Background: Primary prophylaxis started early at the dosage of 25-40 IU/kg three times/week is considered the gold standard for the treatment of patients with severe hemophilia, improving their quality of life (QoL) and reducing bleeds. In the last years different new concentrates have been marketed, allowing a reduction of annual bleeding rate (ABR) with concomitant improvement of compliance to treatment.

Aims: The aim of this study was to compare the differences between the treatment with the single chain rFVIII, lonoctocog-alpha,  and octocog-alpha in terms of total, joint and spontaneous ABR, number of infusions and dosage in a group of patients with hemophilia A (HA).

Methods: All patients with HA, referred to four Hemophilia Centers (Padova, Vicenza, Castelfranco Veneto, Verona) belonging to the same Italian region (Veneto), previously receiving octocog-alpha (2^nd generation), and subsequently switched to lonoctocog-alpha were considered in our study. Statistical analysis were performed considering one year of treatment for any type of treatment.

Results: Twenty-one previously treated patients (PTPs), 10-59 years-old were switched to lonoctocog-alpha, 13/21 of them were previously on prophylaxis with octocog-alpha. 81% had severe hemophilia, 5% moderate, while three (13%) were mild young subjects in prophylactic treatment as practice sport activity. 72.6% were adults (≥14 years). Five PTPs on demand were put on prophylaxis with rFVIII single-chain. Mean ABR were decreased after switch, while the weekly median number of infusion remained unchanged, 2 (range 1-3). A comparison head to head between the 13 patients on prophylaxis with the two different rFVIII concentrates are reported in Figure 1.  

Comparison head to head between 13 prophylaxis with octocog-alpha and lonoctocg-alpha. ABR: annual bleeding rate; AjBR: annual joint bleeding rate; AsBR: annual spontaneous bleeding rate.

Conclusions: In our study the switch to rFVIII single-chain was proved to be more effective compared to prophylaxis with octocog-alpha in all patients. The 13 patients already on prophylaxis with octocog-alpha reduced their ABR by 50% after the switch, while the annual consumption of the product remained similar as was the cost.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/cost-effectiveness-of-rfviii-single-chain-in-an-italian-population-of-hemophilia-a-patients-the-veneto-experience/