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RESEARCH PRODUCT
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.
Silvia LinariAnnarita TagliaferriBrigida AruSamantha PascaEzio ZanonBerardino PollioRenato MarinoAngiola RocinoElena SantagostinoCristina SantoroAntonio CoppolaSergio SiragusaAlessandra Borchiellinisubject
AdultMalemedicine.medical_specialtyPopulationHaemophilia AAlpha (ethology)030204 cardiovascular system & hematologyHaemophiliaHemophilia Ahaemophilia A with inhibitors; immune tolerance induction; moroctocog-alpha; poor-prognosis ITI patients; Adult; Child; Child Preschool; Factor VIII; Female; Hemophilia A; Humans; Immune Tolerance; Italy; Male; Prospective Studies; Retrospective Studies; Risk Factors; RegistriesImmune tolerance03 medical and health sciences0302 clinical medicineRisk FactorsInternal medicineImmune ToleranceMedicineHumansProspective StudiesRegistrieseducationHigh titrePreschoolChildGenetics (clinical)Retrospective Studiesimmune tolerance inductioneducation.field_of_studyFactor VIIIbusiness.industryHematologyGeneral Medicinehaemophilia A with inhibitormedicine.diseasepoor-prognosis ITI patientsRegimenItalymoroctocog-alphaChild PreschoolFemalebusinessComplicationhaemophilia A with inhibitors030215 immunologydescription
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.
year | journal | country | edition | language |
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2019-01-01 | Haemophilia : the official journal of the World Federation of HemophiliaREFERENCES |