VOLIBRIS (ambrisentan) - Hypertension artérielle pulmonaire

Key points

Approval of reimbursement, alone or in association, for the treatment of pulmonary arterial hypertension (PAH) in functional class II and III (WHO classification) adolescent and paediatric patients (aged from 8 years to under 18 years). Efficacy has been demonstrated for idiopathic PAH and for PAH associated with systemic collagenosis.

Therapeutic improvement?

No therapeutic improvement.

Role in therapeutic strategy?

The objective of treatment is primarily to improve patient survival and quality of life.

PAH is treated in children based on risk stratification (symptom progression, WHO functional classification, certain ultrasound parameters). Given that PAH is a progressive disease in the short term, regular follow-up is required to ensure early detection of clinical exacerbation in order to allow therapeutic escalation at the earliest possible opportunity. Prognostic assessment is important for the choice of initial treatment and assessment of treatment response.

The paediatric PAH treatment algorithm is extrapolated from that for adults, despite limited paediatric data based on low-quality methodology (no randomised controlled clinical trials).

As a first-line approach, it is recommended to conduct acute vasoreactivity testing in order to identify potential responders (<10%) to calcium channel blocker treatment.

For non-responders, and low-risk patients, it is recommended to introduce monotherapy treatment (endothelin receptor antagonists, PDE5i inhibitors, or prostacyclin analogues), or bitherapy treatment from the outset. For high-risk patients, it is recommended to use intravenously or subcutaneously administered prostacyclin analogues from the outset. 

At the present time, in addition to ambrisentan, only bosentan (orally administered endothelin receptor antagonist) and sildenafil (orally administered PDE5 inhibitors) have been granted a marketing authorisation for children, and are covered in France for this paediatric indication. 

Creating a Potts shunt is an alternative or bridge to lung transplantation to be proposed for forms failing to respond to tritherapy.

Role of VOLIBRIS (ambrisentan)

VOLIBRIS (ambrisentan) must be used, alone or in association, for the treatment of pulmonary arterial hypertension (PAH) in functional class II and III adolescent and paediatric patients. It is a first-line treatment.

It is an alternative to TRACLEER (bosentan) and to REVATIO (sildenafil).

Note that this treatment must only be initiated and supervised by a medical practitioner experienced in the treatment of pulmonary arterial hypertension.