapy in paediatric pulmonary arterial hypertension: are we ready?

Pulmonary arterial hypertension (PAH) is still a fatal disease, affecting patients of all ages including children [1]. In the last 20 years, significant progress has been achieved in the understanding of the disease, and in particular, in the medical approach, with the introduction of new therapies. Goal-oriented strategy represents a significant progress in PAH management and has emerged as the best treatment approach for PAH in adults [2]. Indeed, it is considered as part of the daily practice and should lead to an increase in therapy when goals are not met. In the European Society of Cardiology/European Respiratory Society guidelines for diagnosis and treatment or pulmonary hypertension treatment, goals were suggested [3, 4]. NICKEL et al. [5] described their own, local, approach towards adults with PAH and showed a significant improvement in survival when a goal-oriented strategy was applied [5]. The concept is based on defining markers of the disease and follows their changes systematically, and then adapting therapy to these changes. To be successful, it requires the identification of the markers along with the changes that correlate to an improved outcome. At the last world symposium on pulmonary hypertension, held in Nice (France), a specific task force was dedicated to the treatment goals of pulmonary hypertension, and all potential treatment goals were discussed [2]. This approach is thought to be valid for all groups of patients included in the group 1 of the clinical classification for pulmonary hypertension. But are treatment goals similar for all groups of PAH patients (group 1 of the clinical classification)? A specific discussion raised the important question: are treatment goals different in different subgroups of pulmonary arterial hypertension? This is a key question as the PAH group is a heterogeneous group of patients. In particular, the paediatric group can be considered as a specific group and at the last world symposium it benefited from a specific task force [1], and part of the published proceedings of this group related to treatment goals.