Immune tolerance induction with moroctocog‐alpha (Refacto/Refacto AF) in a population of Italian haemophilia A patients with high‐titre inhibitors: Data from REF.IT Registry. Issue 6 (11th October 2019)

• Record Type: Journal Article
• Authors: Zanon, Ezio; Pasca, Samantha; Pollio, Berardino; Santagostino, Elena; Linari, Silvia; Tagliaferri, Annarita; Santoro, Cristina; Rocino, Angiola; Marino, Renato; Aru, Brigida; Borchiellini, Alessandra; Siragusa, Sergio; Coppola, Antonio
• Is Part Of: Haemophilia. Volume 25:Issue 6(2019)
• Page Start: 1003
• Page End: 1010
• Publication Date: 2019-10-11
• Subjects: haemophilia A with inhibitors -- immune tolerance induction -- moroctocog‐alpha -- poor‐prognosis ITI patients; Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.13859
• Languages: English
• ISSNs: 1351-8216

Abstract

Abstract: Background: The appearance of inhibitors is the most serious complication in haemophilia A (HA) patients. The primary objective is their eradication. Up to date, immune tolerance induction (ITI) was the only therapeutic option to achieve this. Aim: To assess the efficacy of moroctocog‐alpha as an ITI regimen in a population of HA patients with high‐titre inhibitors. Methods: The REF.IT Registry is a retrospective‐prospective study that collected data on all patients with HA and high‐titre inhibitors treated with moroctocog‐alpha as an ITI regimen at twelve Italian Haemophilia Centres. Results: We enrolled 27 patients, 85.2% were children. All patients were high responders, 88.9% had severe HA. We found 69.3% of them had one or more risk factors for poor ITI prognosis, 14.8% were ITI rescue. Overall 59.3% achieved a complete/partial success (complete in 51.9%). ITI failed in 11 patients, 63.6% of them with poor‐prognosis risk factors. Inhibitors appeared after a mean of 27 exposure days. Mean historical peak was 78.8 BU/mL. The primary ITIs started on average 20.2 months after the diagnosis. A partial or complete success after a mean of 15 months of treatment was achieved in 56.6% of the children while the same result was obtained by 75.0% adults after 22 months from ITI onset. Patients who were treated with high‐dose moroctocog‐alpha (200 UI/kg/day) were 63.0%. Conclusion: Our Registry showed that the use of moroctocog‐alpha in the setting of ITI was effective and safe also in a population of patients with high‐titre inhibitors, presenting one or more risk factors for poor ITI prognosis.