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

Ezio Zanon, Samantha Pasca, Berardino Pollio, Elena Santagostino, Silvia Linari, Annarita Tagliaferri, Cristina Santoro, Angiola Rocino, Renato Marino, Brigida Aru, Alessandra Borchiellini, Sergio Siragusa, Antonio Coppola

Research output: Contribution to journalArticle

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.

Original languageEnglish
Pages (from-to)1003-1010
JournalHaemophilia
Volume25
Issue number6
DOIs
Publication statusPublished - 2019

Keywords

  • haemophilia A with inhibitors
  • immune tolerance induction
  • moroctocog-alpha
  • poor-prognosis ITI patients

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

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    Zanon, E., Pasca, S., Pollio, B., Santagostino, E., Linari, S., Tagliaferri, A., Santoro, C., Rocino, A., Marino, R., Aru, B., Borchiellini, A., Siragusa, S., & Coppola, A. (2019). 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. Haemophilia, 25(6), 1003-1010. https://doi.org/10.1111/hae.13859