BACKGROUND:

Hereditary angioedema due to complement C1-inhibitor deficiency (C1-INH-HAE) is a rare condition resulting in recurrent attacks of angioedema, which can cause temporary disability and be life-threatening. Clinical guidelines recommend the treatment of attacks at onset and several therapies for on-demand treatment are available in Italy. However, there is still little knowledge on different treatments’ behaviours, effectiveness and costs, which can affect coverage and reimbursement decisions.

OBJECTIVES:

To explore treatment behaviours in a cohort of Italian patients with C1-INH-HAE and to estimate how effects and costs of treating attacks in routine practice differed across on-demand treatments.

METHODS:

A one-year prospective cohort study on 167 patients was conducted at the Italian reference center for C1-INH-HAE. Attacks were treated with either icatibant, plasma derived C1 inhibitor (pdC1-INH) or supportive care. Survival analyses were carried out to evaluate treatment efficacy in reducing attack duration. Two-part models were used to estimate the direct costs of acute attacks.

RESULTS: CONCLUSIONS:

Both icatibant and pdC1-INH significantly reduced attack duration compared to no treatment. Icatibant resulted to be more effective but more expensive. Treatment behaviours and sub-optimal dosing of pdC1-INH may account for the differences, but further research is needed to define their role.