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Predictors of inhibitor eradication by primary immune tolerance induction in severe haemophilia A with high responding inhibitors

Authors :
Elena Santagostino
Annarita Tagliaferri
Maria Elisa Mancuso
Giancarlo Castaman
Maurizio Margaglione
Pier Mannuccio Mannucci
Silvia Linari
Ezio Zanon
Giovanni Di Minno
Chiara Biasoli
Antonio Coppola
Angiola Rocino
Cristina Santoro
Source :
Haemophilia : the official journal of the World Federation of HemophiliaREFERENCES. 28(1)
Publication Year :
2021

Abstract

BACKGROUND Immune tolerance induction (ITI) is the only proven strategy to eradicate factor VIII inhibitors in patients with haemophilia A (HA). AIM To identify patients and treatment options with the highest chance of inhibitor eradication by primary ITI. PATIENTS AND METHODS In the frame of the Italian ITI Registry, carried out from 1995 to 2015 (last follow-up 2018), 137 primary ITI courses in severe HA patients (90/137 with poor prognosis) were analysed for predictors of outcome (complete/partial response or failure). Sixty-six of them (48%) were prospectively evaluated. RESULTS ITI was successful in 91/137 patients (66.4%) and 70 (51.1%) achieved complete response within 11 months (median). Historical peak titres ≤200 BU/ml (P = .033), inhibitor titres ≤5 BU/ml at ITI start (P = .001), peak titres ≤100 BU/ml during ITI (P

Details

ISSN :
13652516
Volume :
28
Issue :
1
Database :
OpenAIRE
Journal :
Haemophilia : the official journal of the World Federation of HemophiliaREFERENCES
Accession number :
edsair.doi.dedup.....10794c2ca94b83e42894dc1ba7742b68